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FVIII阻害剤を使用しない軽度または中等度の血友病Aの参加者におけるエミシズマブの安全性、有効性、薬物動態および薬力学を評価するための研究 (HAVEN 6)

2026年8月5日 更新者：Hoffmann-La Roche

FVIII阻害剤を使用していない軽度または中等度の血友病A患者におけるエミシズマブの安全性、有効性、薬物動態、および薬力学を評価するための多施設非盲検試験

これは、第VIII因子（FVIII）に対する阻害剤を持たない軽度または中等度の血友病Aの参加者におけるエミシズマブの安全性、有効性、薬物動態、および薬力学を評価するために設計された多施設、非盲検、単群試験です。

調査の概要

状態

完了

条件

介入・治療

研究の種類

介入

入学 (実際)

73

段階

  • フェーズ 3

連絡先と場所

このセクションには、調査を実施する担当者の連絡先の詳細と、この調査が実施されている場所に関する情報が記載されています。

研究場所

  • アメリカ
    • California
      • Los Angeles、California、アメリカ、90027
        • Childrens Hospital LA
    • Georgia
      • Atlanta、Georgia、アメリカ、30308
        • Hemophilia of Georgia Center for Bleeding & Clotting Disorders
    • Indiana
      • Indianapolis、Indiana、アメリカ、46260
        • Indiana Hemophilia & Thrombosis center
    • Michigan
      • Ann Arbor、Michigan、アメリカ、48109
        • University of Michigan, C.S. Mott Children's Hospital
    • Washington
      • Seattle、Washington、アメリカ、98101
        • Washington Institute for Coagulation
  • イギリス
      • Cardiff、イギリス、CF14 4XW
        • Cardiff and Vale NHS Trust
      • London、イギリス、NW3 2QG
        • Royal Free Hospital
      • London、イギリス、WC1N 3HR
        • Great Ormond Street Hospital for Children NHS Foundation Trust
  • オランダ
      • Amsterdam、オランダ、1105 AZ
        • Amsterdam UMC Location AMC
  • カナダ
    • Alberta
      • Edmonton、Alberta、カナダ、T6G 1Z1
        • Kaye Edmonton Clinic
    • Newfoundland and Labrador
      • St. John's、Newfoundland and Labrador、カナダ、A1B 3V6
        • Eastern Health - General Hospital
  • スペイン
      • Madrid、スペイン、28046
        • Hospital Universitario La Paz
      • Seville、スペイン、41013
        • Hospital Universitario Virgen del Rocío
  • ドイツ
      • Bonn、ドイツ、53127
        • Universitatsklinikum Bonn
      • München、ドイツ、80336
        • Klinikum der Universität München, Campus Innenstadt
  • フランス
      • Bron、フランス、69677
        • Hopital Cardio-vasculaire Louis Pradel
      • Le Kremlin-Bicêtre、フランス、94275
        • CH de Bicêtre
      • Paris、フランス、75015
        • Groupe Hospitalier Necker Enfants Malades
  • ベルギー
      • Brussels、ベルギー、1200
        • Cliniques Universitaires St-Luc
      • Leuven、ベルギー、3000
        • UZ Leuven Gasthuisberg
  • ポーランド
      • Warsaw、ポーランド、02-776
        • Instytut Hematologii i Transfuzjologii
  • 南アフリカ
      • Johannesburg、南アフリカ、2193
        • Charlotte Maxeke Johannesburg Hospital

参加基準

研究者は、適格基準と呼ばれる特定の説明に適合する人を探します。これらの基準のいくつかの例は、人の一般的な健康状態または以前の治療です。

適格基準

就学可能な年齢

  • 子
  • 大人
  • 高齢者

健康ボランティアの受け入れ

いいえ

説明

包含基準:

  • 軽度の診断（FVIIIレベルが5％以上から
  • 体重≧3キログラム（kg）
  • 治験責任医師の評価に基づく予防の必要性
  • インヒビターの陰性検査（すなわち、
  • 文書化された阻害剤（すなわち、
  • -予防的または一時的なFVIII治療の詳細、および登録前の少なくとも過去24週間の出血エピソードの数の文書化
  • -十分な血液学的肝および腎機能
  • -出産の可能性のある女性の場合：治療期間中および治験薬の最終投与後少なくとも24週間、禁欲を続けるか避妊を使用することに同意する（プロトコルで定義）

除外基準:

  • -軽度または中等度の先天性血友病A以外の遺伝性または後天性出血性疾患
  • -スクリーニング前48週間以内の違法薬物またはアルコール乱用の履歴、調査官の判断
  • -以前（過去12か月以内）または現在の血栓塞栓症または血栓塞栓症の兆候の治療
  • 現在、出血または血栓症のリスクを高める可能性のあるその他の状態
  • -モノクローナル抗体療法またはエミシズマブ注射の成分に関連する臨床的に重大な過敏症の病歴
  • -エミシズマブ負荷投与フェーズ中に計画された手術（5週目以降のエミシズマブの参加者の手術は許可されています）
  • -CD4数を伴う既知のHIV感染
  • -付随する疾患、状態、スクリーニング評価または臨床検査における重大な異常、または研究の実施を妨げる可能性のある治療、または研究者の意見では、治験薬を参加者に投与する際に追加の許容できないリスクをもたらす
  • 次のいずれかの領収書: 以前のエミシズマブ予防を除いて、最後の薬物投与から 5 半減期以内に血友病性出血を治療またはリスクを軽減するための治験薬。 -過去30日または5半減期のいずれか短い方以内の非血友病関連の治験薬;または現在投与中または投与予定のその他の治験薬
  • 研究者の意見では、研究プロトコルを遵守できない
  • -妊娠中または授乳中、または研究中に妊娠する予定（出産の可能性のある女性は、治験薬の開始前7日以内に血清妊娠検査結果が陰性でなければなりません）

研究計画

このセクションでは、研究がどのように設計され、研究が何を測定しているかなど、研究計画の詳細を提供します。

研究はどのように設計されていますか？

デザインの詳細

  • 主な目的：処理
  • 割り当て：なし
  • 介入モデル：単一グループの割り当て
  • マスキング：なし（オープンラベル）

武器と介入

参加者グループ / アーム
介入・治療
実験的：エミシズマブ
第VIII因子（FVIII）阻害剤のない軽度および中等度の血友病Aの参加者は、登録されます エミシズマブ負荷用量レジメンに続いて、3つの維持用量レジメンのいずれかの参加者の好み。
薬：エミシズマブ
エミシズマブの4回の負荷用量は、体重1キログラムあたり3ミリグラム（mg / kg）を皮下（SC）に1週間に1回（QW）4週間投与し、その後、参加者は次の3つの維持SC用量レジメンのいずれかを好みます：1.5 mg /kg QW、3 mg/kg を 2 週間に 1 回 (Q2W)、または 6 mg/kg を 4 週間に 1 回 (Q4W)。
他の名前：
  • ヘムライブラ
  • RO5534262
  • RG6013
  • ACE910

この研究は何を測定していますか？

主要な結果の測定

結果測定
メジャーの説明
時間枠
治療出血のモデルベースの年間出血率
時間枠：最初のエミシズマブ投与の日から少なくとも 52 週間のエミシズマブ治療まで (中央値 [範囲、最小-最大] 有効期間: 55.64 [8.6-89.9] 週間)
有効期間中の治療出血数は、異なるフォローアップ時間を考慮した負の二項回帰モデルを使用して、年間出血率 (ABR) として推定されました。 治療された出血は、「出血の治療」であると報告された血友病薬が直接続いた出血として定義されました。 72 時間ルールが実装されました。最初の出血に対する最後の治療から 72 時間以内に 2 回目の出血が発生した場合、同じ種類の同じ解剖学的位置での 2 回の出血は 1 回の出血としてカウントされます。 手術/手順による出血は除外されました。
最初のエミシズマブ投与の日から少なくとも 52 週間のエミシズマブ治療まで (中央値 [範囲、最小-最大] 有効期間: 55.64 [8.6-89.9] 週間)
Mean Calculated ABR for Treated Bleeds
時間枠：From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks)
The number of treated bleeds over the efficacy period is presented here as a calculated ABR that was annualized for each participant using the following formula: ABR = (number of bleeds/number of days during the efficacy period) x 365.25. A treated bleed was defined as a bleed that was directly followed by a hemophilia medication reported to be a "treatment for bleed". The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed. Bleeds due to surgery/procedure were excluded.
From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks)
Median Calculated ABR for Treated Bleeds
時間枠：From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks)
The number of treated bleeds over the efficacy period is presented here as a calculated ABR that was annualized for each participant using the following formula: ABR = (number of bleeds/number of days during the efficacy period) x 365.25. A treated bleed was defined as a bleed that was directly followed by a hemophilia medication reported to be a "treatment for bleed". The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed. Bleeds due to surgery/procedure were excluded.
From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks)

二次結果の測定

結果測定
メジャーの説明
時間枠
治療された自然出血の計算された平均年間出血率
時間枠：最初のエミシズマブ投与の日から少なくとも 52 週間のエミシズマブ治療まで (中央値 [範囲、最小-最大] 有効期間: 55.64 [8.6-89.9] 週間)
有効期間中の治療された自然出血の数は、次の式を使用して各参加者について年換算された計算された年換算出血率 (ABR) としてここに表示されます: ABR = (出血数/有効期間中の日数) x 365.25 . 治療された自然出血は、治療された出血（「出血の治療」と報告されている血友病の薬が直接続く出血）として定義され、外傷や処置/手術などの他の既知の要因はありません。 72 時間ルールが実装されました。最初の出血に対する最後の治療から 72 時間以内に 2 回目の出血が発生した場合、同じ種類の同じ解剖学的位置での 2 回の出血は 1 回の出血としてカウントされます。 手術/手順による出血は除外されました。
最初のエミシズマブ投与の日から少なくとも 52 週間のエミシズマブ治療まで (中央値 [範囲、最小-最大] 有効期間: 55.64 [8.6-89.9] 週間)
治療された自然出血の計算された年間出血率の中央値
時間枠：最初のエミシズマブ投与の日から少なくとも 52 週間のエミシズマブ治療まで (中央値 [範囲、最小-最大] 有効期間: 55.64 [8.6-89.9] 週間)
有効期間中の治療された自然出血の数は、次の式を使用して各参加者について年換算された計算された年換算出血率 (ABR) としてここに表示されます: ABR = (出血数/有効期間中の日数) x 365.25 . 治療された自然出血は、治療された出血（「出血の治療」と報告されている血友病の薬が直接続く出血）として定義され、外傷や処置/手術などの他の既知の要因はありません。 72 時間ルールが実装されました。最初の出血に対する最後の治療から 72 時間以内に 2 回目の出血が発生した場合、同じ種類の同じ解剖学的位置での 2 回の出血は 1 回の出血としてカウントされます。 手術/手順による出血は除外されました。
最初のエミシズマブ投与の日から少なくとも 52 週間のエミシズマブ治療まで (中央値 [範囲、最小-最大] 有効期間: 55.64 [8.6-89.9] 週間)
17週目にエミシズマブ嗜好調査を使用して評価された、エミシズマブSC治療、以前の血友病IV治療を好む、または好みがない参加者の割合
時間枠：17週目
Emicizumab Preference Survey は、皮下 (SC) エミシズマブ、静脈内 (IV) 第 VIII 因子 (FVIII) による治療に対する参加者の好みを記録するためにスポンサーによって開発された、目的に適したアンケートです。 95% 信頼区間は、ピアソン-クロッパー法を使用して計算されました。
17週目
17週目にエミシズマブ選好調査を使用して評価された、エミシズマブSC治療、子供の以前の血友病IV治療を好む、または好まない介護者の割合
時間枠：17週目
Emicizumab Preference Survey は、エミシズマブの皮下投与 (SC)、静脈内投与 (IV) の第 VIII 因子 (FVIII) による子供の治療に対する保護者の好みを記録するためにスポンサーによって開発された、目的に合った質問票です。 95% 信頼区間は、ピアソン-クロッパー法を使用して計算されました。
17週目
時間の経過に伴う 1 日の平均ピーク アクティビティ期間のベースラインからの変化
時間枠：ベースライン（1～2週）および13週（12～13週）、25週（24～25週）、37週（36～37週）、49週（48～49週）
身体活動の評価のために、5 歳以上の参加者は、研究中の指定された 2 週間、毎日 (24 時間/日) 手首に研究用加速度測定装置を装着するように指示されました。 参加者は、研究期間中、指定された 2 週間の各期間の少なくとも 8 日間、毎日継続的に (1 日 8 時間以上) 研究デバイスを着用した場合、身体活動評価に準拠していると見なされました。 この順守基準が特定の時点で達成されなかった場合、参加者は、順守が達成されなかった指定された 2 週間の分析に含まれませんでした。 毎日の測定値は、14 日間の時点で平均化されました。 活動カウントは、デバイスによって測定された加速度の尺度でした。 1 日のピーク活動時間は、1 日あたりの中等度から活発な活動の合計 (分単位) として定義されました。
ベースライン（1～2週）および13週（12～13週）、25週（24～25週）、37週（36～37週）、49週（48～49週）
1 日の平均歩数の経時的なベースラインからの変化
時間枠：ベースライン (1 ～ 2 週) および 13 週 (12 ～ 13 週)、25 (24 ～ 25 週)、37 (36 ～ 37 週)、および 49 (48 ～ 49 週)
身体活動の評価のために、5 歳以上の参加者は、研究中の指定された 2 週間、毎日 (24 時間/日) 手首に研究用加速度測定装置を装着するように指示されました。 参加者は、研究期間中、指定された 2 週間の各期間の少なくとも 8 日間、毎日継続的に (1 日 8 時間以上) 研究デバイスを着用した場合、身体活動評価に準拠していると見なされました。 この順守基準が特定の時点で達成されなかった場合、参加者は、順守が達成されなかった指定された 2 週間の分析に含まれませんでした。 毎日の測定値は、14 日間の時点で平均化されました。 活動カウントは、デバイスによって測定された加速度の尺度でした。
ベースライン (1 ～ 2 週) および 13 週 (12 ～ 13 週)、25 (24 ～ 25 週)、37 (36 ～ 37 週)、および 49 (48 ～ 49 週)
Model-Based ABR for All Bleeds
時間枠：From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks)
The number of all bleeds over the efficacy period was estimated as an ABR using a negative binomial regression model, which accounts for different follow-up times. In this outcome measure, all bleeds are included, irrespective of treatment with coagulation factors, with the following exception: bleeds due to surgery/procedure are excluded. As "all bleeds" comprises both treated and non-treated bleeds, the 72-hour rule was implemented separately for treated and non-treated bleeds. For treated bleeds, the 72-hour rule meant that two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed. For non-treated bleeds, the 72-hour rule was implemented by calculating a treatment-free period of 72 hours from the bleed itself.
From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks)
Mean Calculated ABR for All Bleeds
時間枠：From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks)
The number of all bleeds over the efficacy period is presented here as a calculated ABR that was annualized for each participant using the following formula: ABR = (number of bleeds/number of days during the efficacy period) x 365.25. In this outcome measure, all bleeds are included, irrespective of treatment with coagulation factors, with the following exception: bleeds due to surgery/procedure are excluded. As "all bleeds" comprises both treated and non-treated bleeds, the 72-hour rule was implemented separately for treated and non-treated bleeds. For treated bleeds, the 72-hour rule meant that two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed. For non-treated bleeds, the 72-hour rule was implemented by calculating a treatment-free period of 72 hours from the bleed itself.
From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks)
Median Calculated ABR for All Bleeds
時間枠：From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks)
The number of all bleeds over the efficacy period is presented here as a calculated ABR that was annualized for each participant using the following formula: ABR = (number of bleeds/number of days during the efficacy period) x 365.25. In this outcome measure, all bleeds are included, irrespective of treatment with coagulation factors, with the following exception: bleeds due to surgery/procedure are excluded. As "all bleeds" comprises both treated and non-treated bleeds, the 72-hour rule was implemented separately for treated and non-treated bleeds. For treated bleeds, the 72-hour rule meant that two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed. For non-treated bleeds, the 72-hour rule was implemented by calculating a treatment-free period of 72 hours from the bleed itself.
From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks)
Model-Based ABR for Treated Joint Bleeds
時間枠：From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks)
The number of treated joint bleeds over the efficacy period was estimated as an ABR using a negative binomial regression model, which accounts for different follow-up times. A treated joint bleed was defined as a bleed with type reported as "joint" based on at least one of the following symptoms: increasing swelling or warmth of the skin over the joint and/or increasing pain, decreased range of motion, or difficulty using the joint compared with baseline, and the bleed was directly followed by a hemophilia medication reported to be a "treatment for bleed". The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed. Bleeds due to surgery/procedure were excluded.
From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks)
Mean Calculated ABR for Treated Joint Bleeds
時間枠：From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks)
The number of treated joint bleeds over the efficacy period is presented here as a calculated ABR that was annualized for each participant using the following formula: ABR = (number of bleeds/number of days during the efficacy period) x 365.25. A treated joint bleed was defined as a bleed with type reported as "joint" based on at least one of the following symptoms: increasing swelling or warmth of the skin over the joint and/or increasing pain, decreased range of motion, or difficulty using the joint compared with baseline, and the bleed was directly followed by a hemophilia medication reported to be a "treatment for bleed". The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed. Bleeds due to surgery/procedure were excluded.
From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks)
Median Calculated ABR for Treated Joint Bleeds
時間枠：From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks)
The number of treated joint bleeds over the efficacy period is presented here as a calculated ABR that was annualized for each participant using the following formula: ABR = (number of bleeds/number of days during the efficacy period) x 365.25. A treated joint bleed was defined as a bleed with type reported as "joint" based on at least one of the following symptoms: increasing swelling or warmth of the skin over the joint and/or increasing pain, decreased range of motion, or difficulty using the joint compared with baseline, and the bleed was directly followed by a hemophilia medication reported to be a "treatment for bleed". The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed. Bleeds due to surgery/procedure were excluded.
From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks)
Model-Based ABR for Treated Target Joint Bleeds
時間枠：From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks)
The number of treated target joint bleeds over the efficacy period was estimated as an ABR using a negative binomial regression model, which accounts for different follow-up times. A treated target joint bleed was defined as a joint bleed in a target joint, which is a joint location where at least 3 bleeds have occurred over the last 24 weeks prior to study entry or an unresolved target joint (target joint that does not fulfil ≤2 bleeds into this joint within a consecutive 12-month period), and the bleed was directly followed by a hemophilia medication reported to be a "treatment for bleed". The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed. Bleeds due to surgery/procedure were excluded.
From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks)
Mean Calculated ABR for Treated Target Joint Bleeds
時間枠：From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks)
The number of treated target joint bleeds over the efficacy period is presented here as a calculated ABR that was annualized for each participant using the following formula: ABR = (number of bleeds/number of days during the efficacy period) x 365.25. A treated target joint bleed was defined as a joint bleed in a target joint, which is a joint location where at least 3 bleeds have occurred over the last 24 weeks prior to study entry or an unresolved target joint (target joint that does not fulfil ≤2 bleeds into this joint within a consecutive 12-month period), and the bleed was directly followed by a hemophilia medication reported to be a "treatment for bleed". The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed. Bleeds due to surgery/procedure were excluded.
From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks)
Median Calculated ABR for Treated Target Joint Bleeds
時間枠：From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks)
The number of treated target joint bleeds over the efficacy period is presented here as a calculated ABR that was annualized for each participant using the following formula: ABR = (number of bleeds/number of days during the efficacy period) x 365.25. A treated target joint bleed was defined as a joint bleed in a target joint, which is a joint location where at least 3 bleeds have occurred over the last 24 weeks prior to study entry or an unresolved target joint (target joint that does not fulfil ≤2 bleeds into this joint within a consecutive 12-month period), and the bleed was directly followed by a hemophilia medication reported to be a "treatment for bleed". The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed. Bleeds due to surgery/procedure were excluded.
From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks)
Model-Based ABR for Treated Spontaneous Bleeds
時間枠：From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks)
The number of treated spontaneous bleeds over the efficacy period was estimated as an ABR using a negative binomial regression model, which accounts for different follow-up times. A treated spontaneous bleed was defined as a treated bleed (bleed directly followed by a hemophilia medication reported to be a "treatment for bleed") with no other known contributing factor such as trauma or procedure/surgery. The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed. Bleeds due to surgery/procedure were excluded.
From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks)
Long-term Efficacy of Emicizumab: Model-Based ABR for Treated Bleeds
時間枠：Median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
The number of treated bleeds over the efficacy period was estimated as an ABR using a negative binomial regression model, which accounts for different follow-up times. A treated bleed was defined as a bleed that was directly followed by a hemophilia medication reported to be a "treatment for bleed". The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed. Bleeds due to surgery/procedure were excluded.
Median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
Long-term Efficacy of Emicizumab: Mean Calculated ABR for Treated Bleeds
時間枠：Median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
The number of treated bleeds over the efficacy period is presented here as a calculated ABR that was annualized for each participant using the following formula: ABR = (number of bleeds/number of days during the efficacy period) x 365.25. A treated bleed was defined as a bleed that was directly followed by a hemophilia medication reported to be a "treatment for bleed". The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed. Bleeds due to surgery/procedure were excluded.
Median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
Long-term Efficacy of Emicizumab: Median Calculated ABR for Treated Bleeds
時間枠：Median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
The number of treated bleeds over the efficacy period is presented here as a calculated ABR that was annualized for each participant using the following formula: ABR = (number of bleeds/number of days during the efficacy period) x 365.25. A treated bleed was defined as a bleed that was directly followed by a hemophilia medication reported to be a "treatment for bleed". The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed. Bleeds due to surgery/procedure were excluded.
Median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
Long-term Efficacy of Emicizumab: Model-Based ABR for All Bleeds
時間枠：median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
The number of all bleeds over the efficacy period was estimated as an ABR using a negative binomial regression model, which accounts for different follow-up times. In this outcome measure, all bleeds are included, irrespective of treatment with coagulation factors, with the following exception: bleeds due to surgery/procedure are excluded. As "all bleeds" comprises both treated and non-treated bleeds, the 72-hour rule was implemented separately for treated and non-treated bleeds. For treated bleeds, the 72-hour rule meant that two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed. For non-treated bleeds, the 72-hour rule was implemented by calculating a treatment-free period of 72 hours from the bleed itself.
median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
Long-term Efficacy of Emicizumab: Mean Calculated ABR for All Bleeds
時間枠：median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
The number of all bleeds over the efficacy period is presented here as a calculated ABR that was annualized for each participant using the following formula: ABR = (number of bleeds/number of days during the efficacy period) x 365.25. In this outcome measure, all bleeds are included, irrespective of treatment with coagulation factors, with the following exception: bleeds due to surgery/procedure are excluded. As "all bleeds" comprises both treated and non-treated bleeds, the 72-hour rule was implemented separately for treated and non-treated bleeds. For treated bleeds, the 72-hour rule meant that two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed. For non-treated bleeds, the 72-hour rule was implemented by calculating a treatment-free period of 72 hours from the bleed itself.
median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
Long-term Efficacy: Median Calculated ABR for All Bleeds
時間枠：median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
The number of all bleeds over the efficacy period is presented here as a calculated ABR that was annualized for each participant using the following formula: ABR = (number of bleeds/number of days during the efficacy period) x 365.25. In this outcome measure, all bleeds are included, irrespective of treatment with coagulation factors, with the following exception: bleeds due to surgery/procedure are excluded. As "all bleeds" comprises both treated and non-treated bleeds, the 72-hour rule was implemented separately for treated and non-treated bleeds. For treated bleeds, the 72-hour rule meant that two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed. For non-treated bleeds, the 72-hour rule was implemented by calculating a treatment-free period of 72 hours from the bleed itself.
median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
Long-term Efficacy of Emicizumab: Model-Based ABR for Treated Joint Bleeds
時間枠：median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
The number of treated joint bleeds over the efficacy period was estimated as an ABR using a negative binomial regression model, which accounts for different follow-up times. A treated joint bleed was defined as a bleed with type reported as "joint" based on at least one of the following symptoms: increasing swelling or warmth of the skin over the joint and/or increasing pain, decreased range of motion, or difficulty using the joint compared with baseline, and the bleed was directly followed by a hemophilia medication reported to be a "treatment for bleed". The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed. Bleeds due to surgery/procedure were excluded.
median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
Long-term Efficacy of Emicizumab: Mean Calculated ABR for Treated Joint Bleeds
時間枠：median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
The number of treated joint bleeds over the efficacy period is presented here as a calculated ABR that was annualized for each participant using the following formula: ABR = (number of bleeds/number of days during the efficacy period) x 365.25. A treated joint bleed was defined as a bleed with type reported as "joint" based on at least one of the following symptoms: increasing swelling or warmth of the skin over the joint and/or increasing pain, decreased range of motion, or difficulty using the joint compared with baseline, and the bleed was directly followed by a hemophilia medication reported to be a "treatment for bleed". The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed. Bleeds due to surgery/procedure were excluded.
median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
Long-term Efficacy of Emicizumab: Median Calculated ABR for Treated Joint Bleeds
時間枠：median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
The number of treated joint bleeds over the efficacy period is presented here as a calculated ABR that was annualized for each participant using the following formula: ABR = (number of bleeds/number of days during the efficacy period) x 365.25. A treated joint bleed was defined as a bleed with type reported as "joint" based on at least one of the following symptoms: increasing swelling or warmth of the skin over the joint and/or increasing pain, decreased range of motion, or difficulty using the joint compared with baseline, and the bleed was directly followed by a hemophilia medication reported to be a "treatment for bleed". The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed. Bleeds due to surgery/procedure were excluded.
median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
Long-term Efficacy of Emicizumab: Model-Based ABR for Treated Target Joint Bleeds
時間枠：median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
The number of treated target joint bleeds over the efficacy period was estimated as an ABR using a negative binomial regression model, which accounts for different follow-up times. A treated target joint bleed was defined as a joint bleed in a target joint, which is a joint location where at least 3 bleeds have occurred over the last 24 weeks prior to study entry or an unresolved target joint (target joint that does not fulfil ≤2 bleeds into this joint within a consecutive 12-month period), and the bleed was directly followed by a hemophilia medication reported to be a "treatment for bleed". The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed. Bleeds due to surgery/procedure were excluded.
median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
Long-term Efficacy of Emicizumab: Mean Calculated ABR for Treated Target Joint Bleeds
時間枠：median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
The number of treated target joint bleeds over the efficacy period is presented here as a calculated ABR that was annualized for each participant using the following formula: ABR = (number of bleeds/number of days during the efficacy period) x 365.25. A treated target joint bleed was defined as a joint bleed in a target joint, which is a joint location where at least 3 bleeds have occurred over the last 24 weeks prior to study entry or an unresolved target joint (target joint that does not fulfil ≤2 bleeds into this joint within a consecutive 12-month period), and the bleed was directly followed by a hemophilia medication reported to be a "treatment for bleed". The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed. Bleeds due to surgery/procedure were excluded.
median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
Long-term Efficacy of Emicizumab: Median Calculated ABR for Treated Target Joint Bleeds
時間枠：median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
The number of treated target joint bleeds over the efficacy period is presented here as a calculated ABR that was annualized for each participant using the following formula: ABR = (number of bleeds/number of days during the efficacy period) x 365.25. A treated target joint bleed was defined as a joint bleed in a target joint, which is a joint location where at least 3 bleeds have occurred over the last 24 weeks prior to study entry or an unresolved target joint (target joint that does not fulfil ≤2 bleeds into this joint within a consecutive 12-month period), and the bleed was directly followed by a hemophilia medication reported to be a "treatment for bleed". The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed. Bleeds due to surgery/procedure were excluded.
median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
Long-term Efficacy of Emicizumab: Model-Based ABR for Treated Spontaneous Bleeds
時間枠：median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
The number of treated spontaneous bleeds over the efficacy period was estimated as an ABR using a negative binomial regression model, which accounts for different follow-up times. A treated spontaneous bleed was defined as a treated bleed (bleed directly followed by a hemophilia medication reported to be a "treatment for bleed") with no other known contributing factor such as trauma or procedure/surgery. The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed. Bleeds due to surgery/procedure were excluded.
median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
Long-term Efficacy of Emicizumab: Mean Calculated ABR for Treated Spontaneous Bleeds
時間枠：median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
The number of treated spontaneous bleeds over the efficacy period is presented here as a calculated annualized bleed rate (ABR) that was annualized for each participant using the following formula: ABR = (number of bleeds/number of days during the efficacy period) x 365.25. A treated spontaneous bleed was defined as a treated bleed (bleed directly followed by a hemophilia medication reported to be a "treatment for bleed") with no other known contributing factor such as trauma or procedure/surgery. The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed. Bleeds due to surgery/procedure were excluded.
median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
Long-term Efficacy of Emicizumab: Median Calculated ABR for Treated Spontaneous Bleeds
時間枠：median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
The number of treated spontaneous bleeds over the efficacy period is presented here as a calculated annualized bleed rate (ABR) that was annualized for each participant using the following formula: ABR = (number of bleeds/number of days during the efficacy period) x 365.25. A treated spontaneous bleed was defined as a treated bleed (bleed directly followed by a hemophilia medication reported to be a "treatment for bleed") with no other known contributing factor such as trauma or procedure/surgery. The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed. Bleeds due to surgery/procedure were excluded.
median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
CATCH Questionnaire for Adult Participants: Change From Baseline in the Daily Activity Risk Perception (RP) and Impact Domain Scores Over Time
時間枠：Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169, 181 and Study completion (SC)/Early discontinuation (ED) (up to approximately 274 weeks)
Comprehensive Assessment Tool of Challenges in Hemophilia (CATCH) was used to measure the effect of hemophilia and its treatment on adult participants (aged ≥ 18 years). The adult version has 7 domains (daily activity RP and impact, social activity RP and impact, recreational activity RP and impact, work impact, preoccupation, treatment burden, and pain. Of this, the daily activity RP and impact domain has 48 items with each item scored on 4 or 5 points ordinal scales. Raw domain scores were obtained by calculating the mean of the item scores for all items within the corresponding concept and applying a linear transformation. The transformed scores range from 0-100 scale for both the RP and Impact. Higher scores indicating higher perceived risk of having a bleed while doing daily activities and higher impact of hemophilia on daily activities.
Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169, 181 and Study completion (SC)/Early discontinuation (ED) (up to approximately 274 weeks)
CATCH Questionnaire for Adult Participants: Change From Baseline in the Social Activity RP and Impact Domain Scores Over Time
時間枠：Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169, 181 and SC/ED (up to approximately 274 weeks)
CATCH was used to measure the effect of hemophilia and its treatment on adult participants (aged ≥ 18 years). The adult version has 7 domains (daily activity RP and impact, social activity RP and impact, recreational activity RP and impact, work impact, preoccupation, treatment burden, and pain. Of this, the social activity RP and impact domain has 22 items with each item scored on 4 or 5 points ordinal scales. Raw domain scores were obtained by calculating the mean of the item scores for all items within the corresponding concept and applying a linear transformation. The transformed scores range from 0-100 scale for both the RP and Impact. Higher scores indicating higher perceived risk of having a bleed while doing social activities and higher impact of hemophilia on social activities.
Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169, 181 and SC/ED (up to approximately 274 weeks)
CATCH Questionnaire for Adult Participants: Change From Baseline in the Recreational Activity RP and Impact Domain Scores Over Time
時間枠：Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169 and SC/ED (up to approximately 274 weeks)
CATCH was used to measure the effect of hemophilia and its treatment on adult participants (aged ≥ 18 years). The adult version has 7 domains (daily activity RP and impact, social activity RP and impact, recreational activity RP and impact, work impact, preoccupation, treatment burden, and pain. Of this, the recreational activity RP and impact domain has 32 items and a larger bank of additional items with each item scored on 4 or 5 points ordinal scales. Raw domain scores were obtained by calculating the mean of the item scores for all items within the corresponding concept and applying a linear transformation. The transformed scores range from 0-100 scale for both the RP and Impact. Higher scores indicating higher perceived risk of having a bleed while doing recreational activities and higher impact of hemophilia on recreational activities.
Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169 and SC/ED (up to approximately 274 weeks)
CATCH Questionnaire for Adult Participants: Change From Baseline in the Work Impact (WI) and College Impact (CI) Domains Score Over Time
時間枠：WI: Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169, 181 and SC/ED (up to approximately 274 weeks); CI: Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109, 121, 133, 145, and SC/ED (up to approximately 274 weeks)
CATCH was used to measure the effect of hemophilia and its treatment on adult participants (aged ≥ 18 years). The adult version has 7 domains (daily activity RP and impact, social activity RP and impact, recreational activity RP and impact, work impact, preoccupation, treatment burden, and pain. Of this, work and college impact has 10 items and the items were scored on 4 or 5 points ordinal scales. Raw domain scores were obtained by calculating the mean of the item scores for all items within the corresponding concept & applying a linear transformation. The transformed scores range from 0-100 scale. Higher scores indicated impact of hemophilia on work and college activities.
WI: Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169, 181 and SC/ED (up to approximately 274 weeks); CI: Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109, 121, 133, 145, and SC/ED (up to approximately 274 weeks)
CATCH Questionnaire for Adult Participants: Change From Baseline in the Preoccupation Domain Score Over Time
時間枠：Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169, 181 and SC/ED (up to approximately 274 weeks)
CATCH was used to measure the effect of hemophilia and its treatment on adult participants (aged ≥18 years). The adult version has 7 domains (daily activity RP and impact, social activity RP and impact, recreational activity RP and impact, work impact, preoccupation, treatment burden, and pain. Of this, preoccupation has 10 items which were scored on 4 or 5 points ordinal scales. Raw domain scores were obtained by calculating the mean of the item scores for all items within the corresponding concept & applying a linear transformation. The transformed scores range from 0-100 scale. Higher scores indicated greater preoccupation related to hemophilia.
Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169, 181 and SC/ED (up to approximately 274 weeks)
CATCH Questionnaire for Adult Participants: Change From Baseline in the Treatment Burden Domain Score Over Time
時間枠：Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169, 181 and SC/ED (up to approximately 274 weeks)
CATCH was used to measure the effect of hemophilia and its treatment on adult participants (aged ≥18 years). The adult version has 7 domains (daily activity RP and impact, social activity RP and impact, recreational activity RP and impact, work impact, preoccupation, treatment burden, and pain. Of this, treatment burden has 8 items which were scored on 4 or 5 points ordinal scales. Raw domain scores were obtained by calculating the mean of the item scores for all items within the corresponding concept & applying a linear transformation. The transformed scores range from 0-100 scale. Higher scores indicated greater perceived burden of the hemophilia treatment.
Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169, 181 and SC/ED (up to approximately 274 weeks)
CATCH Questionnaire for Adult Participants: Number of Participants by Responses to Their Level of Pain Associated With a Bleed Over Time
時間枠：Baseline, Weeks 25, 49, 61, 85, 121, 133, 157 and 169 and SC/ED (up to approximately 274 weeks)
CATCH was used to measure the effect of hemophilia and its treatment on adult participants (aged ≥18 years). The adult version has 7 domains (daily activity RP and impact, social activity RP and impact, recreational activity RP and impact, work impact, preoccupation, treatment burden, and pain. Of this, the pain domain consisting of five items, assessed using an 11-point numeric rating scale where 0 = no pain and 10 = pain as bad as you can imagine. The data reported here are from participants who answered the following question: "Did you get a bleed in the past 7 days?" If the response was yes, participants were asked to rate their pain by selecting the number that best described the pain associated with the bleed. Data is reported only for categories/timepoints with non-zero values.
Baseline, Weeks 25, 49, 61, 85, 121, 133, 157 and 169 and SC/ED (up to approximately 274 weeks)
CATCH Questionnaire for Adult Participants: Number of Participants by Responses to Their Level of Pain in Target Joints Over Time
時間枠：Baseline, Weeks 1, 13, 25, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169, 181 and SC/ED (up to approximately 274 weeks)
CATCH was used to measure the effect of hemophilia and its treatment on adult participants (aged ≥18 years). The adult version has 7 domains (daily activity RP and impact, social activity RP and impact, recreational activity RP and impact, work impact, preoccupation, treatment burden, and pain. Of this, the pain domain consists of five items, assessed using an 11-point numeric rating scale where 0 = no pain and 10 = pain as bad as you can imagine. The data reported here are from participants who answered the following question "Do you have target joints?" (If yes, please rate your pain by picking the number that best describes your pain in your target joint(s) over the past 7 days). Data is reported only for categories/timepoints with non-zero values.
Baseline, Weeks 1, 13, 25, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169, 181 and SC/ED (up to approximately 274 weeks)
CATCH Questionnaire for Adult Participants: Number of Participants by Responses to Their Level of Pain at Its Worst Over Time
時間枠：Baseline, Weeks 1, 13, 25, 37, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169, 181 and SC/ED (up to approximately 274 weeks)
CATCH was used to measure the effect of hemophilia and its treatment on adult participants (aged ≥18 years). The adult version has 7 domains (daily activity RP and impact, social activity RP and impact, recreational activity RP and impact, work impact, preoccupation, treatment burden, and pain. Of this, pain domain consists of five items, assessed using an 11-point numeric rating scale where 0 = no pain and 10 = pain as bad as you can imagine. The data reported here are from participants who answered the following question "Please rate your pain by picking the number that best describes your pain at its WORST over the past 7 days". Data is reported only for categories/timepoints with non-zero values.
Baseline, Weeks 1, 13, 25, 37, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169, 181 and SC/ED (up to approximately 274 weeks)
CATCH Questionnaire for Adult Participants: Number of Participants by Responses to Their Level of Pain at Its Least Over Time
時間枠：Baseline, Weeks 1, 13, 25, 37, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169, 181 and SC/ED (up to approximately 274 weeks)
CATCH was used to measure the effect of hemophilia and its treatment on adult participants (aged ≥18 years). The adult version has 7 domains (daily activity RP and impact, social activity RP and impact, recreational activity RP and impact, work impact, preoccupation, treatment burden, and pain. Of this, pain domain consists of five items, assessed using an 11-point numeric rating scale where 0 = no pain and 10 = pain as bad as you can imagine. The data reported here are from participants who answered the following question "Please rate your pain by picking the number that best describes your pain at its LEAST over the past 7 days." Data is reported only for categories/timepoints with non-zero values.
Baseline, Weeks 1, 13, 25, 37, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169, 181 and SC/ED (up to approximately 274 weeks)
CATCH Questionnaire for Adult Participants: Number of Participants by Responses to Their Level of Pain at Its Least on the Average Over Time
時間枠：Baseline, Weeks 1, 13, 25, 37, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169, 181 and SC/ED (up to approximately 274 weeks)
CATCH was used to measure the effect of hemophilia and its treatment on adult participants (aged ≥18 years). The adult version has 7 domains (daily activity RP and impact, social activity RP and impact, recreational activity RP and impact, work impact, preoccupation, treatment burden, and pain. Of this, the pain domain consists of five items, assessed using an 11-point numeric rating scale where 0 = no pain and 10 = pain as bad as you can imagine. The data reported here are from participants who answered the following question "Please rate your pain by picking the number that best describes your pain at its average over the past 7 days". Data is reported only for categories/timepoints with non-zero values.
Baseline, Weeks 1, 13, 25, 37, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169, 181 and SC/ED (up to approximately 274 weeks)
CATCH Questionnaire for Pediatric Participants: Change From Baseline in the Daily Activity RP and Impact Domain Score Over Time
時間枠：Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109,121, 133, 145, 157, 169 and SC/ED (up to approximately 274 weeks)
CATCH was used to measure the effect of hemophilia and its treatment on pediatric participants (aged 8-17 years). The pediatric version has 7 domains (daily activity RP and impact, social activity RP and impact, recreational activity RP and impact, school impact, preoccupation, treatment burden, and pain). Of this, daily activity RP and impact has 38 items with each item scored on 3 or 4 points ordinal scale. Raw domain scores were obtained by calculating the mean of the item scores for all items within the corresponding concept and applying a linear transformation. The transformed scores range from 0-100 scale. Higher scores indicating higher perceived risk of having a bleed while doing daily activities and higher impact of hemophilia on daily activities.
Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109,121, 133, 145, 157, 169 and SC/ED (up to approximately 274 weeks)
CATCH Questionnaire for Pediatric Participants: Change From Baseline in the Social Activity Risk Perception and Impact Domain Score Over Time
時間枠：Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169 and SC/ED (up to approximately 274 weeks)
CATCH was used to measure the effect of hemophilia and its treatment on pediatric participants (aged 8-17 years). The pediatric version has 7 domains (daily activity RP and impact, social activity RP and impact, recreational activity RP and impact, school impact, preoccupation, treatment burden, and pain). Of this, social activity RP and impact has 16 items which were scored on 3 or 4 points ordinal scale. Raw domain scores were obtained by calculating the mean of the item scores for all items within the corresponding concept & applying a linear transformation. The transformed scores range from 0-100 scale. Higher scores indicated higher perceived risk of having a bleed while doing social activities and higher impact of hemophilia on social activities.
Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169 and SC/ED (up to approximately 274 weeks)
CATCH Questionnaire for Pediatric Participants: Change From Baseline in the Recreational Activity RP and Impact Domain Score Over Time
時間枠：Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169 and SC/ED (up to approximately 274 weeks)
CATCH was used to measure the effect of hemophilia and its treatment on pediatric participants (aged 8-17 years). The pediatric version has 7 domains (daily activity RP and impact, social activity RP and impact, recreational activity RP and impact, school impact, preoccupation, treatment burden, and pain. Of this, recreational activity RP & impact has 34 items + larger bank of additional items. The items were scored on 3 or 4 points ordinal scales. Raw domain scores were obtained by calculating the mean of the item scores for all items within the corresponding concept & applying a linear transformation. The transformed scores range from 0-100 scale. Higher scores indicated higher perceived risk of having a bleed while doing recreational activities and higher impact of hemophilia on recreational activities.
Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169 and SC/ED (up to approximately 274 weeks)
CATCH Questionnaire for Pediatric Participants: Change From Baseline in the School Impact Domain Score Over Time
時間枠：Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109,121, 133, 145, 157, 169 and SC/ED (up to approximately 274 weeks)
CATCH was used to measure the effect of hemophilia and its treatment on pediatric participants (aged 8-17 years). The pediatric version has 7 domains (daily activity RP and impact, social activity RP and impact, recreational activity RP and impact, school impact, preoccupation, treatment burden, and pain. Of this, school impact has 11 items and the items were scored on 3 or 4 points ordinal scales. Raw domain scores were obtained by calculating the mean of the item scores for all items within the corresponding concept & applying a linear transformation. The transformed scores range from 0-100 scale. Higher scores indicated higher impact of hemophilia on school activities.
Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109,121, 133, 145, 157, 169 and SC/ED (up to approximately 274 weeks)
CATCH Questionnaire for Pediatric Participants: Change From Baseline in the Preoccupation Domain Score Over Time
時間枠：Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109,121, 133, 145, 157, 169 and SC/ED (up to approximately 274 weeks)
CATCH was used to measure the effect of hemophilia and its treatment on pediatric participants (aged 8-17 years). The pediatric version has 7 domains (daily activity RP and impact, social activity RP and impact, recreational activity RP and impact, school impact, preoccupation, treatment burden, and pain. Of this, preoccupation has 3 items which were scored on 3 or 4 points ordinal scales. Raw domain scores were obtained by calculating the mean of the item scores for all items within the corresponding concept & applying a linear transformation. The transformed scores range from 0-100 scale. Higher scores indicated greater preoccupation related to hemophilia.
Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109,121, 133, 145, 157, 169 and SC/ED (up to approximately 274 weeks)
CATCH Questionnaire for Pediatric Participants: Change From Baseline in the Treatment Burden Domain Score Over Time
時間枠：Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109,121, 133, 145, 157, 169 and SC/ED (up to approximately 274 weeks)
CATCH was used to measure the effect of hemophilia and its treatment on pediatric participants (aged 8-17 years). The pediatric version has 7 domains (daily activity RP and impact, social activity RP and impact, recreational activity RP and impact, school impact, preoccupation, treatment burden, and pain. Of this, treatment burden has 7 items which were scored on 3 or 4 points ordinal scales. Raw domain scores were obtained by calculating the mean of the item scores for all items within the corresponding concept & applying a linear transformation. The transformed scores range from 0-100 scale. Higher scores indicated lower perceived burden of the hemophilia treatment.
Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109,121, 133, 145, 157, 169 and SC/ED (up to approximately 274 weeks)
CATCH Questionnaire for Pediatric Participants: Number of Participants by Responses to Their Level of Hurt Associated With a Bleed Over Time
時間枠：Baseline, Weeks 13, 25, 37, 49, 61, 85, 97, 109, 121, 133, 145 and 157
CATCH was used to measure the effect of hemophilia and its treatment on pediatric participants (aged 8-17 years). The pediatric version has 7 domains (daily activity RP and impact, social activity RP and impact, recreational activity RP and impact, school impact, preoccupation, treatment burden, and pain). Of this, pain domain (Hurt) consists of two items, assessed using an 11-point numeric rating scale where 0 = no hurt and 10 = a lot of hurt. The data reported here are from participants who answered the following question "Did you get a bleed in the past 7 days? ( If yes, how much did the bleed hurt?)." Data is reported only for categories/timepoints with non-zero values.
Baseline, Weeks 13, 25, 37, 49, 61, 85, 97, 109, 121, 133, 145 and 157
CATCH Questionnaire for Pediatric Participants: Number of Participants by Responses to Their Level of Hurt at Its Worst Over Time
時間枠：Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109,121, 133, 145,157, 169 and SC/ED (up to approximately 274 weeks)
CATCH was used to measure the effect of hemophilia and its treatment on pediatric participants (aged 8-17 years). The pediatric version has 7 domains (daily activity RP and impact, social activity RP and impact, recreational activity RP and impact, school impact, preoccupation, treatment burden, and pain. Of this, pain domain (Hurt) consists of two items, assessed using an 11-point numeric rating scale where 0 = no hurt and 10 = a lot of hurt. The data reported here are from participants who answered the following question "What is the WORST that your body has hurt over the past 7 days". Data is reported only for categories/timepoints with non-zero values.
Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109,121, 133, 145,157, 169 and SC/ED (up to approximately 274 weeks)
CATCH Questionnaire for Caregivers: Change From Baseline in the Preoccupation Domain Score Over Time
時間枠：Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109,121, 133, 145, 157, 169 and SC/ED (up to approximately 274 weeks)
CATCH was used to measure the effect of hemophilia and its treatment on caregivers of pediatric participants (aged <8 years). The caregiver version has 2 domains (preoccupation and treatment burden). Of this, preoccupation has 13 items with each of the items were scored on 4 or 5 ordinal scales. Raw domain scores were obtained by calculating the mean of the item scores for all items within the corresponding concept and applying a linear transformation. The transformed scores range from 0-100 scale. Higher scores indicated greater preoccupation related to hemophilia.
Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109,121, 133, 145, 157, 169 and SC/ED (up to approximately 274 weeks)
CATCH Questionnaire for Caregivers: Change From Baseline in the Treatment Burden Domain Score Over Time
時間枠：Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109,121, 133, 145, 157, 169 and SC/ED (up to approximately 274 weeks)
CATCH was used to measure the effect of hemophilia and its treatment on caregivers of pediatric participants (aged <8 years). The caregiver version has 2 domains (preoccupation and treatment burden). Of this, treatment burden has 8 items which were scored on 4 or 5 ordinal scales. Raw domain scores were obtained by calculating the mean of the item scores for all items within the corresponding concept and applying a linear transformation. The transformed scores range from 0-100 scale. Higher scores indicated greater perceived burden of the hemophilia treatment.
Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109,121, 133, 145, 157, 169 and SC/ED (up to approximately 274 weeks)
Change From Baseline in Hemophilia Joint Health Scores Over Time
時間枠：Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109,121, 133, 145, 157, 169 and SC/ED (up to approximately 274 weeks)
The HJHS measures joint health of the joints (knees, ankles, and elbows) most commonly affected by bleeding in hemophilia. The HJHS 2.1 provides joint specific total scores which are added to obtain the sum of joints totals and also a global gait score. These two scores are then added to obtain HJHS total score. The minimum score per joint is 0, the maximum score is 20. In addition, Gait is scored on a scale from 0 to 4 based on the number of skills that are not within the normal limits. The total score is the sum of scores across all six joints plus the gait score (range from 0 to 124, with 0 being normal and 124 being the most severe disease). A higher score indicates worse joint health.
Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109,121, 133, 145, 157, 169 and SC/ED (up to approximately 274 weeks)
Menstrual Bleed Questionnaire (MBQ) for Female Participants of Childbearing Potential: Change From Baseline in the MBQ Total Score Over Time
時間枠：Baseline, Weeks 5, 9, 13, 17, 21, 25, 29, 33, 37, 41, 45, 49, 53, 57, 61, 65, 69, 73, 77, 81, 85, 89, 93, 97, 101, 104, 109, 113, 117, 121, 125, 129, 133, 137, 141 and145
The MBQ is a validated measure for menorrhagia and used to assess impact on the menstrual bleed-related heaviness, pain, irregularity, and quality of life (QoL). The MBQ is a 20-item measure covering four aspects regarding heavy menstrual bleeding: heaviness (8 items), QoL (8 items), irregularity (3 item), and pain (1 item). The MBQ score ranges from 0 to 75. Higher scores indicate a worse quality of life and more severe symptoms.
Baseline, Weeks 5, 9, 13, 17, 21, 25, 29, 33, 37, 41, 45, 49, 53, 57, 61, 65, 69, 73, 77, 81, 85, 89, 93, 97, 101, 104, 109, 113, 117, 121, 125, 129, 133, 137, 141 and145
MBQ for Female Participants of Childbearing Potential: Change From Baseline in the Heaviness Subscale Score Over Time
時間枠：Baseline, Weeks 5, 9, 13, 17, 21, 25, 29, 33, 37, 41, 45, 49, 53, 57, 61, 65, 69, 73, 77, 81, 89, 93, 97, 101, 104, 109, 113, 121, 125, 129, 133 and 137 (up to approximately 274 weeks)
The MBQ is a validated measure for menorrhagia and used to assess impact on the menstrual bleed-related heaviness, pain, irregularity, and quality of life (QoL). The MBQ is a 20-item measure covering four aspects regarding heavy menstrual bleeding: heaviness (8 items), QoL (8 items), irregularity (3 item), and pain (1 item). The total score for heaviness subscale ranges from 0 to 29. Higher score indicates more heavy bleeding.
Baseline, Weeks 5, 9, 13, 17, 21, 25, 29, 33, 37, 41, 45, 49, 53, 57, 61, 65, 69, 73, 77, 81, 89, 93, 97, 101, 104, 109, 113, 121, 125, 129, 133 and 137 (up to approximately 274 weeks)
MBQ for Female Participants of Childbearing Potential: Change From Baseline in the QoL Subscale Score Over Time
時間枠：Baseline, Weeks 5, 9, 13, 17, 21, 25, 29, 33, 37, 41, 45, 49, 53, 57, 61, 65, 69, 73, 77, 81, 85, 89, 93, 97, 101, 104, 109, 113, 117, 121, 125, 129, 133, 137, 141 and 145
The MBQ is a validated measure for menorrhagia and used to assess impact on the menstrual bleed-related heaviness, pain, irregularity, and quality of life (QoL). The MBQ is a 20-item measure covering four aspects regarding heavy menstrual bleeding: heaviness (8 items), QoL (8 items), irregularity (3 item), and pain (1 item). The total score for QoL subscale ranges from 0 to 37. Higher scores indicate a worse QoL.
Baseline, Weeks 5, 9, 13, 17, 21, 25, 29, 33, 37, 41, 45, 49, 53, 57, 61, 65, 69, 73, 77, 81, 85, 89, 93, 97, 101, 104, 109, 113, 117, 121, 125, 129, 133, 137, 141 and 145
MBQ for Female Participants of Childbearing Potential: Change From Baseline in the Irregularity Subscale Score Over Time
時間枠：Baseline, Weeks 5, 9, 13, 17, 21, 25, 29, 33, 37, 41, 45, 49, 53, 57, 61, 65, 69, 73, 77, 81, 85, 89, 93, 97, 101, 104, 109, 113, 117, 121, 125, 129, 133, 137, 141 and 145
The MBQ is a validated measure for menorrhagia and used to assess impact on the menstrual bleed-related heaviness, pain, irregularity, and quality of life (QoL). The MBQ is a 20-item measure covering four aspects regarding heavy menstrual bleeding: heaviness (8 items), QoL (8 items), irregularity (3 item), and pain (1 item). The total score for irregularity subscale ranges from 0 to 6. Higher score indicates more irregularity.
Baseline, Weeks 5, 9, 13, 17, 21, 25, 29, 33, 37, 41, 45, 49, 53, 57, 61, 65, 69, 73, 77, 81, 85, 89, 93, 97, 101, 104, 109, 113, 117, 121, 125, 129, 133, 137, 141 and 145
MBQ for Female Participants of Childbearing Potential: Change From Baseline in the Pain Subscale Score Over Time
時間枠：Baseline, Weeks 5, 9, 13, 17, 21, 25, 29, 33, 37, 41, 45, 49, 53, 57, 61, 65, 69, 73, 77, 81, 89, 93, 97, 101, 104, 109, 113, 121, 125, 129, 133 and 137
The MBQ is a validated measure for menorrhagia and used to assess impact on the menstrual bleed-related heaviness, pain, irregularity, and quality of life (QoL). The MBQ is a 20-item measure covering four aspects regarding heavy menstrual bleeding: heaviness (8 items), QoL (8 items), irregularity (3 item), and pain (1 item). The total score for pain subscale ranges from 0 to 3. Higher score indicates severe pain.
Baseline, Weeks 5, 9, 13, 17, 21, 25, 29, 33, 37, 41, 45, 49, 53, 57, 61, 65, 69, 73, 77, 81, 89, 93, 97, 101, 104, 109, 113, 121, 125, 129, 133 and 137
Menstruation Diary With the Pictorial Blood Assessment Chart (PBAC) for Female Participants of Childbearing Potential: PBAC Scores Over Time
時間枠：Baseline and monthly (on days of menstruation) until Study Completion (up to approximately 247 weeks)
The Menstruation Diary used a PBAC, which has shown good correlation with menstrual blood loss. The PBAC is a chart that records the occurrence and size of clots, and the number of episodes of heavy bleeding (flooding), as well as depicts the amount of blood loss during a cycle.The PBAC records pad and tampon use (as either light [1 point], medium [5 points], or heavy [10 points] flow), clots (small [1 point] or large [5 points]), and flooding episodes (1 point each) which can be recorded as many times as necessary any day of the month. The PBAC is scored from 0 (no bleeding) onwards, with a score of >100 defining abnormal coagulation and heavy menstrual bleeding (corresponds to >80ml of blood loss per menstrual cycle).
Baseline and monthly (on days of menstruation) until Study Completion (up to approximately 247 weeks)
Number of Participants With at Least One Adverse Event by Severity, According to the World Health Organization (WHO) Toxicity Grading Scale
時間枠：From enrollment until final completion date cutoff (median [range, min-max] observation period: 156.21 [24.1 - 274.0] weeks)
An AE was defined as any untoward medical occurrence in a participant administered a pharmaceutical product and which does not necessarily have to have a causal relationship with the treatment. An AE can therefore be any unfavorable and unintended sign (including abnormal laboratory values or abnormal clinical test results), symptoms, or disease temporally associated with the use of a pharmaceutical product, whether or not considered related to the pharmaceutical product. As per WHO toxicity grading, Grade 1= mild, 2= moderate, 3=severe, 4=life threatening.
From enrollment until final completion date cutoff (median [range, min-max] observation period: 156.21 [24.1 - 274.0] weeks)
Number of Participants With Adverse Events Leading to Study Drug Discontinuation
時間枠：From enrollment until final completion date cutoff (median [range, min-max] observation period: 156.21 [24.1 - 274.0] weeks)
An AE was defined as any untoward medical occurrence in a participant administered a pharmaceutical product and which does not necessarily have to have a causal relationship with the treatment. An AE can therefore be any unfavorable and unintended sign (including abnormal laboratory values or abnormal clinical test results), symptoms, or disease temporally associated with the use of a pharmaceutical product, whether or not considered related to the pharmaceutical product.
From enrollment until final completion date cutoff (median [range, min-max] observation period: 156.21 [24.1 - 274.0] weeks)
Number of Participants With at Least One Thromboembolic Event
時間枠：From enrollment until final completion date cutoff (median [range, min-max] observation period: 156.21 [24.1 - 274.0] weeks)
An AE was defined as any untoward medical occurrence in a participant administered a pharmaceutical product and which does not necessarily have to have a causal relationship with the treatment. An AE can therefore be any unfavorable and unintended sign (including abnormal laboratory values or abnormal clinical test results), symptoms, or disease temporally associated with the use of a pharmaceutical product, whether or not considered related to the pharmaceutical product. The following signs and symptoms were recognized as potential thromboembolism (i.e., dyspnea, chest pain, leg pain, or swelling; or if in the head, headache, numbness in the face, eye pain or swelling, or vision impairment).
From enrollment until final completion date cutoff (median [range, min-max] observation period: 156.21 [24.1 - 274.0] weeks)
Number of Participants With at Least One Event of Thrombotic Microangiopathy
時間枠：From enrollment until final completion date cutoff (median [range, min-max] observation period: 156.21 [24.1 - 274.0] weeks)
An AE was defined as any untoward medical occurrence in a participant administered a pharmaceutical product and which does not necessarily have to have a causal relationship with the treatment. An AE can therefore be any unfavorable and unintended sign (including abnormal laboratory values or abnormal clinical test results), symptoms, or disease temporally associated with the use of a pharmaceutical product, whether or not considered related to the pharmaceutical product. TMA is used to describe a group of disorders with clinical features of microangiopathic hemolytic anemia, thrombocytopenia, and organ damage that can include the kidneys, gastrointestinal system, central nervous system, etc.
From enrollment until final completion date cutoff (median [range, min-max] observation period: 156.21 [24.1 - 274.0] weeks)
Number of Participants With at Least One Injection-Site Reaction by Severity, According to the WHO Toxicity Grading Scale
時間枠：From enrollment until final completion date cutoff (median [range, min-max] observation period: 156.21 [24.1 - 274.0] weeks)
An AE was defined as any untoward medical occurrence in a participant administered a pharmaceutical product and which does not necessarily have to have a causal relationship with the treatment. An AE can therefore be any unfavorable and unintended sign (including abnormal laboratory values or abnormal clinical test results), symptoms, or disease temporally associated with the use of a pharmaceutical product, whether or not considered related to the pharmaceutical product. Injection-site reactions are defined as AEs related that occur within 24 hours after study drug administration and are judged to be related to the study drug injection. Local injection-site reactions included erythema, hematoma, rash, discomfort, pain, and pruritus and were mostly of mild and moderate intensity. As per WHO toxicity grading, Grade 1= mild, 2= moderate, 3=severe, 4=life threatening.
From enrollment until final completion date cutoff (median [range, min-max] observation period: 156.21 [24.1 - 274.0] weeks)
Number of Participants With at Least One Severe Hypersensitivity, Anaphylaxis, and Anaphylactoid Event
時間枠：From enrollment until final completion date cutoff (median [range, min-max] observation period: 156.21 [24.1 - 274.0] weeks)
An AE was defined as any untoward medical occurrence in a participant administered a pharmaceutical product and which does not necessarily have to have a causal relationship with the treatment. An AE can therefore be any unfavorable and unintended sign (including abnormal laboratory values or abnormal clinical test results), symptoms, or disease temporally associated with the use of a pharmaceutical product, whether or not considered related to the pharmaceutical product.
From enrollment until final completion date cutoff (median [range, min-max] observation period: 156.21 [24.1 - 274.0] weeks)
Number of Participants With at Least One Shifts in Clinical Laboratory Parameters From Baseline WHO Toxicity Scale Grade 0-2 to Post-baseline Grade 3 or 4
時間枠：Up to approximately 274 weeks
An abnormal laboratory value was defined as a laboratory test result outside of the normal range for hematology or serum chemistry parameters. The WHO toxicity grading scale, which ranges from Grades 1 to 4 (least severe to most severe, respectively; Grade 0 is within normal range), was used for assessing the severity of laboratory abnormalities and adverse events (WHO 2003). Not every laboratory abnormality qualified as an adverse event; an abnormality was reported as an adverse event if it met any of the following criteria: was accompanied by clinical symptoms; resulted in a change in study treatment (e.g., dosage modification, treatment interruption or discontinuation); resulted in a medical intervention or a change in concomitant therapy; or was clinically significant in the investigator's judgment.
Up to approximately 274 weeks
Number of Participants With at Least One Adverse Events of Changes From Baseline in Vital Signs
時間枠：Up to approximately 274 weeks
The number of participants with adverse events of changes from baseline in vital signs is reported here. Vital signs measurements consisted of heart and respiratory rate, temperature, and systolic and diastolic blood pressures, with an abnormal vital sign value being outside of the normal range. An abnormal vital sign result was reported as an adverse event if it met any of the following criteria: was accompanied by clinical symptoms; resulted in a change in study treatment (e.g., dosage modification, treatment interruption or discontinuation); resulted in a medical intervention or a change in concomitant therapy; or was clinically significant in the investigator's judgment. All of the adverse events reported here were assessed independently by the investigator as not related to treatment with emicizumab.
Up to approximately 274 weeks
Change From Baseline in Electrocardiogram (ECG) Parameters Over Time: QT, QTcB, QTcF, RR, PR, and QRS Intervals
時間枠：Baseline, Weeks 5, 25, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169, 181, SC/ED (up to approximately 274 weeks)
Baseline, Weeks 5, 25, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169, 181, SC/ED (up to approximately 274 weeks)
Change From Baseline in Heart Rate Over Time, as Measured by ECG
時間枠：Baseline, Weeks 5, 25, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169, 181, SC/ED (up to approximately 274 weeks)
Baseline, Weeks 5, 25, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169, 181, SC/ED (up to approximately 274 weeks)
Plasma Trough Concentration (Ctrough) of Emicizumab Over Time
時間枠：Pre-dose at Weeks 1, 2, 3, 4, 5, 9, 13, 17, 21, 25, 29, 33, 37, 41, 45, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169 and 181 (up to approximately 274 weeks)
Pre-dose at Weeks 1, 2, 3, 4, 5, 9, 13, 17, 21, 25, 29, 33, 37, 41, 45, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169 and 181 (up to approximately 274 weeks)
Number of Participants With Anti-Drug Antibodies Against Emicizumab at Baseline and Post-Baseline
時間枠：Baseline, Weeks 5, 13, 25, 33, 41, 49, 61, 73, 85, 97, 109, 121, 133, 145, 169, 181 and SC/ED (up to approximately 274 weeks)
Participants were considered to be ADA positive if they were ADA negative at baseline but develop an ADA response following study drug administration (treatment-induced ADA response), or if they were ADA positive at baseline and the titer of one or more post-baseline samples is at least 4-fold greater than the titer of the baseline sample (treatment-enhanced ADA response). The sum of all visits has been reported here.
Baseline, Weeks 5, 13, 25, 33, 41, 49, 61, 73, 85, 97, 109, 121, 133, 145, 169, 181 and SC/ED (up to approximately 274 weeks)
Number of Participants Who Develop Anti-FVIII Inhibitors Over Time
時間枠：Up to approximately 274 weeks
Central laboratory measurement of anti-FVIII inhibitory antibodies (inhibitors) was performed using a Chromogenic Bethesda Assay (CBA). Participants who develop anti-FVIII inhibitors (titer ≥ 0.6 Bethesda units per milliliter [BU/mL]) following study drug administration were summarized.
Up to approximately 274 weeks

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Hoffmann-La Roche

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  • スタディディレクター：Clinical Trials、Hoffmann-La Roche

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一般刊行物

研究記録日

これらの日付は、ClinicalTrials.gov への研究記録と要約結果の提出の進捗状況を追跡します。研究記録と報告された結果は、国立医学図書館 (NLM) によって審査され、公開 Web サイトに掲載される前に、特定の品質管理基準を満たしていることが確認されます。

主要日程の研究

研究開始 (実際)

2020年2月10日

一次修了 (実際)

2021年10月30日

研究の完了 (実際)

2025年12月19日

試験登録日

最初に提出

2019年11月7日

QC基準を満たした最初の提出物

2019年11月7日

最初の投稿 (実際)

2019年11月12日

学習記録の更新

投稿された最後の更新 (実際)

2026年8月27日

QC基準を満たした最後の更新が送信されました

2026年8月5日

最終確認日

2026年8月1日

詳しくは

本研究に関する用語

キーワード

追加の関連 MeSH 用語

その他の研究ID番号

  • BO41423
  • 2019 (米国 NIH グラント/契約：Chief Medical Office (CMO) Alberta Health Services)
  • 2023 (米国 NIH グラント/契約：GRAMMY Museum Foundation)
  • 2019-002179-32 (EudraCT番号)
  • 2023-506610-52-00 (レジストリ識別子：EU CT Number)

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医薬品およびデバイス情報、研究文書

米国FDA規制医薬品の研究

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米国FDA規制機器製品の研究

いいえ

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