CSL312_3003 遺伝性血管性浮腫を有する 2 歳から 11 歳の被験者における安全性および薬物動態研究
2歳から11歳の小児被験者における遺伝性血管性浮腫の予防的治療におけるCSL312（ガラダシマブ）の安全性、薬物動態、薬力学、および有効性を評価する第3相非盲検試験
調査の概要
研究の種類
入学 (実際)
段階
- フェーズ 3
連絡先と場所
研究場所
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Arizona
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Litchfield Park、Arizona、アメリカ、85340
- Research Solutions of Arizona
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Scottsdale、Arizona、アメリカ、85251
- Medical Research of Arizona
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California
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Orange、California、アメリカ、92868
- Donald S. Levy M.D.
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Santa Monica、California、アメリカ、90404
- Raffi Tachdjian MD, Inc.
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Ohio
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Cincinnati、Ohio、アメリカ、45236
- Bernstein Clinical Research
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Pennsylvania
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Hershey、Pennsylvania、アメリカ、17033
- PennState Health Milton S. Hershey Medical Center
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Texas
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Dallas、Texas、アメリカ、75231
- AARA Research Center
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Ashkelon、イスラエル、7830604
- Barzilai University Medical Center
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Campbelltown、オーストラリア、NSW 2560
- Campbelltown Hospital, Western Sydney University
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Ottawa、カナダ、K1H1E4
- Ottawa Allergy Research Corp
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Berlin、ドイツ、12203
- Charité - Universitätsmedizin Berlin
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Frankfurt am Main、ドイツ、60590
- Universitätsklinikum Frankfurt
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Hesse
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Frankfurt am Main、Hesse、ドイツ、60596
- HZRM Hämophilie Zentrum Rhein Main GmbH
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参加基準
適格基準
就学可能な年齢
- 子
健康ボランティアの受け入れ
説明
包含基準:
- 男性か女性
- -年齢に基づいて体重が10パーセンタイル以上の2歳から11歳までの年齢
- -臨床的に確認されたC1-INH HAEと診断されました
- -スクリーニング前の6か月間に2回以上のHAE攻撃を経験した
除外基準:
- 特発性または後天性血管性浮腫、蕁麻疹に関連する再発性血管性浮腫、またはHAEタイプIIIなどの別の形態の血管性浮腫の同時診断
- -臨床試験中に事前に計画された主要な手術または手順
- -C1-INH製品、アンドロゲン、抗線溶薬、承認済みまたは将来承認される医薬品、またはHAE攻撃に対する定期的な予防のためのその他の低分子医薬品の使用
- -別の介入臨床研究への参加
研究計画
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 |
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実験的：CSL312
2 ～ 5 歳および 6 ～ 11 歳には、特定の皮下投与スケジュールがあります。
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完全ヒト免疫グロブリン G サブクラス 4/ラムダ組換え阻害剤モノクローナル抗体を皮下投与 (SC)
他の名前：
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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 |
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Number of Participants With Treatment Emergent Adverse Events (TEAE)
時間枠：Up to Month 12
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Up to Month 12
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Percentage of Participants With TEAE
時間枠：Up to Month 12
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The percentage of participants was rounded to one place of decimal.
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Up to Month 12
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Number of TEAE
時間枠：Up to Month 12
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Up to Month 12
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TEAE Rates Per Injection
時間枠：Up to Month 12
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The TEAE rate per injection was calculated as the number of TEAE/ number of injections.
The number of injections was defined as the total injections a participant received during the Safety Evaluation Period under the dosing regimen to which the TEAE was assigned.
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Up to Month 12
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TEAE Rates Per Participant-Year
時間枠：Up to Month 12
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The TEAE rate per participant year was calculated as number of TEAEs/ participant years.
Participant-years of exposure were calculated as the sum of each participant's exposure duration (in years) under the specified dosing regimen or overall.
For the time assigned to a dosing regimen, each study day was counted under the corresponding regimen.
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Up to Month 12
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Maximum Concentration (Cmax) of CSL312 at Steady-state
時間枠：Up to Month 12
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Up to Month 12
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Trough Concentration (Ctrough) of CSL312 at Steady-state
時間枠：At Months 3, 4, 6, 9, 10, and 12
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At Months 3, 4, 6, 9, 10, and 12
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Time to Maximum Concentration (Tmax) of CSL312 at Steady-State
時間枠：Up to Month 12
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Up to Month 12
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二次結果の測定
結果測定 |
メジャーの説明 |
時間枠 |
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Time-normalized Number of HAE Attacks Per Month
時間枠：Up to Month 12
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Time-normalized number of HAE attacks per month during treatment was calculated per participant as: [Number of HAE attacks / Length of participant treatment in days] * 30.4375.
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Up to Month 12
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Time-normalized Number of HAE Attacks Per Year
時間枠：Up to Month 12
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Time-normalized number of HAE attacks per year during treatment was calculated per participant as: [Number of HAE attacks / Length of participant treatment in days] * 365.25.
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Up to Month 12
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Time-normalized Number of HAE Attacks Treated With On-demand Treatment Per Month
時間枠：Up to Month 12
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The time-normalized number of HAE attacks per month treated with on-demand treatment were calculated as follows: [(Number of HAE attacks treated with on - demand treatment during treatment period)/ Length of participant treatment in days] ∗ 30.4375. |
Up to Month 12
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Time-normalized Number of HAE Attacks Treated With On-demand Treatment Per Year
時間枠：Up to Month 12
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The time-normalized number of HAE attacks per year treated with on-demand treatment were calculated as follows: [(Number of HAE attacks treated with on - demand treatment during treatment period)/ Length of participant treatment in days] ∗ 365.25. |
Up to Month 12
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Time-normalized Number of Moderate and/or Severe HAE Attacks Per Month
時間枠：Up to Month 12
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Time-normalized number of moderate or severe HAE attacks per month during treatment period was calculated per participant as: [number of moderate or severe HAE attacks / length of participant treatment in days] * 30.4375.
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Up to Month 12
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Time-normalized Number of Moderate and/or Severe HAE Attacks Per Year
時間枠：Up to Month 12
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Time-normalized number of moderate or severe HAE attacks per month during treatment period was calculated per participant as: [number of moderate or severe HAE attacks / length of participant treatment in days] * 365.25.
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Up to Month 12
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Percentage Reduction in the Time-normalized Number of HAE Attacks
時間枠：Up to Month 12
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The percentage reduction in the time-normalized number of HAE attacks was calculated within a participant as follows: 100*[ 1 - (Time-normalized number of HAE attacks per month during treatment period/Time-normalized number of HAE attacks per month from historical data)].
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Up to Month 12
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Number of Participants Experiencing at Least Greater Than or Equal to (>=) 50 Percent (%), >= 70%, >= 90%, or Equal to 100% (Attack-free) Reduction in the Time-normalized Number of HAE Attacks
時間枠：Up to Month 12
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A participant was classified as a responder if the percentage reduction in the time-normalized number of HAE attacks under treatment compared to the time-normalized number of HAE attacks documented in the medical records was >= 50%.
Percent Reduction = 100 * [1 - (time-normalized number of HAE attacks during corresponding time window / time-normalized number of HAE attacks based on historical data)].
Here number of participants experiencing at least >= 50%, >= 70%, >= 90%, or equal to 100% (Attack-free) reduction in the time-normalized number of HAE attacks are reported.
The number of responders at each reduction category have been reported.
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Up to Month 12
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Number of Participants Experiencing Serious Adverse Events (SAE), Experiencing Death, Related TEAE, TEAE Leading to Study Discontinuation
時間枠：Up to Month 12
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Up to Month 12
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Percentage of Participants Experiencing SAE, Experiencing Death, Related TEAE, TEAE Leading to Study Discontinuation
時間枠：Up to Month 12
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The percentage of participants was rounded to one decimal place.
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Up to Month 12
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Number of Participants With TEAE by Severity
時間枠：Up to Month 12
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Severity of AE was assessed by the investigator and categorized as mild, moderate and severe where: Mild: AE that is usually transient and may require only minimal treatment or therapeutic intervention. The event does not generally interfere with usual activities of daily living. Moderate: AE that is usually alleviated with additional specific therapeutic intervention. The event interferes with usual activities of daily living, causing discomfort but poses no significant or permanent risk of harm to the research participant. Severe: AE that interrupts usual activities of daily living, significantly affects clinical status, or may require intensive therapeutic intervention. |
Up to Month 12
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Percentage of Participants With TEAE by Severity
時間枠：Up to Month 12
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Severity of AE was assessed by the investigator and categorized as mild, moderate and severe where: Mild: AE that is usually transient and may require only minimal treatment or therapeutic intervention. The event does not generally interfere with usual activities of daily living. Moderate: AE that is usually alleviated with additional specific therapeutic intervention. The event interferes with usual activities of daily living, causing discomfort but poses no significant or permanent risk of harm to the research participant. Severe: AE that interrupts usual activities of daily living, significantly affects clinical status, or may require intensive therapeutic intervention. The percentage of participants was rounded to one place of decimal. |
Up to Month 12
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Number of Participants With Anti-CSL312 Antibodies
時間枠：At Day 1, Months 6 and 12
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At Day 1, Months 6 and 12
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Percentage of Participants With Anti-CSL312 Antibodies
時間枠：At Day 1, Months 6 and 12
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The percentage of participants was rounded to one place of decimal.
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At Day 1, Months 6 and 12
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Number of Participants With Adverse Events of Special Interest (AESI)
時間枠：Up to Month 12
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AESI included severe hypersensitivity including anaphylaxis.
The AESI reported have been identified by investigators and suggestive events were independently identified for further review with a Standardized MedDRA Query (SMQ).
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Up to Month 12
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Percentage of Participants With AESI
時間枠：Up to Month 12
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AESI included severe hypersensitivity including anaphylaxis.
The AESI reported have been identified by investigators and suggestive events were independently identified for further review with an SMQ.
The percentage of participants was rounded to one place of decimal.
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Up to Month 12
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FXIIa-mediated Kallikrein Activity
時間枠：At Months 3, 4, and 12 and pre-dose and post dose at Months 6, 9, and 10
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At Months 3, 4, and 12 and pre-dose and post dose at Months 6, 9, and 10
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Percent of Baseline FXIIa-mediated Kallikrein Activity
時間枠：At Months 3, 4, and 12 and pre-dose and post dose at Months 6, 9, and 10
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Percent of Baseline at Visit [i] = 100 * (actual value at Visit [i] / Baseline value), where Baseline is defined as the most recent, non-missing value before the first IP administration (including unscheduled visits).
Here unit of measure is Percent (%) of FXIIa-mediated Kallikrein Activity.
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At Months 3, 4, and 12 and pre-dose and post dose at Months 6, 9, and 10
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Number of Participants With Laboratory Findings Reported as AE
時間枠：Up to Month 12
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Up to Month 12
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Percentage of Participants With Laboratory Findings Reported as AE
時間枠：Up to Month 12
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The participant data were rounded to one decimal place.
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Up to Month 12
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協力者と研究者
スポンサー
捜査官
- スタディディレクター：Study Director、CSL Behring
研究記録日
主要日程の研究
研究開始 (実際)
一次修了 (実際)
研究の完了 (実際)
試験登録日
最初に提出
QC基準を満たした最初の提出物
最初の投稿 (実際)
学習記録の更新
投稿された最後の更新 (実際)
QC基準を満たした最後の更新が送信されました
最終確認日
詳しくは
本研究に関する用語
追加の関連 MeSH 用語
その他の研究ID番号
- CSL312_3003
- 2022-502386-13-00 (Ctis)
個々の参加者データ (IPD) の計画
個々の参加者データ (IPD) を共有する予定はありますか?
IPD プランの説明
CSL は、システマティック レビュー グループまたは真正な研究者からの個々の患者データ (IPD) の共有要求を検討します。 IPD の任意のデータ共有リクエストを送信するプロセスと要件については、CSL (clinicaltrials@cslbehring.com) までお問い合わせください。
該当する国固有のプライバシーおよびその他の法律および規制が考慮され、IPD の共有が妨げられる場合があります。
リクエストが承認され、研究者が適切なデータ共有契約を締結した場合、適切に匿名化された IPD が利用可能になります。
IPD 共有時間枠
IPD 共有アクセス基準
要求は、IPD の提案された使用が本質的に非営利であり、内部審査委員会によって承認された系統的審査グループまたは善意の研究者によってのみ行うことができます。
IPD 要求は、CSL の内部審査委員会によって決定されたように、提案されたリサーチ クエスチョンが重要かつ未知の医学または患者ケアのクエスチョンに答えようとしない限り、CSL によって考慮されません。
要求側は、IPD が利用可能になる前に、適切なデータ共有契約を締結する必要があります。
IPD 共有サポート情報タイプ
- STUDY_PROTOCOL
- SAP
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
米国FDA規制機器製品の研究
この情報は、Web サイト clinicaltrials.gov から変更なしで直接取得したものです。研究の詳細を変更、削除、または更新するリクエストがある場合は、register@clinicaltrials.gov。 までご連絡ください。 clinicaltrials.gov に変更が加えられるとすぐに、ウェブサイトでも自動的に更新されます。