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遺伝性血管性浮腫タイプIまたはタイプIIの患者における血管性浮腫発作に対する予防のための経口PHA-022121の用量範囲研究 (HAE CHAPTER-1)

2026年8月12日 更新者：Pharvaris Netherlands B.V.

C1阻害剤欠乏による遺伝性血管性浮腫患者の血管性浮腫発作に対する予防のために経口投与されたPHA-022121の安全性と有効性を評価するための第II相、二重盲検、プラセボ対照、無作為化、用量範囲設定、並行群間試験 (タイプIまたはタイプII）

この研究では、遺伝性血管性浮腫 (HAE) 患者の血管性浮腫発作に対する予防のために経口投与された PHA-022121 の安全性と有効性を評価します。 この試験は 2 つのパートで構成され、患者はパート 1 への参加を完了してからパート 2 の治療を開始します。試験のパート 1 には 3 つの並行アームがあり、約 30 人の患者が PHA-022121 の 2 つの投与計画のいずれかに均等に無作為化されます。または一致するプラセボ。 患者は、パート1の完了後、研究のパート2の単一の非盲検アームに進みます。 スクリーニング期間は最大 8 週間で、治療期間は 12 週間 (パート 1) と 50 週間 (パート 2) です。

調査の概要

状態

終了しました

条件

介入・治療

研究の種類

介入

入学 (実際)

34

段階

  • フェーズ2

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

  • アイルランド
      • Dublin、アイルランド、D08NHY1
        • Study Site
  • アメリカ
    • Alabama
      • Birmingham、Alabama、アメリカ、35209
        • Study Site
    • Arizona
      • Paradise Valley、Arizona、アメリカ、85253
        • Study Site
    • Missouri
      • St Louis、Missouri、アメリカ、63141
        • Study Site
  • イギリス
    • England
      • Brighton、England、イギリス、BN2 1ES
        • Study Site
      • Bristol、England、イギリス、BS10 5NB
        • Study Site
      • Cambridge、England、イギリス、CB2 0QQ
        • Study Site
      • London、England、イギリス、E1 1FR
        • Study Site
      • Southampton、England、イギリス、SO16 6YD
        • Study Site
  • イタリア
      • Milan、イタリア、20157
        • Study Site
      • Palermo、イタリア、0146
        • Study Site
    • PD
      • Padua、PD、イタリア、35128
        • Study Site
  • オーストリア
      • Vienna、オーストリア、A-1090
        • Study Site
  • カナダ
    • Ontario
      • Ottawa、Ontario、カナダ、K1H 1E4
        • Study Site
    • Quebec
      • Montreal、Quebec、カナダ、H2W 1R7
        • Study Site
  • ドイツ
      • Berlin、ドイツ、10117
        • Study Site
      • Frankfurt、ドイツ、60323
        • Study Site
  • ブルガリア
      • Sofia、ブルガリア、1680
        • Study Site
  • ポーランド
      • Krakow、ポーランド
        • Study Site

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

18年～75年 (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:

  • 署名および日付入りのインフォームド コンセント フォーム
  • HAEタイプIまたはIIの診断
  • -スクリーニング前の過去3か月連続で少なくとも3回のHAE攻撃の記録された履歴、またはスクリーニング期間中の最低2回のHAE攻撃
  • 標準治療の急性発作治療薬を使用するための信頼できるアクセスと経験

除外基準:

  • 妊娠中または授乳中
  • 臨床的に重大な心電図異常
  • -患者の安全性または研究に参加する能力を妨げる他の全身性疾患または重大な疾患または障害
  • -登録前の定義された期間内のC1-エステラーゼ阻害剤、経口カリクレイン阻害剤、弱毒化アンドロゲン、抗線溶薬、またはモノクローナルHAE療法の使用
  • 肝機能異常
  • 腎機能異常
  • 定義された期間内のアルコールまたは薬物乱用の履歴、または物質依存または乱用の現在の証拠
  • -定義された期間内の他の治験薬研究への参加

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：防止
  • 割り当て：ランダム化
  • 介入モデル：並列代入
  • マスキング：4倍

武器と介入

参加者グループ / アーム
介入・治療
プラセボコンパレーター：パート 1: プラセボ
BID プラセボ
薬：プラセボ
経口使用のための一致するプラセボ カプセル
実験的：パート 1: 低用量
BID 低用量の緩和剤
薬：低用量の解糖剤
経口用Deucrictibantソフトジェルカプセル（PHVS416）
実験的：パート 1: 高用量
BID 高用量の緩和剤
薬：緩和剤の高用量
経口用Deucrictibantソフトジェルカプセル（PHVS416）
実験的：パート 2: オープンラベル
BID 高用量の緩和剤
薬：緩和剤の高用量
経口用Deucrictibantソフトジェルカプセル（PHVS416）

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
Number of Investigator-confirmed Hereditary Angioedema (HAE) Attacks (Expressed as the Normalized Number of Attacks Per Month [4 Weeks] of Exposure) During the Treatment Period in Part 1
時間枠：Up to Day 84
HAE attack was defined as signs or symptoms in ≥1 location: peripheral angioedema (cutaneous swelling involving an extremity, face, neck, torso, and/or genitourinary region), abdominal angioedema (abdominal pain with or without abdominal distension, nausea, vomiting, or diarrhea), or upper airway angioedema (lump sensation, difficulty swallowing, throat tightening, difficulty speaking, hoarseness, stridor, dyspnea, or swelling of tongue, palate, uvula, or larynx). A new attack was counted if symptoms occurred ≥24 hours after complete resolution of a prior attack. Attacks were recorded via daily electronic patient-reported outcome (ePRO) diaries and confirmed by the investigator. Monthly attack rate was calculated as number of attacks during treatment divided by treatment days, multiplied by 28. Mean rate ratio, confidence interval (CI), and p-value were estimated using a generalized linear model with Poisson distribution, with baseline attack rate included as a factor.
Up to Day 84

二次結果の測定

結果測定
メジャーの説明
時間枠
Number of Investigator-confirmed Moderate or Severe HAE Attacks During the Treatment Period in Part 1
時間枠：Up to Day 84
HAE attack was defined as signs or symptoms in ≥1 location: peripheral angioedema (cutaneous swelling involving an extremity, face, neck, torso, and/or genitourinary region), abdominal angioedema (abdominal pain with or without abdominal distension, nausea, vomiting, or diarrhea), or upper airway angioedema (lump sensation, difficulty swallowing, throat tightening, difficulty speaking, hoarseness, stridor, dyspnea, or swelling of tongue, palate, uvula, or larynx). A new attack was counted if symptoms occurred ≥24 hours after complete resolution of a prior attack. Attacks were recorded via daily ePRO diaries and confirmed by the investigator. Monthly attack rate was calculated as number of attacks during treatment divided by treatment days×28. Moderate HAE attack defined as which limits/interferes with the participant's ability to attend work/school or participate in family life and social/recreational activities whereas Severe HAE attack significantly limits the participant's ability.
Up to Day 84
Number of Investigator-confirmed HAE Attacks Requiring Acute Treatment During the Treatment Period in Part 1
時間枠：Up to Day 84
HAE attack was defined as signs or symptoms in ≥1 location: peripheral angioedema (cutaneous swelling involving an extremity, face, neck, torso, and/or genitourinary region), abdominal angioedema (abdominal pain with or without abdominal distension, nausea, vomiting, or diarrhea), or upper airway angioedema (lump sensation, difficulty swallowing, throat tightening, difficulty speaking, hoarseness, stridor, dyspnea, or swelling of tongue, palate, uvula, or larynx). A new attack was counted if symptoms occurred ≥24 hours after complete resolution of a prior attack. Attacks were recorded via daily ePRO diaries and confirmed by the investigator. Monthly attack rate was calculated as number of attacks during treatment divided by treatment days, multiplied by 28.
Up to Day 84
Number of Participants Achieving ≥50%, ≥70% and ≥90% Reduction in Attack Rate Relative to Baseline During the Treatment Period in Part 1
時間枠：Up to Day 84
Baseline attack rate was defined as each participant's normalized HAE attack rate (attacks per 4 weeks) prior to randomization, based on documented history or confirmed during screening. Monthly attack rate was calculated as number of attacks during treatment divided by treatment days, multiplied by 28. Percent reduction from baseline was calculated for each participant by comparing the normalized treatment-period attack rate to the baseline attack rate. Participants were classified as responders if they achieved ≥50%, ≥70%, or ≥90% reduction in attack rate relative to baseline during Part 1.
Up to Day 84
Number of Participants Who Were Investigator-confirmed Attack-free During the Treatment Period in Part 1
時間枠：Up to Day 84
HAE attack was defined as signs or symptoms in ≥1 location: peripheral angioedema (cutaneous swelling involving an extremity, face, neck, torso, and/or genitourinary region), abdominal angioedema (abdominal pain with or without abdominal distension, nausea, vomiting, or diarrhea), or upper airway angioedema (lump sensation, difficulty swallowing, throat tightening, difficulty speaking, hoarseness, stridor, dyspnea, or swelling of tongue, palate, uvula, or larynx). A new attack was counted if symptoms occurred ≥24 hours after complete resolution of a prior attack. Attacks were recorded via daily ePRO diaries and confirmed by the investigator. Participants reporting no confirmed attacks over the treatment period were considered attack-free.
Up to Day 84
Number of Days With Investigator-confirmed HAE Symptoms During the Treatment Period in Part 1
時間枠：Up to Day 84
HAE attack was defined as signs or symptoms in ≥1 location: peripheral angioedema (cutaneous swelling involving an extremity, face, neck, torso, and/or genitourinary region), abdominal angioedema (abdominal pain with or without abdominal distension, nausea, vomiting, or diarrhea), or upper airway angioedema (lump sensation, difficulty swallowing, throat tightening, difficulty speaking, hoarseness, stridor, dyspnea, or swelling of tongue, palate, uvula, or larynx). HAE symptom days were identified from daily participant entries in the ePRO diary and were confirmed by the investigator.
Up to Day 84
Proportion of Days With Investigator-confirmed HAE Symptoms During the Treatment Period in Part 1
時間枠：Up to Day 84
HAE attack was defined as signs or symptoms in ≥1 location: peripheral angioedema (cutaneous swelling involving an extremity, face, neck, torso, and/or genitourinary region), abdominal angioedema (abdominal pain with or without abdominal distension, nausea, vomiting, or diarrhea), or upper airway angioedema (lump sensation, difficulty swallowing, throat tightening, difficulty speaking, hoarseness, stridor, dyspnea, or swelling of tongue, palate, uvula, or larynx). HAE symptom days were identified from daily participant entries in the ePRO diary and were confirmed by the investigator.
Up to Day 84
Time to First Investigator-confirmed HAE Attack in the Treatment Period in Part 1
時間枠：Up to Day 84
Time to first investigator-confirmed HAE attack was defined as days from date of first administration of study drug until the onset date of first Investigator-confirmed HAE attack. Participants without an Investigator-confirmed HAE attack during the treatment period in Part 1 were censored at the date of the last dose of study drug +1 in Part 1 or last day of the study, whichever came sooner.
Up to Day 84
Number of Investigator-confirmed HAE Attacks Resulting in a Visit to the Emergency Department or an Admission to Hospital During the Treatment Period in Part 1
時間枠：Up to Day 84
HAE attack was defined as signs or symptoms in ≥1 location: peripheral angioedema (cutaneous swelling involving an extremity, face, neck, torso, and/or genitourinary region), abdominal angioedema (abdominal pain with or without abdominal distension, nausea, vomiting, or diarrhea), or upper airway angioedema (lump sensation, difficulty swallowing, throat tightening, difficulty speaking, hoarseness, stridor, dyspnea, or swelling of tongue, palate, uvula, or larynx). A new attack was counted if symptoms occurred ≥24 hours after complete resolution of a prior attack. Attacks were recorded via daily ePRO diaries and confirmed by the investigator. Monthly attack rate was calculated as number of attacks during treatment divided by treatment days, multiplied by 28. Only Investigator-confirmed attacks resulting in emergency department visits or hospital admissions were included.
Up to Day 84
Number of Investigator-confirmed HAE Attacks During the Treatment Period in Part 2
時間枠：Day 84 to Day 938
HAE attack was defined as signs or symptoms in ≥1 location: peripheral angioedema (cutaneous swelling involving an extremity, face, neck, torso, and/or genitourinary region), abdominal angioedema (abdominal pain with or without abdominal distension, nausea, vomiting, or diarrhea), or upper airway angioedema (lump sensation, difficulty swallowing, throat tightening, difficulty speaking, hoarseness, stridor, dyspnea, or swelling of tongue, palate, uvula, or larynx). A new attack was counted if symptoms occurred ≥24 hours after complete resolution of a prior attack. Attacks were recorded via daily ePRO diaries and confirmed by the investigator. Monthly attack rate was calculated as number of attacks during treatment divided by treatment days, multiplied by 28. An attack will be counted as in Part 2 if it occurs after the first IMP administration in Part 2 and within 24 hours after last IMP administration in Part 2.
Day 84 to Day 938
Number of Investigator-confirmed Moderate or Severe HAE Attacks During the Treatment Period in Part 2
時間枠：Day 84 to Day 938
HAE attack was defined as signs or symptoms in ≥1 location: peripheral angioedema (cutaneous swelling involving an extremity, face, neck, torso, and/or genitourinary region), abdominal angioedema (abdominal pain with or without abdominal distension, nausea, vomiting, or diarrhea), or upper airway angioedema (lump sensation, difficulty swallowing, throat tightening, difficulty speaking, hoarseness, stridor, dyspnea, or swelling of tongue, palate, uvula, or larynx). Attacks were recorded via daily ePRO diaries and confirmed by the investigator. Monthly attack rate was calculated as number of attacks during treatment divided by treatment days×28. Moderate HAE attack defined as which limits/interferes with the participant's ability to attend work/school or participate in family life and social/recreational activities whereas Severe HAE attack significantly limits the participant's ability. LSM and SE were estimated using a generalized linear model with Poisson distribution.
Day 84 to Day 938
Number of Investigator-confirmed HAE Attacks Requiring Acute Treatment During the Treatment Period in Part 2
時間枠：Day 84 to Day 938
HAE attack was defined as signs or symptoms in ≥1 location: peripheral angioedema (cutaneous swelling involving an extremity, face, neck, torso, and/or genitourinary region), abdominal angioedema (abdominal pain with or without abdominal distension, nausea, vomiting, or diarrhea), or upper airway angioedema (lump sensation, difficulty swallowing, throat tightening, difficulty speaking, hoarseness, stridor, dyspnea, or swelling of tongue, palate, uvula, or larynx). A new attack was counted if symptoms occurred ≥24 hours after complete resolution of a prior attack. Attacks were recorded via daily ePRO diaries and confirmed by the investigator. Monthly attack rate was calculated as number of attacks during treatment divided by treatment days, multiplied by 28. LSM and SE were estimated using a generalized linear model with Poisson distribution.
Day 84 to Day 938
Number of Days With Investigator-confirmed HAE Symptoms During the Treatment Period in Part 2
時間枠：Day 84 to Day 938
HAE attack was defined as signs or symptoms in ≥1 location: peripheral angioedema (cutaneous swelling involving an extremity, face, neck, torso, and/or genitourinary region), abdominal angioedema (abdominal pain with or without abdominal distension, nausea, vomiting, or diarrhea), or upper airway angioedema (lump sensation, difficulty swallowing, throat tightening, difficulty speaking, hoarseness, stridor, dyspnea, or swelling of tongue, palate, uvula, or larynx). HAE symptom days were identified from daily participant entries in the ePRO diary and were confirmed by the investigator.
Day 84 to Day 938
Proportion of Days With Investigator-confirmed HAE Symptoms During the Treatment Period in Part 2
時間枠：Day 84 to Day 938
HAE attack was defined as signs or symptoms in ≥1 location: peripheral angioedema (cutaneous swelling involving an extremity, face, neck, torso, and/or genitourinary region), abdominal angioedema (abdominal pain with or without abdominal distension, nausea, vomiting, or diarrhea), or upper airway angioedema (lump sensation, difficulty swallowing, throat tightening, difficulty speaking, hoarseness, stridor, dyspnea, or swelling of tongue, palate, uvula, or larynx). HAE symptom days were identified from daily participant entries in the ePRO diary and were confirmed by the investigator.
Day 84 to Day 938

協力者と研究者

ここでは、この調査に関係する人々や組織を見つけることができます。

スポンサー

Pharvaris Netherlands B.V.

捜査官

  • 主任研究者：Marc Riedl, MD、UC San Diego, La Jolla, California, United States
  • 主任研究者：Emel Aygören-Pürsün, MD、University Hospital Frankfurt - Goethe University, Frankfurt, Germany

出版物と役立つリンク

研究に関する情報を入力する責任者は、自発的にこれらの出版物を提供します。これらは、研究に関連するあらゆるものに関するものである可能性があります。

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)

2022年4月19日

一次修了 (実際)

2025年6月30日

研究の完了 (実際)

2025年6月30日

試験登録日

最初に提出

2021年9月7日

QC基準を満たした最初の提出物

2021年9月7日

最初の投稿 (実際)

2021年9月17日

学習記録の更新

投稿された最後の更新 (実際)

2026年8月17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12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

  • PHA022121-C301
  • 2021-000227-13 (EudraCT番号)

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?

いいえ

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

はい

米国FDA規制機器製品の研究

いいえ

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。

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