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先天性 C1-INH 欠損症患者における急性遺伝性血管浮腫発作の治療および前処置予防を目的とした IV ヒト血漿由来 C1 エステラーゼ阻害剤濃縮物の第 3 相試験

2026年9月25日 更新者：Octapharma

先天性 C1-INH 欠損症の治療参加者を対象とした、ヒト血漿由来 C1 エステラーゼ阻害剤 (C1-INH) 濃縮物の静脈内投与に関する前向き、多施設共同、無作為化、二重盲検、並行群間、プラセボ対照、有効性と安全性の第 3 相試験急性遺伝性血管浮腫発作の術前予防

Prospective, multicenter, randomized, double-blind, parallel group, placebo- controlled, efficacy and safety phase 3 study of an intravenous human plasma- derived C1 esterase inhibitor (C1-INH) concentrate in participants with congenital C1-INH deficiency for the treatment and pre-procedure prevention of acute hereditary angioedema attacks

調査の概要

状態

募集

条件

介入・治療

研究の種類

介入

入学 (推定)

124

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。

研究連絡先

研究場所

  • アメリカ
    • Colorado
      • Centennial、Colorado、アメリカ、80112
        • 引きこもった
        • Octapharma Research Site
    • Michigan
      • Farmington Hills、Michigan、アメリカ、48334
        • 引きこもった
        • Octapharma Research Site
    • Ohio
      • Toledo、Ohio、アメリカ、43617
        • 引きこもった
        • Octapharma Research Site
  • アルゼンチン
      • Rosario、アルゼンチン
        • 募集
        • Octapharma Research Site
  • アルバニア
      • Tirana、アルバニア
        • 引きこもった
        • Octapharma Research Site
  • アルメニア
      • Yerevan、アルメニア
        • 募集
        • Octapharma Research Site
  • インド
      • Bangalore、インド、560017
        • まだ募集していません
        • Octapharma Research Site
      • Chhatrapati Sambhajinagar、インド、431003
        • まだ募集していません
        • Octapharma Research Site, Chhatrapati Sambhajinagar
      • Patna、インド、801507
        • まだ募集していません
        • Octapharma Research Site
    • Maharashtra
      • Pune、Maharashtra、インド、411040
        • まだ募集していません
        • Octapharma Research Site
  • ウクライナ
      • Kyiv、ウクライナ、03057
        • 募集
        • Octapharma Research Site
      • Lviv、ウクライナ、79035
        • まだ募集していません
        • Octapharma Research Site
      • Lviv、ウクライナ、79010
        • 引きこもった
        • Octapharma Research Site
  • セルビア
      • Kragujevac、セルビア、11221
        • 引きこもった
        • Octapharma Research Site
  • トルコ（Türkiye）
      • Ankara、トルコ（Türkiye）
        • まだ募集していません
        • Octapharma Research Site
      • Istanbul、トルコ（Türkiye）
        • まだ募集していません
        • Octapharma Research Site
      • Izmir、トルコ（Türkiye）
        • 引きこもった
        • Octapharma Research Site
      • Sakarya、トルコ（Türkiye）
        • まだ募集していません
        • Octapharma Research Site
  • ブルガリア
      • Sofia、ブルガリア、1431
        • 募集
        • Octapharma Research Site
  • ペルー
      • Lima、ペルー、15001
        • 募集
        • Octapharma Research Site 5103
      • Lima、ペルー、15001
        • 募集
        • Octapharma Research Site 5102
  • メキシコ
      • Mexico City、メキシコ、06720
        • まだ募集していません
        • Octapharma Research Site
      • México、メキシコ
        • まだ募集していません
        • Octapharma Research Site
  • モンテネグロ
      • Podgorica、モンテネグロ
        • 引きこもった
        • Octapharma Research Site
  • ルーマニア
      • Cluj-Napoca、ルーマニア、400162
        • 引きこもった
        • Octapharma Research 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

2年歳以上 (子、大人、高齢者)

健康ボランティアの受け入れ

いいえ

受講資格のある性別

全て

説明

Inclusion Criteria:

  1. Is at least 18 years of age (applicable for 1st study phase) or is at least 2 years of age (applicable for 2nd study phase)

  2. Has confirmed diagnosis of HAE type I or II

  3. Has had at least 3 moderate or severe HAE attacks (excluding extremity attacks) in the last 3 months before the Screening Visit. For participants ≥2 and ≤12 years of age, has had at least 1 moderate or severe HAE attack (excluding extremity attacks) in the last 6 months before Screening Visit

  4. Has a documented congenital C1-INH functional activity <50% with or without C1-INH deficiency and C4 antigen level below the laboratory reference range

  5. Participant or the participant's legally authorized representative(s) has signed informed consent (as required by local law), with the assent of participants legally capable of providing it, as applicable

  6. States willingness to comply with all study procedures and availability for the duration of the study

  7. If the participant is of childbearing potential (CBP), has a negative pregnancy test and must have been using a highly effective method of contraception and continue to do so until at least 2 weeks after their last dose (for both blinded and open-label doses of IMP). Not of CBP is defined as surgically sterilized (hysterectomy, bilateral oophorectomy) or who are postmenopausal (defined as women with no menses for 12 months without an alternative medical cause). Highly effective methods of contraception:

    • Combined hormonal contraception (estrogens and progesterone) methods such as oral, implantable, intravaginal, injectable, or transdermal contraceptives at a stable dose for a minimum of 1 full cycle (hormonal contraceptives must inhibit ovulation) and for at least 4 weeks before screening
    • Progesterone only hormonal contraception associated with inhibition of ovulation (oral, injectable, implantable)
    • Intrauterine device
    • Intrauterine hormone-releasing system inserted at least 4 weeks before screening
    • Bilateral tubal ligation/occlusion or vasectomized partner (with surgical success confirmed by medical assessment) OR Agrees to abstain from heterosexual intercourse during study participation and to use a highly effective contraceptive (as described above) as backup if they become sexually active during the study. Abstinence is only acceptable if this is the participant's usual lifestyle. Periodic abstinence (calendar, symptothermal, post-ovulation methods), withdrawal (coitus interruptus), spermicides only, and lactational amenorrhea method are not acceptable methods of contraception
    • Note: If a participant of CBP has a positive or suspected positive urine pregnancy test within 72 hours prior to treatment, a serum pregnancy test will be required
    • Male participants must not plan to father a child or donate sperm for 90 days after their last dose of study drug (for both blinded and open-label doses of the IMP). However, there are no official contraception requirements for male participants during the study.

Inclusion Criteria for IMP Dosing for QAT:

  1. Has confirmed QAT per definition criteria
  2. Has a swelling episode that is new and not the continuation of a previous HAE attack

Exclusion Criteria:

  1. Has a history of clinically relevant antibody development against C1-INH
  2. Has a medical history consistent with Type 3 HAE (i.e., onset at age above 40 year, no family history, no known HAE mutation, low C1q level in plasma)
  3. Has a history of allergic reaction to C1-INH or other blood/plasma product
  4. Has a history of B-cell malignancy that was unresolved in the past 5 years
  5. Has a narcotic and/or alcoholic addiction
  6. Has participated in any other investigational drug evaluation within 30 days before screening
  7. Is pregnant or breastfeeding
  8. Has any clinically significant medical or psychiatric condition that, in the investigator's opinion would interfere with the participant's ability to participate in the study
  9. Has a history of thromboembolic events (TEEs), myocardial infarction, unstable angina pectoris, critical aortic stenosis, cerebrovascular accident, transient ischemic attack, severe peripheral vascular disease, or disseminated intravascular coagulation within one year before screening
  10. (applicable until IDMC review of the interim preliminary safety and efficacy data): has clinically significant derangement in measurements of cardiovascular status (i.e. uncontrolled arterial hypertension, cardiac insufficiency New York Heart Association (NYHA) class III-IV), pulmonary status (i.e., COPD GOLD classification 3 and 4, severe asthma) and renal status (i.e., eGFR below 90 ml/min per 1.73 m2)

Exclusion Criteria for IMP Dosing for QAT:

  1. Has received blood or a blood product for prophylactic or acute treatment with any C1-INH (Berinert®, Cinryze®, HAEgarda®, Ruconest®, etc.), non-biological bradykinin and kallikrein pathway inhibitors (e.g., ecallantide, icatibant, berotralstat), or treatment with tranexamic acid within 14 days before dosing with the IMP (or is not willing to abstain from these medications throughout the study)
  2. started or changed hormone replacement therapy or selective estrogen receptor modulators (e.g., tamoxifen) within 14 days before IMP dosing
  3. Started or changed androgen therapy (e.g. testosterone, dehydro- epiandrosterone/androstenedione, oxandrolone, danazol, stanozolol) within 14 days before IMP dosing or is not willing to maintain a stable dose throughout the study
  4. Started or changed the dose of monoclonal antibodies e.g. lanadelumab within 11 weeks before dosing or not willing to maintain a stable dose throughout the study
  5. Has used narcotic pain medications or non-opioid analgesics within 7 days before IMP dosing for a QAT
  6. Has received OCTA-C1-INH within 14 days before IMP dosing

Exclusion Criteria for IMP Dosing for PK:

  1. Has received blood or a blood product for prophylactic or acute treatment with any C1-INH (Berinert®, Cinryze®, HAEgarda®, Ruconest®, etc.), non-biological bradykinin and kallikrein pathway inhibitors (e.g., ecallantide, icatibant, berotralstat), or treatment with tranexamic acid within 14 days before dosing with the IMP (or is not willing to abstain from these medications throughout the study)
  2. Is receiving hormone replacement therapy or selective estrogen receptor modulators (e.g., tamoxifen) and has had their dose changed within 14 days before IMP dosing
  3. Is receiving or has received androgen therapy (e.g., testosterone, dehydroepiandrosterone/androstenedione, oxandrolone, danazol, stanozolol) IN ANY DOSE within 14 days before dosing
  4. Started or changed the dose of monoclonal antibodies e.g lanadelumab within 11 weeks before dosing or not willing to maintain a stable dose throughout the study
  5. Has used narcotic pain medications or non-opioid analgesics within 7 days before IMP dosing
  6. Has received IMP within 14 days before IMP dosing
  7. Has planned dental, medical, or surgical procedures during the PK Period that will require pre-procedural prevention

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：ランダム化
  • 介入モデル：並列代入
  • マスキング：ダブル

武器と介入

参加者グループ / アーム
介入・治療
実験的：OCTA-C1-INH

OCTA-C1-INH injection, 20IU/kg BW after first qualifying attack. Treatment to be administered to blinded as well as open-label subjects.
薬：OCTA-C1-INH

OCTA-C1-INH is a stable, sterile, virus-inactivated, nano-filtered, highly purified concentrate of human C1-INH prepared from pooled human plasma. After reconstitution in 2.5mL water for injection, the solution can be administered as a slow IV injection. OCTA-C1-INH is given as a dose of 20 IU/kg body weight (BW)
プラセボコンパレーター：Placebo

0.1 mL/kg BW 0.9% sodium chloride solution injection after first qualifying attack. Only blinded subjects to receive.
他の：Placebo

0.1 mL/kg BW 0.9% sodium chloride injection

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
Time (h) to beginning of unequivocal symptom relief at the defining site in blinded participants.
時間枠：Within 4 hours after injection

Patient will rate symptom relief for the defining attack site (site of swelling or pain) from 15 minutes after start of the IMP injection every 15 minutes over 4 hours. Unequivocal relief is defined as having 3 consecutive reports of "absent now but present before," "absent now and absent before", or "present, symptoms better" on the 5-grade SRRS.

This measure will also be used for the secondary outcomes in a different context.
Within 4 hours after injection

二次結果の測定

結果測定
メジャーの説明
時間枠
Percentage of participants responding to treatment
時間枠：within 4 hours after injection

Percentage of patients responding to treatment, defined as beginning of unequivocal symptom relief at the defining site within 4 hours after injection (once per participant after first qualifying attack in the study). Unequivocal relief is defined as having 3 consecutive reports of "absent now but present before," "absent now and absent before", or "present, symptoms better" on the 5-grade symptom relief rating.
within 4 hours after injection
Time to beginning of unequivocal symptom relief at all sites involved
時間枠：Within 4 hours after injection

Time to the beginning of unequivocal symptom relief at all sites involved within 4 hours after injection. Unequivocal relief is defined as having 3 consecutive reports of "absent now but present before," "absent now and absent before", or "present, symptoms better" on the 5-grade symptom relief rating.
Within 4 hours after injection
Changes in symptom severity at the defining site by VAS severity rating
時間枠：Within 4 hours after injection

Changes in symptom severity at the defining site by visual analog scale (VAS) rating from pre-injection over 4 hours after injection. The VAS is a self-reported 0-100 pain and swelling rating scale.
Within 4 hours after injection

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

Octapharma

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)

2024年4月30日

一次修了 (推定)

2026年12月1日

研究の完了 (推定)

2027年6月1日

試験登録日

最初に提出

2024年1月11日

QC基準を満たした最初の提出物

2024年4月5日

最初の投稿 (実際)

2024年4月12日

学習記録の更新

投稿された最後の更新 (実際)

2026年9月29日

QC基準を満たした最後の更新が送信されました

2026年9月25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

  • CONE-02

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

米国で製造され、米国から輸出された製品。

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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