A Phase II Exploratory Study of SR604 Injection Evaluating the Safety and Efficacy in the Treatment of Congenital Coagulation Factor VII Deficiency
2026年7月14日 更新者:Shanghai RAAS Blood Products Co., Ltd.
An Open-Label, Multicenter, Phase II Clinical Trial to Evaluate the Efficacy, Safety, and Pharmacokinetic Characteristics of SR604 Injection in Patients With Congenital Coagulation Factor VII Deficiency
This is an open-label, multicenter, exploratory Phase II clinical trial designed to evaluate the efficacy, safety, and pharmacokinetic characteristics of SR604 Injection in patients with congenital coagulation Factor VII deficiency.
調査の概要
研究の種類
介入
入学 (推定)
12
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。
研究連絡先
- 名前:Research and Development
- 電話番号:862122130888
- メール:hanyu@raas-corp.com
研究場所
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Tianjin、中国
- Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences
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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
- 大人
- 高齢者
健康ボランティアの受け入れ
いいえ
説明
Inclusion Criteria:
- Age ≥12 years and ≤65 years at the time of signing the informed consent form; both genders eligible;
- Clinically diagnosed with congenital coagulation Factor VII deficiency, with historical or screening FVII activity <10%, and ≥2 treated new-onset bleeding events within 3 months prior to enrollment;
- No active bleeding symptoms prior to first administration of SR604 Injection;
- The subject and/or legal representative and impartial witness have signed the informed consent form, indicating voluntary agreement to participate in this trial, to provide biological samples for testing as required by the protocol, and to comply with the planned study visits;
- Female subjects (post-menarche) must have a negative serum pregnancy test (HCG) during the screening period; subjects with childbearing potential (females post-menarche or males post-spermarche) must agree to use highly effective contraceptive measures throughout the study period.
Exclusion Criteria:
- Known history of hypersensitivity to the study drug formulation or any of its components;
- Intolerance to subcutaneous injection or other local skin abnormalities or dermatoses that may affect drug administration or safety assessment;
Meeting any one of the following criteria during screening:
- Hemoglobin <60 g/L;
- Platelet count <100×10⁹/L;
- Abnormal hepatic or renal function: alanine aminotransferase (ALT) or aspartate aminotransferase (AST) ≥2.5× upper limit of normal (ULN), or total bilirubin ≥1.5× ULN; or serum creatinine (Cr) ≥1.5× ULN;
- Positive for anti-human immunodeficiency virus (HIV) antibodies.
- Any bleeding disorder other than congenital Factor VII deficiency, or other conditions causing significantly abnormal coagulation parameters (e.g., hemophilia A or B, von Willebrand disease, platelet disorders, vitamin K deficiency, etc.);
- Protein C deficiency or Protein S deficiency;
- History of thrombosis, family history of thrombosis, or history of thrombophilia;
- Intracranial hemorrhage within 2 years prior to signing the informed consent form;
- Severe cardiac disease, such as unstable angina, congestive heart failure (New York Heart Association class ≥III), serious arrhythmia (QTc interval >450 ms, corrected by Fridericia formula), uncontrolled hypertension (systolic blood pressure ≥160 mmHg or diastolic blood pressure ≥100 mmHg), etc.;
- Female patients with menstrual abnormalities caused by organic gynecological conditions (e.g., uterine fibroids, endometriosis, adenomyosis, etc.);
- Received Factor VII-containing products within 48 hours prior to first administration of SR604 Injection; received whole blood or plasma transfusion within 2 weeks prior to first administration of SR604 Injection;
- Used any anticoagulants, antifibrinolytics, or agents affecting platelet function (including chemical drugs, biological products, or traditional Chinese medicine), including aspirin, within 1 week prior to screening, or required use of such agents during the treatment period;
- Underwent major surgery (defined as Grade III or IV surgery) within 1 month prior to signing the informed consent form, or planned to undergo surgery during the study period;
- Enrolled in other clinical trials within 1 month prior to signing the informed consent form;
- Miscarriage or pregnancy termination within 3 months prior to signing the informed consent form; pregnant or breastfeeding women;
- Mental illness or significant psychiatric disorder, or incapacity or lack of cognitive ability due to other causes;
Other conditions deemed by the investigator to be unsuitable for enrollment, such as alcoholism, anticipated poor subject compliance preventing completion of dosing and study follow-up, poorly controlled comorbid chronic diseases, or serious systemic diseases.
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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:並列代入
- 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:SR604:Multiple-dose exploratory efficacy trial consists of 2 cohorts
Participants with FVII deficiency will receive SR604 dose 1/2 as multiple SC injections every 4-weeks
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SR604 は皮下注射として投与されます。
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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 |
時間枠 |
|---|---|
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Treated overall Annualized Bleeding Rate (ABR)
時間枠:Through 28 weeks of treatment.
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Through 28 weeks of treatment.
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二次結果の測定
結果測定 |
時間枠 |
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Treated spontaneous annualized bleeding rate
時間枠:over 28 weeks of treatment
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over 28 weeks of treatment
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Treated overall annualized joint bleeding rate
時間枠:over 28 weeks of treatment
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over 28 weeks of treatment
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Overall annualized bleeding rate including treated and untreated bleeding events
時間枠:over 28 weeks of treatment,
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over 28 weeks of treatment,
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Annualized menorrhagia bleeding rate (menstruating females only)
時間枠:over 28 weeks of treatment
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over 28 weeks of treatment
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Change in PBAC score (menstruating females only);
時間枠:From baseline over 28 weeks of treatment
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From baseline over 28 weeks of treatment
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Change in EQ-5D-5L health index score
時間枠:From pre-treatment over 28 weeks of treatment
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From pre-treatment over 28 weeks of treatment
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Change in EQ-VAS score
時間枠:From baseline over 28 weeks of treatment;
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From baseline over 28 weeks of treatment;
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Subject overall satisfaction score with treatment efficacy
時間枠:over 28 weeks of treatment
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over 28 weeks of treatment
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その他の成果指標
結果測定 |
時間枠 |
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Single-dose pharmacokinetic (PK) parameters:Time to Peak Plasma Concentration (Tmax)
時間枠:over 28 weeks of treatment
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over 28 weeks of treatment
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Single-dose pharmacokinetic (PK) parameters:Peak Plasma Concentration (Cmax)
時間枠:over 28 weeks of treatment
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over 28 weeks of treatment
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Single-dose pharmacokinetic (PK) parameters:Area Under the Concentration-Time Curve from Zero to Last Quantifiable Time Point (AUC0-t)
時間枠:over 28 weeks of treatment
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over 28 weeks of treatment
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Multiple-dose pharmacokinetic parameters-Time to Peak Plasma Concentration (Tmax)
時間枠:over 28 weeks of treatment
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over 28 weeks of treatment
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Multiple-dose pharmacokinetic parameters-Peak Plasma Concentration (Cmax)
時間枠:Over 28 weeks of treatment
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Over 28 weeks of treatment
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Safety and Immunogenicity:Incidence of AEs/SAEs/AESI,
時間枠:over 28 weeks of treatment
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over 28 weeks of treatment
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Incidence of drug-related AEs/SAEs/AESIs
時間枠:over 28 weeks of treatment
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over 28 weeks of treatment
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Safety and Immunogenicity: Anti-drug antibody (ADA) and neutralizing antibody (NAb) - number of subjects and incidence rate.
時間枠:over 28 weeks of treatment
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over 28 weeks of treatment
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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。
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 (推定)
2026年7月1日
一次修了 (推定)
2027年2月1日
研究の完了 (推定)
2027年12月1日
試験登録日
最初に提出
2026年7月14日
QC基準を満たした最初の提出物
2026年7月14日
最初の投稿 (実際)
2026年7月17日
学習記録の更新
投稿された最後の更新 (実際)
2026年7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7月14日
最終確認日
2026年5月1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
- LS-SR604-VII-II01
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
いいえ
米国FDA規制機器製品の研究
いいえ
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