An Open-label, Multicenter Phase I/II Clinical Trial to Evaluate the Safety, Tolerability, Efficacy, and Pharmacokinetic/Pharmacodynamic (PK/PD) Characteristics of SR604 Injection in Patients With Hemophilia A/B and Congenital Factor VII Deficiency
2026年6月8日 更新者:Shanghai RAAS Blood Products Co., Ltd.
The purpose of this study is to evaluate the safety, tolerability, immunogenicity , PK, and PD of a single dose of SR604 in participants with Hemophilia A or Hemophilia B, with or without inhibitors (Part A)and to evaluate the safety, PK, PD, and efficacy of multiple doses of SR604 in participants with Hemophilia A or Hemophilia B, or Factor VII (FVII) deficiency, with or without inhibitors (Part B and Part C).
調査の概要
研究の種類
介入
入学 (推定)
76
段階
- フェーズ2
- フェーズ 1
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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Changsha、中国
- 募集
- Xiangya Hospital of Central South University
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Hefei、中国
- 募集
- The First Affiliated Hospital of University of Science and Technology of China
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Jinan、中国
- 募集
- Jinan Central Hospital
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Lanzhou、中国
- 募集
- The First Hospital of Lanzhou University
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Shanghai、中国
- 完了
- Ruijin Hospital Shanghai Jiaotong University School of Medicine
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Taiyuan、中国
- 募集
- The Second Hospital of Shanxi Medical University
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Tianjin、中国
- 完了
- Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences
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Xi'an、中国
- 募集
- Xian Central Hospital
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Zhengzhou、中国
- 募集
- Zhengzhou People's Hospital
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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 ≥18 years and ≤65 years at the time of signing informed consent, regardless of sex;
Clinically diagnosed with Hemophilia A or B or congenital coagulation Factor VII deficiency, and must meet the following criteria:
- Hemophilia A or B patients with historical or screening FVIII activity level <1% or FIX activity level ≤2%; Note: Hemophilia A or B patients with or without inhibitors may be enrolled. For patients without inhibitors (inhibitor titer <0.6 BU/mL), they must have previously received coagulation factor treatment with exposure days (EDs) >50 days.
- Congenital coagulation Factor VII deficiency patients with historical or screening FVII activity <10%;
- Part A only: Received on-demand treatment with FVIII, FIX, recombinant human coagulation Factor VIIa (rFVIIa), or PCC for bleeding events within 1 month prior to screening;
- Part B/Part C only: Accessible bleeding and treatment records (factor replacement or bypassing agent therapy) for at least 3 months prior to enrollment. Hemophilia A or B patients must have received on-demand treatment with ≥3 treated de novo bleeding episodes within 3 months prior to enrollment. Congenital coagulation Factor VII deficiency patients must have ≥2 treated de novo bleeding episodes within 3 months prior to enrollment;
- No active bleeding symptoms prior to first dosing;
- The subject or a legally acceptable representative has a full understanding of and can comply with the protocol requirements, has the willingness to complete the study as planned, and voluntarily agrees to provide biological samples for testing as required by the protocol;
- The subject is able to understand the procedures and methods of this clinical trial, has been fully informed, and voluntarily participates in the trial by personally signing the informed consent form.
Exclusion Criteria:
- Subjects with a known history of hypersensitivity to the investigational medicinal product or any of its components;
- Intolerance to subcutaneous injection or presence of other local skin abnormalities or dermatological conditions that may affect administration and safety assessment;
Subjects meeting any of the following criteria at screening:
- Hemoglobin <60 g/L;
- Platelet count <100 × 10^9/L;
- Hepatic or renal impairment: 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 result(s) for hepatitis B virus surface antigen (HBsAg), anti-human immunodeficiency virus (HIV) antibody, and/or Treponema pallidum-specific antibody;
- Clinically diagnosed with active hepatitis C;
- Any other bleeding disorder or any other disease causing significant coagulation abnormalities (e.g., platelet disorders, vitamin K deficiency, etc.) other than Hemophilia A or B and congenital coagulation Factor VII deficiency;
- Protein C deficiency or protein S deficiency;
- History of or current thrombosis, family history of thrombosis, or history of thrombophilia prior to signing informed consent;
- Intracranial hemorrhage due to Hemophilia A or B or congenital coagulation Factor VII deficiency within 2 years prior to screening;
- Severe cardiac disease, such as unstable angina, congestive heart failure (New York Heart Association Class ≥III), severe arrhythmia (QTc interval >450 ms, corrected by Fridericia's formula), or uncontrolled hypertension (systolic blood pressure ≥160 mmHg or diastolic blood pressure ≥95 mmHg);
- Received recombinant human coagulation Factor VIIa (rFVIIa) within 48 hours prior to first dosing; received any FVIII-containing product within 72 hours prior to first dosing; received any FIX-containing product within 96 hours prior to first dosing; long-acting products of the above have not completed a washout of 5 half-lives;
- Used or requires use of any anticoagulant, antifibrinolytic agent, or chemical drug, biological product, or traditional Chinese medicine affecting platelet function, including nonsteroidal anti-inflammatory drugs (NSAIDs) such as aspirin, within 1 week prior to first dosing or during the trial;
- Received whole blood or plasma therapy within 2 weeks prior to first dosing;
- Received emicizumab treatment within 6 months prior to first dosing;
- Received or planned to receive vaccination within 4 weeks prior to first dosing or during the trial;
- Underwent major surgery (e.g., orthopedic surgery, abdominal surgery) within 1 month prior to first dosing, or planned to undergo surgery during the study;
- Enrolled in another clinical trial within 1 month prior to first dosing;
- History of drug abuse or alcoholism (alcoholism criteria: long-term drinking history exceeding 5 years, equivalent to ethanol intake ≥40 g/day, or heavy drinking within 2 weeks, equivalent to ethanol intake >80 g/day. Ethanol amount (g) conversion formula = alcohol volume (mL) × ethanol content (%) × 0.8);
- Psychiatric illness or significant mental impairment, or incapacity or lack of cognitive ability due to other reasons;
- Plans to have children or donate sperm during the entire trial period up to 6 months after the last dose, or unwilling to use effective physical contraceptive measures (e.g., condoms);
- Subjects with clinically significant disease or other conditions that the investigator considers unsuitable for participation in the clinical trial (e.g., the patient cannot benefit from the clinical trial);
- Subjects deemed by the investigator to have poor compliance, rendering efficacy evaluation impossible or with low likelihood of completing the planned treatment course and follow-up.
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: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:Part A: Dose escalation trial consists of 6 cohorts
Participants with Hemophilia A or Hemophilia B will receive single subcutaneous (SC) dose from dose 1 to dose 6
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SR604 は皮下注射として投与されます。
SR604 will be administered as SC injection
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実験的:Part B: Multiple-dose exploratory efficacy trial consists of 2 cohorts
Participants with Hemophilia A or Hemophilia B or FVII deficiency will receive SR604 dose 1/2 as multiple SC injections every 2-weeks
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SR604 は皮下注射として投与されます。
SR604 will be administered as SC injection
|
|
実験的:Part C: Multiple-dose exploratory efficacy trial consists of 3 cohorts
Participants with Hemophilia A or Hemophilia B will receive SR604 dose 5 as multiple SC injections every 4-weeks/6-weeks/8-weeks.
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SR604 は皮下注射として投与されます。
SR604 will be administered as SC 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 |
|---|---|---|
|
Part A: Incidence of AEs/SAEs/AESI
時間枠:Part A: From Baseline (Day 1) up to Day 85
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Assessed through clinical signs and symptoms, vital signs, physical examination, laboratory tests (complete blood count, urinalysis, and blood biochemistry), coagulation function [PT, TT, INR, FIB, APTT, D-dimer], FDP, 12-lead electrocardiogram, injection site reactions, hypersensitivity/allergic reactions, thrombotic events, etc.;Safety and Immunogenicity of a single ascending SC dose of SR604 inparticipants with Hemophilia A or Hemophilia B will be evaluated.
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Part A: From Baseline (Day 1) up to Day 85
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PartA: Incidence of drug-related AEs/SAEs/AESIs
時間枠:Part A: From Baseline (Day 1) up to Day 85
|
Safety and Immunogenicity of a single ascending SC dose of SR604 inparticipants with Hemophilia A or Hemophilia B will be evaluated.
|
Part A: From Baseline (Day 1) up to Day 85
|
|
Part A: Number and incidence of patients with anti-drug antibodies (ADA) and neutralizing antibodies
時間枠:Part A: From Baseline (Day 1) up to Day 85
|
Safety and Immunogenicity of a single ascending SC dose of SR604 inparticipants with Hemophilia A or Hemophilia B will be evaluated.
|
Part A: From Baseline (Day 1) up to Day 85
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Part B/ Part C:Treated total annualized bleeding rate (ABR)
時間枠:Part B: From Baseline (Day 1) up to Day 211;Part C: From baseline (Day 1) up to Day 393
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Part B: From Baseline (Day 1) up to Day 211;Part C: From baseline (Day 1) up to Day 393
|
二次結果の測定
結果測定 |
メジャーの説明 |
時間枠 |
|---|---|---|
|
Part A:Single-dose pharmacokinetic (PK) parameters:Peak Plasma Concentration (Cmax)
時間枠:Part A: From Baseline (Day 1) up to Day 85
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PK of a single ascending SC dose of SR604 inparticipants with Hemophilia A or Hemophilia B will be evaluated.
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Part A: From Baseline (Day 1) up to Day 85
|
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Part B/ Part C:Treated spontaneous annualized bleeding rate
時間枠:Part B: From Baseline (Day 1) up to Day 211;Part C: From baseline (Day 1) up to Day 393
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Part B: From Baseline (Day 1) up to Day 211;Part C: From baseline (Day 1) up to Day 393
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Part B/ Part C:Treated total annualized joint bleeding rate
時間枠:Part B: From Baseline (Day 1) up to Day 211;Part C: From baseline (Day 1) up to Day 393
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Part B: From Baseline (Day 1) up to Day 211;Part C: From baseline (Day 1) up to Day 393
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Part B/ Part C:Treated annualized menorrhagia bleeding rate (applicable only to reproductive-age female patients with congenital FVII deficiency and active menstruation)
時間枠:Part B: From Baseline (Day 1) up to Day 211;Part C: From baseline (Day 1) up to Day 393
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Part B: From Baseline (Day 1) up to Day 211;Part C: From baseline (Day 1) up to Day 393
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Part B/ Part C:Change from baseline in Hemophilia Joint Health Score (HJHS) (for hemophilia A/B patients)
時間枠:Part B: From Baseline (Day 1) up to Day 211;Part C: From baseline (Day 1) up to Day 393
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Part B: From Baseline (Day 1) up to Day 211;Part C: From baseline (Day 1) up to Day 393
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Part B/ Part C:Change from baseline in EuroQol-5 Dimensions-5 Levels (EQ-5D-5L) score
時間枠:Part B: From Baseline (Day 1) up to Day 211;Part C: From baseline (Day 1) up to Day 393
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Part B: From Baseline (Day 1) up to Day 211;Part C: From baseline (Day 1) up to Day 393
|
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Part B/ Part C:Multiple-dose pharmacokinetic parameters-Time to Peak Plasma Concentration (Tmax)
時間枠:Part B: From Baseline (Day 1) up to Day 211;Part C: From baseline (Day 1) up to Day 393
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Part B: From Baseline (Day 1) up to Day 211;Part C: From baseline (Day 1) up to Day 393
|
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Part B/ Part C:Safety and Immunogenicity
時間枠:Part B: From Baseline (Day 1) up to Day 211;Part C: From baseline (Day 1) up to Day 393
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Incidence of AEs/SAEs/AESI, Incidence of drug-related AEs/SAEs/AESIs, Number and incidence of patients with anti-drug antibodies (ADA) and neutralizing antibodies
|
Part B: From Baseline (Day 1) up to Day 211;Part C: From baseline (Day 1) up to Day 393
|
|
Part A:Single-dose pharmacokinetic (PK) parameters:Time to Peak Plasma Concentration (Tmax)
時間枠:Part A: From Baseline (Day 1) up to Day 85
|
PK of a single ascending SC dose of SR604 inparticipants with Hemophilia A or Hemophilia B will be evaluated.
|
Part A: From Baseline (Day 1) up to Day 85
|
|
Part A:Single-dose pharmacokinetic (PK) parameters:Area Under the Concentration-Time Curve from Zero to Last Quantifiable Time Point (AUC0-t)
時間枠:Part A: From Baseline (Day 1) up to Day 85
|
PK of a single ascending SC dose of SR604 inparticipants with Hemophilia A or Hemophilia B will be evaluated.
|
Part A: From Baseline (Day 1) up to Day 85
|
|
Part B/ Part C:Multiple-dose pharmacokinetic parameters-Peak Plasma Concentration (Cmax)
時間枠:Part B: From Baseline (Day 1) up to Day 211;Part C: From baseline (Day 1) up to Day 393
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Part B: From Baseline (Day 1) up to Day 211;Part C: From baseline (Day 1) up to Day 393
|
その他の成果指標
結果測定 |
時間枠 |
|---|---|
|
Part A, part B and part C: Pharmacodynamic parameters-protein C
時間枠:Part A: From Baseline (Day 1) up to Day 85;Part B: From Baseline (Day 1) up to Day 211;Part C: From baseline (Day 1) up to Day 393
|
Part A: From Baseline (Day 1) up to Day 85;Part B: From Baseline (Day 1) up to Day 211;Part C: From baseline (Day 1) up to Day 393
|
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Part A, part B and part C: Pharmacodynamic parameters-prothrombin time (PT)
時間枠:Part A: From Baseline (Day 1) up to Day 85;Part B: From Baseline (Day 1) up to Day 211;Part C: From baseline (Day 1) up to Day 393
|
Part A: From Baseline (Day 1) up to Day 85;Part B: From Baseline (Day 1) up to Day 211;Part C: From baseline (Day 1) up to Day 393
|
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 (実際)
2024年5月31日
一次修了 (推定)
2026年12月31日
研究の完了 (推定)
2026年12月31日
試験登録日
最初に提出
2026年5月26日
QC基準を満たした最初の提出物
2026年6月8日
最初の投稿 (実際)
2026年6月12日
学習記録の更新
投稿された最後の更新 (実際)
2026年6月12日
QC基準を満たした最後の更新が送信されました
2026年6月8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-Ⅰ01
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
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。