- ICH GCP
- Registr klinických studií v USA
- Klinická studie NCT07644832
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
8. června 2026 aktualizováno: 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).
Přehled studie
Postavení
Nábor
Podmínky
Intervence / Léčba
Typ studie
Intervenční
Zápis (Odhadovaný)
76
Fáze
- Fáze 2
- Fáze 1
Kontakty a umístění
Tato část poskytuje kontaktní údaje pro ty, kteří studii provádějí, a informace o tom, kde se tato studie provádí.
Studijní kontakt
- Jméno: Research and Development
- Telefonní číslo: 862122130888
- E-mail: hanyu@raas-corp.com
Studijní místa
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Changsha, Čína
- Nábor
- Xiangya Hospital of Central South University
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Hefei, Čína
- Nábor
- The First Affiliated Hospital of University of Science and Technology of China
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Jinan, Čína
- Nábor
- Jinan Central Hospital
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Lanzhou, Čína
- Nábor
- The First Hospital of Lanzhou University
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Shanghai, Čína
- Dokončeno
- Ruijin Hospital Shanghai Jiaotong University School of Medicine
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Taiyuan, Čína
- Nábor
- The Second Hospital of Shanxi Medical University
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Tianjin, Čína
- Dokončeno
- Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences
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Xi'an, Čína
- Nábor
- Xian Central Hospital
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Zhengzhou, Čína
- Nábor
- Zhengzhou People's Hospital
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Kritéria účasti
Výzkumníci hledají lidi, kteří odpovídají určitému popisu, kterému se říká kritéria způsobilosti. Některé příklady těchto kritérií jsou celkový zdravotní stav osoby nebo předchozí léčba.
Kritéria způsobilosti
Věk způsobilý ke studiu
- Dospělý
- Starší dospělý
Přijímá zdravé dobrovolníky
Ne
Popis
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.
Studijní plán
Tato část poskytuje podrobnosti o studijním plánu, včetně toho, jak je studie navržena a co studie měří.
Jak je studie koncipována?
Detaily designu
- Primární účel: Léčba
- Přidělení: Randomizované
- Intervenční model: Paralelní přiřazení
- Maskování: Žádné (otevřený štítek)
Zbraně a zásahy
Skupina účastníků / Arm |
Intervence / Léčba |
|---|---|
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Experimentální: 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 bude podáván jako SC injekce.
SR604 will be administered as SC injection
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Experimentální: 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 bude podáván jako SC injekce.
SR604 will be administered as SC injection
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Experimentální: 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 bude podáván jako SC injekce.
SR604 will be administered as SC injection
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Co je měření studie?
Primární výstupní opatření
Měření výsledku |
Popis opatření |
Časové okno |
|---|---|---|
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Part A: Incidence of AEs/SAEs/AESI
Časové okno: 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
Časové okno: Part A: From Baseline (Day 1) up to Day 85
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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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Part A: Number and incidence of patients with anti-drug antibodies (ADA) and neutralizing antibodies
Časové okno: Part A: From Baseline (Day 1) up to Day 85
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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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Part B/ Part C:Treated total annualized bleeding rate (ABR)
Časové okno: 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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Sekundární výstupní opatření
Měření výsledku |
Popis opatření |
Časové okno |
|---|---|---|
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Part A:Single-dose pharmacokinetic (PK) parameters:Peak Plasma Concentration (Cmax)
Časové okno: 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
Časové okno: 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
Časové okno: 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)
Časové okno: 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)
Časové okno: 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
Časové okno: 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)
Časové okno: 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
Časové okno: 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
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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 A:Single-dose pharmacokinetic (PK) parameters:Time to Peak Plasma Concentration (Tmax)
Časové okno: 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 A:Single-dose pharmacokinetic (PK) parameters:Area Under the Concentration-Time Curve from Zero to Last Quantifiable Time Point (AUC0-t)
Časové okno: 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:Multiple-dose pharmacokinetic parameters-Peak Plasma Concentration (Cmax)
Časové okno: 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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Další výstupní opatření
Měření výsledku |
Časové okno |
|---|---|
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Part A, part B and part C: Pharmacodynamic parameters-protein C
Časové okno: 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: 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)
Časové okno: 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: 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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Spolupracovníci a vyšetřovatelé
Zde najdete lidi a organizace zapojené do této studie.
Termíny studijních záznamů
Tato data sledují průběh záznamů studie a předkládání souhrnných výsledků na ClinicalTrials.gov. Záznamy ze studií a hlášené výsledky jsou před zveřejněním na veřejné webové stránce přezkoumány Národní lékařskou knihovnou (NLM), aby se ujistily, že splňují specifické standardy kontroly kvality.
Hlavní termíny studia
Začátek studia (Aktuální)
31. května 2024
Primární dokončení (Odhadovaný)
31. prosince 2026
Dokončení studie (Odhadovaný)
31. prosince 2026
Termíny zápisu do studia
První předloženo
26. května 2026
První předloženo, které splnilo kritéria kontroly kvality
8. června 2026
První zveřejněno (Aktuální)
12. června 2026
Aktualizace studijních záznamů
Poslední zveřejněná aktualizace (Aktuální)
12. června 2026
Odeslaná poslední aktualizace, která splnila kritéria kontroly kvality
8. června 2026
Naposledy ověřeno
1. května 2026
Více informací
Termíny související s touto studií
Další relevantní podmínky MeSH
- Genetické choroby, vrozené
- Hematologická onemocnění
- Poruchy srážení krve
- Hemoragické poruchy
- Genetická onemocnění, vázaná na X
- Poruchy srážení krve, dědičné
- Poruchy koagulačního proteinu
- Vrozené, dědičné a neonatální nemoci a abnormality
- Hemická a lymfatická onemocnění
- Hemofilie A
- Hemofilie B
- Nedostatek faktoru VII
Další identifikační čísla studie
- LS-SR604-Ⅰ01
Plán pro data jednotlivých účastníků (IPD)
Plánujete sdílet data jednotlivých účastníků (IPD)?
NE
Informace o lécích a zařízeních, studijní dokumenty
Studuje lékový produkt regulovaný americkým FDA
Ne
Studuje produkt zařízení regulovaný americkým úřadem FDA
Ne
Tyto informace byly beze změn načteny přímo z webu clinicaltrials.gov. Máte-li jakékoli požadavky na změnu, odstranění nebo aktualizaci podrobností studie, kontaktujte prosím register@clinicaltrials.gov. Jakmile bude změna implementována na clinicaltrials.gov, bude automaticky aktualizována i na našem webu .
Klinické studie na SR604
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Shanghai RAAS Blood Products Co., Ltd.NáborVon Willebrandova nemoc (VWD)Čína
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Equilibra Bioscience LLCNáborHemofilie A | Hemofilie B | Nedostatek faktoru VII | Zdraví účastníciSpojené státy, Kanada