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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 giugno 2026 aggiornato da: 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).

Panoramica dello studio

Stato

Reclutamento

Intervento / Trattamento

Tipo di studio

Interventistico

Iscrizione (Stimato)

76

Fase

  • Fase 2
  • Fase 1

Contatti e Sedi

Questa sezione fornisce i recapiti di coloro che conducono lo studio e informazioni su dove viene condotto lo studio.

Contatto studio

Luoghi di studio

      • Changsha, Cina
        • Reclutamento
        • Xiangya Hospital of Central South University
      • Hefei, Cina
        • Reclutamento
        • The First Affiliated Hospital of University of Science and Technology of China
      • Jinan, Cina
        • Reclutamento
        • Jinan Central Hospital
      • Lanzhou, Cina
        • Reclutamento
        • The First Hospital of Lanzhou University
      • Shanghai, Cina
        • Completato
        • Ruijin Hospital Shanghai Jiaotong University School of Medicine
      • Taiyuan, Cina
        • Reclutamento
        • The Second Hospital of Shanxi Medical University
      • Tianjin, Cina
        • Completato
        • Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences
      • Xi'an, Cina
        • Reclutamento
        • Xian Central Hospital
      • Zhengzhou, Cina
        • Reclutamento
        • Zhengzhou People's Hospital

Criteri di partecipazione

I ricercatori cercano persone che corrispondano a una certa descrizione, chiamata criteri di ammissibilità. Alcuni esempi di questi criteri sono le condizioni generali di salute di una persona o trattamenti precedenti.

Criteri di ammissibilità

Età idonea allo studio

  • Adulto
  • Adulto più anziano

Accetta volontari sani

No

Descrizione

Inclusion Criteria:

  1. Age ≥18 years and ≤65 years at the time of signing informed consent, regardless of sex;
  2. Clinically diagnosed with Hemophilia A or B or congenital coagulation Factor VII deficiency, and must meet the following criteria:

    1. 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.
    2. Congenital coagulation Factor VII deficiency patients with historical or screening FVII activity <10%;
  3. 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;
  4. 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;
  5. No active bleeding symptoms prior to first dosing;
  6. 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;
  7. 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:

  1. Subjects with a known history of hypersensitivity to the investigational medicinal product or any of its components;
  2. Intolerance to subcutaneous injection or presence of other local skin abnormalities or dermatological conditions that may affect administration and safety assessment;
  3. Subjects meeting any of the following criteria at screening:

    1. Hemoglobin <60 g/L;
    2. Platelet count <100 × 10^9/L;
    3. 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;
  4. Positive result(s) for hepatitis B virus surface antigen (HBsAg), anti-human immunodeficiency virus (HIV) antibody, and/or Treponema pallidum-specific antibody;
  5. Clinically diagnosed with active hepatitis C;
  6. 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;
  7. Protein C deficiency or protein S deficiency;
  8. History of or current thrombosis, family history of thrombosis, or history of thrombophilia prior to signing informed consent;
  9. Intracranial hemorrhage due to Hemophilia A or B or congenital coagulation Factor VII deficiency within 2 years prior to screening;
  10. 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);
  11. 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;
  12. 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;
  13. Received whole blood or plasma therapy within 2 weeks prior to first dosing;
  14. Received emicizumab treatment within 6 months prior to first dosing;
  15. Received or planned to receive vaccination within 4 weeks prior to first dosing or during the trial;
  16. Underwent major surgery (e.g., orthopedic surgery, abdominal surgery) within 1 month prior to first dosing, or planned to undergo surgery during the study;
  17. Enrolled in another clinical trial within 1 month prior to first dosing;
  18. 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);
  19. Psychiatric illness or significant mental impairment, or incapacity or lack of cognitive ability due to other reasons;
  20. 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);
  21. 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);
  22. 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.

Piano di studio

Questa sezione fornisce i dettagli del piano di studio, compreso il modo in cui lo studio è progettato e ciò che lo studio sta misurando.

Come è strutturato lo studio?

Dettagli di progettazione

  • Scopo principale: Trattamento
  • Assegnazione: Randomizzato
  • Modello interventistico: Assegnazione parallela
  • Mascheramento: Nessuno (etichetta aperta)

Armi e interventi

Gruppo di partecipanti / Arm
Intervento / Trattamento
Sperimentale: 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
SR604 verrà somministrato come iniezione SC.
SR604 will be administered as SC injection
Sperimentale: 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
SR604 verrà somministrato come iniezione SC.
SR604 will be administered as SC injection
Sperimentale: 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.
SR604 verrà somministrato come iniezione SC.
SR604 will be administered as SC injection

Cosa sta misurando lo studio?

Misure di risultato primarie

Misura del risultato
Misura Descrizione
Lasso di tempo
Part A: Incidence of AEs/SAEs/AESI
Lasso di tempo: Part A: From Baseline (Day 1) up to Day 85
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.
Part A: From Baseline (Day 1) up to Day 85
PartA: Incidence of drug-related AEs/SAEs/AESIs
Lasso di tempo: 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
Lasso di tempo: 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 B/ Part C:Treated total annualized bleeding rate (ABR)
Lasso di tempo: Part B: From Baseline (Day 1) up to Day 211;Part C: From baseline (Day 1) up to Day 393
Part B: From Baseline (Day 1) up to Day 211;Part C: From baseline (Day 1) up to Day 393

Misure di risultato secondarie

Misura del risultato
Misura Descrizione
Lasso di tempo
Part A:Single-dose pharmacokinetic (PK) parameters:Peak Plasma Concentration (Cmax)
Lasso di tempo: 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:Treated spontaneous annualized bleeding rate
Lasso di tempo: Part B: From Baseline (Day 1) up to Day 211;Part C: From baseline (Day 1) up to Day 393
Part B: From Baseline (Day 1) up to Day 211;Part C: From baseline (Day 1) up to Day 393
Part B/ Part C:Treated total annualized joint bleeding rate
Lasso di tempo: Part B: From Baseline (Day 1) up to Day 211;Part C: From baseline (Day 1) up to Day 393
Part B: From Baseline (Day 1) up to Day 211;Part C: From baseline (Day 1) up to Day 393
Part B/ Part C:Treated annualized menorrhagia bleeding rate (applicable only to reproductive-age female patients with congenital FVII deficiency and active menstruation)
Lasso di tempo: Part B: From Baseline (Day 1) up to Day 211;Part C: From baseline (Day 1) up to Day 393
Part B: From Baseline (Day 1) up to Day 211;Part C: From baseline (Day 1) up to Day 393
Part B/ Part C:Change from baseline in Hemophilia Joint Health Score (HJHS) (for hemophilia A/B patients)
Lasso di tempo: Part B: From Baseline (Day 1) up to Day 211;Part C: From baseline (Day 1) up to Day 393
Part B: From Baseline (Day 1) up to Day 211;Part C: From baseline (Day 1) up to Day 393
Part B/ Part C:Change from baseline in EuroQol-5 Dimensions-5 Levels (EQ-5D-5L) score
Lasso di tempo: Part B: From Baseline (Day 1) up to Day 211;Part C: From baseline (Day 1) up to Day 393
Part B: From Baseline (Day 1) up to Day 211;Part C: From baseline (Day 1) up to Day 393
Part B/ Part C:Multiple-dose pharmacokinetic parameters-Time to Peak Plasma Concentration (Tmax)
Lasso di tempo: Part B: From Baseline (Day 1) up to Day 211;Part C: From baseline (Day 1) up to Day 393
Part B: From Baseline (Day 1) up to Day 211;Part C: From baseline (Day 1) up to Day 393
Part B/ Part C:Safety and Immunogenicity
Lasso di tempo: Part B: From Baseline (Day 1) up to Day 211;Part C: From baseline (Day 1) up to Day 393
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)
Lasso di tempo: 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)
Lasso di tempo: 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)
Lasso di tempo: Part B: From Baseline (Day 1) up to Day 211;Part C: From baseline (Day 1) up to Day 393
Part B: From Baseline (Day 1) up to Day 211;Part C: From baseline (Day 1) up to Day 393

Altre misure di risultato

Misura del risultato
Lasso di tempo
Part A, part B and part C: Pharmacodynamic parameters-protein C
Lasso di tempo: 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
Part A, part B and part C: Pharmacodynamic parameters-prothrombin time (PT)
Lasso di tempo: 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

Collaboratori e investigatori

Qui è dove troverai le persone e le organizzazioni coinvolte in questo studio.

Studiare le date dei record

Queste date tengono traccia dell'avanzamento della registrazione dello studio e dell'invio dei risultati di sintesi a ClinicalTrials.gov. I record degli studi e i risultati riportati vengono esaminati dalla National Library of Medicine (NLM) per assicurarsi che soddisfino specifici standard di controllo della qualità prima di essere pubblicati sul sito Web pubblico.

Studia le date principali

Inizio studio (Effettivo)

31 maggio 2024

Completamento primario (Stimato)

31 dicembre 2026

Completamento dello studio (Stimato)

31 dicembre 2026

Date di iscrizione allo studio

Primo inviato

26 maggio 2026

Primo inviato che soddisfa i criteri di controllo qualità

8 giugno 2026

Primo Inserito (Effettivo)

12 giugno 2026

Aggiornamenti dei record di studio

Ultimo aggiornamento pubblicato (Effettivo)

12 giugno 2026

Ultimo aggiornamento inviato che soddisfa i criteri QC

8 giugno 2026

Ultimo verificato

1 maggio 2026

Maggiori informazioni

Termini relativi a questo studio

Piano per i dati dei singoli partecipanti (IPD)

Hai intenzione di condividere i dati dei singoli partecipanti (IPD)?

NO

Informazioni su farmaci e dispositivi, documenti di studio

Studia un prodotto farmaceutico regolamentato dalla FDA degli Stati Uniti

No

Studia un dispositivo regolamentato dalla FDA degli Stati Uniti

No

Queste informazioni sono state recuperate direttamente dal sito web clinicaltrials.gov senza alcuna modifica. In caso di richieste di modifica, rimozione o aggiornamento dei dettagli dello studio, contattare register@clinicaltrials.gov. Non appena verrà implementata una modifica su clinicaltrials.gov, questa verrà aggiornata automaticamente anche sul nostro sito web .

Prove cliniche su SR604

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