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

Trial statusRecruiting
Trial phasePhase 1, Phase 2
Trial typeInterventional
Biological sexAll
Age18-65
SponsorShanghai RAAS Blood Products Co., Ltd.

About this trial

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).

Eligibility criteria

Qualifiers

Age ≥18 years and ≤65 years at the time of signing informed consent, regardless of sex;

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;

Disqualifiers

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;

Hemoglobin <60 g/L;

Platelet count <100 × 10^9/L;

Trial design

Treatments tested in this trial

  • SR604
  • SR604
  • SR604

Treatment groups

76 Participants
are divided into 3 treatment groups