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

NCT07644832 | Recruiting Phase 1

• 1 other identifier: LS-SR604-Ⅰ01
• Study Type: interventional
• Target: 76
• Locations: 1 country
• Sites: 9

Brief Summary

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).

Conditions

Hemophilia A; Hemophilia B; Factor VII Deficiency

Outcome Measures

Primary Outcomes (4)

• Part A: Incidence of AEs/SAEs/AESI

  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

  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

  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)

  Part B: From Baseline (Day 1) up to Day 211;Part C: From baseline (Day 1) up to Day 393

Secondary Outcomes (11)

• Part A:Single-dose pharmacokinetic (PK) parameters:Peak Plasma Concentration (Cmax)

  Part A: From Baseline (Day 1) up to Day 85

• Part B/ Part C:Treated spontaneous annualized bleeding rate

  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

  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)

  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)

  Part B: From Baseline (Day 1) up to Day 211;Part C: From baseline (Day 1) up to Day 393

Other Outcomes (2)

• Part A, part B and part C: Pharmacodynamic parameters-protein C

  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)

  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

Study Arms (3)

Part A: Dose escalation trial consists of 6 cohorts

EXPERIMENTAL

Participants with Hemophilia A or Hemophilia B will receive single subcutaneous (SC) dose from dose 1 to dose 6

Drug: SR604

Part B: Multiple-dose exploratory efficacy trial consists of 2 cohorts

EXPERIMENTAL

Participants with Hemophilia A or Hemophilia B or FVII deficiency will receive SR604 dose 1/2 as multiple SC injections every 2-weeks

Drug: SR604

Part C: Multiple-dose exploratory efficacy trial consists of 3 cohorts

EXPERIMENTAL

Participants with Hemophilia A or Hemophilia B will receive SR604 dose 5 as multiple SC injections every 4-weeks/6-weeks/8-weeks.

Drug: SR604

Interventions

SR604 DRUG

SR604 will be administered as SC injection

Part A: Dose escalation trial consists of 6 cohorts

Eligibility Criteria

• Age: 18 Years - 65 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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;

Sponsors & Collaborators

• Shanghai RAAS Blood Products Co., Ltd.: lead

Study Sites (9)

Xiangya Hospital of Central South University

Changsha, China

RECRUITING

The First Affiliated Hospital of University of Science and Technology of China

Hefei, China

RECRUITING

Jinan Central Hospital

Jinan, China

RECRUITING

The First Hospital of Lanzhou University

Lanzhou, China

RECRUITING

Ruijin Hospital Shanghai Jiaotong University School of Medicine

Shanghai, China

COMPLETED

The Second Hospital of Shanxi Medical University

Taiyuan, China

RECRUITING

Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences

Tianjin, China

COMPLETED

Xian Central Hospital

Xi'an, China

RECRUITING

Zhengzhou People's Hospital

Zhengzhou, China

RECRUITING

MeSH Terms

Conditions

Hemophilia A; Hemophilia B; Factor VII Deficiency

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Genetic Diseases, X-Linked

Central Study Contacts

Research and Development

CONTACT

862122130888; hanyu@raas-corp.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 26, 2026
• First Posted: June 12, 2026
• Study Start: May 31, 2024
• Primary Completion (Estimated): December 31, 2026
• Study Completion (Estimated): December 31, 2026
• Last Updated: June 12, 2026

Record last verified: 2026-05

Data Sharing

• IPD Sharing: Will not share

Locations

CN (9)