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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. juni 2026 opdateret af: 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).

Studieoversigt

Status

Rekruttering

Intervention / Behandling

Undersøgelsestype

Interventionel

Tilmelding (Anslået)

76

Fase

  • Fase 2
  • Fase 1

Kontakter og lokationer

Dette afsnit indeholder kontaktoplysninger for dem, der udfører undersøgelsen, og oplysninger om, hvor denne undersøgelse udføres.

Studiekontakt

Studiesteder

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

Deltagelseskriterier

Forskere leder efter personer, der passer til en bestemt beskrivelse, kaldet berettigelseskriterier. Nogle eksempler på disse kriterier er en persons generelle helbredstilstand eller tidligere behandlinger.

Berettigelseskriterier

Aldre berettiget til at studere

  • Voksen
  • Ældre voksen

Tager imod sunde frivillige

Ingen

Beskrivelse

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.

Studieplan

Dette afsnit indeholder detaljer om studieplanen, herunder hvordan undersøgelsen er designet, og hvad undersøgelsen måler.

Hvordan er undersøgelsen tilrettelagt?

Design detaljer

  • Primært formål: Behandling
  • Tildeling: Randomiseret
  • Interventionel model: Parallel tildeling
  • Maskning: Ingen (Åben etiket)

Våben og indgreb

Deltagergruppe / Arm
Intervention / Behandling
Eksperimentel: 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 vil blive administreret som SC-injektion.
SR604 will be administered as SC injection
Eksperimentel: 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 vil blive administreret som SC-injektion.
SR604 will be administered as SC injection
Eksperimentel: 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 vil blive administreret som SC-injektion.
SR604 will be administered as SC injection

Hvad måler undersøgelsen?

Primære resultatmål

Resultatmål
Foranstaltningsbeskrivelse
Tidsramme
Part A: Incidence of AEs/SAEs/AESI
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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)
Tidsramme: 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

Sekundære resultatmål

Resultatmål
Foranstaltningsbeskrivelse
Tidsramme
Part A:Single-dose pharmacokinetic (PK) parameters:Peak Plasma Concentration (Cmax)
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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)
Tidsramme: 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)
Tidsramme: 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
Tidsramme: 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)
Tidsramme: 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
Tidsramme: 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)
Tidsramme: 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)
Tidsramme: 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)
Tidsramme: 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

Andre resultatmål

Resultatmål
Tidsramme
Part A, part B and part C: Pharmacodynamic parameters-protein C
Tidsramme: 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)
Tidsramme: 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

Samarbejdspartnere og efterforskere

Det er her, du vil finde personer og organisationer, der er involveret i denne undersøgelse.

Datoer for undersøgelser

Disse datoer sporer fremskridtene for indsendelser af undersøgelsesrekord og resumeresultater til ClinicalTrials.gov. Studieregistreringer og rapporterede resultater gennemgås af National Library of Medicine (NLM) for at sikre, at de opfylder specifikke kvalitetskontrolstandarder, før de offentliggøres på den offentlige hjemmeside.

Studer store datoer

Studiestart (Faktiske)

31. maj 2024

Primær færdiggørelse (Anslået)

31. december 2026

Studieafslutning (Anslået)

31. december 2026

Datoer for studieregistrering

Først indsendt

26. maj 2026

Først indsendt, der opfyldte QC-kriterier

8. juni 2026

Først opslået (Faktiske)

12. juni 2026

Opdateringer af undersøgelsesjournaler

Sidste opdatering sendt (Faktiske)

12. juni 2026

Sidste opdatering indsendt, der opfyldte kvalitetskontrolkriterier

8. juni 2026

Sidst verificeret

1. maj 2026

Mere information

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