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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

maanantai 8. kesäkuuta 2026 päivittänyt: 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).

Tutkimuksen yleiskatsaus

Opintotyyppi

Interventio

Ilmoittautuminen (Arvioitu)

76

Vaihe

  • Vaihe 2
  • Vaihe 1

Yhteystiedot ja paikat

Tässä osiossa on tutkimuksen suorittajien yhteystiedot ja tiedot siitä, missä tämä tutkimus suoritetaan.

Opiskeluyhteys

Opiskelupaikat

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

Osallistumiskriteerit

Tutkijat etsivät ihmisiä, jotka sopivat tiettyyn kuvaukseen, jota kutsutaan kelpoisuuskriteereiksi. Joitakin esimerkkejä näistä kriteereistä ovat henkilön yleinen terveydentila tai aiemmat hoidot.

Kelpoisuusvaatimukset

Opintokelpoiset iät

  • Aikuinen
  • Vanhempi Aikuinen

Hyväksyy terveitä vapaaehtoisia

Ei

Kuvaus

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.

Opintosuunnitelma

Tässä osiossa on tietoja tutkimussuunnitelmasta, mukaan lukien kuinka tutkimus on suunniteltu ja mitä tutkimuksella mitataan.

Miten tutkimus on suunniteltu?

Suunnittelun yksityiskohdat

  • Ensisijainen käyttötarkoitus: Hoito
  • Jako: Satunnaistettu
  • Inventiomalli: Rinnakkaistehtävä
  • Naamiointi: Ei mitään (avoin tarra)

Aseet ja interventiot

Osallistujaryhmä / Arm
Interventio / Hoito
Kokeellinen: 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 annetaan SC-injektiona.
SR604 will be administered as SC injection
Kokeellinen: 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 annetaan SC-injektiona.
SR604 will be administered as SC injection
Kokeellinen: 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 annetaan SC-injektiona.
SR604 will be administered as SC injection

Mitä tutkimuksessa mitataan?

Ensisijaiset tulostoimenpiteet

Tulosmittaus
Toimenpiteen kuvaus
Aikaikkuna
Part A: Incidence of AEs/SAEs/AESI
Aikaikkuna: 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
Aikaikkuna: 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
Aikaikkuna: 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)
Aikaikkuna: 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

Toissijaiset tulostoimenpiteet

Tulosmittaus
Toimenpiteen kuvaus
Aikaikkuna
Part A:Single-dose pharmacokinetic (PK) parameters:Peak Plasma Concentration (Cmax)
Aikaikkuna: 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
Aikaikkuna: 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
Aikaikkuna: 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)
Aikaikkuna: 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)
Aikaikkuna: 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
Aikaikkuna: 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)
Aikaikkuna: 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
Aikaikkuna: 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)
Aikaikkuna: 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)
Aikaikkuna: 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)
Aikaikkuna: 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

Muut tulostoimenpiteet

Tulosmittaus
Aikaikkuna
Part A, part B and part C: Pharmacodynamic parameters-protein C
Aikaikkuna: 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)
Aikaikkuna: 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

Yhteistyökumppanit ja tutkijat

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Opintojen ennätyspäivät

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