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- Essai clinique NCT07644832
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 juin 2026 mis à jour par: 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).
Aperçu de l'étude
Statut
Recrutement
Les conditions
Intervention / Traitement
Type d'étude
Interventionnel
Inscription (Estimé)
76
Phase
- Phase 2
- La phase 1
Contacts et emplacements
Cette section fournit les coordonnées de ceux qui mènent l'étude et des informations sur le lieu où cette étude est menée.
Coordonnées de l'étude
- Nom: Research and Development
- Numéro de téléphone: 862122130888
- E-mail: hanyu@raas-corp.com
Lieux d'étude
-
-
-
Changsha, Chine
- Recrutement
- Xiangya Hospital of Central South University
-
Hefei, Chine
- Recrutement
- The First Affiliated Hospital of University of Science and Technology of China
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Jinan, Chine
- Recrutement
- Jinan Central Hospital
-
Lanzhou, Chine
- Recrutement
- The First Hospital of Lanzhou University
-
Shanghai, Chine
- Complété
- Ruijin Hospital Shanghai Jiaotong University School of Medicine
-
Taiyuan, Chine
- Recrutement
- The Second Hospital of Shanxi Medical University
-
Tianjin, Chine
- Complété
- Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences
-
Xi'an, Chine
- Recrutement
- Xian Central Hospital
-
Zhengzhou, Chine
- Recrutement
- Zhengzhou People's Hospital
-
-
Critères de participation
Les chercheurs recherchent des personnes qui correspondent à une certaine description, appelée critères d'éligibilité. Certains exemples de ces critères sont l'état de santé général d'une personne ou des traitements antérieurs.
Critère d'éligibilité
Âges éligibles pour étudier
- Adulte
- Adulte plus âgé
Accepte les volontaires sains
Non
La description
Inclusion Criteria:
- 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.
Exclusion Criteria:
- 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;
- Received whole blood or plasma therapy within 2 weeks prior to first dosing;
- Received emicizumab treatment within 6 months prior to first dosing;
- Received or planned to receive vaccination within 4 weeks prior to first dosing or during the trial;
- Underwent major surgery (e.g., orthopedic surgery, abdominal surgery) within 1 month prior to first dosing, or planned to undergo surgery during the study;
- Enrolled in another clinical trial within 1 month prior to first dosing;
- 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);
- Psychiatric illness or significant mental impairment, or incapacity or lack of cognitive ability due to other reasons;
- 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);
- 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);
- 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.
Plan d'étude
Cette section fournit des détails sur le plan d'étude, y compris la façon dont l'étude est conçue et ce que l'étude mesure.
Comment l'étude est-elle conçue ?
Détails de conception
- Objectif principal: Traitement
- Répartition: Randomisé
- Modèle interventionnel: Affectation parallèle
- Masquage: Aucun (étiquette ouverte)
Armes et Interventions
Groupe de participants / Bras |
Intervention / Traitement |
|---|---|
|
Expérimental: 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 sera administré par injection SC.
SR604 will be administered as SC injection
|
|
Expérimental: 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 sera administré par injection SC.
SR604 will be administered as SC injection
|
|
Expérimental: 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 sera administré par injection SC.
SR604 will be administered as SC injection
|
Que mesure l'étude ?
Principaux critères de jugement
Mesure des résultats |
Description de la mesure |
Délai |
|---|---|---|
|
Part A: Incidence of AEs/SAEs/AESI
Délai: 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
Délai: 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
Délai: 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)
Délai: 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
|
Mesures de résultats secondaires
Mesure des résultats |
Description de la mesure |
Délai |
|---|---|---|
|
Part A:Single-dose pharmacokinetic (PK) parameters:Peak Plasma Concentration (Cmax)
Délai: 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
Délai: 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
Délai: 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
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Part B/ Part C:Treated annualized menorrhagia bleeding rate (applicable only to reproductive-age female patients with congenital FVII deficiency and active menstruation)
Délai: 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)
Délai: 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
Délai: 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)
Délai: 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
Délai: 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)
Délai: 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)
Délai: 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)
Délai: 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
|
Autres mesures de résultats
Mesure des résultats |
Délai |
|---|---|
|
Part A, part B and part C: Pharmacodynamic parameters-protein C
Délai: 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)
Délai: 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
|
Collaborateurs et enquêteurs
C'est ici que vous trouverez les personnes et les organisations impliquées dans cette étude.
Parrainer
Dates d'enregistrement des études
Ces dates suivent la progression des dossiers d'étude et des soumissions de résultats sommaires à ClinicalTrials.gov. Les dossiers d'étude et les résultats rapportés sont examinés par la Bibliothèque nationale de médecine (NLM) pour s'assurer qu'ils répondent à des normes de contrôle de qualité spécifiques avant d'être publiés sur le site Web public.
Dates principales de l'étude
Début de l'étude (Réel)
31 mai 2024
Achèvement primaire (Estimé)
31 décembre 2026
Achèvement de l'étude (Estimé)
31 décembre 2026
Dates d'inscription aux études
Première soumission
26 mai 2026
Première soumission répondant aux critères de contrôle qualité
8 juin 2026
Première publication (Réel)
12 juin 2026
Mises à jour des dossiers d'étude
Dernière mise à jour publiée (Réel)
12 juin 2026
Dernière mise à jour soumise répondant aux critères de contrôle qualité
8 juin 2026
Dernière vérification
1 mai 2026
Plus d'information
Termes liés à cette étude
Termes MeSH pertinents supplémentaires
- Maladies génétiques, innées
- Maladies hématologiques
- Troubles de la coagulation sanguine
- Troubles hémorragiques
- Maladies génétiques liées à l'X
- Troubles de la coagulation sanguine, héréditaires
- Troubles des protéines de la coagulation
- Maladies et anomalies congénitales, héréditaires et néonatales
- Maladies hémiques et lymphatiques
- Hémophilie A
- Hémophilie B
- Déficit en facteur VII
Autres numéros d'identification d'étude
- LS-SR604-Ⅰ01
Plan pour les données individuelles des participants (IPD)
Prévoyez-vous de partager les données individuelles des participants (DPI) ?
NON
Informations sur les médicaments et les dispositifs, documents d'étude
Étudie un produit pharmaceutique réglementé par la FDA américaine
Non
Étudie un produit d'appareil réglementé par la FDA américaine
Non
Ces informations ont été extraites directement du site Web clinicaltrials.gov sans aucune modification. Si vous avez des demandes de modification, de suppression ou de mise à jour des détails de votre étude, veuillez contacter register@clinicaltrials.gov. Dès qu'un changement est mis en œuvre sur clinicaltrials.gov, il sera également mis à jour automatiquement sur notre site Web .