A Phase II Exploratory Study of SR604 Injection Evaluating the Safety and Efficacy in the Treatment of Congenital Coagulation Factor VII Deficiency
An Open-Label, Multicenter, Phase II Clinical Trial to Evaluate the Efficacy, Safety, and Pharmacokinetic Characteristics of SR604 Injection in Patients With Congenital Coagulation Factor VII Deficiency
Panoramica dello studio
Stato
Stato
Condizioni
Condizioni
Intervento / Trattamento
Intervento / Trattamento
Tipo di studio
Tipo di studio
Iscrizione (Stimato)
Iscrizione
Fase
Fase
- Fase 2
Contatti e Sedi
Contatto studio
Contatto studio
- Nome: Research and Development
- Numero di telefono: 862122130888
- Email: hanyu@raas-corp.com
Luoghi di studio
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Tianjin, Cina
- Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences
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Criteri di partecipazione
Criteri di ammissibilità
Criteri di ammissibilità
Età idonea allo studio
- Adulto
- Adulto più anziano
Accetta volontari sani
Descrizione
Inclusion Criteria:
- Age ≥12 years and ≤65 years at the time of signing the informed consent form; both genders eligible;
- Clinically diagnosed with congenital coagulation Factor VII deficiency, with historical or screening FVII activity <10%, and ≥2 treated new-onset bleeding events within 3 months prior to enrollment;
- No active bleeding symptoms prior to first administration of SR604 Injection;
- The subject and/or legal representative and impartial witness have signed the informed consent form, indicating voluntary agreement to participate in this trial, to provide biological samples for testing as required by the protocol, and to comply with the planned study visits;
- Female subjects (post-menarche) must have a negative serum pregnancy test (HCG) during the screening period; subjects with childbearing potential (females post-menarche or males post-spermarche) must agree to use highly effective contraceptive measures throughout the study period.
Exclusion Criteria:
- Known history of hypersensitivity to the study drug formulation or any of its components;
- Intolerance to subcutaneous injection or other local skin abnormalities or dermatoses that may affect drug administration or safety assessment;
Meeting any one of the following criteria during screening:
- Hemoglobin <60 g/L;
- Platelet count <100×10⁹/L;
- Abnormal hepatic or renal function: 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 for anti-human immunodeficiency virus (HIV) antibodies.
- Any bleeding disorder other than congenital Factor VII deficiency, or other conditions causing significantly abnormal coagulation parameters (e.g., hemophilia A or B, von Willebrand disease, platelet disorders, vitamin K deficiency, etc.);
- Protein C deficiency or Protein S deficiency;
- History of thrombosis, family history of thrombosis, or history of thrombophilia;
- Intracranial hemorrhage within 2 years prior to signing the informed consent form;
- Severe cardiac disease, such as unstable angina, congestive heart failure (New York Heart Association class ≥III), serious arrhythmia (QTc interval >450 ms, corrected by Fridericia formula), uncontrolled hypertension (systolic blood pressure ≥160 mmHg or diastolic blood pressure ≥100 mmHg), etc.;
- Female patients with menstrual abnormalities caused by organic gynecological conditions (e.g., uterine fibroids, endometriosis, adenomyosis, etc.);
- Received Factor VII-containing products within 48 hours prior to first administration of SR604 Injection; received whole blood or plasma transfusion within 2 weeks prior to first administration of SR604 Injection;
- Used any anticoagulants, antifibrinolytics, or agents affecting platelet function (including chemical drugs, biological products, or traditional Chinese medicine), including aspirin, within 1 week prior to screening, or required use of such agents during the treatment period;
- Underwent major surgery (defined as Grade III or IV surgery) within 1 month prior to signing the informed consent form, or planned to undergo surgery during the study period;
- Enrolled in other clinical trials within 1 month prior to signing the informed consent form;
- Miscarriage or pregnancy termination within 3 months prior to signing the informed consent form; pregnant or breastfeeding women;
- Mental illness or significant psychiatric disorder, or incapacity or lack of cognitive ability due to other causes;
Other conditions deemed by the investigator to be unsuitable for enrollment, such as alcoholism, anticipated poor subject compliance preventing completion of dosing and study follow-up, poorly controlled comorbid chronic diseases, or serious systemic diseases.
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Piano di studio
Come è strutturato lo studio?
Dettagli di progettazione
- Scopo principale: Trattamento
- Assegnazione: N / A
- Modello interventistico: Assegnazione parallela
- Mascheramento: Nessuno (etichetta aperta)
Numero di armi
Armi e interventi
Gruppo di partecipanti / ArmGruppo di partecipanti / Arm |
Intervento / TrattamentoIntervento / Trattamento |
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Sperimentale: SR604:Multiple-dose exploratory efficacy trial consists of 2 cohorts
Participants with FVII deficiency will receive SR604 dose 1/2 as multiple SC injections every 4-weeks
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SR604 verrà somministrato come iniezione SC.
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Cosa sta misurando lo studio?
Misure di risultato primarie
Misure di risultato primarie
Misura del risultato |
Lasso di tempo |
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Treated overall Annualized Bleeding Rate (ABR)
Lasso di tempo: Through 28 weeks of treatment.
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Through 28 weeks of treatment.
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Misure di risultato secondarie
Misure di risultato secondarie
Misura del risultato |
Lasso di tempo |
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Treated spontaneous annualized bleeding rate
Lasso di tempo: over 28 weeks of treatment
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over 28 weeks of treatment
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Treated overall annualized joint bleeding rate
Lasso di tempo: over 28 weeks of treatment
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over 28 weeks of treatment
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Overall annualized bleeding rate including treated and untreated bleeding events
Lasso di tempo: over 28 weeks of treatment,
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over 28 weeks of treatment,
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Annualized menorrhagia bleeding rate (menstruating females only)
Lasso di tempo: over 28 weeks of treatment
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over 28 weeks of treatment
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Change in PBAC score (menstruating females only);
Lasso di tempo: From baseline over 28 weeks of treatment
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From baseline over 28 weeks of treatment
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Change in EQ-5D-5L health index score
Lasso di tempo: From pre-treatment over 28 weeks of treatment
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From pre-treatment over 28 weeks of treatment
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Change in EQ-VAS score
Lasso di tempo: From baseline over 28 weeks of treatment;
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From baseline over 28 weeks of treatment;
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Subject overall satisfaction score with treatment efficacy
Lasso di tempo: over 28 weeks of treatment
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over 28 weeks of treatment
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Altre misure di risultato
Altre misure di risultato
Misura del risultato |
Lasso di tempo |
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Single-dose pharmacokinetic (PK) parameters:Time to Peak Plasma Concentration (Tmax)
Lasso di tempo: over 28 weeks of treatment
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over 28 weeks of treatment
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Single-dose pharmacokinetic (PK) parameters:Peak Plasma Concentration (Cmax)
Lasso di tempo: over 28 weeks of treatment
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over 28 weeks of treatment
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Single-dose pharmacokinetic (PK) parameters:Area Under the Concentration-Time Curve from Zero to Last Quantifiable Time Point (AUC0-t)
Lasso di tempo: over 28 weeks of treatment
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over 28 weeks of treatment
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Multiple-dose pharmacokinetic parameters-Time to Peak Plasma Concentration (Tmax)
Lasso di tempo: over 28 weeks of treatment
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over 28 weeks of treatment
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Multiple-dose pharmacokinetic parameters-Peak Plasma Concentration (Cmax)
Lasso di tempo: Over 28 weeks of treatment
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Over 28 weeks of treatment
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Safety and Immunogenicity:Incidence of AEs/SAEs/AESI,
Lasso di tempo: over 28 weeks of treatment
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over 28 weeks of treatment
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Incidence of drug-related AEs/SAEs/AESIs
Lasso di tempo: over 28 weeks of treatment
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over 28 weeks of treatment
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Safety and Immunogenicity: Anti-drug antibody (ADA) and neutralizing antibody (NAb) - number of subjects and incidence rate.
Lasso di tempo: over 28 weeks of treatment
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over 28 weeks of treatment
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Collaboratori e investigatori
Sponsor
Sponsor
Studiare le date dei record
Studia le date principali
Inizio studio (Stimato)
Inizio studio
Completamento primario (Stimato)
Completamento primario
Completamento dello studio (Stimato)
Completamento dello studio
Date di iscrizione allo studio
Primo inviato
Primo inviato
Primo inviato che soddisfa i criteri di controllo qualità
Primo inviato che soddisfa i criteri di controllo qualità
Primo Inserito (Effettivo)
Primo Inserito
Aggiornamenti dei record di studio
Ultimo aggiornamento pubblicato (Effettivo)
Ultimo aggiornamento pubblicato
Ultimo aggiornamento inviato che soddisfa i criteri QC
Ultimo aggiornamento inviato che soddisfa i criteri QC
Ultimo verificato
Ultimo verificato
Maggiori informazioni
Termini relativi a questo studio
Termini MeSH pertinenti aggiuntivi
- Malattie genetiche, congenite
- Malattie ematologiche
- Disturbi della coagulazione del sangue
- Disturbi emorragici
- Disturbi della coagulazione del sangue, ereditari
- Disturbi delle proteine della coagulazione
- Malattie e anomalie congenite, ereditarie e neonatali
- Malattie emiche e linfatiche
- Carenza di fattore VII
Altri numeri di identificazione dello studio
Altri numeri di identificazione dello studio
- LS-SR604-VII-II01
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