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
Studienübersicht
Status
Status
Bedingungen
Bedingungen
Intervention / Behandlung
Intervention / Behandlung
Studientyp
Studientyp
Einschreibung (Geschätzt)
Einschreibung
Phase
Phase
- Phase 2
Kontakte und Standorte
Studienkontakt
Studienkontakt
- Name: Research and Development
- Telefonnummer: 862122130888
- E-Mail: hanyu@raas-corp.com
Studienorte
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Tianjin, China
- Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences
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Teilnahmekriterien
Zulassungskriterien
Zulassungskriterien
Studienberechtigtes Alter
- Erwachsene
- Älterer Erwachsener
Akzeptiert gesunde Freiwillige
Beschreibung
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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Studienplan
Wie ist die Studie aufgebaut?
Designdetails
- Hauptzweck: Behandlung
- Zuteilung: N / A
- Interventionsmodell: Parallele Zuordnung
- Maskierung: Keine (Offenes Etikett)
Anzahl der Arme
Waffen und Interventionen
Teilnehmergruppe / ArmTeilnehmergruppe / Arm |
Intervention / BehandlungIntervention / Behandlung |
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Experimental: 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 wird als SC-Injektion verabreicht.
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Was misst die Studie?
Primäre Ergebnismessungen
Primäre Ergebnismessungen
Ergebnis Maßnahme |
Zeitfenster |
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Treated overall Annualized Bleeding Rate (ABR)
Zeitfenster: Through 28 weeks of treatment.
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Through 28 weeks of treatment.
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Sekundäre Ergebnismessungen
Sekundäre Ergebnismessungen
Ergebnis Maßnahme |
Zeitfenster |
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Treated spontaneous annualized bleeding rate
Zeitfenster: over 28 weeks of treatment
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over 28 weeks of treatment
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Treated overall annualized joint bleeding rate
Zeitfenster: 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
Zeitfenster: 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)
Zeitfenster: 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);
Zeitfenster: 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
Zeitfenster: 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
Zeitfenster: 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
Zeitfenster: over 28 weeks of treatment
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over 28 weeks of treatment
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Andere Ergebnismessungen
Andere Ergebnismessungen
Ergebnis Maßnahme |
Zeitfenster |
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Single-dose pharmacokinetic (PK) parameters:Time to Peak Plasma Concentration (Tmax)
Zeitfenster: 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)
Zeitfenster: 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)
Zeitfenster: 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)
Zeitfenster: 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)
Zeitfenster: 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,
Zeitfenster: 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
Zeitfenster: 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.
Zeitfenster: over 28 weeks of treatment
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over 28 weeks of treatment
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Mitarbeiter und Ermittler
Sponsor
Sponsor
Studienaufzeichnungsdaten
Haupttermine studieren
Studienbeginn (Geschätzt)
Studienbeginn
Primärer Abschluss (Geschätzt)
Primärer Abschluss
Studienabschluss (Geschätzt)
Studienabschluss
Studienanmeldedaten
Zuerst eingereicht
Zuerst eingereicht
Zuerst eingereicht, das die QC-Kriterien erfüllt hat
Zuerst eingereicht, das die QC-Kriterien erfüllt hat
Zuerst gepostet (Tatsächlich)
Zuerst gepostet
Studienaufzeichnungsaktualisierungen
Letztes Update gepostet (Tatsächlich)
Letztes Update gepostet
Letztes eingereichtes Update, das die QC-Kriterien erfüllt
Letztes eingereichtes Update, das die QC-Kriterien erfüllt
Zuletzt verifiziert
Zuletzt verifiziert
Mehr Informationen
Begriffe im Zusammenhang mit dieser Studie
Zusätzliche relevante MeSH-Bedingungen
Andere Studien-ID-Nummern
Andere Studien-ID-Nummern
- LS-SR604-VII-II01
Plan für individuelle Teilnehmerdaten (IPD)
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Studiert ein von der US-amerikanischen FDA reguliertes Arzneimittelprodukt
Studiert ein von der US-amerikanischen FDA reguliertes Geräteprodukt
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