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Safety and Efficacy of Long-term Treatment With SCT800 in Previously Treated Hemophilia A Patients.

Sponsor
Sinocelltech Ltd. (Industry)
Overall Status
Recruiting
CT.gov ID
NCT03947567
Collaborator
(none)
240
Enrollment
1
Location
1
Arm
68.2
Anticipated Duration (Months)
3.5
Patients Per Site Per Month

Study Details

Study Description

Brief Summary

This study is a multi-center, open-label, extension trial to evaluate the safety, efficacy of SCT800 in long term prophylaxis and on-demand treatment in patients with severe hemophilia A who have been previously treated with coagulation factor VIII(FVIII) . This study includes two phases: the screening period and prophylaxis period.Prophylaxis with 25 - 50 IU/kg of SCT800 shall be administered once every other day or three times per week starting from V1, on-demand treatment is given when bleeding episodes occur. The study period is 120 consecutive weeks.

Condition or Disease Intervention/Treatment Phase
  • Drug: Recombinant Human Coagulation FVIII
 Phase 4

Study Design

Study Type:
Interventional
Anticipated Enrollment :
240 participants
Allocation:
N/A
Intervention Model:
Single Group Assignment
Masking:
None (Open Label)
Primary Purpose:
Prevention
Official Title:
A Multicenter, Open, Extension Trial to Evaluate Safety and Efficacy of Recombinant Human Coagulation Factor VIII (SCT800) During Long Term Treatment in Previously Treated Patients With Severe Haemophilia A .
Actual Study Start Date :
Jul 26, 2019
Anticipated Primary Completion Date :
Apr 1, 2025
Anticipated Study Completion Date :
Apr 1, 2025

Arms and Interventions

Arm Intervention/Treatment
Experimental: Recombinant Human Coagulation FVIII

Drug: Recombinant Human Coagulation FVIII
Participants received SCT800 for prophylaxis and/or on-demand treatment for 120 weeks.
Other Names:
  • SCT800

    		•

    Outcome Measures

    Primary Outcome Measures

    1. Incidence of FVIII inhibitors [up to 120 weeks]

      The Nijmegen-Bethesda assay shall be used to monitor the production of FVIII inhibitors during the trial.

    Secondary Outcome Measures

    1. Annualized Bleeding Rate [up to 120 weeks]

      Annualized Bleeding Rate（ABR） can be calculated using the following formula: Number of bleeding events in efficacy evaluation period/(number of days in treatment period/365.25)

    2. FVIII incremental in-vivo recovery [up to 120 weeks]

      Incremental recovery is determined as the peak factor level recorded in the first hour after infusion and is reported as [IU/ml]/[IU/kg]

    3. Bleeding event treatment efficacy [up to 120 weeks]

      The investigator shall evaluate the hemostatic effect after the treatment of every bleeding event of subjects based on a four-point scale(excellent, good, moderate, not relieved).

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    N/A and Older
    Sexes Eligible for Study:
    Male
    Accepts Healthy Volunteers:
    No
    Inclusion Criteria:

    • Male patients who are diagnosed with severe (laboratory tested FVIII:C <2%) hemophilia A;

    • Patients Who Completed trial of SCT800-A302 or SCT800-A 303 and New PTP(previously treated patients)(Previously received FVIII prophylactic or bleeding treatment, have the relevant records and are verified to have accumulated exposures days( EDs) ≥150 days;

    • Negative FVIII inhibitor assay results (laboratory tested Nijmegen-Bethesda assay result <0.6 BU(Bethesda unit)/mL);

    • The treatment records of at least 50EDs before screening can be obtained;

    • HIV negative; if HIV positive, the viral load <200 particles/uL or <400,000 copies/mL, and HIV patients must satisfy CD4+ count >200/μL;

    • The patient or his guardian voluntarily signed the Informed Consent Form.

    Exclusion Criteria:

    • Known allergy to any coagulation factor VIII or any excipient; known allergy to bovine, rodent or hamster bovine;

    • Has a history or family history of blood coagulation factor VIII inhibitor;

    • Patients with other coagulation dysfunction diseases in addition to hemophilia A;

    • Patients with other clinically significant diseases, alcoholism, drug abuse, mental disorders or intellectual disabilities;

    • Patients with other severe or clinical significant diseases verified by the investigator to be unable to benefit from the clinical study;

    • Patients who participated in other clinical studies within one month before the first drug administration (except FVIII trials) and patients who participated in other FVIII clinical trials after signing the Informed Consent Form;

    Contacts and Locations

    Locations

    Site City State Country Postal Code
    1 Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College Tianjin China

    Sponsors and Collaborators

    • Sinocelltech Ltd.

    Investigators

    • Principal Investigator: Renchi Yang, Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College

    Study Documents (Full-Text)

    None provided.

    More Information

    Publications

    None provided.
    Responsible Party:
    Sinocelltech Ltd.
    ClinicalTrials.gov Identifier:
    NCT03947567
    Other Study ID Numbers:
    • SCT800-A401
    First Posted:
    May 13, 2019
    Last Update Posted:
    Apr 28, 2020
    Last Verified:
    May 1, 2019
    Studies a U.S. FDA-regulated Drug Product:
    No
    Studies a U.S. FDA-regulated Device Product:
    No
    Additional relevant MeSH terms:
    Hemophilia A

    Study Results

    No Results Posted as of Apr 28, 2020