Safety,Efficacy and Pharmacokinetics Evaluation of SCT800 in Previously Treated Paediatric Patients With Severe Haemophilia A.

Brief Title

Safety,Efficacy and Pharmacokinetics Evaluation of SCT800 in Previously Treated Paediatric Patients With Severe Haemophilia A.

Official Title

A Multicenter Phase III Uncontrolled Open-label Trial to Evaluate Safety and Efficacy and Pharmacokinetics of Recombinant Human Coagulation Factor VIII (SCT800) in Previously Treated Paediatric Patients With Severe Haemophilia A.

Brief Summary

      This study is a multicenter phase III uncontrolled open-label trial to evaluate the
      efficacy,safety and pharmacokinetics of SCT800 in regular prophylaxis and perioperative
      treatment in patients (<12 years old) 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 Visit 1 and prophylaxis with
      SCT800 shall continue for 24 consecutive weeks.

Study Phase

Phase 3

Study Type

Interventional

Primary Outcome

Annualized Bleeding Rate

Secondary Outcome

 Annualized joint bleeding rate

Condition

Hemophilia A

Intervention

Recombinant Human Coagulation FVIII

Study Arms / Comparison Groups

 Recombinant Human Coagulation FVIII

Description:  Participant receivedSCT800 for prophylaxis with 25 - 50 IU/kg injection once every other day or three times per week for 6 months.

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information

Recruitment Status

Drug

Estimated Enrollment

Start Date

December 20, 2019

Completion Date

September 20, 2020

Primary Completion Date

September 20, 2020

Eligibility Criteria

        Inclusion Criteria:

          -  Aged <12 years ;

          -  Male severe (central laboratory tested FVIII:C <1%) hemophilia A patients;

          -  Previously received FVIII treatment (prophylactic or bleeding treatment), have the
             relevant records and are verified to have accumulated EDs ≥150 days（6≤age<12years
             old）and EDs >50 days（age <6 years old）;

          -  The bleeding treatment records of at least 3 months before screening can be obtained;

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

          -  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;

          -  Platelet count <100 × 109/L;

          -  Clinical liver function test ((glutamic-pyruvic transaminase, glutamic-pyruvic
             transaminase) ≥ five times the upper limit of normal (ULN) or clinical kidney function
             test （creatinine) ≥ two times the ULN;

          -  International normalized ratio (INR) >1.5;

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

          -  Patients who used any anticoagulant or anti-platelet treatment (including
             non-steroidal anti-inflammatory drugs [NSAIDs]) within 1 weeks before the first drug
             administration or who regularly (e.g., daily, every other day) use anticoagulant or
             anti-platelet treatment within the clinical trial period;

          -  Patients who used immunomodulator(e.g., immunoglobulin,
             corticosteroids,alpha-interferon, prednisone [>10 mg/day and >7 days], or comparable
             drugs, other than anti-retroviral chemotherapy) within two weeks before the first
             administration of the study drug or during the clinical trial period;

          -  Administration of any cryoprecipitate, whole blood or plasma within 30 days prior to
             administration of study drug.

          -  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;

Gender

Male

Ages

N/A - 12 Years

Accepts Healthy Volunteers

No

Contacts

, ,

Administrative Informations

NCT ID

NCT03947320

Organization ID

SCT800-A303

Responsible Party

Sponsor

Study Sponsor

Sinocelltech Ltd.

Study Sponsor

, ,

Verification Date

May 2019