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.

Sinocelltech Ltd.0 sites70 target enrollmentDecember 20, 2019

ConditionsHemophilia A

InterventionsRecombinant Human Coagulation FVIII

DrugsRecombinant Human Coagulation FVIII

Overview

Phase: Phase 3
Intervention: Recombinant Human Coagulation FVIII
Conditions: Hemophilia A
Sponsor: Sinocelltech Ltd.
Enrollment: 70
Primary Endpoint: Annualized Bleeding Rate
Last Updated: 6 years ago

Overview Investigators Eligibility Criteria Arms & Interventions Outcomes Similar Trials

Overview

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.

Registry

clinicaltrials.gov

Start Date

December 20, 2019

End Date

September 20, 2020

Last Updated

6 years ago

Study Type

Interventional

Study Design

Single Group

Sex

Male

Investigators

Sponsor

Sinocelltech Ltd.

Responsible Party

Sponsor

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;

Arms & Interventions

Recombinant Human Coagulation FVIII

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

Intervention: Recombinant Human Coagulation FVIII

Outcomes

Primary Outcomes

Annualized Bleeding Rate

Time Frame: up to 24 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)

Secondary Outcomes

Clearance(Predose within 30 min，15 min±2 min、1 hour±5 min，10 hours±30 min，24 hours±1hours and 48 hours±2 hours post-dose)
Annualized joint bleeding rate(up to 24 weeks)
FVIII incremental in-vivo recovery(Predose within 30 min，15 min±2 min)
Elimination Half Life(Predose within 30 min，15 min±2 min、1 hour±5 min，10 hours±30 min，24 hours±1hours and 48 hours±2 hours post-dose)
Bleeding event treatment efficacy(up to 24 weeks)