Clinical trial NCT03947567: Safety and Efficacy of Long-term Treatment With SCT800 in Previously Treated Hemophilia A Patients.
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 .
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: PHASE4
Conditions: Hemophilia A
Lead sponsor: Sinocelltech Ltd.
Sponsor class: INDUSTRY
First posted: 2019-05-13
Enrollment: 240
Sex: MALE
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: PREVENTION

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.


Eligibility criteria: 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;

Primary outcomes:
measure: Incidence of FVIII inhibitors
timeFrame: up to 120 weeks
Secondary outcomes:
measure: Annualized Bleeding Rate
timeFrame: up to 120 weeks
measure: FVIII incremental in-vivo recovery
timeFrame: up to 120 weeks
measure: Bleeding event treatment efficacy
timeFrame: up to 120 weeks
Other outcomes:
Locations:
city: Tianjin
country: China
facility: Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College
Study officials:
affiliation: Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College
name: Renchi Yang
role: PRINCIPAL_INVESTIGATOR
References: