Evaluation of SCT800 in Prophylaxis Therapy on Previous Treated Patients With Severe Hemophilia A

• Status: Completed
• Trial end date: 2020-01-16
• Target enrollment: 0
• Participant gender: Male

Summary

This study is a multi-center, open-label, single-arm 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:C) . 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 and prophylaxis with SCT800 shall continue for 24 consecutive weeks.

• Phase: Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Sinocelltech Ltd.
• Collaborators: Chinese Academy of Medical Sciences; Parexel; Q2 Solutions
• Treatments: Factor VIII

Criteria

Inclusion Criteria:

• Aged ≥12 years old and ≤65 years old;

• Male patients who are clinically diagnosed with severe (laboratory tested FVIII:C <1%) hemophilia A, including historical FVIII:C <1%;

• Previously received FVIII treatment (prophylactic or bleeding treatment), have the relevant records and are verified to have accumulated exposures days( EDs) ≥150 days;

• 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(Bethesda unit)/mL);

• The prothrombin time is normal or international normalized ratio (INR) ≤1.5; 7. Platelet count ≥100 × 109/L;

Exclusion Criteria:

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

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

• Clinical liver function test (glutamic-pyruvic transaminase, glutamic-pyruvic transaminase) ≥ three times the upper limit of normal (ULN) or clinical kidney function test (blood urea nitrogen, creatinine) ≥ three times the ULN;

• Patients clinically diagnosed with active Hepatitis B or Hepatitis C;

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

• Patients with severe heart disease, including myocardial infarction and cardiac dysfunction of class III or above;

• Patients who previously experienced intracranial bleeding;