Multicenter, Randomized Phase III Inhibitor Prevention Trial, Comparing Eloctate vs. Emicizumab to Prevent Inhibitor Formation in Severe Hemophilia A

PRO19040140

This is a multi-center randomized phase III clinical trial, the Inhibitor Prevention Trial, in which Eloctate will be compared with Emicizumab, using adaptive design, to prevent inhibitors in patients with severe hemophilia A.

2021-10-11

2022-04-06

2022-06-16

Terminated

Early phase I

1

Inclusion Criteria: 1. Male children \>= 4 months and up to 4 years of age. 2. Severe hemophilia A (FVIII \< 0.01 U/ml). 3. No evidence of an inhibitor i.e. anti-FVIII \< 0.6 B.U. 4. No more than 3 FVIII exposures (Factor VIII concentrate, cryoprecipitate, or fresh frozen plasma), including circumcision. Exclusion Criteria: 1. Acquired hemophilia or any bleeding disorder other than hemophilia A. 2. Treatment with clotting factor or emicizumab previously. 3. Use of an experimental drug(s). 4. Surgery anticipated in the next 48 weeks. 5. Life expectancy less than 5 years. 6. Parent/caretaker unable or unwilling to keep a personal diary of bleeding frequency and study drug treatment, make monthly visits and blood draws at weeks 4, 12, 24, 36, and 48. 7. Other illness, condition or reason in the opinion of the investigator that would make the patient unsuitable for the trial.

{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE3'], 'designInfo': {'allocation': 'RANDOMIZED', 'interventionModel': 'PARALLEL', 'interventionModelDescription': 'This is a phase III open-label, randomized controlled trial comparing two drugs in the prevention of hemophilia inhibitor formation.', 'primaryPurpose': 'PREVENTION', 'maskingInfo': {'masking': 'NONE'}}, 'enrollmentInfo': {'count': 1, 'type': 'ACTUAL'}}

2023-04-11