Phase III Multi-Center, Randomized, Controlled Inhibitor Eradication Trial, Comparing Eloctate Immune Tolerance Induction (ITI) Plus Emicizumab vs. Eloctate ITI Alone to Eradicate Inhibitor Formation in Severe Hemophilia A

PRO19070080

This is a multi-center randomized phase III clinical trial, the Inhibitor Eradication Trial, in which Eloctate ITI plus Emicizumab will be compared with Eloctate ITI alone to eradicate inhibitors in severe hemophilia A.

2021-11-01

2022-06-27

2022-06-27

Terminated

Early phase I

1

Inclusion Criteria: 1. Male adults or children \> 4 months of age. 2. Severe hemophilia A (FVIII \< 0.01 U/ml). 3. Current or past high-responding inhibitor, anti-FVIII \>= 5.0 B.U., ITI-refractory or ITI-naive. Exclusion Criteria: 1. Acquired hemophilia or any bleeding disorder other than hemophilia A. 2. Current use of Emicizumab, or if used, \> 8 weeks since last treatment. 3. Use of an experimental drug(s). 4. Surgery anticipated in the next 48 weeks. 5. Life expectancy less than 5 years. 6. Patient/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, 8, 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 immune tolerance induction with one drug, with or without a second drug in the eradication of hemophilia inhibitors.', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'NONE'}}, 'enrollmentInfo': {'count': 1, 'type': 'ACTUAL'}}

2023-02-21