Rescue Immunotolerance Study in Induction of Immune Tolerance (ITI)-Experienced Patients (RES.I.S.T. Experienced)

• Status: Completed
• Trial end date: 2020-10-21
• Target enrollment: 0
• Participant gender: Male

Summary

The purpose of this study is to evaluate whether a concentrate containing both FVIII and von Willebrand Factor (VWF) given at a high dose will induce immune tolerance in subjects who have already experienced and failed ITI with VWF-free FVIII concentrates. The treatment on this study is expected to last up to 33 months.

• Phase: N/A
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: City of Hope Medical Center
• Collaborators: Biotest Pharmaceuticals Corporation; Charta Fondazione; Charta Foundation; CSL Behring; Grifols Biologicals Inc.; Grifols Biologicals, LLC; Grifols Therapeutics Inc.; Grifols Therapeutics LLC

Criteria

Inclusion Criteria:

1. severe hemophilia A (FVIII<1%) with high responding inhibitors (peak levels >5 BU)

2. male, any age;

3. any inhibitor level at study enrollment;

4. ability and willingness to participate to the study;

5. previous ITI course of at least 9 months with a VWF-free FVIII concentrate at any dosage, such as recombinant FVIII and/or monoclonally purified FVIII.

Exclusion Criteria:

1. concomitant systemic treatment with immunosuppressive drugs;

2. concomitant experimental treatment;

3. previous history of myocardial infarction and/or cerebral stroke