Study of First TIME Immunotolerance Induction in Severe Hemophilia A Patients With Inhibitor at High Risk of Failure: Comparison With FVIII Concentrates With or Without Von Willebrand Factor - RES.I.S.T. Naive

• Status: Withdrawn
• Trial end date: 2020-06-25
• Target enrollment: 0
• Participant gender: Male

Summary

This is a prospective, controlled, randomized, open label study, aimed at comparing FVIII/VWF concentrates with FVIII concentrates at 200 IU/kg daily in their ability to induce immune tolerance in Haemophilia A patients with high responding inhibitors and poor prognosis for success.

• 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
• Treatments: Factor VIII

Criteria

Inclusion Criteria:

1. severe hemophilia A (FVIII<1%);

2. male, any age;

3. high responders (peak inhibitor levels > 5 BU);

4. any inhibitor level at study enrolment;

5. ability and willingness to participate in the study;

6. at least one of the following risk factors for ITI failure:

• peak inhibitor titer > 200 BU

• titer at ITI start > 10 BU

• age > 7 years

• time between inhibitor occurrence and ITI > 2 years

7. absence of high risk of cardiovascular, cerebrovascular or other thromboembolic events as deemed by the treating clinician.

Exclusion Criteria:

1. concomitant systemic treatment with immunosuppressive drugs;

2. concomitant experimental treatment;

3. previous ITI attempt;

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