Immune Tolerance Induction study in patients with severe type A haemophilia with inhibitor after failure of a previous induction of immune tolerance with FVIII concentrates without Von Willebrand factor. (RESIST-EXPERIENCED)

Phase 4

Withdrawn

• Conditions: Factor VIII deficiency; Haemophilia; 10064477; 10005330

• Registration Number: NL-OMON35902
• Lead Sponsor: niversitair Medisch Centrum Utrecht

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Last Update Posted: 29 April 2024

Recruitment & Eligibility

• Status: Withdrawn
• Sex: Not specified
• Target Recruitment: 3

Inclusion Criteria

- severe hemophilia A (FVIII<1%)
- male patients, 18 years or older;
- high responders (peak inhibitor levels > 5 BU);
- any inhibitor level at study enrollment
- with ability and willingness to participate to the study;
- previous ITI course of at least 9 months with a VWF-free FVIII concentrate (recombinant FVIII and/or monoclonally purified FVIII) at any dosage (patients who initially succeeded to clear the inhibitor and then relapsed can be included)

Exclusion Criteria

- Concomitant systemic treatment with drugs with immunosuppressive side effects
- Concomitant experimental treatment
- Previous history of myocardial infarction and/or cerebral stroke
- High risk of cardiovascular, cerebrovascular or other thromboembolic events as deemed by the treating clinician

Study & Design

• Study Type: Interventional
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
<p>abolition of the inhibitor to < 0.6 BU within 33 months of ITI with a factor<br /><br>VIII recovery >= 66% and half-life >= 6 hrs</p><br>

Secondary Outcome Measures

Name	Time	Method
<p>Maintenance of immune tolerance<br /><br>Time to achieve success<br /><br>Safety<br /><br>Compliance to treatment<br /><br>Cost of care</p><br>