Therapy of Refractory Inhibitors with immUne Modulation in Patients with Hemophilia. The TRIUMPH trial

Phase 2

Withdrawn

• Conditions: Haemophilia A; FVIII deficiency; 10064477; 10027665

• Registration Number: NL-OMON42326
• Lead Sponsor: niversitair Medisch Centrum Utrecht

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Last Update Posted: 23 April 2024

Recruitment & Eligibility

• Status: Withdrawn
• Sex: Not specified
• Target Recruitment: 5

Inclusion Criteria

Adult patients with mild, moderate or severe hemophilia A with a current anti-FVIII inhibitor that have failed previous regular ITI, independent of titre height.

Exclusion Criteria

- Patients with active, severe or uncontrolled infection
- HIV positivity and/or CD4 < 400 mm3/ml
- Significant hepatic dysfunction (total bilirubin * 30 µmol/l or transaminases * 2.5x upper normal limit)
- Significant renal dysfunction requiring hemodialysis
- Intolerance of exogenous protein administration
- Currently participating in interventional hemophilia studies
- Known uncontrolled toxicity for DMSO
- Any psychological, familial, sociological and/or geographical condition potentially hampering compliance with the study protocol and follow-up schedule
- Life expectancy <3 years
- History of active cancer during the past 5 years, except basal carcinoma of the skin

Study & Design

• Study Type: Interventional
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
<p>Primary objective is to evaluate the efficacy, safety and feasibility of<br /><br>combined immune modulation with rituximab, ITI and MSCs in terms of eradication<br /><br>of FVIII inhibitory activity in hemophilia A patients.</p><br>

Secondary Outcome Measures

Name	Time	Method
<p>Secondary endpoints are to evaluate the immune changes during the TRIUMPH<br /><br>protocol in terms of T-cell and B-cell proliferation and modulation, time to<br /><br>Complete Response and Partial Response, time to Relapse and adverse events</p><br>