Safety/Efficacy Study to Assess Whether FVIII/VWF Concentrate Can Induce Immune Tolerance in Haemophilia A Patients

• Status: Unknown status
• Trial end date: 2020-01-01
• Target enrollment: 0
• Participant gender: Male

Summary

The purpose of this study is to assess the role of a FVIII/VWF complex concentrate (Emoclot) in successfully inducing immune tolerance (I.T.I.) in patients with Haemophilia A with inhibitors, including patients at high risk of failure.

• Phase: Phase 4
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico
• Collaborator: Sintesi Research Srl
• Treatments: Factor VIII

Criteria

Inclusion Criteria:

1. Subjects (his/her parent/legal representative), must have given a written informed consent.

2. Male children: age <12 years.

3. Severe or moderate Haemophilia A (FVIII <2%).

4. High responders (clinical history of inhibitor peak > 5BU) or low-responders (clinical history of inhibitor peak < 5 BU) with potential bleedings, assessed by responsible physicians as not to be treated with high FVIII doses.

5. Any level of inhibitor at study enrollment.

6. Willingness and ability to participate in the study.

7. No other experimental treatments (involving or not FVIII concentrates).

Exclusion Criteria:

1. Any clinically relevant abnormality, in hematological, biochemical and urinary routine examinations, or any condition or treatment which in the investigator's opinion, makes the patient not eligible for the study.

2. Intolerance to active substances or to any of the excipients of FVIII / VWF concentrates.

3. Concomitant systemic treatment with immunosuppressive drugs.