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

• Register: ClinicalTrials.gov
• Last refreshed on: 3 August 2015
• Main ID: NCT02479087
• Date of registration: 16/06/2015
• Prospective Registration: No
• Primary sponsor: Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico
• Public title: Safety/Efficacy Study to Assess Whether FVIII/VWF Concentrate Can Induce Immune Tolerance in Haemophilia A Patients ITI
• Scientific title: IMMUNE TOLERANCE INDUCTION, BY FACTOR VIII CONCENTRATE CONTAINING VON WILLEBRAND FACTOR, IN SEVERE OR MODERATE HAEMOPHILIA A PATIENTS WITH INHIBITORS
• Date of first enrolment: January 2015
• Target sample size: 20
• Recruitment status: Recruiting
• URL: https://clinicaltrials.gov/show/NCT02479087
• Study type: Interventional
• Study design: Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
• Phase: Phase 4

Countries of recruitment

Egypt, India

Contacts

• Name: Flora Peyvandi, MD
• Affiliation: Università di Milano, IRCCS Fondazione Ca' Granda Ospedale Maggiore Policlinico

• Name: Elena Santagostino, MD
• Affiliation: Centro Emofilia e Trombosi Angela Bianchi Bonomi, IRCCS Fondazione Ospedale Maggiore Policlinico

• Name: Pier Mannuccio Mannucci, MD
• Affiliation: IRCCS Fondazione Ca' Granda Ospedale Maggiore Policlinico

• Name: Pier Mannuccio Mannucci, MD

Key inclusion & exclusion 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.



Age minimum: N/A
Age maximum: 12 Years
Gender: Male

Health Condition(s) or Problem(s) studied

Hemophilia A

Intervention(s)

Drug: Plasma-derived FVIII/VWF concentrate

Primary Outcome(s)

Efficacy: evaluation of the success of IT induction [Time Frame: Up to33 months]

Secondary Outcome(s)

Efficacy evaluation - IT persistence [Time Frame: Up to 33 months+ 12 months FU]

Efficacy evaluation - Role of an immediate IT to delayed IT in IT induction. [Time Frame: Up to 33 months]

Efficacy evaluation - FVIII genetic defect role in IT achievement [Time Frame: Up to 33 months]

Efficacy evaluation - Time to achieve ITI [Time Frame: Up to 33 months]

Analysis of treatment compliance [Time Frame: Up to 33 months]

Safety (adverse events) [Time Frame: Up to 33 months]

Evaluation of the cost of therapy [Time Frame: Up to 33 months]

Secondary ID(s)

FCG-CNS-001

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Sintesi Research Srl