Study to evaluate if switching from plasma-derived factor VIII products to recombinant factor VIII products lead to an increased inhibitor development in patients with hemophilia A

Phase 1

• Conditions: Severe hemophilia A; MedDRA version: 20.0 Level: LLT Classification code 10018938 Term: Haemophilia A (Factor VIII) System Organ Class: 100000004850; Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

• Registration Number: EUCTR2017-005197-19-IT
• Lead Sponsor: FONDAZIONE IRCCS CA' GRANDA OSPEDALE MAGGIORE POLICLINICO

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2019-03-14
• Last Update Posted: 30 April 2019

Recruitment & Eligibility

• Status: Authorised-recruitment may be ongoing or finished
• Sex: Not specified
• Target Recruitment: 200

Inclusion Criteria

1. Male subjects
2. Any ethnicity
3. Age < 6 years
4. Severe haemophilia A (FVIII:C < 1% as confirmed at enrolment by the central laboratory (those patients diagnosed locally as severe but subsequently found to have measurable FVIII ( = 1%) will be considered screening failure).
5. Previously untreated patients (0 ED to any FVIII concentrates or blood products) or minimally treated (< 5 EDs) with blood components (whole blood, packed red blood cells, platelets, cryoprecipitate or fresh frozen plasma)
6. Negative inhibitor measurement at both local and central laboratory at screening, i.e., before any treatment with a FVIII concentrate
7. Ability to comply with study requirements
8. Signed informed consent of legal tutors

Are the trial subjects under 18? yes
Number of subjects for this age range: 1
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

1. Previous history of inhibitor
2. Other congenital or acquired bleeding defects
3. Plasma FVIII level = 1% as assayed at the central laboratory. Patients diagnosed locally as severe and not confirmed by the central evaluation will be considered screening failure and excluded from the study.
4. Concomitant congenital or acquired immunodeficiencies
5. Concomitant treatment with systemic immunosuppressive drugs

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified