Immune Tolerance Induction in Haemophilia A Patients Using Wilate or Nuwiq (PREVAIL)

Octapharma

Status

Terminated

Conditions

Hemophilia A

Treatments

Drug: Wilate or Nuwiq

Study type

Observational

Funder types

Industry

Identifiers

NCT03344003

WIL-26

Details and patient eligibility

About

Uncontrolled, multi-centre, non-interventional study with a prospective and a retrospective cohort, to evaluate the efficacy of Wilate or Nuwiq in achieving complete or partial immune tolerance induction (ITI) success in severe and moderate haemophilia A patients with inhibitors

Enrollment

14 patients

Sex

Male

Volunteers

No Healthy Volunteers

Inclusion criteria

Male patients of any age with moderate or severe haemophilia A.
Patients with a first occurrence of inhibitors, inhibitors refractory to previous ITI attempt(s), or relapsed inhibitors to FVIII, with an inhibitor titre of ≥0.6 BU measured on 2 separate occasions at least 2 weeks apart.
Informed written consent from the patient and/or the patient's parent(s) or legal guardian(s)

For patients in the prospective cohort:

Patients who are currently on Wilate or Nuwiq ITI, have just initiated ITI, or are planned to initiate ITI treatment with Wilate or Nuwiq.

For patients in the retrospective cohort:

Patients having received Wilate or Nuwiq ITI before entry into this study. Retrospective data will be collected for a maximum of 3 years before enrolment into the study. To be eligible, the following information is needed:
Wilate or Nuwiq treatment details (start date, dose, treatment frequency, and dose change).
Reliably documented bleeding frequency.
FVIII inhibitor titres.
FVIII half-life.
FVIII IVR.

Exclusion criteria

Patients who meet any of the following criteria are not eligible for the study:

Congenital or acquired bleeding disorders other than haemophilia A.
A history of hypersensitivity to blood products and/or plasma-derived FVIII concentrates.
Inability to speak/read English or French well enough to provide consent and adhere to the study.
People who are receiving other non-factor therapies, e.g. concizumab