Status:

TERMINATED

Immune Tolerance Induction in Haemophilia A Patients Using Wilate or Nuwiq

Lead Sponsor:

Octapharma

Conditions:

Hemophilia A

Eligibility:

MALE

Brief Summary

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 induct...

Eligibility Criteria

Inclusion

  • 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

  • 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

Key Trial Info

Start Date :

June 28 2018

Trial Type :

OBSERVATIONAL

Allocation :

ACTUAL

End Date :

November 20 2020

Estimated Enrollment :

14 Patients enrolled

Trial Details

Trial ID

NCT03344003

Start Date

June 28 2018

End Date

November 20 2020

Last Update

August 9 2024

Active Locations (3)

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Page 1 of 1 (3 locations)

1

Stollery children's hospital, University of Alberta

Edmonton, Alberta, Canada

2

Children's Hospital of Eastern Ontario

Ottawa, Ontario, Canada

3

Hamilton Health Science center

Toronto, Ontario, Canada