Clinical trial NCT03344003: Immune Tolerance Induction in Haemophilia A Patients Using Wilate or Nuwiq
Official title: Immune Tolerance Induction in Haemophilia A Patients Using Wilate or Nuwiq - A Canadian Study
Study type: OBSERVATIONAL
Overall status: TERMINATED
Conditions: Hemophilia A
Lead sponsor: Octapharma
Sponsor class: INDUSTRY
First posted: 2017-11-17
Enrollment: 14
Sex: MALE
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: OTHER
Sampling method: NON_PROBABILITY_SAMPLE
Study population: A total of at least 80 patients were planned, at least 40 in the prospective cohort and 40 in the retrospective cohort. Due to study termination, only 14 patients were enrolled.

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 induction (ITI) success in severe and moderate haemophilia A patients with inhibitors


Eligibility criteria: 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

Primary outcomes:
measure: Number of Moderate and Severe Haemophilia A Patients With Inhibitors Achieving Complete or Partial Immune Tolerance Induction (ITI) Success
timeFrame: From ITI start until termination of study, a maximum of 2 years
Secondary outcomes:
measure: Time Necessary to Achieve Complete or Partial ITI Success
timeFrame: A maximum period of 5 years from ITI start
measure: In Case of Complete or Partial ITI Success, Duration of Immune Tolerance
timeFrame: A maximum period of 5 years from ITI start
measure: Bleeding Frequency While on Wilate or Nuwiq ITI Treatment
timeFrame: A maximum period of 5 years from ITI start
measure: Association of Inhibitor Titres With the Probability of ITI Success
timeFrame: A maximum period of 5 years from ITI start
measure: Use of Bypassing Agents Before and During ITI Treatment With Wilate or Nuwiq
timeFrame: 12 months before the start of ITI with Wilate or Nuwiq to a maximum of 5 years from starting ITI with Wilate or Nuwiq
measure: Use of Emicizumab (Hemlibra) During ITI Treatment With Wilate or Nuwiq
timeFrame: A maximum period of 5 years from ITI start
measure: Relapse Rate Following Complete or Partial Successful ITI Using Wilate or Nuwiq
timeFrame: A maximum period of 5 years from ITI start
measure: Time to Relapse Following Complete or Partial Successful ITI Using Wilate or Nuwiq
timeFrame: A maximum period of 5 years from ITI start
measure: Adherence With the ITI Regimen
timeFrame: A maximum period of 5 years from ITI start
Other outcomes:
Locations:
city: Edmonton
country: Canada
facility: Stollery children's hospital, University of Alberta
state: Alberta
city: Ottawa
country: Canada
facility: Children's Hospital of Eastern Ontario
state: Ontario
city: Toronto
country: Canada
facility: Hamilton Health Science center
state: Ontario
Study officials:
affiliation: Octapharma
name: Sri Adapa
role: STUDY_DIRECTOR
References: