Clinical trial NCT02718677: Patient-reported Outcomes in Subjects Treated With ReFacto AF Routine Prophylaxis
Official title: NON-INTERVENTIONAL STUDY TO ASSESS THE HEALTH-RELATED QUALITY OF LIFE IN SEVERE OR MODERATELY SEVERE HEMOPHILIA A SUBJECTS TREATED WITH REFACTO AF ROUTINE PROPHYLAXIS
Study type: OBSERVATIONAL
Overall status: WITHDRAWN
Conditions: Hemophilia A
Lead sponsor: Pfizer
Sponsor class: INDUSTRY
First posted: 2016-03-24
Enrollment: 0
Sex: MALE
Minimum age (years): 2
Maximum age (years): 30
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Observational model: OTHER
Time perspective: OTHER
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Subjects receiving routine prophylaxis treatment with ReFacto AF as determined by the inclusion criteria will be included in the study.

Brief summary: This NIS aims to assess the patient-reported outcomes (PROs) in enrolled subjects

Detailed description: The design of the study is observational: both prospective and retrospective, national, multicenter, non- interventional. During this observational study, subjects diagnosed with severe or moderately severe (with severe phenotype) hemophilia A receiving routine prophylaxis treatment with ReFacto AF as per local standard clinical care, and in line with local labeling, are to be observed for 12 months.

The primary objective is to assess the health-related quality of life in subjects treated with ReFacto AF routine prophylaxis at baseline and after 6 and 12 months.

The secondary objectives are:

* To assess the subjective physical functioning of subjects treated with ReFacto AF routine prophylaxis at baseline and after 6 and 12 months;
* To assess the treatment satisfaction of caregivers of children and subjects above 18 years treated with ReFacto AF routine prophylaxis at baseline and after 6 and 12 months;
* To determine in routine clinical practice the mean annualized bleeding rate (ABR) in subjects treated with ReFacto AF routine prophylaxis;
* To describe the status of joint health measured by HJHS before and after starting routine prophylaxis for subjects above 18 years.

Eligibility criteria: Inclusion Criteria:

1. Severe (FVIII: C \<1%) or moderately severe (FVIII: C ≥1 - ≤2% with severe phenotype \[at least 4 spontaneous bleeds clinically documented during a 6 months period\] and without inhibitors) male subjects with hemophilia A receiving routine prophylaxis treatment.
2. Age:

   * From 2 to 18 years;
   * Subjects above 18 years who began routine prophylaxis treatment in childhood (and were part of the national prophylaxis program when turned 18 years).
3. Previously treated patients (PTPs) with hemophilia A. Previously treated patient (PTP) is considered in this study as a subject who has at least 50 exposure days to any FVIII product. An exposure day (ED) is a 24-hour period during which a dose of FVIII concentrate has been administered, irrespective of size and frequency.
4. No history and no current FVIII inhibitor defined as a titer ≥0.6 BU/mL or any measured Bethesda inhibitor titer greater than the upper limit of normal for the laboratory performing the assay.
5. Subjects who are scheduled by their treating physician to initiate prophylaxis with ReFacto AF or to continue previously initiated prophylaxis with ReFacto AF, and subjects who are switching from prophylaxis with another FVIII product to ReFacto AF.
6. Evidence of a personally signed and dated informed consent and assent (for children 6-17 years of age) document indicating that the subject (or a legally acceptable representative) has been informed of all pertinent aspects of the study.

Main Exclusion Criteria:

1. Subject has known hypersensitivity to the active substance or any of the excipients.
2. Subject has known allergic reaction to hamster proteins.
3. Presence of any bleeding disorder in addition to hemophilia A.
4. Treatment with any investigational agent or device within the past 30 days.
5. Any other contraindications according to Summary of Product Characteristics (SPC).
6. Unsuitable to participate in study for any other reason as assessed by the investigator.
7. Subjects (or a legally acceptable representative) not able to understand study documents and study procedure.

Primary outcomes:
measure: The health-related quality of life in subjects treated with ReFacto AF routine prophylaxis
timeFrame: Baseline and after 6 and 12 months.
measure: The health-related quality of life
timeFrame: Baseline and after 6 and 12 months.
Secondary outcomes:
measure: The subjective physical functioning of subjects treated with ReFacto AF routine prophylaxis
timeFrame: Baseline and after 6 and 12 months;
measure: The treatment satisfaction of caregivers of children and subjects above 18 years treated with ReFacto AF routine prophylaxis
timeFrame: Baseline and after 6 and 12 months;
measure: The mean annualized bleeding rate (ABR) in subjects treated with ReFacto AF routine prophylaxis;
timeFrame: After 12 months
measure: Joint health for subjects above 18 years
timeFrame: After 12 months
Other outcomes:
Locations:
Study officials:
affiliation: Pfizer
name: Pfizer CT.gov Call Center
role: STUDY_DIRECTOR
References: