Brief Title
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
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.
Study Type
Observational
Primary Outcome
The health-related quality of life in subjects treated with ReFacto AF routine prophylaxis
Secondary Outcome
The subjective physical functioning of subjects treated with ReFacto AF routine prophylaxis
Condition
Hemophilia A
Intervention
Refacto AF
Study Arms / Comparison Groups
1. Refacto AF (NIS)
Description: Non-Interventional Study
Publications
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
Recruitment Information
Recruitment Status
Drug
Estimated Enrollment
0
Start Date
September 1, 2019
Completion Date
January 1, 2022
Primary Completion Date
May 1, 2021
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.
Gender
Male
Ages
2 Years - 30 Years
Accepts Healthy Volunteers
No
Contacts
Pfizer CT.gov Call Center, ,
Administrative Informations
NCT ID
NCT02718677
Organization ID
B1831088
Responsible Party
Sponsor
Study Sponsor
Pfizer
Study Sponsor
Pfizer CT.gov Call Center, Study Director, Pfizer
Verification Date
November 2020