Patient-reported Outcomes in Subjects Treated With ReFacto AF Routine Prophylaxis

NCT02718677 | Withdrawn FDA Drug

• 1 other identifier: B1831088
• Study Type: observational
• Target: N/A
• Locations: 0 countries
• Sites: N/A

Brief Summary

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

Conditions

Hemophilia A

Outcome Measures

Primary Outcomes (2)

• The health-related quality of life in subjects treated with ReFacto AF routine prophylaxis

  HRQoL will be assessed by applying the self-reported hemophilia-specific quality of life Haemo-QoL questionnaire. The endpoint will include the domains and total scores of the questionnaire. The results of this study will be presented using descriptive statistics and there will be no hypothesis testing. The domain scores and total scores will be summarized by: N, mean (sd), median, min-max and the 95% confidence interval at baseline, end of study, and the change from baseline.

  Baseline and after 6 and 12 months.

• The health-related quality of life

  HRQoL will be be evaluated also by recording the number of days of absence from kindergarten, school or high-school. There are no a priori hypotheses specified and results will be presented as descriptive statistics.

  Baseline and after 6 and 12 months.

Secondary Outcomes (4)

• The subjective physical functioning of subjects treated with ReFacto AF routine prophylaxis

  Baseline and after 6 and 12 months;

• The treatment satisfaction of caregivers of children and subjects above 18 years treated with ReFacto AF routine prophylaxis

  Baseline and after 6 and 12 months;

• The mean annualized bleeding rate (ABR) in subjects treated with ReFacto AF routine prophylaxis;

  After 12 months

• Joint health for subjects above 18 years

  After 12 months

Study Arms (1)

1. Refacto AF (NIS)

Non-Interventional Study

Drug: Refacto AF

Interventions

Refacto AF DRUG

Observational - Non-Interventional Study

Also known as: moroctocog alfa

1. Refacto AF (NIS)

Eligibility Criteria

• Age: 2 Years - 30 Years
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)
• 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.

You may qualify if:

• 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.
• 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).
• 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.
• 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.
• 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.
• 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.

You may not qualify if:

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

Sponsors & Collaborators

• Pfizer: lead

Related Links

• To obtain contact information for a study center near you, click here.

MeSH Terms

Conditions

Hemophilia A

Interventions

Factor VIII; recombinant factor VIII SQ

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Blood Coagulation Factors; Blood Proteins; Proteins; Amino Acids, Peptides, and Proteins; Protein Precursors; Biological Factors

Study Officials

• Pfizer CT.gov Call Center

  Pfizer

  STUDY DIRECTOR

Study Design

• Study Type: observational
• Observational Model: OTHER
• Time Perspective: OTHER
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 22, 2016
• First Posted: March 24, 2016
• Study Start: September 1, 2019
• Primary Completion: May 1, 2021
• Study Completion: January 1, 2022
• Last Updated: November 16, 2020

Record last verified: 2020-11

Data Sharing

• IPD Sharing: Will not share