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

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ClinicalTrials.gov Identifier: NCT02718677
Recruitment Status : Withdrawn (Company decision to not conduct study)
First Posted : March 24, 2016
Last Update Posted : November 16, 2020
Sponsor:
Information provided by (Responsible Party):
Pfizer

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

Condition or disease Intervention/treatment
Hemophilia A Drug: Refacto AF

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.

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Study Type : Observational
Actual Enrollment : 0 participants
Observational Model: Other
Time Perspective: Other
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
Estimated Study Start Date : September 1, 2019
Estimated Primary Completion Date : May 1, 2021
Estimated Study Completion Date : January 1, 2022

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Hemophilia

Group/Cohort Intervention/treatment
1. Refacto AF (NIS)
Non-Interventional Study
Drug: Refacto AF
Observational - Non-Interventional Study
Other Name: moroctocog alfa




Primary Outcome Measures :
  1. The health-related quality of life in subjects treated with ReFacto AF routine prophylaxis [ Time Frame: Baseline and after 6 and 12 months. ]

    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.


  2. The health-related quality of life [ Time Frame: Baseline and after 6 and 12 months. ]
    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.


Secondary Outcome Measures :
  1. The subjective physical functioning of subjects treated with ReFacto AF routine prophylaxis [ Time Frame: Baseline and after 6 and 12 months; ]

    Subjects will be asked to report their subjective physical functioning using the HEP-Test-Q.

    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.


  2. The treatment satisfaction of caregivers of children and subjects above 18 years treated with ReFacto AF routine prophylaxis [ Time Frame: Baseline and after 6 and 12 months; ]

    Caregivers will evaluate their satisfaction with the treatment of their child and the above 18 years subjects will evaluate their own satisfaction with the treatment using the Hemo-Satp 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.


  3. The mean annualized bleeding rate (ABR) in subjects treated with ReFacto AF routine prophylaxis; [ Time Frame: After 12 months ]
    ABR will be calculated at the end of the study based on the number of bleeds recorded during the 12-months observation period. The ABR will be presented as N, mean (sd), median, min-max and the 95% confidence interval.

  4. Joint health for subjects above 18 years [ Time Frame: After 12 months ]

    Status of joint health will be described for subjects above 18 years using the HJHS (Hemophilia Joint Health Score) before and after starting routine prophylaxis. HJHS for one particular joint (elbow, knee or ankle) represents the sum between the joint totals and the global gait score.The minimum score is 0 and the maximum is 24.

    The joint totals is the sum between the scores for swelling, duration of swelling, muscle atrophy, crepitus on motion, flexion loss, extension loss, joint pain and strength; the maximum score is 20.

    The global gait score evaluates walking, stairs, running and hopping in one leg; the maximum score is 4.




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Ages Eligible for Study:   2 Years to 30 Years   (Child, Adult)
Sexes Eligible for Study:   Male
Gender Based Eligibility:   Yes
Accepts Healthy Volunteers:   No
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.
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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02718677


Sponsors and Collaborators
Pfizer
Investigators
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Study Director: Pfizer CT.gov Call Center Pfizer
Additional Information:
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Responsible Party: Pfizer
ClinicalTrials.gov Identifier: NCT02718677    
Other Study ID Numbers: B1831088
First Posted: March 24, 2016    Key Record Dates
Last Update Posted: November 16, 2020
Last Verified: November 2020
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No
Plan Description: Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests.

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Product Manufactured in and Exported from the U.S.: No
Additional relevant MeSH terms:
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Hemophilia A
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn
Factor VIII
Coagulants