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

NCT02718677

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Eligibility Criteria
condition
The disease, disorder, syndrome, illness, or injury that is being studied.
Hemophilia A
Sex
Male
Age
Pediatric Trials: 0-17 Years
Adult Trials: 18+ Years
2-30 years
Inclusion Criteria
The factors, or reasons, that allow a person to participate in a clinical study.
Show details

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
The factors, or reasons, that prevent a person from participating in a clinical study.
Show details


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.

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Advanced Information
Descriptive Information
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
Study Design Observational Model: Other
Time Perspective: Other
Target Follow-Up Duration Not Provided
Biospecimen Not Provided
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.
Condition Hemophilia A
Intervention Drug: Refacto AF
Observational - Non-Interventional Study
Other Name: moroctocog alfa
Study Groups/Cohorts 1. Refacto AF (NIS)
Non-Interventional Study
Intervention: Drug: Refacto AF
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status Not yet recruiting
Estimated Enrollment
 (submitted: February 5, 2019)
26
Original Estimated Enrollment
 (submitted: March 18, 2016)
33
Estimated Study Completion Date May 1, 2021
Estimated Primary Completion Date May 1, 2021   (Final data collection date for primary outcome measure)
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.
Sex/Gender
Sexes Eligible for Study:Male
Gender Based Eligibility:Yes
Ages 2 Years to 30 Years   (Child, Adult)
Accepts Healthy Volunteers No
Contacts
Contact: Pfizer CT.gov Call Center1-800-718-1021[email protected]
Listed Location Countries Not Provided
Removed Location Countries  
 
Administrative Information
NCT Number NCT02718677
Other Study ID Numbers B1831088
Has Data Monitoring Committee No
U.S. FDA-regulated Product
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
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/d….
Responsible Party Pfizer
Study Sponsor Pfizer
Collaborators Not Provided
Investigators
Study Director:Pfizer CT.gov Call CenterPfizer
PRS Account Pfizer
Verification Date August 2019