Hemophilia ANCT02718677
Patient-reported Outcomes in Subjects Treated With ReFacto AF Routine Prophylaxis
An observational study of Hemophilia A, run by Pfizer.
Trial summary
This NIS aims to assess the patient-reported outcomes (PROs) in enrolled subjects Read more about this study
- Who is it for?
- Ages 2 to 30 years, men only
- Conditions the study is for
- Hemophilia A
- What is being tested?
- Refacto AF
- How many people
- 0 participants
- Where
- No sites listed yet
- Study dates
- 1 Sep 2019to 1 Jan 2022
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 2 to 30 years
- Men only
- No healthy volunteers
- Child, Adult
Study population: Subjects receiving routine prophylaxis treatment with ReFacto AF as determined by the inclusion criteria will be included in the study.
Who may be able to join
- 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.
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- 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.
- Main Exclusion Criteria:
- 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.
Who cannot join
None listed.
About this study
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
This NIS aims to assess the patient-reported outcomes (PROs) in enrolled subjects
Read the 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.
- Conditions
- Hemophilia A
- Being tested
- Refacto AF
- Medical subject headings
- Hemophilia A
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Refacto AFDrug
Also called moroctocog alfa
Observational - Non-Interventional Study
| Study group | Role | What they receive |
|---|---|---|
| 1. Refacto AF (NIS) | Non-Interventional Study Receives: Drug: Refacto AF |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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.
Measured: Baseline and after 6 and 12 months.
Secondary outcomes (4)
The subjective physical functioning of subjects treated with ReFacto AF routine prophylaxis
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.
Measured: 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
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.
Measured: Baseline and after 6 and 12 months;
The mean annualized bleeding rate (ABR) in subjects treated with ReFacto AF routine prophylaxis;
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.
Measured: After 12 months
Joint health for subjects above 18 years
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.
Measured: After 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Mar 2016 (estimated)
- Study start1 Sep 2019 (estimated)
- Primary completion1 May 2021 (estimated)
- Study completion1 Jan 2022 (estimated)
Why it stopped: Company decision to not conduct study
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hemophilia AStudies of the same condition, open and under way.
- More trials run by PfizerThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Other
- Time perspective
- Other
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Pfizer
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Pfizer CT.gov Call Center, Study director, Pfizer
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02718677
- Sponsor's protocol ID
- B1831088
- Status last verified
- Nov 2020
- First submitted
- 22 Feb 2016
- Last updated
- 16 Nov 2020
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No: 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.
Study at a glance
From the registry record, last updated by the sponsor on 16 Nov 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT02718677
- Acronym / sponsor ID
- B1831088
- Conditions
- Hemophilia A
- Study type
- Observational
- Recruitment status
- WithdrawnReason stopped: Company decision to not conduct study
- Enrolment
- 0 participants (actual)
- Lead sponsor
- PfizerIndustry (pharma, biotech, devices)
- Study start
- 1 Sep 2019 (estimated)
- Primary completion
- 1 May 2021 (estimated)
- Study completion
- 1 Jan 2022 (estimated)
- First posted
- 24 Mar 2016 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Observational model
- Other
- Time perspective
- Other
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| 1. Refacto AF (NIS) | – | Drug: Refacto AFNon-Interventional Study |
Who may qualify
- Ages
- 2 to 30 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
- Study population
- Subjects receiving routine prophylaxis treatment with ReFacto AF as determined by the inclusion criteria will be included in the study.
Key inclusion criteria (16 listed)
- 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:
- 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.
Key exclusion criteria (0 listed)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Refacto AFAlso: moroctocog alfaObservational - Non-Interventional Study | Drug | 1. Refacto AF (NIS) |
Primary outcome
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.
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.
4 secondary outcomes
The subjective physical functioning of subjects treated with ReFacto AF routine prophylaxis
Time frame: 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
Time frame: Baseline and after 6 and 12 months;
The mean annualized bleeding rate (ABR) in subjects treated with ReFacto AF routine prophylaxis;
Time frame: After 12 months
Joint health for subjects above 18 years
Time frame: After 12 months
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 Nov 2020.
- To obtain contact information for a study center near you, click here.Link provided by the sponsor.
- Individual participant dataSharing planned: No. 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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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Pfizer CT.gov Call Center study director
Pfizer
Questions about this trial
What is this clinical trial studying?
An observational study of Hemophilia A, run by Pfizer.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 16 inclusion and 0 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: withdrawn, as of the sponsor's last update on 16 Nov 2020. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study was withdrawn before enrolling participants, so there are no results.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
The health-related quality of life in subjects treated with ReFacto AF routine prophylaxis (time frame: Baseline and after 6 and 12 months.). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 16 Nov 2020.