Hemophilia ANCT06634836
Patient Interview Study to Explore the Impact of Gene Therapy in Hemophilia A & B
An observational study of Hemophilia A and Hemophilia B, run by Pfizer.
Trial summary
The purpose of this study is to learn about experiences of patients with hemophilia A and B after taking gene therapy. The experiences of patients will be studied through online interviews. Read more about this study
- Who is it for?
- Ages 18 to 85 years, men only
- Conditions the study is for
- Hemophilia A, Hemophilia B
- How many people
- 0 participants
- Where
- No sites listed yet
- Study dates
- 17 Mar 2025to 31 Dec 2025
- 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 18 to 85 years
- Men only
- No healthy volunteers
- Adult, Older adult
Study population: Patients who are participating in the Pfizer's gene therapy clinical trials for Hemophilia A and B or are in the long-term follow up for these clinical programs
Who may be able to join
- Participation in the Pfizer's gene therapy clinical trials for Hemophilia A or B .
- Participants will be one year or more post-gene therapy
- Participants will be willing to provide written consent and will be willing to comply with the study requirements
Who cannot join
- -No exclusion criteria
About this study
Official title: In-trial Qualitative Interviews to Explore the Impact of Gene Therapy in Hemophilia A & B
The purpose of this study is to learn about experiences of patients with hemophilia A and B after taking gene therapy. The experiences of patients will be studied through online interviews.
This study is seeking participants who are:
- part of the Pfizer's gene therapy clinical studies or
- in the long-term follow up for these clinical programs. Participants will have one study visit at the clinic and one online interview. The planned duration for each participant will be 1 to 2 months. This covers the time from entering the study to end of the online interview.
- Conditions
- Hemophilia A, Hemophilia B
- Keywords
- Hemophilia A, Hemophilia B, Genetic Therapy
- Medical subject headings
- Hemophilia A, Hemophilia B
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| Hemophilia A and Hemophilia B patients | Patients who are participating in the Pfizer's gene therapy clinical trials for Hemophilia A or Hemophilia B or are in the long-term follow up for these clinical programs |
What is being measured
The primary outcome is the main question the study is designed to answer.
Frequency of self-reported symptoms pre and post hemophilia gene therapy
Measured: From date of dosing in the gene therapy clinical trial until the interview date, no less than 52 weeks.
Frequency of self-reported severity pre and post hemophilia gene therapy
Measured: From date of dosing in the gene therapy clinical trial until the interview date, no less than 52 weeks.
Frequency of self-reported impacts pre and post hemophilia gene therapy
Measured: From date of dosing in the gene therapy clinical trial until the interview date, no less than 52 weeks.
Change in the proportion of patients answering 5-point Likert scale questions about the burden of hemophilia on the participant's life pre-post gene therapy
Measured: From date of dosing in the gene therapy clinical trial until the interview date, no less than 52 weeks.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Oct 2024
- Study start17 Mar 2025 (estimated)
- Primary completion31 Dec 2025 (estimated)
- Study completion31 Dec 2025 (estimated)
Why it stopped: Business decision
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
- Case only
- Time perspective
- Prospective
- 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
- NCT06634836
- Sponsor's protocol ID
- C3731011
- Other IDs
- NCT06634836
- Status last verified
- Aug 2025
- First submitted
- 1 Oct 2024
- Last updated
- 29 Aug 2025
- Results posted
- No
- Data monitoring committee
- 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 29 Aug 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- In-trial Qualitative Interviews to Explore the Impact of Gene Therapy in Hemophilia A & B
- ClinicalTrials.gov ID
- NCT06634836
- Acronym / sponsor ID
- C3731011
- Conditions
- Hemophilia A, Hemophilia B
- Keywords
- Hemophilia A, Hemophilia B, Genetic Therapy
- Study type
- Observational
- Recruitment status
- WithdrawnReason stopped: Business decision
- Enrolment
- 0 participants (actual)
- Lead sponsor
- PfizerIndustry (pharma, biotech, devices)
- Study start
- 17 Mar 2025 (estimated)
- Primary completion
- 31 Dec 2025 (estimated)
- Study completion
- 31 Dec 2025 (estimated)
- First posted
- 10 Oct 2024 (actual)
- 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.
The purpose of this study is to learn about experiences of patients with hemophilia A and B after taking gene therapy. The experiences of patients will be studied through online interviews.
This study is seeking participants who are:
- part of the Pfizer's gene therapy clinical studies or
- in the long-term follow up for these clinical programs. Participants will have one study visit at the clinic and one online interview. The planned duration for each participant will be 1 to 2 months. This covers the time from entering the study to end of the online interview.
Study design
- Observational model
- Case only
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Hemophilia A and Hemophilia B patients | – | –Patients who are participating in the Pfizer's gene therapy clinical trials for Hemophilia A or Hemophilia B or are in the long-term follow up for these clinical programs |
Who may qualify
- Ages
- 18 to 85 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- Patients who are participating in the Pfizer's gene therapy clinical trials for Hemophilia A and B or are in the long-term follow up for these clinical programs
Key inclusion criteria (3 listed)
- Participation in the Pfizer's gene therapy clinical trials for Hemophilia A or B .
- Participants will be one year or more post-gene therapy
- Participants will be willing to provide written consent and will be willing to comply with the study requirements
Key exclusion criteria (1 listed)
- -No exclusion criteria
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Frequency of self-reported symptoms pre and post hemophilia gene therapy
Time frame: From date of dosing in the gene therapy clinical trial until the interview date, no less than 52 weeks.
Frequency of self-reported severity pre and post hemophilia gene therapy
Time frame: From date of dosing in the gene therapy clinical trial until the interview date, no less than 52 weeks.
Frequency of self-reported impacts pre and post hemophilia gene therapy
Time frame: From date of dosing in the gene therapy clinical trial until the interview date, no less than 52 weeks.
Change in the proportion of patients answering 5-point Likert scale questions about the burden of hemophilia on the participant's life pre-post gene therapy
Time frame: From date of dosing in the gene therapy clinical trial until the interview date, no less than 52 weeks.
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 29 Aug 2025.
- 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.
Related trials for Hemophilia A
RecruitingObservational study
The CACHE Study: Coronary Artery Care in HaEmophilia
Haemophilia, Cardiovascular Diseases
Active, not recruitingObservational study
A Study to Learn About the Study Medicine -Hympavzi in Congenital Hemophilia Patients Without Inhibitors in Japan.
Hemophilia A or B
RecruitingPhase 1/2
A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of NXT007 in Persons With Severe or Moderate Hemophilia A
Hemophilia A
Active, not recruitingPhase 3
A Study to Evaluate the Efficacy and Safety of Factor IX Gene Therapy With PF-06838435 in Adult Males With Moderately Severe to Severe Hemophilia B
Hemophilia B
Not yet recruiting
Safety of Intraosseous Anaesthesia in Children With Haemophilia
Haemophilia A, Haemophilia B
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 and Hemophilia B, 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 3 inclusion and 1 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 29 Aug 2025. 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?
Frequency of self-reported symptoms pre and post hemophilia gene therapy (time frame: From date of dosing in the gene therapy clinical trial until the interview date, no less than 52 weeks.). The study lists 4 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 29 Aug 2025.