Patient Interview Study to Explore the Impact of Gene Therapy in Hemophilia A & B

Not yet recruiting

• Conditions: Hemophilia A; Hemophilia B

• Registration Number: NCT06634836
• Lead Sponsor: Pfizer

Brief 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.

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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2024-10-01
• Study First Submitted QC: 2024-10-08
• Study First Posted: 2024-10-10
• Status Verified: 2025-02
• Last Update Submitted: 2025-03-03
• Last Update Posted: 2025-03-05
• Study Start: 2025-03-17
• Primary Completion: 2025-12-31
• Study Completion: 2025-12-31

Recruitment & Eligibility

• Status: NOT_YET_RECRUITING
• Sex: Male
• Target Recruitment: 40

Inclusion Criteria

• 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

Exclusion Criteria

-No exclusion criteria

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Frequency of self-reported symptoms pre and post hemophilia gene therapy	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	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	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	From date of dosing in the gene therapy clinical trial until the interview date, no less than 52 weeks.