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

In-trial Qualitative Interviews to Explore the Impact of Gene Therapy in Hemophilia A & B

Status

Withdrawn

Phases

Unknown

Study type

Observational

Source

ClinicalTrials.gov

Registry ID

NCT06634836

Enrollment

Registered

2024-10-10

Start date

2025-03-17

Completion date

2025-12-31

Last updated

2025-08-29

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Hemophilia A, Hemophilia B

Keywords

Hemophilia A, Hemophilia B, Genetic Therapy

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.

Interventions

None listed

Study design

Observational model

CASE_ONLY

Time perspective

PROSPECTIVE

Eligibility

Sex/Gender

MALE

Age

18 Years to 85 Years

Healthy volunteers

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

Design outcomes

Primary

Measure	Time frame
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.

Outcome results