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

NCT06634836 | Withdrawn

• 2 other identifiers: C3731011NCT06634836
• Study Type: observational
• Target: N/A
• Locations: 0 countries
• Sites: N/A

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.

Conditions

Hemophilia A; Hemophilia B

Keywords

Hemophilia A; Hemophilia B; Genetic Therapy

Outcome Measures

Primary Outcomes (4)

• 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.

Study Arms (1)

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

Eligibility Criteria

• Age: 18 Years - 85 Years
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

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

You may qualify if:

• 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

Sponsors & Collaborators

• Pfizer: lead

MeSH Terms

Conditions

Hemophilia A; Hemophilia B

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Genetic Diseases, X-Linked

Study Officials

• Pfizer CT.gov Call Center

  Pfizer

  STUDY DIRECTOR

Study Design

• Study Type: observational
• Observational Model: CASE ONLY
• Time Perspective: PROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 1, 2024
• First Posted: October 10, 2024
• Study Start: March 17, 2025
• Primary Completion: December 31, 2025
• Study Completion: December 31, 2025
• Last Updated: August 29, 2025

Record last verified: 2025-08

Data Sharing

• IPD Sharing: Will not share