NCT04723680

An Exploration of the Impact of Gene Therapy on the Lives of People With Haemophilia and Their Families

• Status: Completed
• Phase: —
• Study type: Observational
• Enrollment: 69 (actual)
• Sponsor: Haemnet · Academic / Other
• Sex: Male
• Age: 16 Years – 100 Years
• Healthy volunteers: Not accepted

Summary

This study programme aims to examine the real-world experience and impact of gene therapy in a diverse community of people and families affected by haemophilia who have been or will be exposed to gene therapy.

Detailed description

This is a prospective observational multiple cohort qualitative research study to be conducted among diverse groups within the haemophilia community whose lives may have been impacted by gene therapy. The study is designed to allow English-speaking patients and their families to tell their own life stories through narrative accounts. The narratives represent a true sharing of experiences and offer insight into how these patients and families cope with haemophilia.

Conditions

• Hemophilia

Interventions

Type	Name	Description
OTHER	Qualitative interview	Qualitative semi structured interview

Timeline

• Start date: 2020-08-13
• Primary completion: 2023-02-28
• Completion: 2023-02-28
• First posted: 2021-01-26
• Last updated: 2023-03-01

Locations

1 site across 1 country: United Kingdom