NCT04723680

Brief 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.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
69

participants targeted

Target at P25-P50 for all trials

Timeline
Completed

Started Aug 2020

Typical duration for all trials

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

August 13, 2020

Completed
5 months until next milestone

First Submitted

Initial submission to the registry

January 15, 2021

Completed
11 days until next milestone

First Posted

Study publicly available on registry

January 26, 2021

Completed
2.1 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

February 28, 2023

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

February 28, 2023

Completed
Last Updated

March 1, 2023

Status Verified

February 1, 2023

Enrollment Period

2.5 years

First QC Date

January 15, 2021

Last Update Submit

February 28, 2023

Conditions

Hemophilia

Keywords

Haemophilia, Gene Therapy, Quality of Life

Outcome Measures

Primary Outcomes (1)

  • patient expectations

    To explore the expectations that patients, and families in the UK have of gene therapy and its position in contemporary and future haemophilia management

    Each participant & family member will take part in a 1 hour semi structured qualitative interview where his experiences of his condition, previous treatment & experience of gene therapy will be discussed & analysed using a grounded theory approach

Secondary Outcomes (5)

  • Lived experience

    Each participant & family member will take part in a 1 hour semi structured qualitative interview where his experiences of his condition, previous treatment & experience of gene therapy will be discussed & analysed using a grounded theory approach

  • Impact of gene therapy

    Each participant & family member will take part in a 1 hour semi structured qualitative interview where his experiences of his condition, previous treatment & experience of gene therapy will be discussed & analysed using a grounded theory approach

  • Impact of ineligibility

    Each participant & family member will take part in a 1 hour semi structured qualitative interview where his experiences of his condition, previous treatment & experience of gene therapy will be discussed & analysed using a grounded theory approach

  • Impact of withdrawal

    Each participant & family member will take part in a 1 hour semi structured qualitative interview where his experiences of his condition, previous treatment & experience of gene therapy will be discussed & analysed using a grounded theory approach

  • Why not interested

    Each participant & family member will take part in a 1 hour semi structured qualitative interview where his experiences of his condition, previous treatment & experience of gene therapy will be discussed & analysed using a grounded theory approach

Study Arms (5)

Early dose finding cohort

People with Haemophilia who took part in the early dose finding studies

Other: Qualitative interview

Subsequent studies group cohort

People with haemophilia who took part in subsequent gene therapy studies

Other: Qualitative interview

Withdrawn/ineligible Cohort

People with haemophilia who were withdrawn or proved ineligible

Other: Qualitative interview

Not interested cohort

People with haemophilia who are definitely not interested in gene therapy

Other: Qualitative interview

Not offered cohort

People with haemophilia who are interested in gene therapy but have not been the opportunity to take part

Other: Qualitative interview

Interventions

Qualitative interviewOTHER

Qualitative semi structured interview

Early dose finding cohortNot interested cohortNot offered cohortSubsequent studies group cohortWithdrawn/ineligible Cohort

Eligibility Criteria

Age16 Years - 100 Years
Sexmale
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)
Sampling MethodNon-Probability Sample
Study Population

People with haemophilia and a member of their family

You may qualify if:

  • People with haemophilia A or B who consented to and have undergone gene therapy in the early dose-finding studies and a member of their family
  • People with haemophilia A or B who consented to a gene therapy trial following the results of the early studies and a member of their family
  • People with haemophilia A or B who consented to a gene therapy trial but who withdrew, were withdrawn from, or were ineligible for the study, and a member of their family
  • People with haemophilia A or B who are definitely not interested in or unaware of gene therapy and a member of their family
  • People with haemophilia A or B who are interested in but have not been offered gene therapy
  • Those who have given written consent to be in the study
  • All participants will be ≥16 years.

You may not qualify if:

  • Participants will be excluded if they do not speak English (for the interviews) or do not consent to be in the study.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Oxford University Hospitals NHS Foundation Trust

Oxford, Oxfordshire, OX3 7LE, United Kingdom

Location

MeSH Terms

Conditions

Hemophilia A

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, InheritedBlood Coagulation DisordersHematologic DiseasesHemic and Lymphatic DiseasesCoagulation Protein DisordersHemorrhagic DisordersGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

  • Simon P Fletcher

    Researcher

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
observational
Observational Model
COHORT
Time Perspective
PROSPECTIVE
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

January 15, 2021

First Posted

January 26, 2021

Study Start

August 13, 2020

Primary Completion

February 28, 2023

Study Completion

February 28, 2023

Last Updated

March 1, 2023

Record last verified: 2023-02

Data Sharing

IPD Sharing
Will not share

Locations

GB(1)