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

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ClinicalTrials.gov Identifier: NCT04723680
Recruitment Status : Recruiting
First Posted : January 26, 2021
Last Update Posted : January 27, 2021
Sponsor:
Collaborator:
UniQure Biopharma B.V.
Information provided by (Responsible Party):
Haemnet

Study Description
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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.

Condition or disease Intervention/treatment
Hemophilia Other: Qualitative interview

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.

Study Design
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Study Type : Observational
Estimated Enrollment : 65 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: An Exploration of the Impact of Gene Therapy on the Lives of People With Haemophilia and Their Families
Actual Study Start Date : August 13, 2020
Estimated Primary Completion Date : December 31, 2021
Estimated Study Completion Date : May 31, 2022

Resource links provided by the National Library of Medicine


Groups and Cohorts
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Group/Cohort Intervention/treatment
Early dose finding cohort
People with Haemophilia who took part in the early dose finding studies
 Other: Qualitative interview
Qualitative semi structured interview
Subsequent studies group cohort
People with haemophilia who took part in subsequent gene therapy studies
 Other: Qualitative interview
Qualitative semi structured interview
Withdrawn/ineligible Cohort
People with haemophilia who were withdrawn or proved ineligible
 Other: Qualitative interview
Qualitative semi structured interview
Not interested cohort
People with haemophilia who are definitely not interested in gene therapy
 Other: Qualitative interview
Qualitative semi structured 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
Qualitative semi structured interview


Outcome Measures
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Primary Outcome Measures :
  1. patient expectations [ Time Frame: 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 ]
    To explore the expectations that patients, and families in the UK have of gene therapy and its position in contemporary and future haemophilia management


Secondary Outcome Measures :
  1. Lived experience [ Time Frame: 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 ]
    To understand the lived experience of people who have undergone gene therapy

  2. Impact of gene therapy [ Time Frame: 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 ]
    To understand the as yet "unseen" impact of gene therapy on the extended family

  3. Impact of ineligibility [ Time Frame: 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 ]
    To understand the impact of ineligibility for gene therapy trials

  4. Impact of withdrawal [ Time Frame: 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 ]
    To understand the impact of withdrawal from gene therapy on individuals and their ongoing attitude to their haemophilia care

  5. Why not interested [ Time Frame: 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 ]
    To understand why some patients and families opt not to participate in gene therapy trials as a treatment option


Eligibility Criteria
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Ages Eligible for Study:   16 Years to 100 Years   (Child, Adult, Older Adult)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
People with haemophilia and a member of their family
Criteria

Inclusion Criteria:

  • 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.

Exclusion Criteria:

  • Participants will be excluded if they do not speak English (for the interviews) or do not consent to be in the study.
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04723680


Contacts
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Contact: Michael Holland 02078199615 mike@haemnet.com
Contact: Kate Kahir 07515900812 kate@haemnet.com

Locations
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United Kingdom
Oxford University Hospitals NHS Foundation Trust Recruiting
Oxford, Oxfordshire, United Kingdom, OX3 7LE
Contact: Shahista Hussain    01865 572236    shahista.hussain@ouh.nhs.uk   
Principal Investigator: Simon P Fletcher, MA         
Sponsors and Collaborators
Haemnet
UniQure Biopharma B.V.
Investigators
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Principal Investigator: Simon P Fletcher Researcher
  Study Documents (Full-Text)

Documents provided by Haemnet:
Study Protocol  [PDF] November 13, 2020

More Information
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Responsible Party: Haemnet
ClinicalTrials.gov Identifier: NCT04723680    
Other Study ID Numbers: v3Nov2020
First Posted: January 26, 2021    Key Record Dates
Last Update Posted: January 27, 2021
Last Verified: January 2021
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Haemnet:
Haemophilia, Gene Therapy, Quality of Life
Additional relevant MeSH terms:
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Hemophilia A
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
 Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn