Learning to Live With Non-severe Haemophilia

NCT05314751 | Completed

• 1 other identifier: L2L
• Study Type: observational
• Target: 165
• Locations: 1 country
• Sites: 1

Brief Summary

While the burden of standard treatment may be reduced through the use of gene therapy, converting those with severe haemophilia to a mild or moderate phenotype, the long-term sequelae of previous joint bleeds and associated limitations imposed on those with severe haemophilia may not translate to lessen the biomedical burden of living with a history of severe haemophilia. We wish to explore these issues further in the Learning to Live study. The study will also seek to identify the ongoing support needs of those who transition to a milder bleeding phenotype.

Conditions

Hemophilia

Outcome Measures

Primary Outcomes (1)

• To Identify and understand the differences between those with genetically mild/moderate haemophilia and those with previously severe haemophilia who have received phenotype altering treatment

  Survey using the validated PROBE questionnaire (using 7 point Likert scale)

  3 months

Secondary Outcomes (1)

• To assess the impact of phenotype altering treatments on those who receive them, and to compare this with those people with genetically mild/moderate haemophilia.

  3 months

Interventions

Questionnaire and interview BEHAVIORAL

An online survey and an in-depth interview.

Eligibility Criteria

• Age: 16 Years - 100 Years
• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Probability Sample

Study Population

People with haemophilia A or B either mild moderate or severe living in the UK.

You may qualify if:

• Confirmed diagnosis of haemophilia A or B of any severity

You may not qualify if:

• Diagnosis of any other bleeding disorder

Sponsors & Collaborators

• Haemnet: lead
• BioMarin Pharmaceutical: collaborator

Study Sites (1)

Oxford University Hospitals NHS Foundation Trust

Oxford, Oxfordshire, OX3 9DU, United Kingdom

Location

MeSH Terms

Conditions

Hemophilia A

Interventions

Surveys and Questionnaires; Interviews as Topic

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

Intervention Hierarchy (Ancestors)

Data Collection; Epidemiologic Methods; Investigative Techniques; Health Care Evaluation Mechanisms; Quality of Health Care; Health Care Quality, Access, and Evaluation; Public Health; Environment and Public Health

Study Design

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

Study Record Dates

• First Submitted: March 29, 2022
• First Posted: April 6, 2022
• Study Start: December 21, 2022
• Primary Completion: September 29, 2023
• Study Completion: September 29, 2023
• Last Updated: March 13, 2024

Record last verified: 2024-03

Data Sharing

• IPD Sharing: Will not share

Locations

GB (1)