The Experiences of People Who Live With Glanzmanns Thrombasthenia.

NCT05315232 | Completed

• 1 other identifier: Glanzmanns 360
• Study Type: observational
• Target: 122
• Locations: 1 country
• Sites: 1

Brief Summary

To understand the lived experiences of people with Glanzmanns Thrombasthenia

Conditions

Glanzmann Thrombasthenia

Outcome Measures

Primary Outcomes (1)

• to understand the impact of GT on quality of life of affected individuals and their families.

  Incidence of reported impact of GT on daily life using validated questionnaires EQ5D, MIQ, PHQ9, Rosenbergs self esteem scale.

  one year

Secondary Outcomes (2)

• Identify levels of acceptability of current treatments and management approaches

  one year

• Identify areas of unmet need among people with GT

  one year

Study Arms (2)

Qualitative assessment

Survey (using validated quality of life assessment questionnaires) and one to one interviews of people and family members living with Glanzmanns Thrombasthenia

Other: survey and interview

Bleed diary

Daily bleed diary completion over 12 weeks

Other: survey and interview

Interventions

survey and interview OTHER

qualitative assessment

Also known as: diary completion

Bleed diary; Qualitative assessment

Eligibility Criteria

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

Study Population

People with Glanzmanns Thrombasthenia or parents of children with Glanzmanns Thrombasthenia.

You may qualify if:

• Clinical diagnosis of congenital (inherited) Glanzmanns Thrombasthenia

You may not qualify if:

• Acquired Glanzmanns Thrombasthenia

Sponsors & Collaborators

• Haemnet: lead
• Hemab ApS: collaborator

Study Sites (1)

Oxford University Hospitals NHS Foundation Trust

Oxford, Oxfordshire, OX3 7LE, United Kingdom

Location

MeSH Terms

Conditions

Thrombasthenia

Interventions

Surveys and Questionnaires; Interviews as Topic

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Blood Platelet 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 Officials

• Mike Holland, BSC

  Haemnet

  STUDY DIRECTOR

Study Design

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

Study Record Dates

• First Submitted: March 15, 2022
• First Posted: April 7, 2022
• Study Start: April 27, 2022
• Primary Completion: April 27, 2023
• Study Completion: April 27, 2023
• Last Updated: March 13, 2024

Record last verified: 2024-03

Data Sharing

• IPD Sharing: Will share
• Shared Documents: ICF, CSR
• Time Frame: 3-6 months

Locations

GB (1)