A Qualitative Study on the Pain and Quality of Life of Patients With Fabry Disease

NCT05679076 | Unknown

• 1 other identifier: 4-2022-1347
• Study Type: observational
• Target: 10
• Locations: 1 country
• Sites: 1

Brief Summary

Background It is necessary to find out the difficulties experienced by patients with Fabry disease and their families, and to seek solutions for them. For this purpose, qualitative research with a patient-centered approach is useful. Objectives The Objective of this study is to understand the various difficulties of patients with Fabry disease and to help them establish a comprehensive support system, medical, health, and welfare service system, and develop the direction and components of social and educational programs. Methods The subjects of this study are patients who have been diagnosed with Fabry disease, and among them, are selected considering the selection and exclusion criteria, confirm consent for participation in the study after sufficient explanation, and then enroll. The target number of subjects is 10 people. Subjects who consented to the study had an in-depth interview centered on the in-depth interview questionnaire through the HA Research Institute, a specialized institution.

• The principle of saturation is observed by conducting 3 interviews per research subject.
• The duration of each interview per research subject is 50 to 60 minutes.
• All dictations of the research subjects were recorded with prior consent and then transcribed into a written copy.
• For 3 interviews, the number of interviews can be reduced to 1 or 2 by extending the interview time according to the research subject's request.
• As for the place of interview, the research counseling room at Severance Cardiovascular Hospital or the in-depth interview room in the HA Research Institute can be used, and the interview can be conducted at other places desired by the research subject at the request of the research subject.

Conditions

Fabry Disease

Outcome Measures

Primary Outcomes (3)

• Identify negative problems experienced by patients with Fabry disease

  up to 5 months

• Check the quality of life of patients with Fabry disease

  up to 5 months

• Identifying the structural causes of policies for patients with Fabry disease

  up to 5 months

Study Arms (1)

Fabry patients

Fabry patients

Eligibility Criteria

• Age: 19 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Fabry patients

You may qualify if:

• Subjects who have delayed diagnosis of Fabry disease or have experienced treatment refusal.
• Subjects who have been diagnosed with Fabry name for more than 3 years or who have been treated for more than 1 year
• Adults between the ages of 19 and 70 who have the ability to consent to participate in the research (excluding those subject to adult guardianship) and have the cognitive ability to dictate their experiences
• Subjects who have experienced disadvantages and support in health-related quality of life, academic, job, family, social, and heterosexual relationships due to Fabry disease
• Subjects who voluntarily gave written consent after hearing the explanation of the purpose and method of this clinical study

You may not qualify if:

• \. Subjects who, in the opinion of the researcher, are unlikely to complete the study

Sponsors & Collaborators

• Yonsei University: lead

Study Sites (1)

Division of Cardiology, Yonsei Cardiovascular Hospital, Yonsei University College of Medicine

Seoul, 03722, South Korea

RECRUITING

MeSH Terms

Conditions

Fabry Disease

Condition Hierarchy (Ancestors)

Sphingolipidoses; Lysosomal Storage Diseases, Nervous System; Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Cerebral Small Vessel Diseases; Cerebrovascular Disorders; Vascular Diseases; Cardiovascular Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Metabolism, Inborn Errors; Lipidoses; Lipid Metabolism, Inborn Errors; Lysosomal Storage Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases; Lipid Metabolism Disorders

Study Officials

• Geu-Ru Hong

  Severance Hospital

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Geu-Ru Hong

CONTACT

02-2228-8443; grhong@yuhs.ac

Study Design

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

Study Record Dates

• First Submitted: December 28, 2022
• First Posted: January 10, 2023
• Study Start: January 11, 2023
• Primary Completion: May 12, 2023
• Study Completion: May 12, 2023
• Last Updated: January 20, 2023

Record last verified: 2023-01

Data Sharing

• IPD Sharing: Will not share

Locations

KR (1)