Fabry DiseaseNCT05679076
A Qualitative Study on the Pain and Quality of Life of Patients With Fabry Disease
An observational study of Fabry Disease, run by Yonsei University at 1 site in South Korea.
Trial 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. Read more about this study
- Who is it for?
- Ages 19 years and older
- Conditions the study is for
- Fabry Disease
- How many people
- 10 participants
- Where
- 1 siteSouth Korea
- Study dates
- 11 Jan 2023to 12 May 2023
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 19 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: Fabry patients
Who may be able to join
- 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
Who cannot join
- 1. Subjects who, in the opinion of the researcher, are unlikely to complete the study
Trial locations and contacts
1 site in 1 country.
South Korea1 site, 1 recruiting
- Recruiting
Division of Cardiology, Yonsei Cardiovascular Hospital, Yonsei University College of Medicine
Seoul, 03722
Geu-Ru Hong, MD, PhD, 82-2-2228-8443, GRHONG@yuhs.ac
About this study
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.
Read more
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
- Medical subject headings
- Fabry Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| Fabry patients | Fabry patients |
What is being measured
The primary outcome is the main question the study is designed to answer.
Identify negative problems experienced by patients with Fabry disease
Measured: up to 5 months
Check the quality of life of patients with Fabry disease
Measured: up to 5 months
Identifying the structural causes of policies for patients with Fabry disease
Measured: up to 5 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Jan 2023
- Study start11 Jan 2023
- Primary completion12 May 2023 (estimated)
- Study completion12 May 2023 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Fabry DiseaseStudies of the same condition, open and under way.
- More trials run by Yonsei UniversityThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Cross-sectional
- Enrolment
- 10 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Yonsei University
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Geu-Ru Hong, Principal investigator, Severance Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05679076
- Sponsor's protocol ID
- 4-2022-1347
- Status last verified
- Jan 2023
- First submitted
- 28 Dec 2022
- Last updated
- 20 Jan 2023
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 20 Jan 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Qualitative Study on the Pain and Quality of Life of Patients With Fabry Disease
- ClinicalTrials.gov ID
- NCT05679076
- Acronym / sponsor ID
- 4-2022-1347
- Conditions
- Fabry Disease
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 10 participants (estimated)
- Lead sponsor
- Yonsei UniversityUniversity, hospital or other
- Study start
- 11 Jan 2023 (actual)
- Primary completion
- 12 May 2023 (estimated)
- Study completion
- 12 May 2023 (estimated)
- First posted
- 10 Jan 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Observational model
- Cohort
- Time perspective
- Cross-sectional
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Fabry patients | – | –Fabry patients |
Who may qualify
- Ages
- 19 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- Fabry patients
Key inclusion criteria (5 listed)
- 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
Key exclusion criteria (1 listed)
- 1. Subjects who, in the opinion of the researcher, are unlikely to complete the study
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Identify negative problems experienced by patients with Fabry disease
Time frame: up to 5 months
Check the quality of life of patients with Fabry disease
Time frame: up to 5 months
Identifying the structural causes of policies for patients with Fabry disease
Time frame: up to 5 months
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 20 Jan 2023.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Geu-Ru Hong principal investigator
Severance Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Fabry Disease, run by Yonsei University at 1 site in South Korea.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 20 Jan 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in South Korea. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Geu-Ru Hong (02-2228-8443, grhong@yuhs.ac), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Identify negative problems experienced by patients with Fabry disease (time frame: up to 5 months). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 20 Jan 2023.