Quality of Life in Adults With Untreated Fabry Disease in Sweden (QoLUF)

Sponsor
Vastra Gotaland Region
Study ID
NCT07560956
Status
Not Yet Recruiting

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Conditions

Eligibility Criteria

Sex
ALL
Age
18 Years - N/A
Healthy Volunteers
Not accepted

Interventions

  • Qualitative Interview — OTHER
    Non-interventional

Study Details

Fabry disease is a rare, inherited condition that can affect multiple organs, including the heart, kidneys, and nervous system. Many people with Fabry disease live for a period of time without receiving disease-specific treatment, for example because treatment criteria have not yet been met. There is limited knowledge about how patients experience this period without treatment. The purpose of this study is to improve understanding of how adults with Fabry disease who are not yet receiving disease-specific treatment experience their quality of life, their daily lives, and their contacts with health care services. This is a national interview study in which participants will take part in one individual digital interview lasting approximately 45-60 minutes. During the interview, participants will be invited to describe their experiences in their own words, including symptoms, how the disease affects everyday life, psychological and emotional well-being, and experiences of follow-up and support from health care providers. Participants will also complete a short questionnaire with basic background information, such as age, education, and living situation. By collecting and analyzing patients' own narratives, the study aims to identify needs, challenges, and support resources during the period without treatment. The results are expected to contribute to increased knowledge and to the development of more person-centered care for individuals living with Fabry disease.

Key Dates

Start date
May 1, 2026
Status verified
Apr 2026
Primary completion
Dec 31, 2027
Completion
Dec 31, 2027

Study Design

Enrollment
35 participants (estimated)

Arms

  • Arm: Adults with Fabry Disease Not Receiving Disease-Specific Treatment
    This group includes adult patients diagnosed with Fabry disease who are not currently receiving disease-specific treatment (such as enzyme replacement therapy or chaperone therapy). Participants are followed at national highly specialized care centers in Sweden and take part in a single qualitative interview to describe their experiences of living with Fabry disease without current treatment.

Primary Outcome Measure

Patient-reported experiences of quality of life while living with untreated Fabry disease [ Time Frame: Single assessment during one qualitative interview (approximately 45-60 minutes) ]

Central Contacts

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