Study of the Quality of Life of Patients With Fabry Disease Aged 65 and Over With and Without Specific Treatment

Plain-language summary

This study looks at the quality of life of people aged 65 and over living with Fabry disease, comparing those who receive specific treatment with those who do not. Men with a proven alpha-galactosidase A deficiency or a pathogenic GLA gene variant, and women with an identified pathogenic GLA variant, aged 65 and over, can take part. It is a study without an assigned phase and is currently recruiting participants.

The summary above is a plain-language rendering of the official record. The original English title is shown for reference.

Trial details

Phase

N/A

Status

Recruiting

Sponsor

Wladimir MAUHIN, Dr

Start date

10/8/2024

Estimated completion

10/14/2031

Countries

France

Related genes

Locations

• Paris, France

Eligibility criteria

Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.

Inclusion Criteria:

* Men and women aged 65 and over with a diagnosis of Fabry disease with, for men, a proven alpha-galactosidase A deficiency or an identified pathogenic GLA genetic variant, and for women, an identified pathogenic GLA variant.
* Minimum work-up available: ECG, 24h holterECG, cardiac ultrasound, creatinemia, proteinuria and/or microalbuminuria.
* Have received written and oral information about the protocol and have not expressed any opposition to participating in the study.
* Affiliated to a social security scheme or entitled to benefits (excluding AME).

Exclusion Criteria:

* Inability to understand the information provided,
* Under guardianship, curatorship or safeguard of justice,
* Under restraint or deprived of liberty by judicial or administrative decision.