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

Summary

Fabry disease is a rare genetic disorder affecting 1 in 10,000 individuals, leading to complications such as chronic pain, heart and kidney failure, and strokes, ultimately impacting life expectancy. People with this disease are increasingly being diagnosed later in life, around the age of 65, as the condition progresses slowly with irreversible organ damage. The effectiveness of treatments for Fabry disease remains controversial, but early initiation is recommended for long-term benefits. Despite the high cost and inconvenience of treatments, there is limited research on their efficacy in older people or on the quality of life for those aged 65 and over with Fabry disease. This study aims to assess the quality of life in this age group both with and without treatment over a period of 5 years to determine the benefits of treatment beyond the age of 65.

Conditions

• Fabry Disease
• Aged 65 Years or Older
• Alpha Galactosidase A Deficiency
• Galactosidase A Gene Mutation

Sponsors & Collaborators

• University Hospital, Angers

  collaborator OTHER_GOV

• University Hospital, Bordeaux

  collaborator OTHER

• Centre Hospitalier Bretagne Atlantique

  collaborator OTHER

• Centre Hospitalier Universitaire de Caen

  collaborator OTHER

• University Hospital, Clermont-Ferrand

  collaborator OTHER

• Centre Hospitalier Universitaire Dijon

  collaborator OTHER

• University Hospital, Lille

  collaborator OTHER

• University Hospital, Limoges

  collaborator OTHER

• University Hospital, Marseille

  collaborator OTHER

• Nantes University Hospital

  collaborator OTHER

• Necker Hospital, 75015 Paris

  collaborator UNKNOWN

• Rennes University Hospital

  collaborator OTHER

• University Hospital, Tours

  collaborator OTHER

• Hôpital Raymond Poincaré

  collaborator OTHER

• University Hospital, Rouen

  collaborator OTHER

• Hospital, Vannes

  collaborator UNKNOWN

• University Hospital, Brest

  collaborator OTHER

• University Hospital, Grenoble

  collaborator OTHER

• University Hospital, Strasbourg

  collaborator OTHER

• Hospices Civils de Lyon

  collaborator OTHER

• Centre Hospitalier de la côte Basque

  collaborator OTHER

• University Hospital, Montpellier

  collaborator OTHER

• Centre Hospitalier Universitaire de Nice

  collaborator OTHER

• Wladimir MAUHIN, Dr

  lead OTHER

Principal Investigators

• Wladimir MAUHIN, Doctor · Groupe Hospitalier Diaconesses Croix Saint-Simon

Eligibility

Min Age

65 Years

Sex

ALL

Healthy Volunteers

No

Timeline & Regulatory

Start

2024-10-08

Primary Completion

2029-10-14

Completion

2031-10-14

Countries

• France

Study Locations