Mucopolysaccharidosis Patients in France in the Era of Specific Therapeutics
Status: Recruiting
Location: See all (23) locations...
Study Type: Observational
SUMMARY
The goal of this observational study is to characterize the epidemiology and natural history of MPS diseases by building a retrospective and prospective collection of extensive phenotypic data from French MPS patients.
Eligibility
Participation Requirements
Sex: All
Healthy Volunteers: f
View:
• Confirmed diagnosis of MPS based on clinically relevant enzyme deficiency, with abnormally elevated GAG urinary excretion and/or identification of pathogenic mutations.
• Signed informed consent or parents/guardian non-opposition for deceased patients (minor or protected major)
Locations
Other Locations
France
Centre Hospitalier Universitaire d'Angers
RECRUITING
Angers
Hôpital des Enfants - Groupe Hospitalier Pellegrin
NOT_YET_RECRUITING
Bordeaux
Hôpital Morvan
RECRUITING
Brest
Hôpital d'Estaing
NOT_YET_RECRUITING
Clermont-ferrand
Hôpital Beaujon
RECRUITING
Clichy
Hôpital Raymond-Poincaré
NOT_YET_RECRUITING
Garches
Hôpital Jeanne de Flandre
RECRUITING
Lille
Hôpital de la Timone
RECRUITING
Marseille
Hôpital Gui de Chauliac
RECRUITING
Montpellier
Hôpital Brabois
RECRUITING
Nancy
Hôpital Armand Trousseau
RECRUITING
Paris
Hôpital de la Croix Saint-Simon
NOT_YET_RECRUITING
Paris
Hôpital de la Pitié-Salpêtrière
RECRUITING
Paris
Hôpital Necker-Enfants Malades
RECRUITING
Paris
Hôpital Robert Debré
RECRUITING
Paris
Centre Hospitalier de Pau
RECRUITING
Pau
American Memorial Hospital
NOT_YET_RECRUITING
Reims
Hôpital Pontchaillou
RECRUITING
Rennes
Hôpital Charles Nicolle
NOT_YET_RECRUITING
Rouen
Hôpital de Hautepierre
NOT_YET_RECRUITING
Strasbourg
Clinique Monié
NOT_YET_RECRUITING
Toulouse
Hôpital des Enfants
NOT_YET_RECRUITING
Toulouse
Hôpital Clocheville
RECRUITING
Tours
Contact Information
Primary
Bénédicte HERON
benedicte.heron@aphp.fr
01 44 73 65 75
Time Frame
Start Date: 2017-12-20
Estimated Completion Date: 2026-12
Participants
Target number of participants: 1000
Related Therapeutic Areas
Sponsors
Leads: Institut National de la Santé Et de la Recherche Médicale, France