Status:
RECRUITING
MPS (RaDiCo Cohort) (RaDiCo-MPS)
Lead Sponsor:
Institut National de la Santé Et de la Recherche Médicale, France
Conditions:
Mucopolysaccharidosis I
Mucopolysaccharidosis II
Eligibility:
All Genders
Brief 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 Fr...
Eligibility Criteria
Inclusion
- Inclusion Criteria:
- 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)
- There are no non-inclusion criteria.
Exclusion
Key Trial Info
Start Date :
December 20 2017
Trial Type :
OBSERVATIONAL
Allocation :
ESTIMATED
End Date :
December 1 2026
Estimated Enrollment :
1000 Patients enrolled
Trial Details
Trial ID
NCT06036693
Start Date
December 20 2017
End Date
December 1 2026
Last Update
January 31 2025
Active Locations (23)
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1
Centre Hospitalier Universitaire d'Angers
Angers, France
2
Hôpital des Enfants - Groupe Hospitalier Pellegrin
Bordeaux, France
3
Hôpital Morvan
Brest, France
4
Hôpital d'Estaing
Clermont-Ferrand, France