Clinical Trials in France / NCT06036693
Recruiting
Observational
MPS (RaDiCo Cohort) (RaDiCo-MPS)
NCT06036693 · tracked via the Priya Life Science France tracker
Condition(s) studied
Mucopolysaccharidosis IMucopolysaccharidosis IIMucopolysaccharidosis IIIMucopolysaccharidosis IVMucopolysaccharidosis VIMucopolysaccharidosis VIIMucopolysaccharidosis IXMultiple Sulfatase Deficiency Disease
Study 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
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.
Primary outcome measure(s)
- Evaluation of the clinical data of MPS like growth for each system — Through study completion, an average of 5 years
- Evaluation of the clinical data of MPS like signs for each system — Through study completion, an average of 5 years
- Evaluation of the clinical data of MPS like symptoms for each system — Through study completion, an average of 5 years
- Evaluation of the clinical data of MPS like complications for each system — Through study completion, an average of 5 years
- Evaluation of the clinical data of MPS like psychomotor milestones — Through study completion, an average of 5 years
- Evaluation of the clinical data of MPS like cognitive evolution — Through study completion, an average of 5 years
- Evaluation of the clinical data of MPS like handicap using scales adapted to multivisceral disease for all types of MPS — Through study completion, an average of 5 years
- Evaluation of the clinical data of MPS like handicap using scales adapted to cognitive and neurologic disease for the types I, II, III VII — Through study completion, an average of 5 years
- Evaluation of the radiological data of MPS like standard bone radiographs — Through study completion, an average of 5 years
- Evaluation of the radiological data of MPS like abdominal echography — Through study completion, an average of 5 years
- Evaluation of the radiological data of MPS like echocardiography — Through study completion, an average of 5 years
- Evaluation of the radiological data of MPS like cerebral and medullar tomodensitometry — Through study completion, an average of 5 years
- Evaluation of the radiological data of MPS like magnetic resonance imaging — Through study completion, an average of 5 years
- Evaluation of the electrophysiological data of MPS like EMG — Through study completion, an average of 5 years
- Evaluation of the electrophysiological data of MPS like EEG — Through study completion, an average of 5 years
- Evaluation of the electrophysiological data of MPS like ERG — Through study completion, an average of 5 years
- Evaluation of the biochemical data of MPS like urinary GAG before specific treatment — Through study completion, an average of 5 years
- Evaluation of the biochemical data of MPS like urinary GAG during specific treatment — Through study completion, an average of 5 years
- Evaluation of the biochemical data of MPS like enzyme activities before specific treatment — Through study completion, an average of 5 years
- Evaluation of the biochemical data of MPS like enzyme activities during specific treatment — Through study completion, an average of 5 years
- Evaluation of the biochemical data of MPS like specific antibodies — Through study completion, an average of 5 years
- Evaluation of the molecular data of MPS — Through study completion, an average of 5 years
Trial sites (23)
| Facility | City | Region | Status |
|---|---|---|---|
| Centre Hospitalier Universitaire d'Angers | Angers | France | Recruiting |
| Hôpital des Enfants - Groupe Hospitalier Pellegrin | Bordeaux | France | Not Yet Recruiting |
| Hôpital Morvan | Brest | France | Recruiting |
| Hôpital d'Estaing | Clermont-Ferrand | France | Not Yet Recruiting |
| Hôpital Beaujon | Clichy | France | Recruiting |
| Hôpital Raymond-Poincaré | Garches | France | Not Yet Recruiting |
| Hôpital Jeanne de Flandre | Lille | France | Recruiting |
| Hôpital de la Timone | Marseille | France | Recruiting |
| Hôpital Gui de Chauliac | Montpellier | France | Recruiting |
| Hôpital Brabois | Nancy | France | Recruiting |
| Hôpital Armand Trousseau | Paris | France | Recruiting |
| Hôpital de la Croix Saint-Simon | Paris | France | Not Yet Recruiting |
| Hôpital de la Pitié-Salpêtrière | Paris | France | Recruiting |
| Hôpital Necker-Enfants Malades | Paris | France | Recruiting |
| Hôpital Robert Debré | Paris | France | Recruiting |
| Centre Hospitalier de Pau | Pau | France | Recruiting |
| American Memorial Hospital | Reims | France | Not Yet Recruiting |
| Hôpital Pontchaillou | Rennes | France | Recruiting |
| Hôpital Charles Nicolle | Rouen | France | Not Yet Recruiting |
| Hôpital de Hautepierre | Strasbourg | France | Not Yet Recruiting |
| Clinique Monié | Toulouse | France | Not Yet Recruiting |
| Hôpital des Enfants | Toulouse | France | Not Yet Recruiting |
| Hôpital Clocheville | Tours | France | Recruiting |
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Official registry record
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
View NCT06036693 on ClinicalTrials.gov ↗ ← All trials in France