An observational study in Mucopolysaccharidosis I, Mucopolysaccharidosis II and Mucopolysaccharidosis III, sponsored by Institut National de la Santé Et de la Recherche Médicale, France. Recruiting at 23 sites in France. Per ClinicalTrials.gov, last updated 2026-02-11.
Sponsored by Institut National de la Santé Et de la Recherche Médicale, France · Observational
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.
74 studies on the registry are indexed under Mucopolysaccharidosis I; 7 are open to participants now.
This study's planned enrollment of 1,000 is above the median of 41 across 21 observational studies indexed under Mucopolysaccharidosis I.
Browse Mucopolysaccharidosis I studies →Institut National de la Santé Et de la Recherche Médicale, France is the lead sponsor of 378 studies on the registry; 81 are open to participants now.
Counted across the registry records on this site, refreshed daily.
Prevalent and incident patients will be included in the cohort RaDiCo-MPS.
Inclusion Criteria:
There are no non-inclusion criteria.
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Clinically significant
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Evaluation of the clinical data of MPS like growth for each system
Time frame: Through study completion, an average of 5 years
Evaluation of the clinical data of MPS like signs for each system
Time frame: Through study completion, an average of 5 years
Evaluation of the clinical data of MPS like symptoms for each system
Time frame: Through study completion, an average of 5 years
Evaluation of the clinical data of MPS like complications for each system
Time frame: Through study completion, an average of 5 years
Evaluation of the clinical data of MPS like psychomotor milestones
Time frame: Through study completion, an average of 5 years
Evaluation of the clinical data of MPS like cognitive evolution
Time frame: 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
Time frame: 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
Time frame: Through study completion, an average of 5 years
Evaluation of the radiological data of MPS like standard bone radiographs
Time frame: Through study completion, an average of 5 years
Evaluation of the radiological data of MPS like abdominal echography
Time frame: Through study completion, an average of 5 years
Evaluation of the radiological data of MPS like echocardiography
Time frame: Through study completion, an average of 5 years
Evaluation of the radiological data of MPS like cerebral and medullar tomodensitometry
Time frame: Through study completion, an average of 5 years
Evaluation of the radiological data of MPS like magnetic resonance imaging
Time frame: Through study completion, an average of 5 years
Evaluation of the electrophysiological data of MPS like EMG
Time frame: Through study completion, an average of 5 years
Evaluation of the electrophysiological data of MPS like EEG
Time frame: Through study completion, an average of 5 years
Evaluation of the electrophysiological data of MPS like ERG
Time frame: Through study completion, an average of 5 years
Evaluation of the biochemical data of MPS like urinary GAG before specific treatment
Time frame: Through study completion, an average of 5 years
Evaluation of the biochemical data of MPS like urinary GAG during specific treatment
Time frame: Through study completion, an average of 5 years
Evaluation of the biochemical data of MPS like enzyme activities before specific treatment
Time frame: Through study completion, an average of 5 years
Evaluation of the biochemical data of MPS like enzyme activities during specific treatment
Time frame: Through study completion, an average of 5 years
Evaluation of the biochemical data of MPS like specific antibodies
Time frame: Through study completion, an average of 5 years
Evaluation of the molecular data of MPS
Time frame: Through study completion, an average of 5 years
Description of the management of MPS diseases without specific treatment
Time frame: Through study completion, an average of 5 years
Description of the management of MPS diseases before specific treatment
Time frame: Through study completion, an average of 5 years
Description of the management of MPS diseases under specific treatment.
Time frame: Through study completion, an average of 5 years
Description of the outcome of MPS diseases without specific treatment
Time frame: Through study completion, an average of 5 years
Description of the outcome of MPS diseases before specific treatment
Time frame: Through study completion, an average of 5 years
Description of the outcome of MPS diseases under specific treatment.
Time frame: Through study completion, an average of 5 years
Identification of mutation(s) in each MPS type
Time frame: Through study completion, an average of 5 years
Establishment of genotype/phenotype relationships in each MPS type.
Time frame: Through study completion, an average of 5 years
Eligibility is decided by the study team. Share this record with your doctor or contact the team directly.
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