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MPS (RaDiCo Cohort) (RaDiCo-MPS)

NCT ID: NCT06036693

Last Updated: 2025-01-31

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Total Enrollment

1000 participants

Study Classification

OBSERVATIONAL

Study Start Date

2017-12-20

Study Completion Date

2026-12-31

Brief Summary

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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.

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Detailed Description

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Conditions

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Mucopolysaccharidosis I Mucopolysaccharidosis II Mucopolysaccharidosis III Mucopolysaccharidosis IV Mucopolysaccharidosis VI Mucopolysaccharidosis VII Mucopolysaccharidosis IX Multiple Sulfatase Deficiency Disease

Study Design

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Observational Model Type

COHORT

Study Time Perspective

OTHER

Eligibility Criteria

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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)
Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Institut National de la Santé Et de la Recherche Médicale, France

OTHER_GOV

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Thierry BILLETTE DE VILLEMEUR

Role: PRINCIPAL_INVESTIGATOR

INSERM UMR 1141

Locations

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Centre Hospitalier Universitaire d'Angers

Angers, , France

Site Status RECRUITING

Hôpital des Enfants - Groupe Hospitalier Pellegrin

Bordeaux, , France

Site Status NOT_YET_RECRUITING

Hôpital Morvan

Brest, , France

Site Status RECRUITING

Hôpital d'Estaing

Clermont-Ferrand, , France

Site Status NOT_YET_RECRUITING

Hôpital Beaujon

Clichy, , France

Site Status RECRUITING

Hôpital Raymond-Poincaré

Garches, , France

Site Status NOT_YET_RECRUITING

Hôpital Jeanne de Flandre

Lille, , France

Site Status RECRUITING

Hôpital de la Timone

Marseille, , France

Site Status RECRUITING

Hôpital Gui de Chauliac

Montpellier, , France

Site Status RECRUITING

Hôpital Brabois

Nancy, , France

Site Status RECRUITING

Hôpital Armand Trousseau

Paris, , France

Site Status RECRUITING

Hôpital de la Croix Saint-Simon

Paris, , France

Site Status NOT_YET_RECRUITING

Hôpital de la Pitié-Salpêtrière

Paris, , France

Site Status RECRUITING

Hôpital Necker-Enfants Malades

Paris, , France

Site Status RECRUITING

Hôpital Robert Debré

Paris, , France

Site Status RECRUITING

Centre Hospitalier de Pau

Pau, , France

Site Status RECRUITING

American Memorial Hospital

Reims, , France

Site Status NOT_YET_RECRUITING

Hôpital Pontchaillou

Rennes, , France

Site Status RECRUITING

Hôpital Charles Nicolle

Rouen, , France

Site Status NOT_YET_RECRUITING

Hôpital de Hautepierre

Strasbourg, , France

Site Status NOT_YET_RECRUITING

Clinique Monié

Toulouse, , France

Site Status NOT_YET_RECRUITING

Hôpital des Enfants

Toulouse, , France

Site Status NOT_YET_RECRUITING

Hôpital Clocheville

Tours, , France

Site Status RECRUITING

Countries

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France

Central Contacts

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Bénédicte HERON

Role: CONTACT

[email protected]
01 44 73 65 75

Facility Contacts

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Magalie BARTH

Role: primary

Didier LACOMBE

Role: primary

Elise SACAZE

Role: primary

Marc BERGER

Role: primary

Nadia BELMATOUG

Role: primary

Dominique-Paul GERMAIN

Role: primary

Anne-Sophie GUEMANN

Role: primary

Brigitte CHABROL

Role: primary

Agathe ROUBERTIE

Role: primary

François FEILLET

Role: primary

Bénédicte HERON

Role: primary

Wladimir MAUHIN

Role: primary

Yann NADJAR

Role: primary

Anaïs BRASSIER

Role: primary

Samia PICHARD

Role: primary

Pierre BEZE-BEYRIE

Role: primary

Nathalie BEDNAREK

Role: primary

Bérengère CADOR

Role: primary

Stéphanie TORRE

Role: primary

Marie-Thérèse ABI WARDE

Role: primary

Francis GACHES

Role: primary

Guy TOUATI

Role: primary

Marine TARDIEU

Role: primary

Other Identifiers

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C16-53

Identifier Type: -

Identifier Source: org_study_id

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