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Status:

COMPLETED

Respiratory Cathepsins, Proteases Inhibitors and Glycosaminoglycans (GAG) in Mucopolysaccharidosis

Lead Sponsor:

University Hospital, Tours

Conditions:

Mucopolysaccharidoses

Eligibility:

All Genders

Up to 17 years

Brief Summary

Mucopolysaccharidosis (MPS) are a group of inherited, metabolic diseases caused by a deficiency of lysosomal enzymes that degrade glycosaminoglycans (GAGs). Loss of their activity results in cellular ...

Detailed Description

Mucopolysaccharidosis (MPS) are a group of inherited, metabolic diseases caused by a deficiency of lysosomal enzymes that degrade glycosaminoglycans (GAGs). Loss of their activity results in cellular ...

Eligibility Criteria

Inclusion

  • Patients with MPS:
  • Patient with a confirmed diagnosis (enzymatic activity, and/or genotyping) of MPS, all types (I, II, III, IV, VI, VII, IX)
  • Aged from 0 to 17 years old
  • Non inclusion Criteria:
  • Chronic respiratory disease independent of MPS disease (potential interferences with our analyzes)
  • Inability to obtain pulmonary samples
  • Refusal of the patient, parent or legal representative to participate in this study

Exclusion

  • Impossibility to use pulmonary samples (insufficient volume, conservation problem, etc.)
  • Non-MPS patients:
  • Inclusion Criteria:
  • Patient with no respiratory disease
  • Aged from 0 to 17 years old
  • Non inclusion Criteria:
  • Emergency medical situation
  • Inability to obtain pulmonary expectoration
  • Refusal of the patient, parent or legal representative to participate in this study

Key Trial Info

Start Date :

November 12 2019

Trial Type :

OBSERVATIONAL

Allocation :

ACTUAL

End Date :

June 26 2020

Estimated Enrollment :

27 Patients enrolled

Trial Details

Trial ID

NCT04112602

Start Date

November 12 2019

End Date

June 26 2020

Last Update

January 4 2022

Active Locations (7)

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Page 1 of 2 (7 locations)

1

Metabolic Disease Competence Centre - Medical Genetics Department - University Hospital, Angers

Angers, France

2

Metabolic Disease Reference Centre - Medical Genetics Department - University Hospital, Bordeaux

Bordeaux, France

3

Metabolic Disease Competence Centre - Medical Genetics Department - University Hospital, Brest

Brest, France

4

Metabolic Disease Competence Centre - Pediatrics Department - University Hospital, Rennes

Rennes, France