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NCT Number: NCT00144794

Mucopolysaccharidosis I (MPS I) Registry

The Mucopolysaccharidosis I (MPS I) Registry is an ongoing, observational database that tracks the outcomes of patients with MPS I. The data collected by the MPS I Registry will provide information to better characterize the natural history and progression of MPS I as well as the clinical responses of patients receiving enzyme replacement therapy, such as Aldurazyme (Recombinant Human Alpha-L-Iduronidase), or other treatment modalities.

The objectives of the Registry are:

* To evaluate the long-term effectiveness and safety of Aldurazyme® (laronidase) * To characterize and describe the MPS I population as a whole, including the variability, progression, and natural history of MPS I * To help the MPS I medical community with the development of recommendations for monitoring patients and reports on patient outcomes to optimize patient care

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Key information

Conditions

Mucopolysaccharidosis I (MPS I) Carbohydrate Metabolism, Inborn Errors Congenital, Hereditary, and Neonatal Diseases and Abnormalities Connective Tissue Diseases Genetic Diseases, Inborn Lysosomal Storage Diseases Metabolic Diseases Metabolism, Inborn Errors Mucinoses Mucopolysaccharidoses Mucopolysaccharidosis I Nutritional and Metabolic Diseases Skin and Connective Tissue Diseases

Sex eligibility

All sexes

Study type

Observational

Primary location

Investigational Site Number : 153032, Buenos Aires, Argentina

Loading trial locations.

About this study

The MPS I Registry is an international program; in addition to the central contact information provided under the "Location" heading, patients may contact:

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • All patients with a confirmed diagnosis of MPS I are eligible for inclusion. Confirmed diagnosis is defined as: A. documented biochemical evidence of a deficiency in alpha (a)-L-iduronidase enzyme activity and/or B. mutation(s) in the gene coding for a-L-iduronidase, or measurable clinical signs and symptoms of MPS I
  • For all patients there should be a completed patient authorization form

Exclusion criteria

  • No exclusion criteria for participation in the MPS I Registry. NOTE: Registry participation does not exclude participation in other clinical studies.

Treatment and study plan

Primary outcomes

  1. To evaluate the long-term effectiveness of Aldurazyme

    Time frame: Approximately 17 Years

Sponsors and collaborators

Lead sponsor

Genzyme, a Sanofi Company

Industry

Registry information

Important dates

Study start
2003
Primary completion
2024
Study completion
2024
First posted
Sep 5, 2005
Registry last updated
Feb 27, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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