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RECRUITING

NCT05619900

Registry of Patients Diagnosed With Lysosomal Storage Diseases

Sponsor: University of California, San Francisco

View on ClinicalTrials.gov

Summary

This is an international prospective and retrospective registry of patients with Lysosomal Storage Diseases (LSDs) to understand the natural history of the disease and the outcomes of fetal therapies, with the overall goal of improving the prenatal management of patients with LSDs.

Key Details

Gender

All

Age Range

Any - 64 Years

Study Type

OBSERVATIONAL

Enrollment

250

Start Date

2022-05-31

Completion Date

2050-05-31

Last Updated

2026-04-08

Healthy Volunteers

Conditions

Mucopolysaccharidosis I Mucopolysaccharidosis II Mucopolysaccharidosis IV A Mucopolysaccharidosis VI Mucopolysaccharidosis VII Pompe Disease Infantile-Onset Neuronopathic Gaucher Disease Wolman Disease

Interventions

OTHER

There is no intervention

This is an observational study. There is no intervention. The purpose of the project is to create a database of patients diagnosed either prenatally or after birth with a lysosomal storage disease. The database will be utilized to assess patient outcomes, build on existing clinical management, improve medical decision making, and improve quality of care.

Locations (1)

University of California San Francisco

San Francisco, California, United States