NCT05619900View on ClinicalTrials.gov

Registry of Patients Diagnosed With Lysosomal Storage Diseases

RecruitingOBSERVATIONAL
Enrollment

250

Participants

Timeline

Start Date

May 31, 2022

Primary Completion Date

May 31, 2050

Study Completion Date

May 31, 2050

Conditions
Mucopolysaccharidosis IMucopolysaccharidosis IIMucopolysaccharidosis IV AMucopolysaccharidosis VIMucopolysaccharidosis VIIPompe Disease Infantile-OnsetNeuronopathic Gaucher DiseaseWolman 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.

Trial Locations (1)

94143

RECRUITING

University of California San Francisco, San Francisco

All Listed Sponsors
lead

University of California, San Francisco

OTHER