Registry of Patients Diagnosed With Lysosomal Storage Diseases — LSD Registry  

Mucopolysaccharidosis II Clinical Trial

Official title: Registry of Patients Diagnosed With Lysosomal Storage Diseases

Status Recruiting

Clinical Trial 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.

Clinical Trial Description

The need for methods to track patient outcomes, clinical management, medical decision making, and quality of care are all part of current national mandates in patient safety and quality of care delivery.

The aim of this registry is to prospectively and retrospectively collect data on patients who are diagnosed with Lysosomal Storage Disease and other LSD mutations. Data collected will be used to:

1. Identify patient outcomes of therapies.

2. Improve clinical management of patients with LSDs.

3. Improve medical decision making.

4. Improve quality of care. ;

Related Conditions & MeSH terms

• Gaucher Disease
• Glycogen Storage Disease Type II
• Lysosomal Storage Diseases
• Mucopolysaccharidoses
• Mucopolysaccharidosis I
• Mucopolysaccharidosis II
• Mucopolysaccharidosis IV
• Mucopolysaccharidosis IV A
• Mucopolysaccharidosis VI
• Mucopolysaccharidosis VII
• Pompe Disease Infantile-Onset
• Wolman Disease

• NCT number: NCT05619900
• Study type: Observational [Patient Registry]
• Source: University of California, San Francisco
• Contact: Billie Lianoglou, MS
• Phone: 415-476-2461
• Email: billie.lianoglou@ucsf.edu
• Status: Recruiting
• Start date: May 31, 2022
• Completion date: May 31, 2050