NCT05619900Recruiting
Registry of Patients Diagnosed With Lysosomal Storage Diseases
Source: ClinicalTrials.gov · Last updated 8 Apr 2026
Brief summary (registry, in English)
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.
- Status (registry)
- Recruiting
- Study type
- Observational
- Enrollment
- 250 (estimated)
- First submitted
- 9 Nov 2022
- Start date
- 31 May 2022
- Primary completion date
- 31 May 2050
- Lead sponsor
- University of California, San Francisco
- Diseases
- Pompe disease, Gaucher disease
- NCT number
- NCT05619900
What you commit to
- Study type: Observational
- Planned participants: 250
- From 31 May 2022 to the primary completion date, 31 May 2050
- 1 site in 1 country
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Other
- There are no current exclusion criteria
Who the study is looking for
- Patients aged 0-64 with a diagnosis of a lysosomal storage disease
- Pregnant patients whose fetus has a diagnosis of a lysosomal storage disease
Age in the registry: - to 64 years · Sex: All · Does not accept healthy volunteers
Sites (1)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| University of California San Francisco Coordinator of this site? Confirm its status | San Francisco | United States | Recruiting |
This page is information, not medical advice. Discuss any trial with your doctor.