RecruitingNCT05619900

Registry of Patients Diagnosed With Lysosomal Storage Diseases


Sponsor

University of California, San Francisco

Enrollment

250 participants

Start Date

May 31, 2022

Study Type

OBSERVATIONAL

Conditions

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.


Eligibility

Max Age: 64 Years

Plain Language Summary

Simplified for easier understanding

This is a long-term patient registry collecting information from patients with lysosomal storage diseases (LSDs) — a group of rare inherited conditions where the body cannot break down certain substances properly due to missing or faulty enzymes. Examples include Gaucher disease, Fabry disease, Pompe disease, and Mucopolysaccharidoses. The registry helps researchers understand how these diseases progress and how treatments work over time. You may be eligible if: - You are between 0 and 64 years of age - You have been diagnosed with a lysosomal storage disease - OR you are pregnant and your unborn baby has been diagnosed with a lysosomal storage disease You may NOT be eligible if: - There are no current exclusion criteria for this registry Talk to your doctor to see if this trial is right for you.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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Interventions

OTHERThere 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

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NCT05619900


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