Gaucher Disease Trial, Recruiting
Who may be able to join
AI generated eligibility summary. Written by an AI model from the official source data and checked on a sample basis. It can contain mistakes, so confirm anything important against the original source. How we use AI
Who might be able to join this trial:
- People aged 0 to 64 years old who have been diagnosed with a lysosomal storage disease (a type of inherited condition where the body cannot break down certain substances properly)
- Pregnant people whose unborn baby has been diagnosed with a lysosomal storage disease
Who may not be able to join:
- There are currently no stated reasons that would prevent someone from joining this trial
Important: Always verify eligibility with the trial site directly before applying.
Based on publicly available eligibility criteria from ClinicalTrials.gov. Verify directly with the trial site before acting. This is not medical advice.
Contact this trial
Principal Investigator: Tippi C MacKenzie, MD, University of California, San Francisco
Phone: 415-476-2461
Contact details sourced from ClinicalTrials.gov. Verify directly with the trial site before attending.
GP referral letter
Print a one-page summary to share with your doctor.
Trial details
Where this trial is recruiting
Primary endpoints
Number of patients with and types of prenatal features of Lysosomal Storage Diseases; Number of participants with the presence and levels of glycosaminoglycans (GAGs) in urine.; Number of participants that show measured levels of antibodies against the enzyme.; Number of participants that show functional cardiac, growth, mobility, and neurocognitive function.
Can't join this trial?
Data last synced from ClinicalTrials.gov: 28 July 2026. Trial status can change. Always verify current status directly with the trial site before making any decision.