NCT05619900 Registry of Patients Diagnosed With Lysosomal Storage Diseases
| NCT ID | NCT05619900 |
| Status | Recruiting |
| Phase | — |
| Sponsor | University of California, San Francisco |
| Condition | Mucopolysaccharidosis I |
| Study Type | OBSERVATIONAL |
| Enrollment | 250 participants |
| Start Date | 2022-05-31 |
| Primary Completion | 2050-05-31 |
Eligibility & Interventions
Eligibility Fast-Check
Enter your details for a quick preliminary check. This does not replace medical advice.
What to Expect as a Participant
This is an observational study. You will not receive an experimental treatment; researchers will collect data based on your existing condition or standard treatment.
This trial targets 250 participants in total. It began in 2022-05-31 with a primary completion date of 2050-05-31.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief 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 Criteria
Inclusion Criteria: * Patients aged 0-64 with a diagnosis of a lysosomal storage disease * Pregnant patients whose fetus has a diagnosis of a lysosomal storage disease Exclusion Criteria: * There are no current exclusion criteria
Contact & Investigator
Tippi C MacKenzie, MD
PRINCIPAL INVESTIGATOR
University of California, San Francisco
Frequently Asked Questions
Who can join the NCT05619900 clinical trial?
This trial is open to participants of all sexes, up to 64 Years, studying Mucopolysaccharidosis I. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
Is NCT05619900 currently recruiting?
Yes, NCT05619900 is actively recruiting participants. Contact the research team at billie.lianoglou@ucsf.edu for enrollment information.
Where is the NCT05619900 trial being conducted?
This trial is being conducted at San Francisco, United States.
Who is sponsoring the NCT05619900 clinical trial?
NCT05619900 is sponsored by University of California, San Francisco. The principal investigator is Tippi C MacKenzie, MD at University of California, San Francisco. The trial plans to enroll 250 participants.