Registry of Patients Diagnosed With Lysosomal Storage Diseases
Launched by UNIVERSITY OF CALIFORNIA, SAN FRANCISCO · Nov 9, 2022
Trial Information
Current as of June 26, 2025
Recruiting
Keywords
ClinConnect Summary
This clinical trial is studying patients with lysosomal storage diseases (LSDs), which are a group of conditions that affect how the body breaks down certain substances. The goal of the study is to learn more about these diseases, how they progress, and how fetal therapies can improve outcomes for babies diagnosed with these conditions. This information will help doctors provide better care before and after birth for families affected by LSDs.
If you're a patient aged 0-64 with a diagnosed lysosomal storage disease, or if you are pregnant and your baby has been diagnosed with one of these diseases, you may be eligible to participate. There are no specific exclusions, so many individuals may be able to join. Participants will be asked to share their experiences and medical information, which will contribute to a better understanding of these diseases. The study is currently recruiting, and everyone's involvement can help improve future treatments and management for others with similar conditions.
Gender
ALL
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
About University Of California, San Francisco
The University of California, San Francisco (UCSF) is a leading academic institution renowned for its commitment to advancing healthcare through innovative research and clinical trials. With a focus on translating scientific discoveries into effective therapies, UCSF collaborates with a diverse array of stakeholders, including healthcare professionals, industry partners, and patient communities. The university's research programs emphasize interdisciplinary approaches and leverage cutting-edge technology to address complex medical challenges. As a sponsor of clinical trials, UCSF is dedicated to maintaining the highest standards of ethical conduct and scientific rigor, ensuring the safety and well-being of participants while contributing to the broader medical knowledge base.
Contacts
Jennifer Cobb
Immunology at National Institute of Allergy and Infectious Diseases (NIAID)
Locations
San Francisco, California, United States
Patients applied
Trial Officials
Tippi C MacKenzie, MD
Principal Investigator
University of California, San Francisco
Timeline
First submit
Trial launched
Trial updated
Estimated completion
Not reported
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