Stem Cell Gene Therapy for Cystinosis | MicrobiomeTrials← Back to trialsNCT03897361
Stem Cell Gene Therapy for Cystinosis
CompletedPHASE1, PHASE2INTERVENTIONAL
Trial Details
Start: 2019-07-08Est. Completion: 2024-09-18
Enrollment target: 7 participants Locations- University of California San Diego, La Jolla, California, United States
Other microbiome trials you might be interested in
Lysosomal DiseasesGangliosidosisGM1
Background:
GM1 gangliosidosis is a disorder that destroys nerve cells. It is fatal. There is no treatment. People with…
Bethesda, United StatesEst. completion Jan 1, 2030
Lysosomal Storage DisordersGaucher DiseaseParkinson Disease
The purpose of this study is to identify genetic, biochemical, and clinical factors that are associated with disease sev…
Bethesda, United States
Cystinosis is an inherited disease resulting in poor growth and kidney failure. There is no known cure for cystinosis, a…