Phase 1/2 Clinical Trial of PR001 in Infants With Type 2 Gaucher Disease (PROVIDE)
Phase 1/2 Clinical Trial of PR001 in Infants With Type 2 Gaucher Disease (PROVIDE)
Active, not recruiting · NCT04411654 · Registry updated 2026-08-19Clinical trials
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| Trial | Conditions | Status | Phase | Updated | More info | NCT ID |
|---|---|---|---|---|---|---|
| Phase 1/2 Clinical Trial of PR001 in Infants With Type 2 Gaucher Disease (PROVIDE) | Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Gaucher Disease, Type 2, Gaucher disease, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Lysosomal Storage Diseases, Nervous System, Nutritional and Metabolic Diseases, brain disorder, central nervous system disorder, hereditary disease, inborn errors of metabolism, lysosomal lipid storage disorder, lysosomal storage disease, metabolic disease, nervous system disorder, sphingolipidosis | Active, not recruiting | Phase 1 / Phase 2 | 2026-08-19 | NCT04411654 |
2 results · Page 1 of 1
Organizations
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| Organizations | Linked trials |
|---|---|
| Eli Lilly and Company | 370 |
| Prevail Therapeutics | 5 |
6 results · Page 1 of 1
Research sites
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| Research sites | City | Country | Linked trials |
|---|---|---|---|
| Children's Hospital of Pittsburgh, 4401 Penn Avenue | Pittsburgh | United States | 1 |
| Lysosomal & Rare Disorders Research and Treatment Center | Fairfax | United States | 1 |
| Manchester Centre for Genomic Medicine, 6th Floor, St Mary's Hospital, Oxford Road | Manchester | United Kingdom | 1 |
| NYU Medical Center, 305 Second Ave, Suite 16 | New York | United States | 1 |
| UCSF Benioff Children's Hospital, 747 52nd St | Oakland | United States | 1 |
| University of Minnesota Masonic Children's Hospital, 2450 Riverside Avenue | Minneapolis | United States | 1 |