Phase 3 Study to Evaluate Intravenous Trappsol(R) Cyclo(TM) in Pediatric and Adult Patients With Niemann-Pick Disease Type C1
Condition: Niemann-Pick Disease, Type C1 · Sponsor: Cyclo Therapeutics, Inc.
About this study
A prospective, randomized, double-blind, placebo controlled, multi-center therapeutic study for patients age 3 and older with confirmed diagnosis of Niemann Pick disease type C1 (NPC1). The objective of this study is to evaluate the safety, tolerability and efficacy of 2000 mg/kg dose of Trappsol Cyclo (hydroxypropyl betacyclodextrin) administered intravenously compared to standard of care. An open-label sub-study in countries following European Medicines Agency (EMA) guidance will enroll asymptomatic or symptomatic patients from infancy up to age 3 to evaluate safety in that population.
This description comes directly from the study's public registry record.
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No contact information is available for this study as per the public registry record.
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Locations (35)
| UCSF Benioff Children's Hospital Oakland | Oakland, California, United States | — |
| University of Florida | Jacksonville, Florida, United States | — |
| Emory | Atlanta, Georgia, United States | — |
| Cincinnati Children's Hospital Medical Center | Cincinnati, Ohio, United States | — |
| UPMC Children's Hospital | Pittsburgh, Pennsylvania, United States | — |
| University Utah | Salt Lake City, Utah, United States | — |
| Lysosomal and Rare Disorders Research & Treatment Center, Inc. | Fairfax, Virginia, United States | — |
| Hospital de Alta Complejidad en Red "El Cruce" | Buenos Aires, Argentina | — |
| Hospital de Niños de la Santísima Trinidad | Córdoba, Argentina | — |
| Melbourne Children's Trials Centre Murdoch Children's Research Institute | Parkville, Victoria, Australia | — |
| Royal Melbourne Hospital | Parkville, Victoria, Australia | — |
| Metabolic Clinical Trials Unit | Adelaide, Australia | — |
+ 23 more locations — full list on the registry record.
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Source record: clinicaltrials.gov/study/NCT04860960