Study of Lithium Carbonate to Treat Niemann-Pick Type C1 Disease

Phase 1: Researchers test a new drug or treatment in a small group of people for the first time to evaluate its safety, determine a safe dosage range, and identify side effects.
Phase 2: The drug or treatment is given to a larger group of people to see if it is effective and to further evaluate its safety.
Phase 3: The drug or treatment is given to large groups of people to confirm its effectiveness, monitor side effects, compare it to commonly used treatments, and collect information that will allow the drug or treatment to be used safely.
Phase 4: Studies are done after the drug or treatment has been marketed to gather information on the drug's effect in various populations and any side effects associated with long-term use.

Niemann-Pick Disease, Type C1 Clinical Trial

Official title:
a Single-center, Prospective, Open, and Non-randomized Case-control Study of Lithium Carbonate Effect on Niemann Disease C1 Type

Status Active, not recruiting

Clinical Trial Summary

This study is planned to study whether lithium carbonate has protective effect on the brain of Niemann-Pick disease type C1.

n/a

NCT number	NCT03201627
Study type	Interventional
Source	Xinhua Hospital, Shanghai Jiao Tong University School of Medicine
Contact
Status	Active, not recruiting
Phase	Early Phase 1
Start date	July 5, 2017
Completion date	June 30, 2021

See also
Status	Clinical Trial	Phase
Enrolling by invitation	`NCT03655223` - Early Check: Expanded Screening in Newborns
Completed	`NCT02939547` - Study of the Pharmacokinetics of Trappsol and Effects on Potential Biomarkers of Niemann-Pick C1 (NPC1)	Phase 1
Active, not recruiting	`NCT03893071` - Open-Label Study of Long-Term Safety and Efficacy of Intravenous Trappsol Cyclo (HPβCD) in Niemann-Pick Disease Type C	Phase 1
Completed	`NCT02912793` - Safety and Efficacy of Intravenous Trappsol Cyclo (HPBCD) in Niemann-Pick Type C Patients	Phase 1/Phase 2
Active, not recruiting	`NCT04860960` - Phase 3 Study to Evaluate Intravenous Trappsol(R) Cyclo(TM) in Pediatric and Adult Patients With Niemann-Pick Disease Type C1	Phase 3
Terminated	`NCT03887533` - Combined Intrathecal and Intravenous VTS-270 Therapy for Liver and Neurological Disease Associated With Niemann-Pick Disease, Type C1	Phase 1/Phase 2
Completed	`NCT01747135` - Hydroxypropyl Beta Cyclodextrin for Niemann-Pick Type C1 Disease	Phase 1