Niemann-Pick disease, type C1
Information
- Disease name
- Niemann-Pick disease, type C1
- Disease ID
- Description
Disease area statistics
[No Data.]
Chromosome band
[No Data.]
Annotation
| Genes | Mutation | Description | Source | Links |
|---|
| NCT ID | Status | Phase | Summary | Start date | Completion date |
|---|---|---|---|---|---|
| NCT04860960 | Active, not recruiting | Phase 3 | Phase 3 Study to Evaluate Intravenous Trappsol(R) Cyclo(TM) in Pediatric and Adult Patients With Niemann-Pick Disease Type C1 | July 20, 2021 | June 2026 |
| NCT02912793 | Completed | Phase 1/Phase 2 | Safety and Efficacy of Intravenous Trappsol Cyclo (HPBCD) in Niemann-Pick Type C Patients | March 20, 2017 | March 3, 2021 |
| NCT02939547 | Completed | Phase 1 | Study of the Pharmacokinetics of Trappsol and Effects on Potential Biomarkers of Niemann-Pick C1 (NPC1) | October 11, 2017 | February 10, 2020 |
| NCT01747135 | Completed | Phase 1 | Hydroxypropyl Beta Cyclodextrin for Niemann-Pick Type C1 Disease | January 2013 | March 2017 |
| NCT03655223 | Enrolling by invitation | Early Check: Expanded Screening in Newborns | October 15, 2018 | December 31, 2025 | |
| NCT03887533 | Terminated | Phase 1/Phase 2 | Combined Intrathecal and Intravenous VTS-270 Therapy for Liver and Neurological Disease Associated With Niemann-Pick Disease, Type C1 | January 6, 2020 | October 25, 2021 |
| NCT03893071 | Unknown status | Phase 1 | Open-Label Study of Long-Term Safety and Efficacy of Intravenous Trappsol Cyclo (HPβCD) in Niemann-Pick Disease Type C | May 23, 2019 | March 2022 |
| NCT03201627 | Unknown status | Early Phase 1 | Study of Lithium Carbonate to Treat Niemann-Pick Type C1 Disease | July 5, 2017 | June 30, 2021 |