Niemann-Pick disease, type C
Information
- Disease name
- Niemann-Pick disease, type C
- 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 |
|---|---|---|---|---|---|
| NCT05163288 | Active, not recruiting | Phase 3 | A Pivotal Study of N-Acetyl-L-Leucine on Niemann-Pick Disease Type C | June 30, 2022 | November 2024 |
| NCT05758922 | Active, not recruiting | Phase 2 | Phase 2 Study Evaluating the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Oral AZ-3102 in Patients With GM2 Gangliosidosis or Niemann-Pick Type C Disease | April 24, 2023 | December 2024 |
| NCT02612129 | Active, not recruiting | Phase 2/Phase 3 | Arimoclomol Prospective Study in Participants Diagnosed With Niemann-Pick Disease Type C | June 14, 2016 | January 31, 2025 |
| NCT04316637 | Available | Early Access Program With Arimoclomol in US Patients With NPC | |||
| NCT02534844 | Completed | Phase 2/Phase 3 | VTS-270 to Treat Niemann-Pick Type C1 (NPC1) Disease | October 2015 | March 28, 2018 |
| NCT03759639 | Completed | Phase 2 | N-Acetyl-L-Leucine for Niemann-Pick Disease, Type C (NPC) | September 4, 2019 | November 7, 2022 |
| NCT00975689 | Completed | Phase 1/Phase 2 | Biomarker Validation for Niemann-Pick Disease, Type C: Safety and Efficacy of N-Acetyl Cysteine | August 2009 | November 2010 |
| NCT01899950 | Completed | Longitudinal Study of Cognition With Niemann-Pick Disease, Type C | March 2011 | September 2014 | |
| NCT02435030 | Completed | A Prospective Non-therapeutic Study in Patients Diagnosed With Niemann-Pick Disease Type C | September 2015 | May 2017 | |
| NCT03910621 | Completed | Phase 4 | Safety and Efficacy of Miglustat in Chinese NPC Patients | April 2, 2020 | March 25, 2022 |
| NCT05368038 | Enrolling by invitation | ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program | May 10, 2021 | July 31, 2026 | |
| NCT03471143 | Recruiting | Phase 1/Phase 2 | Study of IV VTS-270 for Infantile Liver Disease Associated With Niemann-Pick Disease, Type C | February 22, 2019 | June 2024 |
| NCT00344331 | Recruiting | Evaluation of Biochemical Markers and Clinical Investigation of Niemann-Pick Disease, Type C | August 14, 2006 | ||
| NCT05588167 | Recruiting | Establishment of Genomic and Phenotypic Database for Niemann-Pick Disease, Type C | November 28, 2022 | September 30, 2024 | |
| NCT04958642 | Terminated | Phase 2/Phase 3 | Adrabetadex to Treat Niemann-Pick Type C1 (NPC1) Disease | December 23, 2015 | April 11, 2022 |
| NCT03879655 | Terminated | Phase 2/Phase 3 | Open-label Study of VTS-270 in Participants With Neurologic Manifestations of Niemann-Pick Type C1 | December 2, 2019 | October 18, 2021 |
| NCT00668564 | Terminated | Phase 2 | Hematopoietic Stem Cell Transplantation (HCT) for Inborn Errors of Metabolism | March 2008 | February 2010 |
| NCT01306604 | Withdrawn | Biomarker for Niemann Pick Type C Disease (BioNPC) | August 20, 2018 | February 28, 2021 | |
| NCT04189601 | Withdrawn | Complement Activation in the Lysosomal Storage Disorders | September 30, 2020 | April 30, 2021 | |
| NCT03687476 | Withdrawn | Phase 2 | Safety and Tolerability Study of VTS-270 in Pediatric Participants With Niemann-Pick Type C (NPC) Disease | May 2020 | October 30, 2022 |
- MeSH unique ID (MeSH (Medical Subject Headings))
- D052556