Disease · MONDO_0018982
Niemann-Pick disease type C
6 sourcesdiseases-2026-09-18-0.1.0
Cite this record
ConductScience Library. Niemann-Pick disease type C. Diseases, record MONDO_0018982. Release diseases-2026-09-18-0.1.0, 18 Sept 2026. https://conductscience.org/library/diseases/MONDO_0018982
On this page
Disease
Names and IDs as Open Targets gives them. Its IDs in MeSH, Orphanet, the Disease Ontology and the NCI Thesaurus are those Open Targets states for it.
Drugs9
The drugs Open Targets' clinical reports study for the disease, the highest stage first, then the most trial reports. The stage is Open Targets', the highest any of its sources reports for the pair, and each row names the sources that report it.
| Drug | ChEMBL ID | Type | Open Targets' stage | Stage reported by | Reports | Of them trials | Trials shown here | Source |
|---|---|---|---|---|---|---|---|---|
| MIGLUSTAT | CHEMBL1029 | Small molecule | Approval | PMDA | 5 | 4 | 4 | Drugs and targets |
| LEVACETYLLEUCINE | CHEMBL56021 | Small molecule | Approval | FDA | 4 | 2 | 1 | Drugs and targets |
| ARIMOCLOMOL CITRATE | CHEMBL4298149 | Small molecule | Approval | FDA | 2 | 0 | 0 | Drugs and targets |
| NIZUBAGLUSTAT | CHEMBL3354637 | Small molecule | Phase 3 | ClinicalTrials.gov | 4 | 4 | 3 | Drugs and targets |
| ADRABETADEX | CHEMBL4297678 | Oligosaccharide | Phase 3 | ClinicalTrials.gov | 1 | 1 | 1 | Drugs and targets |
| ARIMOCLOMOL | CHEMBL2107726 | Small molecule | Phase 2/3 | ClinicalTrials.gov | 4 | 2 | 2 | Drugs and targets |
| 2-HYDROXYPROPYL-BETA-CYCLODEXTRIN | CHEMBL577294 | Oligosaccharide | Phase 2 | ClinicalTrials.gov | 3 | 3 | 3 | Drugs and targets |
| TRENONACOG ALFA | CHEMBL3039538 | Phase 2 | ClinicalTrials.gov | 1 | 1 | 1 | Drugs and targets | |
| ACETYLCYSTEINE | CHEMBL600 | Small molecule | Phase 1/2 | ClinicalTrials.gov | 1 | 1 | 0 | Drugs and targets |
Targets2
The targets ChEMBL's mechanism records give for the disease's drugs, through Open Targets, the most drugs first. A mechanism record says what a drug acts on, not that the target causes the disease.
| Target | Protein | Its drugs that act on it | Mechanisms | Highest stage of those drugs | Gene in Genes | Source |
|---|---|---|---|---|---|---|
| F9 | Coagulation factor IX | 1 | Coagulation factor IX exogenous protein | Phase 2 | https://conductscience.org/library/genes/2158 | Drugs and targets |
| UGCG | Ceramide glucosyltransferase | 1 | Ceramide glucosyltransferase inhibitor | Approval | https://conductscience.org/library/genes/7357 | Drugs and targets |
Trials13
Trials an Open Targets clinical report links to the disease, posted results first, then the largest. A trial is shown when the release's checks confirm its link to the disease and Drugs and targets shows its link to one of the disease's drugs. 3 more are not shown, since the checks do not confirm them.
| Trial | Title | Phase | Participants | Results first posted | Conditions | Drugs | Linked by | Source |
|---|---|---|---|---|---|---|---|---|
| NCT02612129 | Arimoclomol Prospective Study in Participants Diagnosed With Niemann-Pick Disease Type C | Phase 2/3 | 50 | 2023-06-09 | Niemann-Pick Disease, Type C | ARIMOCLOMOL | Its record names the disease | ClinicalTrials.gov |
| NCT03759639 | N-Acetyl-L-Leucine for Niemann-Pick Disease, Type C (NPC) | Phase 2 | 33 | 2023-11-28 | Niemann-Pick Disease, Type C | LEVACETYLLEUCINE, TRENONACOG ALFA | Its record names the disease | ClinicalTrials.gov |
| NCT03643562 | Niemann-Pick Type C Treatment With Adrabetadex for Symptoms of Brain and Nervous System | Phase 3 | 27 | 2024-01-17 | Niemann-Pick Type C Disease | ADRABETADEX | Open Targets' reading of its record | ClinicalTrials.gov |
| NCT03471143 | Study of IV VTS-270 for Infantile Liver Disease Associated With Niemann-Pick Disease, Type C | Phase 1/2 | 4 | 2025-12-12 | Niemann-Pick Disease, Type C | 2-HYDROXYPROPYL-BETA-CYCLODEXTRIN | Its record names the disease | ClinicalTrials.gov |
| NCT07054515 | A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of Niemann-Pick Type C Disease, GM1 Gangliosidosis or GM2 Gangliosidosis | Phase 3 | 147 | Niemann-Pick Type C Disease, GM1 Gangliosidosis, GM2 Gangliosidosis | NIZUBAGLUSTAT | Open Targets' reading of its record | ClinicalTrials.gov | |
| NCT07082725 | A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of Niemann-Pick Type C Disease (NPC) | Phase 3 | 72 | Niemann-Pick Type C Disease | NIZUBAGLUSTAT | Open Targets' reading of its record | ClinicalTrials.gov | |
| NCT00517153 | Miglustat in Niemann-Pick Type C Disease | Phase 2 | 29 | Niemann-Pick Type C Disease | MIGLUSTAT | Open Targets' reading of its record | ClinicalTrials.gov | |
| NCT07399704 | A Study to Evaluate the Safety and Efficacy of Nizubaglustat (AZ-3102) in Patients With GM2 Gangliosidosis or Niemann-Pick Type C Disease | Phase 2 | 21 | GM2 Gangliosidosis, Niemann-Pick Type C Disease | MIGLUSTAT, NIZUBAGLUSTAT | Open Targets' reading of its record | ClinicalTrials.gov | |
| NCT03910621 | Safety and Efficacy of Miglustat in Chinese NPC Patients | Phase 4 | 17 | Niemann-Pick Disease, Type C | MIGLUSTAT | Its record names the disease | ClinicalTrials.gov | |
| NCT02939547 | Study of the Pharmacokinetics of Trappsol and Effects on Potential Biomarkers of Niemann-Pick C1 (NPC1) | Phase 1 | 13 | Niemann-Pick Disease, Type C1 | 2-HYDROXYPROPYL-BETA-CYCLODEXTRIN | Its record names the disease | ClinicalTrials.gov |
1 to 10 of 13
See all 13 trialsComparisons in its trials4
Comparisons Trial comparisons calculated in the trials shown here, most participants first. A comparison in one of these trials does not always compare a treatment for the disease, so read the two groups.
| Comparison | Group 1, with the outcome | Group 2, with the outcome | Participants | Trial | Source |
|---|---|---|---|---|---|
| Treatment With IB1001 vs Post-Treatment Washout: deaths from any cause | 0 of 33 | 0 of 31 | 64 | NCT03759639 | Trial comparisons |
| Treatment With IB1001 vs Post-Treatment Washout: serious adverse events | 4 of 33 | 0 of 31 | 64 | NCT03759639 | Trial comparisons |
| Treatment Naive vs Previously Treated: deaths from any cause | 0 of 9 | 0 of 18 | 27 | NCT03643562 | Trial comparisons |
| Treatment Naive vs Previously Treated: serious adverse events | 5 of 9 | 9 of 18 | 27 | NCT03643562 | Trial comparisons |
Papers from its trials7
The papers the trials shown here list on ClinicalTrials.gov as their results (RESULT) or as drawn from them (DERIVED), results first, newest first.
Data dates
The date of each source this release read.
Sources 6
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ChEMBL, through Open TargetsCC BY-SA 3.0
- ClinicalTrials.govNo license stated
- NLM's MeSHNLM's terms
- Open TargetsCC0 1.0
- PubMedNLM's terms
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ChEMBL, through Open TargetsCC BY-SA 3.0, for the parts from ChEMBL. Read on 8 Nov 2026 at https://platform-docs.opentargets.org/licence
- ClinicalTrials.govnone stated
- NLM's MeSHNLM's
- Open TargetsCC0 1.0. Open Targets asks that users cite its latest paper: Buniello et al. 2025, Open Targets Platform: facilitating therapeutic hypotheses building in drug discovery, Nucleic Acids Research 53(D1):D1467, PMID 39657122. Read on 8 Nov 2026 at https://platform-docs.opentargets.org/licence
- PubMedNLM's terms
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this disease from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/diseases/v1/MONDO_0018982
GET https://conductscience.org/api/diseases/v1/MONDO_0018982/drugs