Disease · MONDO_0009757

Niemann-Pick disease, type C1

6 sourcesdiseases-2026-09-18-0.1.0

Cite this record

ConductScience Library. Niemann-Pick disease, type C1. Diseases, record MONDO_0009757. Release diseases-2026-09-18-0.1.0, 18 Sept 2026. https://conductscience.org/library/diseases/MONDO_0009757

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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.

Name: Niemann-Pick disease, type C1; Open Targets
Open Targets ID: MONDO_0009757; Open Targets
Other names: type C1 Niemann-Pick disease; Open Targets
Disease Ontology: DOID:0070113; Disease Ontology
NCI Thesaurus: C126864; NCI Thesaurus
Highest stage of its drugs: Phase 3, for 1 of its 4 drugs, from ClinicalTrials.gov; Open Targets

Drugs4

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
2-HYDROXYPROPYL-BETA-CYCLODEXTRIN	CHEMBL577294	Oligosaccharide	Phase 3	ClinicalTrials.gov	5	5	5	Drugs and targets
ADRABETADEX	CHEMBL4297678	Oligosaccharide	Phase 2/3	ClinicalTrials.gov	3	3	3	Drugs and targets
VORINOSTAT	CHEMBL98	Small molecule	Phase 1/2	ClinicalTrials.gov	1	1	1	Drugs and targets
LITHIUM CARBONATE	CHEMBL1200826	Small molecule	Early phase 1	ClinicalTrials.gov	1	1	1	Drugs and targets

Targets7

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
GSK3A	Glycogen synthase kinase-3	1	Glycogen synthase kinase-3 inhibitor	Early phase 1	https://conductscience.org/library/genes/2931	Drugs and targets
GSK3B	Glycogen synthase kinase-3	1	Glycogen synthase kinase-3 inhibitor	Early phase 1	https://conductscience.org/library/genes/2932	Drugs and targets
HDAC1	Histone deacetylase 1	1	Histone deacetylase 1 inhibitor	Phase 1/2	https://conductscience.org/library/genes/3065	Drugs and targets
HDAC2	Histone deacetylase 2	1	Histone deacetylase 2 inhibitor	Phase 1/2	https://conductscience.org/library/genes/3066	Drugs and targets
HDAC3	Histone deacetylase 3	1	Histone deacetylase 3 inhibitor	Phase 1/2	https://conductscience.org/library/genes/8841	Drugs and targets
HDAC6	Protein deacetylase HDAC6	1	Histone deacetylase 6 inhibitor	Phase 1/2	https://conductscience.org/library/genes/10013	Drugs and targets
IMPA1	Inositol monophosphatase 1	1	Inositol-1(or 4)-monophosphatase 1 inhibitor	Early phase 1	https://conductscience.org/library/genes/3612	Drugs and targets

Trials10

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.

Trial	Title	Phase	Participants	Results first posted	Conditions	Drugs	Linked by	Source
NCT04958642	Adrabetadex to Treat Niemann-Pick Type C1 (NPC1) Disease	Phase 2/3	66	2023-08-29	Niemann-Pick Disease, Type C	ADRABETADEX	Open Targets' reading of its record	ClinicalTrials.gov
NCT02534844	VTS-270 to Treat Niemann-Pick Type C1 (NPC1) Disease	Phase 2/3	56	2023-02-22	Niemann-Pick Disease, Type C	ADRABETADEX	Open Targets' reading of its record	ClinicalTrials.gov
NCT02124083	Phase 1/2 Study of Vorinostat Therapy in Niemann-Pick Disease, Type C1	Phase 1/2	12	2018-02-22	Neimann-Pick Disease	VORINOSTAT	Its record names the disease	ClinicalTrials.gov
NCT03879655	Open-label Study of VTS-270 in Participants With Neurologic Manifestations of Niemann-Pick Type C1	Phase 2/3	2	2023-12-26	Niemann-Pick Disease, Type C	2-HYDROXYPROPYL-BETA-CYCLODEXTRIN	Open Targets' reading of its record	ClinicalTrials.gov
NCT03887533	Combined Intrathecal and Intravenous VTS-270 Therapy for Liver and Neurological Disease Associated With Niemann-Pick Disease, Type C1	Phase 1/2	2	2022-08-30	Niemann-Pick Disease, Type C1	ADRABETADEX	Its record names the disease	ClinicalTrials.gov
NCT04860960	Phase 3 Study to Evaluate Intravenous Trappsol(R) Cyclo(TM) in Pediatric and Adult Patients With Niemann-Pick Disease Type C1	Phase 3	94		Niemann-Pick Disease, Type C1	2-HYDROXYPROPYL-BETA-CYCLODEXTRIN	Its record names the disease	ClinicalTrials.gov
NCT03201627	Study of Lithium Carbonate to Treat Niemann-Pick Type C1 Disease	Early phase 1	18		Niemann-Pick Disease, Type C1	LITHIUM CARBONATE	Its record names the disease	ClinicalTrials.gov
NCT01747135	Hydroxypropyl Beta Cyclodextrin for Niemann-Pick Type C1 Disease	Phase 1	14		Niemann-Pick Disease, Type C1	2-HYDROXYPROPYL-BETA-CYCLODEXTRIN	Its record names the disease	ClinicalTrials.gov
NCT02912793	Safety and Efficacy of Intravenous Trappsol Cyclo (HPBCD) in Niemann-Pick Type C Patients	Phase 1/2	12		Niemann-Pick Disease, Type C1	2-HYDROXYPROPYL-BETA-CYCLODEXTRIN	Its record names the disease	ClinicalTrials.gov
NCT03893071	Open-Label Study of Long-Term Safety and Efficacy of Intravenous Trappsol Cyclo (HPβCD) in Niemann-Pick Disease Type C	Phase 1	12		Niemann-Pick Disease, Type C1	2-HYDROXYPROPYL-BETA-CYCLODEXTRIN	Its record names the disease	ClinicalTrials.gov

Comparisons in its trials11

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
Adrabetadex vs Sham Control: Parts A/B: Number of Participants Treated for at Least 6 Months Who Qualified for the Rescue Option	2 of 38	2 of 18	56	NCT02534844	Trial comparisons
Adrabetadex vs Sham Control: Parts A/ B: Number of Participants Classified as CGIC Responders at Week 52	23 of 38	7 of 18	56	NCT02534844	Trial comparisons
Adrabetadex vs Sham Control: Parts A/B: Number of Participants Classified as Responders on NPC-SS Total Score (Excluding Hearing and ABR) at Week 52	22 of 38	13 of 18	56	NCT02534844	Trial comparisons
VTS-270 at 500 mg/kg vs VTS-270 at 1000 mg/kg: Participants With Reduction in Liver Stiffness (kPa)	0 of 1	0 of 1	2	NCT03887533	Trial comparisons
VTS-270 at 500 mg/kg vs VTS-270 at 1000 mg/kg: Participants With Reduction in Plasma Bile Acid B (5α)	1 of 1	0 of 1	2	NCT03887533	Trial comparisons
VTS-270 at 500 mg/kg vs VTS-270 at 1000 mg/kg: Participants With Reduction in Aspartate Aminotransferase	1 of 1	1 of 1	2	NCT03887533	Trial comparisons
VTS-270 at 500 mg/kg vs VTS-270 at 1000 mg/kg: Participants With Reduction in Plasma Cholestane-3β	1 of 1	0 of 1	2	NCT03887533	Trial comparisons
VTS-270 at 500 mg/kg vs VTS-270 at 1000 mg/kg: Participants With Reduction in C-Triol (6β-triol)	1 of 1	0 of 1	2	NCT03887533	Trial comparisons
VTS-270 at 500 mg/kg vs VTS-270 at 1000 mg/kg: Participants With Reduction in Alanine Aminotransferase Level	0 of 1	0 of 1	2	NCT03887533	Trial comparisons
VTS-270 at 500 mg/kg vs VTS-270 at 1000 mg/kg: deaths from any cause	0 of 1	0 of 1	2	NCT03887533	Trial comparisons

1 to 10 of 11

See all 11 comparisons in its trials

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.

PMID	Listed as	Trials listing it	Source
40525490	DERIVED	2	PubMed
37670901	DERIVED	1	PubMed
36279795	DERIVED	2	PubMed
34810067	DERIVED	1	PubMed
34389161	DERIVED	1	PubMed
34177581	DERIVED	1	PubMed
31187454	DERIVED	1	PubMed

Data dates

The date of each source this release read.

Open Targets: 2026-09-18; Open Targets
Drug links and their checks, from Drugs and targets: 2026-09-18; Drugs and targets
ClinicalTrials.gov, from Trials: 2026-11-12; Trials
Comparisons, from Trial comparisons: 2026-03-23; Trial comparisons
PubMed, for the checks: 2026-11-06; PubMed
MeSH, for the checks: 2026-11-10; MeSH

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_0009757

GET https://conductscience.org/api/diseases/v1/MONDO_0009757/drugs

API reference For AI assistants