Disease · MONDO_0007739

Huntington disease

6 sourcesdiseases-2026-09-18-0.1.0

Cite this record

ConductScience Library. Huntington disease. Diseases, record MONDO_0007739. Release diseases-2026-09-18-0.1.0, 18 Sept 2026. https://conductscience.org/library/diseases/MONDO_0007739

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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: Huntington disease; Open Targets
Open Targets ID: MONDO_0007739; Open Targets
Other names: AKINETIC RIGID VARIANT HUNTINGTON DIS, Akinetic Rigid Variant of Huntington Disease, Chorea, Chronic Progressive Hereditary (Huntington), Chorea, Huntington, Chorea, Huntington's, Chronic Progressive Hereditary Chorea (Huntington), Chronic progressive chorea, Chronic progressive hereditary chorea, HC - Huntington chorea, HD, HD - Huntington chorea, HUNTINGTON DIS, HUNTINGTON DIS AKINETIC RIGID VARIANT, HUNTINGTON DIS JUVENILE, HUNTINGTON DIS JUVENILE ONSET, HUNTINGTON DIS LATE ONSET, HUNTINGTONS DIS, Huntington Chronic Progressive Hereditary Chorea, Huntington Disease, Akinetic Rigid Variant, Huntington Disease, Juvenile, Huntington Disease, Juvenile Onset, Huntington Disease, Late Onset, Huntington chorea, Huntington's, Huntington's Disease, Huntington's chorea, Huntington's chorea (disorder), Huntington's disease pathway, Huntingtons disease, JUVENILE HUNTINGTON DIS, JUVENILE ONSET HUNTINGTON DIS, Juvenile Huntington Disease, Juvenile Onset Huntington Disease, LATE ONSET HUNTINGTON DIS, Late Onset Huntington Disease, Progressive Chorea, Chronic Hereditary (Huntington), Progressive Chorea, Hereditary, Chronic (Huntington); Open Targets
Disease Ontology: DOID:12858; Disease Ontology
MeSH: D006816; MeSH
NCI Thesaurus: C82342; NCI Thesaurus
Orphanet: ORPHA:399; Orphanet
Highest stage of its drugs: Approval, for 6 of its 62 drugs, from DailyMed, PMDA and TTD; Open Targets

Drugs62

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
TETRABENAZINE	CHEMBL117785	Small molecule	Approval	DailyMed, PMDA and TTD	8	4	4	Drugs and targets
DEUTETRABENAZINE	CHEMBL3137326	Small molecule	Approval	DailyMed and TTD	6	4	4	Drugs and targets
ATOMOXETINE HYDROCHLORIDE	CHEMBL1702	Small molecule	Approval	PMDA	1	0	0	Drugs and targets
GUANFACINE HYDROCHLORIDE	CHEMBL1200494	Small molecule	Approval	PMDA	1	0	0	Drugs and targets
LISDEXAMFETAMINE DIMESYLATE	CHEMBL1201178	Small molecule	Approval	PMDA	1	0	0	Drugs and targets
METHYLPHENIDATE HYDROCHLORIDE	CHEMBL1722	Small molecule	Approval	PMDA	1	0	0	Drugs and targets
TOMINERSEN	CHEMBL4297581	Oligonucleotide	Phase 3	ClinicalTrials.gov and TTD	9	6	6	Drugs and targets
PRIDOPIDINE	CHEMBL596802	Small molecule	Phase 3	ClinicalTrials.gov and TTD	8	6	6	Drugs and targets
CREATINE	CHEMBL283800	Small molecule	Phase 3	ClinicalTrials.gov	5	5	4	Drugs and targets
DALZANEMDOR	CHEMBL5095095	Small molecule	Phase 3	ClinicalTrials.gov	4	4	4	Drugs and targets

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Targets99

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
DRD2	D(2) dopamine receptor	5	D2-like dopamine receptor antagonist; D2-like dopamine receptor inverse agonist; Dopamine D2 receptor antagonist; Dopamine D2 receptor modulator	Phase 3	https://conductscience.org/library/genes/1813	Drugs and targets
SLC6A2	Sodium-dependent noradrenaline transporter	4	Norepinephrine transporter inhibitor; Norepinephrine transporter releasing agent	Approval	https://conductscience.org/library/genes/6530	Drugs and targets
DRD3	D(3) dopamine receptor	3	D2-like dopamine receptor antagonist; D2-like dopamine receptor inverse agonist; Dopamine D3 receptor antagonist	Phase 3	https://conductscience.org/library/genes/1814	Drugs and targets
HTR2A	5-hydroxytryptamine receptor 2A	3	Serotonin 2a (5-HT2a) receptor antagonist	Phase 3	https://conductscience.org/library/genes/3356	Drugs and targets
SLC18A2	Synaptic vesicular amine transporter	3	Synaptic vesicular amine transporter inhibitor	Approval	https://conductscience.org/library/genes/6571	Drugs and targets
CNR1	Cannabinoid receptor 1	2	Cannabinoid CB1 receptor agonist; Cannabinoid CB1 receptor negative allosteric modulator	Phase 2	https://conductscience.org/library/genes/1268	Drugs and targets
DRD4	D2-like dopamine receptor	2	D2-like dopamine receptor antagonist; D2-like dopamine receptor inverse agonist	Phase 3	https://conductscience.org/library/genes/1815	Drugs and targets
FDPS	Farnesyl pyrophosphate synthase	2	Farnesyl diphosphate synthase inhibitor	Phase 1	https://conductscience.org/library/genes/2224	Drugs and targets
GRIN1	Glutamate [NMDA] receptor	2	Glutamate [NMDA] receptor antagonist; Glutamate [NMDA] receptor negative allosteric modulator	Phase 2	https://conductscience.org/library/genes/2902	Drugs and targets
GRIN2A	Glutamate [NMDA] receptor	2	Glutamate [NMDA] receptor antagonist; Glutamate [NMDA] receptor negative allosteric modulator	Phase 2	https://conductscience.org/library/genes/2903	Drugs and targets

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Trials72

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. 4 more are not shown, since the checks do not confirm them.

Trial	Title	Phase	Participants	Results first posted	Conditions	Drugs	Linked by	Source
NCT03761849	A Study to Evaluate the Efficacy and Safety of Intrathecally Administered RO7234292 (RG6042) in Participants With Manifest Huntington's Disease	Phase 3	899	2023-11-07	Huntingtons Disease	TOMINERSEN	Its record names the disease	ClinicalTrials.gov
NCT00608881	Coenzyme Q10 in Huntington's Disease (HD)	Phase 3	609	2016-03-30	Huntington's Disease	UBIDECARENONE	Its record names the disease	ClinicalTrials.gov
NCT04556656	PRidopidine's Outcome On Function in Huntington Disease, PROOF- HD	Phase 3	499	2025-03-12	Huntington Disease	PRIDOPIDINE	Its record names the disease	ClinicalTrials.gov
NCT00665223	A Study of Treatment With Pridopidine (ACR16) in Participants With Huntington's Disease	Phase 3	437	2023-08-29	Huntington's Disease	PRIDOPIDINE	Its record names the disease	ClinicalTrials.gov
NCT02006472	A Phase 2, to Evaluating the Safety and Efficacy of Pridopidine Vs Placebo for Symptomatic Treatment in Patients With Huntington's Disease	Phase 2	408	2021-07-19	Huntington's Disease	PRIDOPIDINE	Its record names the disease	ClinicalTrials.gov
NCT02215616	A Clinical Study in Participants With Huntington's Disease (HD) to Assess Efficacy and Safety of Three Oral Doses of Laquinimod	Phase 2	352	2019-06-19	Huntington's Disease	LAQUINIMOD	Its record names the disease	ClinicalTrials.gov
NCT02197130	Randomized, Placebo Controlled Study Of The Efficacy And Safety Of PF-02545920 In Subjects With Huntington's Disease	Phase 2	272	2017-11-17	Huntington's Disease	MARDEPODECT	Its record names the disease	ClinicalTrials.gov
NCT02494778	A Study Evaluating if Pridopidine is Safe, Efficacious, and Tolerable in Patients With Huntington's Disease	Phase 2	248	2021-09-17	Huntington's Disease	PRIDOPIDINE	Its record names the disease	ClinicalTrials.gov
NCT03842969	An Open-Label Extension Study to Evaluate Long-Term Safety and Tolerability of RO7234292 (RG6042) in Huntington's Disease Participants Who Participated in Prior Roche and Genentech Sponsored Studies	Phase 3	236	2023-08-31	Huntington Disease	TOMINERSEN	Its record names the disease	ClinicalTrials.gov
NCT00724048	A Study of Pridopidine (ACR16) for the Treatment of Participants With Huntington's Disease	Phase 2	227	2023-08-31	Huntington Disease	PRIDOPIDINE	Its record names the disease	ClinicalTrials.gov

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Comparisons in its trials28

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
A - Coenzyme Q10 2400 mg/Day vs B - Placebo: serious adverse events	76 of 303	83 of 306	609	NCT00608881	Trial comparisons
Pridopidine vs Placebo: deaths from any cause	4 of 250	1 of 249	499	NCT04556656	Trial comparisons
Pridopidine vs Placebo: serious adverse events	34 of 250	21 of 249	499	NCT04556656	Trial comparisons
SAGE-718 vs Placebo: deaths from any cause	0 of 95	0 of 94	189	NCT05107128	Trial comparisons
SAGE-718 vs Placebo: serious adverse events	3 of 95	6 of 94	189	NCT05107128	Trial comparisons
Cohort 1 (Direct Rollover) vs Cohort 2 (Gap Rollover): Number of Participants Who Withdrew From Study Due to TEAEs	2 of 72	3 of 81	153	NCT05655520	Trial comparisons
Cohort 1 (Direct Rollover) vs Cohort 2 (Gap Rollover): deaths from any cause	0 of 72	0 of 81	153	NCT05655520	Trial comparisons
Cohort 1 (Direct Rollover) vs Cohort 2 (Gap Rollover): Number of Participants With at Least One Treatment-Emergent Adverse Events (TEAEs)	46 of 72	46 of 81	153	NCT05655520	Trial comparisons
Cohort 1 (Direct Rollover) vs Cohort 2 (Gap Rollover): serious adverse events	3 of 72	6 of 81	153	NCT05655520	Trial comparisons
Rollover Cohort: SD-809 ER vs Switch Cohort: SD-809 ER: deaths from any cause	1 of 82	0 of 37	119	NCT01897896	Trial comparisons

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Papers from its trials47

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
39804569	RESULT	1	PubMed
27434297	RESULT	1	PubMed
25568297	RESULT	1	PubMed
42912861	DERIVED	1	PubMed
42857615	DERIVED	1	PubMed
42032152	DERIVED	1	PubMed
41491108	DERIVED	1	PubMed
40913168	DERIVED	1	PubMed
39825184	DERIVED	2	PubMed
38988035	DERIVED	1	PubMed

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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_0007739

GET https://conductscience.org/api/diseases/v1/MONDO_0007739/drugs?page=2

API reference For AI assistants