Huntington's Disease Pipeline 2026: Drugs in Clinical Trials

Remission & chronicity

Spontaneous remission

Inevitable progressive neurodegeneration in all carriers of HTT CAG expansion >36 repeats; no spontaneous remission; motor, cognitive, psychiatric decline universally progressive

Best-intervention remission

Tetrabenazine/deutetrabenazine (VMAT2 inhibitors): 25-30% reduction in chorea score (FIRST-HD); no disease-modifying treatment proven; genetic CSF biomarker (mutant huntingtin, mHTT) reduction achieved with tominersen ASO — but Phase 3 halted 2021 (higher dose harmful); lower-dose tominersen now in Phase 2/3 after exploratory biomarker signal

Gap size

Very large — no approved disease-modifying treatment; symptomatic treatment only

Primary barrier

biological

HD is unique in having a perfectly predictive genetic test allowing pre-symptomatic intervention — the challenge is that trials of mHTT-lowering agents (tominersen, WVE-003) have faced setbacks; the therapeutic window (pre-manifest vs manifest disease) and appropriate dose remain unclear; genetic counselling for presymptomatic testing is complex

Biomarker Targets & Therapeutic Agents

Gene targets queried against DGIdb, Open Targets, ChEMBL, PubMed, and Europe PMC. Agents ranked: Clinical > Mechanistic > Correlative.

DRD2 372 FAN1 0 GRIN2B 236 HDAC4 184 HTT 234 MSH3 3 PDE10A 201 PPARGC1A 0 SEMA4D 1 SLC18A2 123

DRD2

UniProt Ensembl DGIdb Open Targets

Therapeutic Agent	Effect Direction	Evidence Tier	Potency	Source
LOXAPINE HYDROCHLORIDE	↓ inhibits	Mechanistic		DGIdb
MINAPRINE	↑ activates	Mechanistic		DGIdb
SULPIRIDE	↓ inhibits	Mechanistic		DGIdb
HALOPERIDOL DECANOATE	↓ inhibits	Mechanistic		DGIdb
ZIPRASIDONE MESYLATE	↓ inhibits	Mechanistic		DGIdb
METHYLDOPA ANHYDROUS	? unclear	Mechanistic		DGIdb
BENPERIDOL	↓ inhibits	Mechanistic		DGIdb
FLUSPIRILENE	↓ inhibits	Mechanistic		DGIdb
VERALIPRIDE	↓ inhibits	Mechanistic		DGIdb
CAFFEINE	? unclear	Mechanistic		DGIdb
SUMANIROLE	↑ activates	Mechanistic	9.0 nM (Ki)	DGIdb
ZIPRASIDONE	↓ inhibits	Mechanistic		DGIdb
METOCLOPRAMIDE HYDROCHLORIDE	? unclear	Mechanistic		DGIdb
LUMATEPERONE	↓ inhibits	Mechanistic		DGIdb
FLUPENTHIXOL	↓ inhibits	Mechanistic		DGIdb
PROCHLORPERAZINE	↓ inhibits	Mechanistic		DGIdb
PERAZINE	? unclear	Mechanistic		DGIdb
FLUPHENAZINE HYDROCHLORIDE	↓ inhibits	Mechanistic		DGIdb
ROPINIROLE	↑ activates	Mechanistic		DGIdb
RACLOPRIDE	↓ inhibits	Mechanistic		DGIdb
PROMAZINE	↓ inhibits	Mechanistic		DGIdb
[3H]PD128907	↑ activates	Mechanistic		DGIdb
MORPHINE SULFATE	? unclear	Mechanistic		DGIdb
ALIZAPRIDE	↓ inhibits	Mechanistic		DGIdb
PHENYLTOLOXAMINE	? unclear	Mechanistic		DGIdb
LOXAPINE	↓ inhibits	Mechanistic		DGIdb
CABERGOLINE	↑ activates	Mechanistic		DGIdb
BREXPIPRAZOLE	↑ activates	Mechanistic		DGIdb
PALIPERIDONE	↓ inhibits	Mechanistic		DGIdb
APLINDORE FUMARATE	↑ activates	Mechanistic		DGIdb
APOMORPHINE	↑ activates	Mechanistic		DGIdb
CARIPRAZINE	↑ activates	Mechanistic		DGIdb
MOLINDONE	? unclear	Mechanistic		DGIdb
TIAPRIDE	↓ inhibits	Mechanistic		DGIdb
ZOTEPINE	↓ inhibits	Mechanistic		DGIdb
DOPAMINE	↑ activates	Mechanistic		DGIdb
XP21279	? unclear	Mechanistic		DGIdb
PROMAZINE HYDROCHLORIDE	↓ inhibits	Mechanistic		DGIdb
VILAZODONE HYDROCHLORIDE	↑ activates	Mechanistic		DGIdb
TRIFLUPROMAZINE	↓ inhibits	Mechanistic		DGIdb

Showing top 40 of 372 agents ranked by evidence tier.

FAN1

UniProt Ensembl DGIdb Open Targets

Pipeline results not yet available for this target. Run the pipeline to populate.

GRIN2B

UniProt Ensembl DGIdb Open Targets

Therapeutic Agent	Effect Direction	Evidence Tier	Potency	Source
APIMOSTINEL	↑ activates	Mechanistic		DGIdb
L701324	↓ inhibits	Mechanistic		DGIdb
FELBAMATE	↓ inhibits	Mechanistic		DGIdb
METHYLPHENIDATE	? unclear	Mechanistic		DGIdb
CLOZAPINE	? unclear	Mechanistic		DGIdb
NVP-AAM077	↓ inhibits	Mechanistic		DGIdb
GLYCINE	↑ activates	Mechanistic		DGIdb
SELFOTEL	↓ inhibits	Mechanistic		DGIdb
AMANTADINE HYDROCHLORIDE	↓ inhibits	Mechanistic		DGIdb
QUETIAPINE FUMARATE	? unclear	Mechanistic		DGIdb
L689560	↓ inhibits	Mechanistic		DGIdb
LATREPIRDINE	? unclear	Mechanistic		DGIdb
MG2+	↓ inhibits	Mechanistic		DGIdb
TRAXOPRODIL	↓ inhibits	Mechanistic	74.0 nM (IC50)	DGIdb
VALPROIC ACID	? unclear	Mechanistic		DGIdb
[3H]DIZOCILPINE	↓ inhibits	Mechanistic		DGIdb
CNS-5161	? unclear	Mechanistic		DGIdb
NEBOGLAMINE	~ modulates	Mechanistic		DGIdb
NP-10679	? unclear	Mechanistic		DGIdb
5,7-DICHLOROKYNURENIC ACID	↓ inhibits	Mechanistic		DGIdb
GV196771A	↓ inhibits	Mechanistic		DGIdb
DIZOCILPINE	↓ inhibits	Mechanistic		DGIdb
INDANTADOL	↓ inhibits	Mechanistic		DGIdb
ESKETAMINE HYDROCHLORIDE	~ modulates	Mechanistic		DGIdb
SEVOFLURANE	? unclear	Mechanistic		DGIdb
LANICEMINE	↓ inhibits	Mechanistic		DGIdb
ASPARTIC ACID	↑ activates	Mechanistic		DGIdb
UBP141	↓ inhibits	Mechanistic		DGIdb
DELUCEMINE	↓ inhibits	Mechanistic		DGIdb
S-DESCHLOROKETAMINE	↓ inhibits	Mechanistic		DGIdb
RAPASTINEL	↑ activates	Mechanistic		DGIdb
[3H]L689560	↓ inhibits	Mechanistic		DGIdb
EVT-101	? unclear	Mechanistic		DGIdb
[3H]D-ASPARTIC ACID	↑ activates	Mechanistic		DGIdb
[3H]MDL105519	↓ inhibits	Mechanistic		DGIdb
ELIPRODIL	? unclear	Mechanistic	13.0 nM (Ki)	DGIdb
D-CCPENE	↓ inhibits	Mechanistic		DGIdb
N1-DANSYL-SPERMINE	↓ inhibits	Mechanistic		DGIdb
ESKETAMINE	~ modulates	Mechanistic		DGIdb
KETAMINE HYDROCHLORIDE	↓ inhibits	Mechanistic		DGIdb

Showing top 40 of 236 agents ranked by evidence tier.

HDAC4

UniProt Ensembl DGIdb Open Targets

Therapeutic Agent	Effect Direction	Evidence Tier	Potency	Source
KA1010	↓ inhibits	Mechanistic		DGIdb
MOCETINOSTAT	? unclear	Mechanistic		DGIdb
TRICHOSTATIN A	↓ inhibits	Mechanistic		DGIdb
TACEDINALINE	↓ inhibits	Mechanistic		DGIdb
PANOBINOSTAT LACTATE	↓ inhibits	Mechanistic		DGIdb
ENTINOSTAT	↓ inhibits	Mechanistic	510.0 nM (IC50)	DGIdb
BELINOSTAT	↓ inhibits	Mechanistic	24.0 nM (IC50)	DGIdb
LARGAZOLE	? unclear	Mechanistic		DGIdb
GIVINOSTAT	↓ inhibits	Mechanistic		DGIdb
ROMIDEPSIN	↓ inhibits	Mechanistic	510.0 nM (IC50)	DGIdb
MARTINOSTAT	↓ inhibits	Mechanistic		DGIdb
SCRIPTAID	↓ inhibits	Mechanistic		DGIdb
CUDC-101	↓ inhibits	Mechanistic		DGIdb
GIVINOSTAT HYDROCHLORIDE	↓ inhibits	Mechanistic		DGIdb
APICIDIN	? unclear	Mechanistic	3600.0 nM (IC50)	DGIdb
SUBEROHYDROXAMIC ACID	? unclear	Mechanistic		DGIdb
TEMOZOLOMIDE	? unclear	Mechanistic		DGIdb
VORINOSTAT	↓ inhibits	Mechanistic	540.0 nM (IC50)	DGIdb
TMP269	↓ inhibits	Mechanistic		DGIdb
PHENYLBUTYRYLHYDROXAMIC ACID	? unclear	Mechanistic		DGIdb
SPIRUCHOSTATIN C	? unclear	Mechanistic		DGIdb
PANOBINOSTAT	↓ inhibits	Mechanistic		DGIdb
FIMEPINOSTAT	↓ inhibits	Mechanistic		DGIdb
4-PHENYLBUTYRIC ACID	↓ inhibits	Mechanistic		DGIdb
SN-38	? unclear	Mechanistic		DGIdb
PSAMMAPLIN A	? unclear	Mechanistic		DGIdb
CHEMBL:CHEMBL117487	? unclear	Mechanistic		DGIdb
DACINOSTAT	↓ inhibits	Mechanistic	4.4 nM (IC50)	DGIdb
CHEMBL:CHEMBL1650245	? unclear	Mechanistic		DGIdb
HINOKITIOL	? unclear	Mechanistic		DGIdb
CHEMBL:CHEMBL324053	? unclear	Mechanistic		DGIdb
RESMINOSTAT	? unclear	Mechanistic		DGIdb
SANTACRUZAMATE A	↓ inhibits	Mechanistic		DGIdb
TASQUINIMOD	~ modulates	Mechanistic		DGIdb
AR-42	? unclear	Mechanistic		DGIdb
BUTYRIC ACID	? unclear	Mechanistic		DGIdb
DAUNORUBICIN LIPOSOMAL	? unclear	Mechanistic		DGIdb
URSOLIC ACID	? unclear	Mechanistic		DGIdb
ABEXINOSTAT	↓ inhibits	Mechanistic		DGIdb
QUISINOSTAT	↓ inhibits	Mechanistic		DGIdb

Showing top 40 of 184 agents ranked by evidence tier.

HTT

UniProt Ensembl DGIdb Open Targets

Therapeutic Agent	Effect Direction	Evidence Tier	Potency	Source
CLOMIPRAMINE	↓ inhibits	Mechanistic	70.0 nM (IC50)	DGIdb
CITALOPRAM	↓ inhibits	Mechanistic	1.6 nM (Ki)	DGIdb
FLUVOXAMINE	↓ inhibits	Mechanistic		DGIdb
MILNACIPRAN	↓ inhibits	Mechanistic		DGIdb
PHENELZINE	↓ inhibits	Mechanistic		DGIdb
FLUOXETINE HYDROCHLORIDE	↓ inhibits	Mechanistic		DGIdb
3,4-METHYLENEDIOXYMETHAMPHETAMINE	? unclear	Mechanistic		DGIdb
BL-1021	? unclear	Mechanistic		DGIdb
FLUVOXAMINE MALEATE	? unclear	Mechanistic		DGIdb
VENLAFAXINE	↓ inhibits	Mechanistic	8.9 nM (Kd)	DGIdb
DESVENLAFAXINE FUMARATE	↓ inhibits	Mechanistic		DGIdb
DESVENLAFAXINE SUCCINATE	↓ inhibits	Mechanistic		DGIdb
NORTRIPTYLINE	↓ inhibits	Mechanistic		DGIdb
ACETAMINOPHEN	? unclear	Mechanistic		DGIdb
FAXELADOL	↓ inhibits	Mechanistic		DGIdb
TEDATIOXETINE	? unclear	Mechanistic		DGIdb
MINAPRINE	? unclear	Mechanistic		DGIdb
TRAZODONE	? unclear	Mechanistic		DGIdb
SERTRALINE HYDROCHLORIDE	↓ inhibits	Mechanistic		DGIdb
LIAFENSINE	↓ inhibits	Mechanistic		DGIdb
DAPOXETINE	↓ inhibits	Mechanistic		DGIdb
MAZINDOL	↓ inhibits	Mechanistic	50.0 nM (Ki)	DGIdb
VORTIOXETINE HYDROBROMIDE	↓ inhibits	Mechanistic		DGIdb
COCAINE	? unclear	Mechanistic	270.0 nM (IC50)	DGIdb
DULOXETINE HYDROCHLORIDE	↓ inhibits	Mechanistic		DGIdb
PAROXETINE HYDROCHLORIDE, HEMIHYDRATE	↓ inhibits	Mechanistic		DGIdb
NORTRIPTYLINE HYDROCHLORIDE	↓ inhibits	Mechanistic		DGIdb
TRIMIPRAMINE	↓ inhibits	Mechanistic		DGIdb
LUMATEPERONE	↓ inhibits	Mechanistic		DGIdb
AMITIFADINE	↓ inhibits	Mechanistic		DGIdb
NEFAZODONE	↓ inhibits	Mechanistic	200.0 nM (Kd)	DGIdb
ATOMOXETINE HYDROCHLORIDE	↓ inhibits	Mechanistic		DGIdb
CHLORPHENTERMINE HYDROCHLORIDE	? unclear	Mechanistic		DGIdb
TRAZODONE HYDROCHLORIDE	↓ inhibits	Mechanistic		DGIdb
DEXFENFLURAMINE	? unclear	Mechanistic		DGIdb
DOXEPIN HYDROCHLORIDE	↓ inhibits	Mechanistic		DGIdb
N-DESMETHYLCLOZAPINE	? unclear	Mechanistic		DGIdb
IBOGAINE	↓ inhibits	Mechanistic		DGIdb
LEVOMILNACIPRAN	↓ inhibits	Mechanistic		DGIdb
PSEUDOEPHEDRINE POLISTIREX	? unclear	Mechanistic		DGIdb

Showing top 40 of 234 agents ranked by evidence tier.

MSH3

UniProt Ensembl DGIdb Open Targets

Therapeutic Agent	Effect Direction	Evidence Tier	Source
METHOTREXATE	? unclear	Mechanistic	DGIdb
AZATHIOPRINE	? unclear	Mechanistic	DGIdb
MERCAPTOPURINE	? unclear	Mechanistic	DGIdb

PDE10A

UniProt Ensembl DGIdb Open Targets

Therapeutic Agent	Effect Direction	Evidence Tier	Potency	Source
BALIPODECT	? unclear	Mechanistic		Open Targets
PBF-999	? unclear	Mechanistic		Open Targets
MARDEPODECT	? unclear	Mechanistic	1.34 nM (IC50)	Open Targets
DIPYRIDAMOLE	? unclear	Mechanistic	1000.0 nM (Ki)	Open Targets
IBUDILAST	? unclear	Mechanistic		Open Targets
ZAPRINAST	↓ inhibits	Mechanistic	22000.0 nM (IC50)	ChEMBL
CHEMBL373102	↓ inhibits	Mechanistic	2200.0 nM (IC50)	ChEMBL
SILDENAFIL	↓ inhibits	Mechanistic	3100.0 nM (IC50)	ChEMBL
VARDENAFIL	↓ inhibits	Mechanistic	790.0 nM (IC50)	ChEMBL
CHEMBL245736	↓ inhibits	Mechanistic	400.0 nM (IC50)	ChEMBL
CHEMBL394442	↓ inhibits	Mechanistic	1300.0 nM (IC50)	ChEMBL
CHEMBL246345	↓ inhibits	Mechanistic	4000.0 nM (IC50)	ChEMBL
CHEMBL246344	↓ inhibits	Mechanistic	79.0 nM (IC50)	ChEMBL
CHEMBL246143	↓ inhibits	Mechanistic	160.0 nM (IC50)	ChEMBL
CHEMBL246142	↓ inhibits	Mechanistic	34.0 nM (IC50)	ChEMBL
CHEMBL437183	↓ inhibits	Mechanistic	120.0 nM (IC50)	ChEMBL
CHEMBL246141	↓ inhibits	Mechanistic	4.0 nM (IC50)	ChEMBL
CHEMBL245939	↓ inhibits	Mechanistic	18.0 nM (IC50)	ChEMBL
CHEMBL245938	↓ inhibits	Mechanistic	100.0 nM (IC50)	ChEMBL
CHEMBL245937	↓ inhibits	Mechanistic	1900.0 nM (IC50)	ChEMBL
CHEMBL245737	↓ inhibits	Mechanistic	21.0 nM (IC50)	ChEMBL
CHEMBL397774	↓ inhibits	Mechanistic	6.0 nM (IC50)	ChEMBL
CHEMBL248059	↓ inhibits	Mechanistic	3.0 nM (IC50)	ChEMBL
CHEMBL439572	↓ inhibits	Mechanistic	18.0 nM (IC50)	ChEMBL
CHEMBL248058	↓ inhibits	Mechanistic	2.0 nM (IC50)	ChEMBL
CHEMBL395205	↓ inhibits	Mechanistic	2.0 nM (IC50)	ChEMBL
CHEMBL440954	↓ inhibits	Mechanistic	22.0 nM (IC50)	ChEMBL
CHEMBL397600	↓ inhibits	Mechanistic	4.0 nM (IC50)	ChEMBL
CHEMBL248380	↓ inhibits	Mechanistic	6.0 nM (IC50)	ChEMBL
CHEMBL248379	↓ inhibits	Mechanistic	16.0 nM (IC50)	ChEMBL
CHEMBL247364	↓ inhibits	Mechanistic	1.0 nM (IC50)	ChEMBL
CHEMBL392183	↓ inhibits	Mechanistic	10.0 nM (IC50)	ChEMBL
CHEMBL247159	↓ inhibits	Mechanistic	0.3 nM (IC50)	ChEMBL
CHEMBL245710	↓ inhibits	Mechanistic	3.0 nM (IC50)	ChEMBL
CHEMBL245709	↓ inhibits	Mechanistic	2.0 nM (IC50)	ChEMBL
CHEMBL246799	↓ inhibits	Mechanistic	5.0 nM (IC50)	ChEMBL
CHEMBL246798	↓ inhibits	Mechanistic	6.0 nM (IC50)	ChEMBL
CHEMBL391364	↓ inhibits	Mechanistic	120.0 nM (IC50)	ChEMBL
CHEMBL245984	↓ inhibits	Mechanistic	7.0 nM (IC50)	ChEMBL
CHEMBL245788	↓ inhibits	Mechanistic	4.0 nM (IC50)	ChEMBL

Showing top 40 of 201 agents ranked by evidence tier.

PPARGC1A

UniProt Ensembl DGIdb Open Targets

Pipeline results not yet available for this target. Run the pipeline to populate.

SEMA4D

UniProt Ensembl DGIdb Open Targets

Therapeutic Agent	Effect Direction	Evidence Tier	Potency	Source
PEPINEMAB	? unclear	Mechanistic		DGIdb

SLC18A2

UniProt Ensembl DGIdb Open Targets

Therapeutic Agent	Effect Direction	Evidence Tier	Potency	Source
RAUWOLFIA SERPENTINA (USP)	? unclear	Mechanistic		DGIdb
DESERPIDINE	↓ inhibits	Mechanistic		DGIdb
METHAMPHETAMINE	? unclear	Mechanistic		DGIdb
DEUTETRABENAZINE	↓ inhibits	Mechanistic		DGIdb
DEXTROAMPHETAMINE SULFATE	↓ inhibits	Mechanistic		DGIdb
[3H]TBZOH	↓ inhibits	Mechanistic		DGIdb
VALBENAZINE	↓ inhibits	Mechanistic		DGIdb
[11C]DTBZ	↓ inhibits	Mechanistic		DGIdb
LISDEXAMFETAMINE DIMESYLATE	↓ inhibits	Mechanistic		DGIdb
ALKAVERVIR	? unclear	Mechanistic		DGIdb
RESERPINE	↓ inhibits	Mechanistic	5.26 nM (Ki)	DGIdb
[125I]IODOVINYL-TBZ	↓ inhibits	Mechanistic		DGIdb
[125I]7-AZIDO-8-IODOKETANSERINE	↓ inhibits	Mechanistic		DGIdb
BENZPHETAMINE HYDROCHLORIDE	↓ inhibits	Mechanistic		DGIdb
LOBELINE	? unclear	Mechanistic	2760.0 nM (Ki)	DGIdb
VALBENAZINE TOSYLATE	? unclear	Mechanistic		DGIdb
3,4-METHYLENEDIOXYMETHAMPHETAMINE	? unclear	Mechanistic		DGIdb
BEVANTOLOL HYDROCHLORIDE	? unclear	Mechanistic		DGIdb
BUPROPION HYDROCHLORIDE	? unclear	Mechanistic		DGIdb
DEXTROAMPHETAMINE ADIPATE	↓ inhibits	Mechanistic		DGIdb
DEXTROAMPHETAMINE SACCHARATE	↓ inhibits	Mechanistic		DGIdb
TETRABENAZINE	↓ inhibits	Mechanistic	19.8 nM (IC50)	DGIdb
RESCINNAMINE	↓ inhibits	Mechanistic		DGIdb
KETANSERIN	↓ inhibits	Mechanistic	353.0 nM (Ki)	DGIdb
BENZPHETAMINE	? unclear	Mechanistic		Open Targets
FLORBENAZINE	↓ inhibits	Mechanistic	0.1 nM (Ki)	ChEMBL
CHEMBL190691	↓ inhibits	Mechanistic	630.0 nM (Ki)	ChEMBL
CHEMBL425617	↓ inhibits	Mechanistic	5160.0 nM (Ki)	ChEMBL
CHEMBL124219	↓ inhibits	Mechanistic	6060.0 nM (Ki)	ChEMBL
LOBELANE	↓ inhibits	Mechanistic	970.0 nM (Ki)	ChEMBL
CHEMBL4206824	↓ inhibits	Mechanistic	152.0 nM (Ki)	ChEMBL
CHEMBL508	↓ inhibits	Mechanistic	6400.0 nM (Ki)	ChEMBL
CHEMBL213814	↓ inhibits	Mechanistic	83.0 nM (Ki)	ChEMBL
SEROTONIN	↓ inhibits	Mechanistic	7690.0 nM (Ki)	ChEMBL
CHEMBL4214975	↓ inhibits	Mechanistic	176.0 nM (Ki)	ChEMBL
CHEMBL4207658	↓ inhibits	Mechanistic	70.0 nM (Ki)	ChEMBL
CHEMBL4202787	↓ inhibits	Mechanistic	74.0 nM (Ki)	ChEMBL
CHEMBL4205254	↓ inhibits	Mechanistic	62.0 nM (Ki)	ChEMBL
CHEMBL4208779	↓ inhibits	Mechanistic	990.0 nM (Ki)	ChEMBL
CHEMBL4208250	↓ inhibits	Mechanistic	450.0 nM (Ki)	ChEMBL

Showing top 40 of 123 agents ranked by evidence tier.

Clinical Trials

Drug, biologic and supplement trials (treatment or prevention) from ClinicalTrials.gov whose main condition is Huntington's Disease: recruiting, active or completed, most recently updated first. Data retrieved 2026-10-28.

Pridopidine Phase 3 Study in Huntington's Disease Recruiting

NCT07609108 Phase: 3 Started: 2026-07-01

The goal of this clinical trial is to learn if pridopidine can slow the clinical decline of Huntington's Disease (HD) in adult participants. It will also inform about the safety of pridopidine. The main questions the study aims to answer are: Does pridopidine slow the overall worsening of HD over 1 ...

A First-in-human Study of the Effects of SRP-1005 in Participants With Huntington's Disease Recruiting

NCT07536061 Phase: 1 Started: 2026-05-12

This is a first-in-human, multi-center trial studying the effects of SRP-1005 in participants with Huntington's disease (HD).

Study to Evaluate the Pharmacodynamics, Safety and Efficacy of SKY-0515 in Participants With Huntington's Disease Recruiting

NCT07378644 Phase: 2/3 Started: 2026-01-06

The goal of this clinical trial is to test if the drug SKY-0515, an oral medication, can lower harmful proteins linked to Huntington's Disease (HD) and improve the symptoms of participants with HD. This study includes men and women aged 25 and older who have HD confirmed by genetic testing and meet ...

A Study to Evaluate ALN-HTT02 in Adult Patients With Huntington's Disease Recruiting

NCT06585449 Phase: 1 Started: 2024-10-14

The purpose of this study is to evaluate the safety, tolerability, pharmacodynamics (PD) and pharmacokinetics (PK) of single or repeat doses of ALN-HTT02.

A Study to Investigate the Efficacy, Safety and Tolerability of Votoplam in Participants With Huntington's Disease Recruiting

NCT07326709 Phase: 3 Started: 2026-03-24

The purpose is to assess safety and tolerability of votoplam and to determine whether votoplam slows disease progression in patients with early symptomatic Huntington's disease (HD) compared to the control arm. HTT227 - current compound code (former code is PTC518 from PTC Therapeutics), HTT227 is N...

A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of RG6496 in Huntington's Disease Active Not Recruiting

NCT07246941 Phase: 1 Started: 2025-11-19

This is a first-in-human (FIH) study of RG6496 that will assess the safety and tolerability of single-ascending doses of RG6496 administered to huntington's disease gene expansion carriers (HDGECs). The study consists of two parts: Part 1 \[single-ascending dose\] followed by Part 2 \[open-label ext...

Real World Effectiveness and Safety of Deutetrabenazine in Adult Chinese Participants With Huntington's Disease (HD) Chorea in China Completed

NCT07601516 Phase: 4 Started: 2024-02-02

The Primary Objective: To evaluate the real-world effectiveness of deutetrabenazine in adult participants with chorea associated with Huntington's disease in China. The Secondary Objectives: To evaluate the real-world safety of deutetrabenazine in adult participants with chorea associated with Hunti...

An Open Label Study of ANX005 in Participants With, or at Risk for, Manifest Huntington's Disease Completed

NCT04514367 Phase: 2 Started: 2020-09-29

This study is a multi-center, open-label study of intravenous (IV) ANX005 in participants with, or at risk for, manifest Huntington's Disease (HD).

GENERATION HD2. A Study to Evaluate the Safety, Biomarkers, and Efficacy of Tominersen Compared With Placebo in Participants With Prodromal and Early Manifest Huntington's Disease Active Not Recruiting

NCT05686551 Phase: 2 Started: 2023-02-03

This study will evaluate the safety, biomarkers, and efficacy of tominersen compared with placebo in participants with prodromal and early manifest Huntington's Disease (HD).

Impact of Deutetrabenazine on Functional Speech and Gait Dynamics in Huntington Disease Active Not Recruiting

NCT04713982 Phase: 2/3 Started: 2021-11-19

Examine the effects of deutetrabenazine on functional speech and gait impairment

Open-Label Rollover Study for Continuing Valbenazine Administration for the Treatment of Chorea Associated With Huntington Disease Completed

NCT04400331 Phase: 3 Started: 2020-09-18

This is a Phase 3, open-label study to evaluate the long-term safety and tolerability of valbenazine, and to provide participants continued access to valbenazine for the treatment of chorea associated with Huntington disease.

A Study to Evaluate the Safety and Efficacy of PTC518 in Participants With Huntington's Disease (HD) Completed

NCT05358717 Phase: 2 Started: 2022-06-03

The primary goal of this study is to evaluate the safety and pharmacodynamic effects of PTC518 compared with placebo in participants with HD.

The Safety and Tolerability Study With ER2001 Intravenous Injection Repetitive Treatment in Adults With Early Manifest Huntington's Disease. Completed

NCT07344519 Phase: Early 1 Started: 2024-06-25

This is an open label, dose escalation clinic trial to evaluate safety, tolerability and pharmacokinetics with ER2001 Intravenous Injection repetitive treatment in Huntington's Disease patients who Completed Study ER2001-001（NCT06024265）. Furthermore, pharmacodynamics in particular target engagement...

Study of SKY-0515 for Safety, Efficacy, and Pharmacodynamics in Participants With Huntington's Disease Active Not Recruiting

NCT06873334 Phase: 2/3 Started: 2025-05-27

Safety and Tolerability Study of Human Neural Stem Cells for Huntington's Disease Recruiting

NCT07451613 Phase: 1/2 Started: 2026-04-06

The purpose of this research study is to determine whether an implantation of hNSC-01 is a safe and tolerable study intervention for Huntington's disease. This study is the first time that hNSC-01 is being tested in people.

Agents Found By Disease-Level Search

Searched directly from “Huntington's Disease” via ClinicalTrials.gov interventions and Open Targets' disease→drug data — independent of the 10-gene target panel above. This surfaces agents whose mechanism doesn't route through a curated gene (combination therapies, standard-of-care drugs, targets outside the panel).

47 agents shown (approved or with trial evidence) — 43 not found by the gene-target search above.

dimebonNOT in gene-target list

· 3 trials · source: ClinicalTrials.gov
NCT00831506 NCT00824590 NCT00824590

Creatine monohydrateNOT in gene-target list

· 3 trials · source: ClinicalTrials.gov
NCT01412151 NCT01411163 NCT01411150

Deutetrabenazine

Approved · 2 trials · source: ClinicalTrials.gov, Open Targets
NCT07601516 NCT04713982

FuRST 2.0NOT in gene-target list

· 2 trials · source: ClinicalTrials.gov
NCT02881931 NCT03709173

digoxinNOT in gene-target list

· 2 trials · source: ClinicalTrials.gov
NCT00831506 NCT00831506

PF-02545920NOT in gene-target list

· 2 trials · source: ClinicalTrials.gov
NCT01806896 NCT01806896

RO7234292 (RG6042)NOT in gene-target list

· 2 trials · source: ClinicalTrials.gov
NCT04000594 NCT03842969

PBT2NOT in gene-target list

· 2 trials · source: ClinicalTrials.gov
NCT01590888 NCT01590888

SEN0014196NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT01485965

PBF-999

Phase 1 · 1 trial · source: ClinicalTrials.gov, Open Targets
NCT02208934

SPK-10001NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT06826612

Non invasive prenatal diagnosisNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT03087526

CreatineNOT in gene-target list

Phase 3 · 1 trial · source: ClinicalTrials.gov, Open Targets
NCT00026988

ActiGraph LEAPNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT07010705

Axivity AX6NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT07010705

Radioligand [11C]CHDI-00491009NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT06634628

Votoplam (blinded)NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT07326709

LithiumNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT00095355

DivalproexNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT00095355

MindTrails-MovementNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT07213648

MindTrailsNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT05126862

VO659NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT05822908

Amantadine

Phase 2 · 1 trial · source: ClinicalTrials.gov, Open Targets
NCT00001930

ER2001 intravenous injectionNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT07339514

ALN-HTT02NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT06585449

NACNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT05509153

deutetrabenazine (SD-809)NOT in gene-target list

Approved · 1 trial · source: ClinicalTrials.gov
NCT01795859

sodium phenylbutyrateNOT in gene-target list

Phase 2 · 1 trial · source: ClinicalTrials.gov, Open Targets
NCT00212316

pridopidine (90 mg)NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT03019289

SRTNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT02814201

Fish OilNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT01690312

pridopidine

Phase 3 · 1 trial · source: ClinicalTrials.gov, Open Targets
NCT01306929

Radioligand [18F]MNI-659NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT02956148

SOM3355 200 mg capsulesNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT05475483

SOM3355 300 mg capsulesNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT05475483

Tetrabenazine withdrawalNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT01834911

LPM3770164 sustained release tabletNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT06216054

LPM3770164 sustained release tablet simulantNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT06216054

TominersenNOT in gene-target list

Phase 3 · 1 trial · source: ClinicalTrials.gov, Open Targets
NCT05686551

SRX246NOT in gene-target list

Phase 2 · 1 trial · source: ClinicalTrials.gov, Open Targets
NCT02507284

Group Drumming Music TherapyNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT05157074

CardioLux™HDLNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT04529174

SEN0014196 (Low Dose)NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT01485952

SEN0014196 (High Dose)NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT01485952

Dimebon IR TabletNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT00988624

Dimebon MR1NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT00988624

Dimebon MR2NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT00988624

Generated by the Open Source Medicine Foundation Biomarker Pipeline — 2026-10-08. Data sources: DGIdb, Open Targets, ChEMBL, PubMed, ClinicalTrials.gov. Not medical advice.

Huntington's Disease

Remission & chronicity

Biomarker Targets & Therapeutic Agents

DRD2

FAN1

GRIN2B

HDAC4

HTT

MSH3

PDE10A

PPARGC1A

SEMA4D

SLC18A2

Clinical Trials

Agents Found By Disease-Level Search

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