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. 2019 Aug 27;8(3):363–371. doi: 10.3233/JHD-199003

Table 2.

Ongoing pharmacological clinical trials registered at the World Health Organization (WHO) International Clinical Trials Research Platform (ICTRP) for people with Huntington’s disease (HD)

Registration ID Trial name Intervention Mechanism of Action Population Comparison Main outcome Study design Estimated Enrolment Sponsor Location
NCT03854019* – Dextromethorphan/quinidine Morphinan/class I antiarrhythmic agent HD with irritability Placebo Clinical efficacy at 6 and 13 weeks Randomized, double-blind, placebo-controlled, cross-over trial 22 University of Texas Health Science Center, Cures Within Reach USA (single centre)
NCT03842969* GEN-EXTEND RG6042 Allele- nonselective antisense oligonucleotide HD None Safety and tolerability at up to 5 years Open-label extension 950 Hoffmann-La Roche USA, Canada, Europe (multi centre)
NCT03761849 GENERATION-HD1 RG6042 Allele- nonselective antisense oligonucleotide HD Placebo Clinical efficacy at 101 weeks Randomized, double-blind, placebo-controlled, parallel trial 660 Hoffmann-La Roche USA, Canada, Europe (multi centre)
NCT03787758 – SAGE-718 NMDA positive allosteric modulator HD Placebo Safety at 21 days Randomized, double-blind, placebo-controlled, multiple ascending dose trial 10 Sage Therapeutics N/S
NCT03575676 – SOM3355 VMAT2 inhibitor and β1 antagonist Early and moderate HD with chorea Placebo Chorea at 6 months Randomized, double-blind, placebo-controlled, cross-over trial 30 SOM Biotech SL Spain (multi centre)
NCT03515213 – Fenofibrate PPARα agonist HD Placebo Pharmacodynamics at 6 months Randomized, double-blind, placebo-controlled, parallel trial 20 University of California, Irvine USA (single centre)
NCT03764215 Tasigna HD Nilotinib Selective Bcr-Abl tyrosine kinase inihbitor HD None Safety, tolerability and pharmacodynamics at 3 months Open label, multiple ascending dose 20 Georgetown University USA (single centre)
NCT03342053 IONIS-HTTRX OLE ISIS 443139 Allele- nonselective antisense oligonucleotide HD None Safety and tolerability at 74 weeks Open label extension 46 Ionis Pharmaceuticals Inc. Canada, Germany and UK (multi-centre)
NCT03225833 PRECISION-HD1 WVE-120102 Allele-selective antisense oligonucleotide HD Placebo Safety and tolerability at 1 and 120 days Randomized, double-blind, placebo-controlled, combined single ascending dose/multiple ascending dose trial 48 Wave Life Sciences Ltd. Canada and Poland (multi-centre)
NCT03225846 PRECISION-HD2 WVE-120102 Allele-selective antisense oligonucleotide HD Placebo Safety and tolerability at 1 and 120 days Randomized, double-blind, placebo-controlled, combined single ascending dose/multiple ascending dose trial 48 Wave Life Sciences Ltd. Canada and Poland (multi-centre)
NCT02453061 TRIHEP 3 Triheptanoin Anaplerotic therapy HD Safflower oil Pharmacodynamic efficacy at 6 months Randomized, double-blind, controlled, parallel trial 100 Institut National de la Santé Et de la Recherche Médicale, Ultragenyx Pharmaceutical Inc France, Netherlands (multi centre)
NCT02509793 – Tetrabenazine VMAT2 inhibitor HD with impulsivity None Cognitive and behavioural effects at 8 weeks Single group, open-label trial 20 University of Texas Health Science Center, and H. Lundbeck A/S USA (single centre)
NCT02481674 SIGNAL VX15/2503 Anti-semaphorin 4D monoclonal antibody Late premanifest or early HD Placebo Safety and tolerability at 15 and 21 months Randomized, double-blind, placebo-controlled, parallel trial 240 Vaccinex Inc., Huntington Study Group USA (multi centre)
NCT02336633 REVHD Resveratrol Dietary supplement HD Placebo Neuroimaging biomarkers at 1 year Randomized, double-blind, placebo-controlled, parallel trial 102 Assistance Publique - Hôpitaux de Paris France (multi centre)
EUCTR2013-002545-10-SE OSU6162Open1309 (-)-OSU616 Monoaminergic stabilizer HD, PD, brain trauma, stroke, myalgic encephalomyelitis and narcolepsy None Safety at 3, 6 and 12 months Single group, open-label trial 240 A. Carlsson Research AB Sweden (multi centre)
NCT00514774 UDCA-HD Ursodiol Bile acid HD Placebo Safety, tolerability and pharmacokinetics at 35 days Randomized, double-blind, placebo-controlled, parallel trial 21 Oregon Health and Science University, Huntington Study Group, Huntington Society of Canada N/S

N/S, not specified; PD, Parkinson’s disease; VMAT2, Vesicular Monoamine Transporter 2. Note: IONIS-HTTRx, ISIS 443139 and RG6042 refer to the same molecule. New trials since the last Clinical Trials Corner are indicated by*.