Novel targets for Huntington’s disease: future prospects

Figures & data

Table 1 Ongoing trials

Sponsor	Compound	End point	Design	Status
Phase I
Ionis Pharmaceuticals	Isis-HTTRx (dose unknown)	Safety, tolerability	Phase I, Phase II/placebo	Recruiting
Cambridge University Hospitals NHS Foundation Trust and University of Cambridge	Rilmenidine (1 mg)	Safety, tolerability, motor, cognition, biomarker	Phase I/open label	Ongoing
Azevan Pharmaceuticals	SRX246 (dose unknown)	Safety, tolerability, psychiatric, motor	Phase I, Phase II/placebo	Not yet open
Oregon Health and Science University	Ursodiol (600–1,200 mg)	Safety, tolerability, pharmacokinetics	Phase I/placebo	Unknown
Phase II
Ipsen	BN82451 (40–80 mg)	Safety, pharmacokinetics, motor	Phase II/placebo	Recruiting
Teva Pharmaceutical Industries	Laquinimod (0.5–1.5 mg)	Motor, MRI	Phase II/placebo	Recruiting
Carlsson Research AB/Sahlgrenska University Hospital	OSU6162 (5–90 mg)	Safety, motor, cognition, psychiatric, function	Phase II/open label	Ongoing
Pfizer	PF-0254920 (40 mg)	Motor, safety, psychiatric	Phase II/placebo and open label	Ongoing
Teva Pharmaceutical Industries	Pridopidine (90–225 mg)	Safety, motor	Phase II/placebo and open label	Recruiting
Vaccinex Inc	VX15/2503 (20 mg/kg)	Safety, tolerability, pharmacokinetics, MRI, cognition, motor, function, psychiatric	Phase II/placebo	Ongoing

Abbreviation: MRI, magnetic resonance imaging.

Table 2 Completed trials

Sponsor	Compound	Endpoint	Design	Status
Phase II
Novartis Pharmaceuticals	AFQ056 (25 mg)a	Safety, tolerability, chorea	Phase II/placebo	Terminatedb
University of Iowa	Atomoxetine (80 mg)	Cognitive, psychiatric	Phase II/placebo	Completed
Charité University, Berlin, Germany	Bupropion (150–300 mg)	Apathy	Phase II/placebo	Completed
University of Iowa	Citalopram (20 mg)	Cognition, function, MRI	Phase II/placebo	Completed
National Institute of Neurological Disorders and Stroke (NINDS)	Lithium and divalproex (dose unknown)	Safety, CSF biomarkers	Phase II/placebo	Completed
University of British Columbia	Memantine (dose unknown)	Novel TRACK-HD end points, psychiatric, cognitive	Phase II/placebo	Completed
Omeros Corporation	OMS643762	Safety, tolerability, pharmacokinetics	Phase II/placebo	Suspended
Prana Biotechnology	PBT2 (100–250 mg)	Safety, tolerability, cognition, motor, psychiatric, function, biomarkers, MRI	Phase II/placebo	Completed
Siena Biotech SPA	SEN0014196 (50–200 mg)	Safety, tolerability, motor, cognition, psychiatric, function, pharmacokinetic	Phase II/placebo	Completed
University of Rochester	Sodium phenylbutyrate	Safety, tolerability, motor, cognition, biomarkers	Phase II/placebo	Completed
University of Angers	Cysteamine (RP103)	Safety, tolerability, motor, function	Phase II/placebo	Completed

Notes:

a Well tolerated, not effective;

b a study which was terminated early due to a failure to demonstrate efficacy.

Abbreviations: MRI, magnetic resonance imaging; CSF, cerebrospinal fluid.