Table 3.
Ongoing clinical trials for treatment of chorea in Huntington’s disease
| Study | Phase | Design | Study agent | Outcome measures | Trial length |
|---|---|---|---|---|---|
| ARC-HD (NCT01897896) | III | Multicenter, open-label, long-term safety extension study of FIRST-HD | Deutetrabenazine 6, 9, or 12 mg/day | Incidence of adverse events, change from baseline in clinical laboratory parameters, vital signs and UHDRS | Up to 58 weeks |
| NCT02197130 | II | Randomized, double-blind, placebo-controlled efficacy and safety study | PF-02545920 5 mg and 20 mg/day | Change from baseline in UHDRS- TMS and total maximum chorea score | 26 weeks |
| NEUROHD (NCT00632645) | III | Multicenter, randomized, controlled comparison study | Olanzapine 2.5–20 mg/day Tetrabenazine 25–200 mg/day Tiapride 300–800 mg/day |
Change in independence, motor, psychiatric and cognitive scales, assessment of adverse effects | 52 weeks |
| PRIDE-HD (NCT02006472) | II b | Multicenter, randomized, double-blind, placebo-controlled safety and efficacy study | Pridopidine 45, 67.5, 90, 112.5 mg twice daily | Change from baseline in UHDRS-TMS and mPPT | 26 and 52 weeks |
| Open-Hart (NCT01306929) | II | North-American, long-term open-label extension study of PRIDE-HD | Pridopidine 45 mg | Number of adverse events, development of UHDRS-TMS over time | Up to 2 years |
| Open-Pride (NCT02494778) | II | Multicenter, open-label safety, tolerability and efficacy extension study of PRIDE-HD | Pridopidine 45 mg twice daily | Percentage of participants with adverse events, change in UHDRS-TMS over time | 2 years |
| Legato (NCT02215616) | II | Multicenter, randomized, double-blind, placebo-controlled efficacy and safety study | Laquinimod 0.5, 1.0, and 1.5 mg/day | Change from baseline in UHDRS-TMS, percent change in caudate volume and HD-CAB total score | 12 months |
| Trihep3 (NCT02453061) | II | Randomized, double-blind, controlled study | Triheptanoin oil 1 g/kg/day | Change in index of brain energy restoration using 31-P MRS and change of caudate atrophy rate using MRI. Change in motor function | 6 and 12 months |
Information on current ongoing clinical trials investigating the efficacy on chorea in patients with Huntington’s disease obtained from http://www.clinicaltrials.gov [accessed 16 August 2016]
HD Huntington’s disease, UHDRS–TMS Unified Huntington’s Disease Rating Scale–Total Motor Score, mPPT modified Physical Performance Test, 31-P MRS 31-Phosophorus Magnetic Resonance Spectroscopy, MRI magnetic resonance imaging, HD-CAB Huntington’s Disease Cognitive Assessment Battery