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Key Clinical Trials

Huntington's Disease


90 Participants

A randomized, double blind, placebo controlled study of SD-809 extended release for the treatment of chorea associated with Huntington’s disease.

FDA Approval April 2017


72 Participants

A randomized, double-blind, placebo-controlled, study of tetrabenazine for the treatment of Huntington’s chorea.

FDA Approval August 2008


301 Participants

A study in individuals with late prodromal and early manifest Huntington’s disease to access the safety, tolerability, pharmacokinetics, and efficacy of pepinemab. (VX15/2503)

Parkinson's Disease


800 Participants

A 2 x 2 factorial, double-blind, placebo-controlled, phase III multi-center clinical trial in participants with early Parkinson’s disease to assess the efficacy of tocopherol and deprenyl.


75 Participants

A randomized, double-blind, placebo-controlled, phase III, study to define the safety, tolerability, clinical and exploratory biological activity of the chronic administration of Nilotinib in participants with Parkinson's disease.


336 Participants

A Phase III double-blind placebo-controlled parallel group study of isradipine as a disease modifying agent in participants with early Parkinson’s disease.


1,700 Participants

The Parkinson’s Progression Markers Initiative is a global, longitudinal observational study seeking markers of progression in Parkinson’s disease.

Other Rare Neurological Disorders


42 Participants

A randomized, controlled study of acetazolamide vs. dichlorphenamide vs. placebo in individuals with hyperkalemic and hypokalemic periodic paralysis.

FDA Approval August 2015


1000+ Participants

Friedreich’s Ataxia Clinical Outcome Measure Study.


Randomized, placebo-controlled clinical trial evaluating the safety, pharmacokinetics, and efficacy of Actimmune in children and young adults with Friedreich's ataxia.

We have conducted additional clinical trials for other conditions, including dental caries, epilepsy, HIV, influenza, intracranial hypertension, stroke, and testicular cancer.