Clinical Trials

Inclusion Criteria:

• Subjects with a known or suspected diagnosis of the following specific hereditary ataxias:
  • SCA1, SCA2, SCA3, SCA6, SCA7, SCA8 and SCA10.
    • A subject has clinical evidence that supports diagnosis of one of the aforementioned SCA genotypes but does not have producible test results from a CLIA certified lab from either a family member or for his or herself and the subject must be willing to undergo such testing to confirm the SCA diagnosis (in this case, site must wait for results of genotypic testing prior to randomization).
• Ability to ambulate 8 meters without human assistance (canes and other devices allowed).
• Screening total SARA score ≥ 8.
• Determined by the investigator to be medically stable at Baseline/randomization as assessed by medical history, physical examination, laboratory test results, and electrocardiogram testing. Subjects must be physically able and expected to complete the trial as designed.
• Subjects must have adequate hearing, vision, and language skills to perform SARA ratings and other neuropsychiatric testing and interviews as specified in the protocol.

Exclusion Criteria:

• MMSE score <24.
• Any medical condition other than one of the hereditary ataxias specified in the inclusion criteria that could predominantly significantly to the subjects' symptoms of ataxia (for example, alcoholism, vitamin deficiencies, multiple sclerosis, vascular disease, tumors, paraneoplastic disease, head injury, idiopathic late onset ataxia, multisystem atrophy) or that can confound assessment of ataxia symptoms (for example, stroke, arthritis).
• SARA total score of >30 points at screening.
• Clinical history of stroke.
• Immunocompromised subjects.
• Active liver disease or a history of hepatic intolerance to medications that in the investigator's judgment, is medically significant.