Open Pilot Trial of BHV-4157

Who cannot join

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• Target Disease Exceptions
• Any medical condition other than one of the hereditary ataxias specified in the inclusion criteria that could predominantly explain or contribute 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); b. MMSE score < 24; c. Subjects with prominent spasticity or dystonia that meet either of the following criteria: i. In the opinion of the investigator will compromise the ability of the SARA instrument to assess underlying ataxia severity; or, ii. Are associated on the INAS instrument at screening with moderate or severe scores on dystonia (at least 3 of 5 items) or spasticity (at least 2 of 3 items) or rigidity (at least 2 of 3 items) d. SARA total score of > 30 points at screening; e. Subjects may not have started physical or occupational therapy within one month of screening and are not expected to start such therapy during the initial 12 week treatment phase. Subjects with ongoing occupational or physical therapy may be allowed to continue as long as the intensity remains unchanged from two months prior to screening throughout the randomization period.
• Medical History Exclusions
• Clinical history of stroke. Note: Subjects with a history of transient ischemic attack (TIA) may be enrolled, if it occurred at least 3 months prior to screening and the subject is prescribed appropriate treatment [e.g., platelet aggregation inhibitors]; b. Immunocompromised subjects. Note: Subjects taking a systemic immunosuppressive agent may enter treatment phase if they are on a stable dose, have no clinically relevant immunosuppression, and have a white blood count (WBC) within normal limits; c. Active liver disease or a history of hepatic intolerance to medications that in the investigator's judgment, is medically significant; d. History of medically significant gastrointestinal (GI) illnesses including: i. A current diagnosis of active, peptic ulceration or gastrointestinal bleeding within the last 6 months and/or chronic inflammatory bowel disease at screening; ii. A history of any gastrointestinal surgery that impacts the absorption of study drug; iii. Chronic or frequent episodes of loose stools; bowel movements; e. Vitamin B12 or folate deficiency Note: Subjects with a B12 deficiency can participate in the study if they are on stable Vitamin B12 replacement for at least 3 months prior to randomization and their B12 levels are within normal limits prior to randomization; f. Hematologic or solid malignancy diagnosis within 5 years prior to screening. (Note: Subjects with a history of localized skin cancer, basal cell or squamous cell carcinoma, may be enrolled in the study as long as they are cancer free prior to randomization. Subjects with other localized cancers (without metastatic spread) who have previously completed their course of treatment more than 2 years prior to baseline, are not currently receiving treatment and have been in remission may be enrolled only if, in the opinion of the investigator, there is no expectation for recurrence or further cancer treatment during the study period. Antihormonal therapy (e.g., tamoxifen) is allowed if the subject's cancer is in remission and the subject is on maintenance therapy to reduce their risk of recurrence; g. Any unstable cardiovascular (includes uncontrolled hypertension), pulmonary, gastrointestinal, or hepatic disease 30 days prior to screening; h. End-stage cardiovascular disease (e.g., Congestive Heart Failure New York Heart Association/CHF NYHA Class III or IV or unstable angina); i. Treated for, or have had a lifetime diagnosis of, schizophrenia or bipolar disorder; j. Active major depressive episode within the past 6 months. Note: Subjects on a stable maintenance dose of a non- tricyclic, non-monoamine oxidase inhibitor (MAOI) antidepressant medication (e.g., serotonin reuptake inhibitor, bupropion) with symptoms in remission may be eligible; k. History of neurosyphilis (as indicated by a positive rapid plasma reagin [RPR] test and a positive confirmatory test); l. History of drug or alcohol abuse within 12 months as defined by DSM-IV-TR-TR criteria; m. History or evidence of any medical, neurological or psychological condition that would expose the subject to an undue risk of a significant adverse event or interfere with assessments of safety and efficacy during the course of the trial as determined by the clinical judgment of the investigator; n. History of chronic pulmonary disease or chronic pulmonary symptoms
• Physical and Laboratory Test Findings