Promising drug for rare balance disorder enters final testing phase

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: * Genetic diagnosis of spinocerebellar ataxia SCA27B caused by an expansion ≥ 250 GAA repeats in the FGF14 gene * At least 18 years of age * SARA total score \> 3 and score ≥ 1 on the "gait" item of the SARA scale. * Physically able and expected to complete the trial as designed and having the ability to take oral medication * Signature of informed consent * Covered by social security Exclusion Criteria: * Hypersensitivity to fampridine * Hypersensitivity to any excipients present in fampridine * Serious systemic illnesses or conditions known for enhancing the side-effects of fampridine (i.e., creatinine clearance \< 50 ml/min, hepatic insufficiency, medically significant heart conduction disorders such as occurrence of torsades de pointes or another severe ventricular arrhythmia, high-degree atrioventricular block (Mobitz II or complete), Brugada pattern, QTcF time of \>480 msec in 3 consecutive ECG recordings taken at least 5 minutes apart, uncompensated cardiovascular disorder, epilepsy) * Unstable, clinically significant neurologic (other than the disease being studied; eg, recurrent strokes), psychiatric, cardiovascular (eg, pulmonary arterial hypertension, cardiac valvulopathy, orthostatic hypotension/tachycardia), pulmonary, hepatic, renal, metabolic, gastrointestinal, urologic, immunologic, hematopoietic, or endocrine disease or other abnormality which may impact the ability of the participant to participate or potentially confound the study results. * Patients with known recurrent, active, or chronic infections. * Patients with prior history of seizure. * Concurrent treatment with other medicinal products containing fampridine (4-aminopyridine). * Concomitant use of Fampyra with medicinal products that are inhibitors or substrates of Organic Cation Transporter 2 (OCT2) for example, cimetidine. * Participation in another clinical trial with an investigational drug or receipt of an investigational product within 12 weeks or 5 times the half-life of the product (whichever is longer) prior to Baseline visit * Previous treatment with fampridine * Patients considered at risk of suicidal behavior based on the Columbia-Suicide Severity Rating Scale (C-SSRS), defined as reporting suicidal ideation with intent to act (C-SSRS items 4 or 5) within the 6 months prior to randomization, or any suicidal behavior (including actual, aborted, or interrupted attempts) within the past 12 months. * Pregnancy and breastfeeding (women in childbearing potential will have a urine pregnancy test at each visit) * Sexual non abstinence or absence of effective contraception (for child-bearing aged women, contraception using highly effective methods (see section 6.2 of the protocol) for the duration of treatment and up to 7 days after the last dose of treatment) * Inability to understand information about the protocol * Legally incapacitated adults (e.g., individuals under legal protection such as guardianship or curatorship) * Persons deprived of their liberty by judicial decision * Other ataxic syndromes than SCA27B