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A Randomized, Parallel-arm, Double Blind, Placebo-controlled Study to Assess the Efficacy of Fampridine for Patients With Spinocerebellar Ataxia SCA27B Caused by a GAA Expansion in the FGF14 Gene

RecruitingPhase 3Ages 18 Years - anyAllAssistance Publique - Hôpitaux de Paris
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In plain words

What the trial is, and whether it could suit you

Spinocerebellar ataxias 27B (SCA27B) is caused by an expansion of ≥ 250 GAA triplets in the FGF14 gene and accounts for 15% of cerebellar ataxias (around 500 patients in France). It is a late-onset form often presenting paroxysmal episodes of ataxia and/or diplopia. The disease progresses slowly, with an average increase of 0.10 points/year on the Friedreich's Ataxia Rating Scale (FARS) - Functional Staging and by 0.23 points/year on the Scale for the Assessment and Rating of Ataxia (SARA). To date, no treatment has been proven to be effective in these patients. Three open-label studies using 4-aminopyridine, have shown improvements in visual symptoms and gait in a total of 36 out of 44 patients, although these improvements were evaluated through diverse methodologies. In a subgroup of patients (n=7), administration of 4-aminopyridine resulted in a reduction in FARS - Functional Staging,

Sign in to get the eligibility rules as simple questions to take to your doctor.

📋 Eligibility rules (as published)
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
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What it tests

  • Fampridine 10 mg prolonged-release tablet (per os)
  • Placebo (tablets per os)
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Where

9 sites
Neurology Department, CHU d'Angers
Angers, France
Genetics Department, CHU de Bordeaux
Bordeaux, France
Neurology and Gentics Department, CHU de Dijon
Dijon, France
Neurology Department, Hôpital Pierre Wertheimer Hospital
Lyon, France
Neurology Department, Gui De Chauliac Hospital
Montpellier, France
Genetics Department, Pitié-Salpêtrière University Hospital
Paris, France
Genetics Department, CHU de Rouen
Rouen, France
Neurology Department, CHRU de Strasbourg
Strasbourg, France

…and 1 more on ClinicalTrials.gov.

🤝 Taking part is your choice, and you can leave at any time. Only the trial team can confirm you are eligible — always decide together with your own doctor.
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