Fampridine versus placebo for adults with SCA27B caused by an FGF14 GAA expansion

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This is a randomized, parallel-arm, double-blind, placebo-controlled Phase 3 trial comparing fampridine 10 mg prolonged-release tablets to matched placebo in adults with genetically confirmed SCA27B due to an FGF14 GAA expansion. Eligible participants must be ≥18 years old, have a SARA total score >3 with at least 1 point on the gait item, and be able to take oral medication and attend clinic visits. Clinical outcomes will be measured using standardized ataxia and functional rating scales and structured clinical assessments at participating university hospitals in France. The trial follows from small open-label reports that suggested symptomatic benefit but aims to provide rigorous, controlled evidence of effect and safety.

Why it matters

Who can join: the full rules

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

Where it's running

• Genetics Department, CHU de Bordeaux
  Bordeaux, France Recruiting
• Neurology and Gentics Department, CHU de Dijon
  Dijon, France Recruiting
• Neurology Department, Gui De Chauliac Hospital
  Montpellier, France Recruiting
• Genetics Department, Pitié-Salpêtrière University Hospital
  Paris, France Recruiting
• Neurology Department, CHU d'Angers
  Angers, France Not yet recruiting
• Neurology Department, Hôpital Pierre Wertheimer Hospital
  Lyon, France Not yet recruiting

Study contacts

• Principal investigator: Giulia COARELLI — Assistance Publique - Hôpitaux de Paris (AP-HP)
• Study coordinator: Giulia COARELLI
• Email: giulia.coarelli@aphp.fr
• Phone: + 33 (0)1 57 27 46 82

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Other studies for the same condition or treatment. Recruiting studies are listed first.

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.