Status:
RECRUITING
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
Lead Sponsor:
Assistance Publique - Hôpitaux de Paris
Conditions:
Spinocerebellar Ataxia 27B (SCA27B)
Eligibility:
All Genders
18+ years
Phase:
PHASE3
Brief Summary
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 fo...
Eligibility Criteria
Inclusion
- 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
- 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
Key Trial Info
Start Date :
October 21 2025
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
May 21 2027
Estimated Enrollment :
70 Patients enrolled
Trial Details
Trial ID
NCT07185347
Start Date
October 21 2025
End Date
May 21 2027
Last Update
December 10 2025
Active Locations (9)
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1
Neurology Department, CHU d'Angers
Angers, France
2
Genetics Department, CHU de Bordeaux
Bordeaux, France
3
Neurology and Gentics Department, CHU de Dijon
Dijon, France
4
Neurology Department, Hôpital Pierre Wertheimer Hospital
Lyon, France