Fampridine versus placebo for adults with SCA27B caused by an FGF14 GAA expansion
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
PHASE3 · Assistance Publique - Hôpitaux de Paris · NCT07185347
This trial tests whether fampridine (4-aminopyridine) can improve walking and visual symptoms in adults who have SCA27B from a GAA expansion in the FGF14 gene.
Quick facts
| Phase | PHASE3 |
|---|---|
| Study type | Interventional |
| Enrollment | 70 (estimated) |
| Ages | 18 Years and up |
| Sex | All |
| Sponsor | Assistance Publique - Hôpitaux de Paris (other) |
| Locations | 9 sites (Angers and 8 other locations) |
| Trial ID | NCT07185347 on ClinicalTrials.gov |
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.
Who should consider this trial
Good fit: Adults (≥18) with a confirmed FGF14 GAA expansion of ≥250 repeats, measurable ataxia (SARA total >3 and gait item ≥1), able to take oral medication and attend clinic visits are ideal candidates.
Not a fit: People without the specific FGF14 GAA expansion or with contraindications to fampridine (for example severe renal impairment, hepatic insufficiency, certain cardiac conduction disorders, extreme QTc prolongation, or known hypersensitivity) are unlikely to benefit or be eligible.
Why it matters
Potential benefit: If successful, fampridine could improve gait and visual symptoms and produce measurable gains on ataxia rating scales for people with SCA27B.
How similar studies have performed: Small open-label studies of 4-aminopyridine reported improvements in visual symptoms and gait in many SCA27B patients, but these were uncontrolled, used diverse methods, and benefits were often transient.
Eligibility criteria
Show full inclusion / exclusion criteria
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 this trial is running
Angers and 8 other locations
- Neurology Department, CHU d'Angers — Angers, France (NOT_YET_RECRUITING)
- Genetics Department, CHU de Bordeaux — Bordeaux, France (RECRUITING)
- Neurology and Gentics Department, CHU de Dijon — Dijon, France (RECRUITING)
- Neurology Department, Hôpital Pierre Wertheimer Hospital — Lyon, France (NOT_YET_RECRUITING)
- Neurology Department, Gui De Chauliac Hospital — Montpellier, France (RECRUITING)
- Genetics Department, Pitié-Salpêtrière University Hospital — Paris, France (RECRUITING)
- Genetics Department, CHU de Rouen — Rouen, France (NOT_YET_RECRUITING)
- Neurology Department, CHRU de Strasbourg — Strasbourg, France (NOT_YET_RECRUITING)
- Neurology Department, CHU de Toulouse — Toulouse, 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
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
Conditions: Spinocerebellar Ataxia 27B, SCA27B, Fampridine, Ataxia, Diplopia, FARS-Functional Staging