ClinicalTrials.gov record NCT07185347

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

Record copied from ClinicalTrials.gov: Oct 10, 2026AI summaries generated: Sep 17, 2026

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

StatusRecruitingThe study is currently enrolling participants. Only the study team can say whether a particular person can join.
Study typeInterventionalA study in which researchers assign participants to one or more interventions, such as a drug, procedure, device, behavioural programme or other approach, to measure their effects.
PhasePhase 3A later-stage study, usually with more participants, designed to give stronger evidence about how well a treatment works and how safe it is. It often compares the treatment with standard care or a placebo, and results may support an application for approval.
Age18 years old and older
SexAll
LocationFrance
AI summaryWhat this study is about, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

This Phase 3 randomized, double-blind, placebo-controlled study compares fampridine (10 mg sustained-release, twice daily) with placebo in adults with genetically confirmed SCA27B. Participants take study tablets for 12 weeks and have clinical assessments at weeks 2 and 12, with a follow-up visit at week 16. The main outcome is the proportion of people with at least a 0.5-point improvement on the FARS-Functional Staging at week 12; other measures include SARA, walking tests, diplopia frequency, quality-of-life questionnaires, and safety monitoring.

What it is studying. The study tests whether fampridine (sustained-release 10 mg twice daily) affects functional and symptom measures compared with placebo in adults who have genetically confirmed SCA27B (GAA expansion in FGF14).

What the researchers hope to learn. To determine whether fampridine changes functional handicap in SCA27B at 12 weeks (primary outcome: FARS-Functional Staging) and to measure effects on ataxia scales (SARA, mFARS, CCFS), oculomotor signs, diplopia frequency, walking speed, quality of life, and drug safety/tolerability, including whether any effects persist 4 weeks after stopping treatment.

What this study is about

The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.

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, ranging from 0.5 to 2 points. Notably, this beneficial effect rapidly disappearing in all patients stopping the drug. 4-aminopyridine, a potassium channel blocker, may involve restoration of cerebellar Purkinje cell rhythmic firing property, impaired with the loss of FGF14 function. Although these results appear very promising, the positive effect of 4-aminopyridine is reported only in restricted sample sizes and open-label experiences. Therefore, a robust clinical trial is necessary to provide the level of evidence required for a definitive conclusion on the benefit-risk of fampridine and before introducing the treatment into the regular patient clinical management. Hence, to confirm the beneficial effect of 4-aminopyridine treatment, this study will compare fampridine 10 mg bid (sustained-release form) to placebo during a 3-month treatment in a randomized, double-blind, multicenter, placebo-controlled study, on functional handicap in SCA27B cerebellar ataxia patients.

Interventions being studied

Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.

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

Eligibility

The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.

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

Study description and what participation involves

The registry record has no detailed description beyond the summary above.

AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

Participants will be randomly assigned to take either fampridine 10 mg prolonged‑release tablets or matching placebo, one tablet twice daily on an empty stomach for 12 weeks. Study visits include baseline, week 2, week 12, and a follow-up visit at week 16 (four weeks after stopping treatment) with neurological exams and ataxia assessments (FARS, SARA, mFARS, CCFS), walking tests (Timed 25‑foot walk), eye‑movement assessments and recordings, diplopia ratings, patient and clinician global impression scales, and quality‑of‑life questionnaires (PROM‑ATAXIA, SF‑36). Safety monitoring includes ECGs and blood tests at week 2 and week 12, continuous recording of adverse events, and pregnancy testing for women of childbearing potential at each visit. Participants must be able to take oral medication and attend the scheduled visits.

How the study is structured. Adults are randomly assigned to one of two groups and neither participants nor study staff know who receives fampridine or placebo. Those assigned to active treatment take one tablet twice a day on an empty stomach for 12 weeks; the placebo group follows the same schedule. Study visits include clinical exams, walking and ataxia scales, oculomotor (eye movement) assessments, patient questionnaires, and safety checks such as blood tests and ECGs at specified visits; there is an additional assessment 4 weeks after stopping treatment.

Questions you could ask

These questions are suggestions for a conversation with the study team or your own clinician, not advice.

  • Some participants receive a placebo. How is that decided, and what happens for placebo-group participants when the study ends?
  • The listed sites are in France. Is any of them reachable for me, and does the study help with travel?
  • Which of the eligibility criteria would need to be checked with tests before I could join?

More on spinocerebellar ataxia

Clinical trials30 trials listed for spinocerebellar ataxiaEvery trial the registry lists for spinocerebellar ataxia, with recruitment status, phase and sites, updated from ClinicalTrials.gov.See all trials →Research updatesWhat changed in spinocerebellar ataxia researchLatest monthly update: September 2026. Includes: NBP trial result. New research summarized each month in plain language, with links to every source paper.Read the latest update →

Important dates

Study start
Oct 21, 2025
Primary completion
May 21, 2027 (estimated)
Study completion
May 21, 2027 (estimated)
Target enrollment
70 participants (estimated)
Last updated on registry
Dec 10, 2025
Data verified
Oct 10, 2026

Sponsor and research team

The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.

Lead sponsor
Assistance Publique - Hôpitaux de Paris

Central study contact

If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.

Participating sites (9)

  • Neurology Department, CHU d'AngersNot Yet Recruiting

    Angers · France

    • Christophe VERNY
  • Genetics Department, CHU de BordeauxRecruiting

    Bordeaux · France

    • Cyril GOIZET
  • Neurology and Gentics Department, CHU de DijonRecruiting

    Dijon · France

    • Quentin THOMAS
  • Neurology Department, Hôpital Pierre Wertheimer HospitalNot Yet Recruiting

    Lyon · France

    • Caroline FROMENT
    • Stéphane THOBOIS
  • Neurology Department, Gui De Chauliac HospitalRecruiting

    Montpellier · France

    • Cecilia MARELLI TOSI
  • Genetics Department, Pitié-Salpêtrière University HospitalRecruiting

    Paris · France

  • Genetics Department, CHU de RouenNot Yet Recruiting

    Rouen · France

    • Gael NICOLAS
  • Neurology Department, CHRU de StrasbourgNot Yet Recruiting

    Strasbourg · France

    • Mathieu ANHEIM
  • Neurology Department, CHU de ToulouseNot Yet Recruiting

    Toulouse · France

    • Fabienne ORY-MAGNE

Terms used on this page · explanations by MedicalUpdates.ai

open-label
No masking is used, so the assigned intervention is not deliberately hidden from participants or study staff.
placebo
A treatment that looks like the study treatment but contains no active ingredient. Some participants receive it instead of the study treatment, usually without knowing which they got.
randomized
Participants are assigned to the study groups by chance, like a coin toss, rather than chosen by the doctor or the participant. This is done so the groups are comparable.
double-blind
Two types of people involved in the study do not know which intervention a participant is receiving. The ClinicalTrials.gov record specifies who is masked, for example participants, care providers, investigators or outcome assessors.
placebo-controlled
A study design in which one group receives a placebo so that the study treatment's effects can be compared with no active treatment.
informed consent
The process of explaining a study, including its risks and what participation involves, so that a person can decide freely whether to take part. Consent is usually documented in writing, and a participant can choose to leave the study later.
creatinine clearance
A measure of kidney function, calculated from a blood creatinine level together with age, weight and sex.
QTcF
A recording of the heart's electrical activity. QTc is one measurement from it; some drugs are avoided if it is prolonged.
investigational drug
A treatment that is being tested and has not been approved for this use. It may be approved for a different condition. Being studied does not mean it works or is available outside the study.
half-life
The time it takes for the amount of a drug in the body to fall by half. 'Five half-lives' is often used as the point at which a previous drug has essentially left the body.
Baseline
Measurements taken at the start of the study, before any study treatment, so that later results can be compared with them.
childbearing potential
Studies often ask participants who could become pregnant, or could father a child, to use contraception during the study and for a period afterwards, because the effect of the treatment on a pregnancy is unknown.
Inclusion Criteria
The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
Exclusion Criteria
The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.

The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.

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