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Study of fampridine treatment for patients with spinocerebellar ataxia SCA27B caused by FGF14 gene mutation

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What is this trial about?

A plain-language summary of the goals, design and what participants do

This study focuses on patients with Spinocerebellar Ataxia, specifically type SCA27B, which is a rare genetic condition affecting movement and balance due to problems in a part of the brain called the cerebellum. The study will test a medication called Fampridine, given as prolonged-release tablets, to see if it can help improve symptoms in people with this condition.

The main purpose of this research is to determine if taking Fampridine tablets twice daily for 12 weeks helps patients with SCA27B. During the study, some patients will receive Fampridine while others will receive placebo. The medication or placebo will be taken by mouth, with a maximum daily dose of 20 mg of Fampridine for those in the treatment group.

The study will last 16 weeks in total, including a 12-week treatment period followed by 4 weeks of observation after stopping the medication. Throughout the study, doctors will monitor various aspects of the participants' movement abilities, balance, daily activities, and overall well-being. They will also check for any potential side effects through regular medical examinations and blood tests.

The research process

The trial runs in 4 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Initial treatment start

    You will receive either fampridine 10 mg or a placebo (inactive substance) twice a day

    The medication should be taken orally (by mouth)

    You will not know whether you are receiving the actual medication or placebo

  2. Step 2

    First evaluation (Week 2)

    Your walking ability will be assessed

    Eye movement tests will be performed

    Your overall condition will be evaluated by your doctor

    Blood tests and heart examination (ECG) will be conducted

    You will be asked about any side effects you may have experienced

  3. Step 3

    Main evaluation (Week 12)

    A comprehensive assessment of your condition will be performed

    Your daily living activities will be evaluated

    Quality of life questionnaires will be completed

    Blood tests and heart examination (ECG) will be repeated

    Your doctor will evaluate your overall progress

  4. Step 4

    Final follow-up (Week 16)

    This evaluation occurs 4 weeks after stopping the treatment

    Your neurological condition will be assessed

    Quality of life assessment will be performed

    This marks the end of your participation in the study

Who can join the trial?

9 criteria

  • You must have a confirmed genetic diagnosis of cerebellar ataxia SCA27B with at least 250 GAA repeats in the FGF14 gene (a specific genetic marker that confirms the condition)
  • You must be at least 18 years old
  • You must have a SARA total score above 3 and at least a score of 1 for walking ability on the SARA scale (SARA is a scale that measures movement and coordination difficulties)
  • You must be able and willing to sign an informed consent form to participate in the study
  • You must have active social security coverage
  • You must be physically capable of:
    • Completing all study activities
    • Taking medication by mouth
    • Following the study schedule for the entire duration
    • Both men and women can participate in this study

Who cannot join the trial?

12 criteria

  • Age below 18 years or above 65 years
  • Pregnancy or breastfeeding women
  • History of seizures or epilepsy (conditions causing uncontrolled electrical activity in the brain)
  • Moderate to severe kidney disease (problems with kidney function)
  • Current treatment with medications that may interact with fampridine
  • Participation in another clinical trial within the past 30 days
  • Known allergic reactions to fampridine or similar medications
  • Significant heart problems or uncontrolled high blood pressure
  • Severe mental health conditions that could affect participation
  • Unable to follow study procedures or attend scheduled visits
  • History of drug or alcohol abuse within the past year
  • Any medical condition that the study doctor considers unsafe for participation
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Investigated drugs

Fampridine is a medication that helps improve walking and movement in people with certain neurological conditions. It works by helping nerve signals travel better through damaged nerve fibers. In this trial, it is being studied to see if it can help patients with a specific type of spinocerebellar ataxia called SCA27B, which affects balance and coordination. The medication is taken twice daily and aims to improve the symptoms of this rare genetic condition.

What is already known about the treatment

Fampridine - A potassium channel blocker medication administered orally in tablet form twice daily, specifically studied for neurological conditions. This drug works by improving signal conduction in nerve fibers where myelin is damaged, helping to enhance nerve impulse transmission in the central nervous system. Currently approved for improving walking ability in multiple sclerosis patients, it is being investigated for potential benefits in spinocerebellar ataxia, particularly SCA27B type, where it may help with movement coordination and balance. The medication belongs to the class of potassium channel antagonists and acts by blocking potassium channels in nerve fibers, which helps strengthen the electrical signals traveling through neurons.

Investigated diseases

Spinocerebellar ataxia - A rare genetic disorder that affects the nervous system, particularly the cerebellum and spinal cord. It causes progressive problems with movement, balance, and coordination. People with this condition experience increasing difficulty with walking, speech, and fine motor tasks. The condition can also affect eye movements and cause involuntary movements. The symptoms typically develop gradually over time and can vary in severity among different individuals.
Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase IIITrial ID2024-520413-53-00Protocol codeAPHP240921Estimated enrolment70 patientsSponsorAssistance Publique Hopitaux De Paris

sourced from the EU Clinical Trials Register and site verification

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