A Phase 3 interventional study of Fampridine 10 mg prolonged-release tablet (per os) and Placebo (tablets per os) in Spinocerebellar Ataxia 27B (SCA27B), sponsored by Assistance Publique - Hôpitaux de Paris. Recruiting at 9 sites in France. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2025-12-10.
Sponsored by Assistance Publique - Hôpitaux de Paris · Phase 3, Interventional, and Treatment
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.
149 studies on the registry are indexed under Ataxia; 37 are open to participants now.
This study's planned enrollment of 70 is above the median of 30 across 100 interventional studies indexed under Ataxia.
Browse Ataxia studies →Assistance Publique - Hôpitaux de Paris is the lead sponsor of 3,592 studies on the registry; 1,033 are open to participants now.
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Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
The other ingredients in a medicine besides the active drug, such as fillers, colours or preservatives.
How quickly the kidneys clear creatinine, a waste product, from the blood. A study may estimate it from a blood test with age, weight and sex (often by the Cockcroft–Gault formula), or measure it using blood and urine collected over a set time. Given in mL/min; higher is better.
On an ECG, the QT interval is the time the heart’s lower chambers take to activate electrically and recover in each beat. QTc is that time adjusted for heart rate (QTcF by Fridericia’s formula, QTcB by Bazett’s). Some medicines lengthen it, and a long QTc can raise the risk of a dangerous heart rhythm.
The study may name a correction formula and a limit in milliseconds, often around 450–480.
A recording of the heart’s electrical activity, taken with stickers on the chest, arms and legs. It takes a few minutes and does not hurt.
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hypersensitivity
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Excipients
The other ingredients in a medicine besides the active drug, such as fillers, colours or preservatives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Creatinine clearance (CrCl)
How quickly the kidneys clear creatinine, a waste product, from the blood. A study may estimate it from a blood test with age, weight and sex (often by the Cockcroft–Gault formula), or measure it using blood and urine collected over a set time. Given in mL/min; higher is better.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
QT and QTc
On an ECG, the QT interval is the time the heart’s lower chambers take to activate electrically and recover in each beat. QTc is that time adjusted for heart rate (QTcF by Fridericia’s formula, QTcB by Bazett’s). Some medicines lengthen it, and a long QTc can raise the risk of a dangerous heart rhythm.
The study may name a correction formula and a limit in milliseconds, often around 450–480.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ECG (electrocardiogram)
A recording of the heart’s electrical activity, taken with stickers on the chest, arms and legs. It takes a few minutes and does not hurt.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Clinically significant
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Childbearing potential
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Drug: Fampridine 10 mg prolonged-release tablet (per os)
Drug: Placebo (tablets per os)
Patients randomized in the experimental arm will take 1 tablet twice a day, 12 hours apart, on an empty stomach, for 12 weeks.
Patients randomized in the control arm will take 1 tablet twice a day, 12 hours apart, on an empty stomach, for 12 weeks.
Proportion of patients showing an improvement of at least 0.5 point on the Friedreich's Ataxia Rating Scale (FARS) -Functional Staging at week 12
Time frame: At week 12
Improvement of at least 0.5 point on the Friedreich's Ataxia Rating Scale (FARS) -Functional Staging at week 2
Time frame: At week 2
Variation in cerebellar syndrome assessed by the Scale for the assessment and rating of ataxia (SARA) at week 2 and week 12
Time frame: At week 2 and week 12
Variation in cerebellar syndrome assessed by the modified Friedreich Ataxia Rating Scale (mFARS) at week 2 and week 12
Time frame: At week 2 and week 12
Variation in cerebellar syndrome assessed by the Composite Cerebellar Functional Severity Score (CCFS score) at week 2 and week 12
Time frame: At week 2 and week 12
Variation in extracerebellar signs assessed by the Inventory of Non-Ataxia Signs (INAS) at week 12
Time frame: At week 12
Variation on Timed 25-foot walk (T25FW) at week 2 and week 12
Time frame: At week 2 and week 12
Variation in oculomotor signs assessed by the Scale for Ocular motor Disorders in Ataxia (SODA) at week 2 and week 12
Time frame: At week 2 and week 12
Variation in oculomotor signs assessed by oculomotor recording (OMR) at week 2 and week 12
Time frame: At week 2 and week 12
Variation in daily frequency of diplopia assessed on a Numerical Diplopia Rating Scale (NDRS) at week 2 and week 12
Time frame: At week 2 and week 12
Variation in daily living activities evaluated by the FARS -Activities of Daily Living scale (FARS-ADL) at week 12
Time frame: At week 12
Quality of life evaluated by the Patient Reported Outcome Measure of Ataxia (PROM-ATAXIA) questionnaire at week 12
Time frame: At week 12
Quality of life evaluated by the 36-Item Short Form Health Survey (SF-36) questionnaire at week 12
Time frame: At week 12
Patient's impression evaluated by the Patient Global Impression of change (PGI-C) at week 2 and week 12
Time frame: At week 2 and week 12
Clinician's impression evaluated by the Clinician Global Impression of Change (CGI-C) at week 2 and week 12
Time frame: At week 2 and week 12
Tolerance to the investigational drug
Tolerance will be evaluated through clinical examination, blood analysis, and electrocardiogram (ECG) performed at Week 2 and Week 12. In addition, adverse events will be systematically recorded throughout the study period.
Time frame: At week 2 and week 12
Clinical assessments at week 16, i.e. 4 weeks after treatment interruption
Time frame: At week 16
Variation in cerebellar syndrome assessed by the FARS-Functional Staging at week 16, i.e. 4 weeks after treatment interruption
Time frame: At week 16
Variation in cerebellar syndrome assessed by the SARA scale at week 16, i.e. 4 weeks after treatment interruption
Time frame: At week 16
Variation in cerebellar syndrome assessed by the mFARS scale at week 16, i.e. 4 weeks after treatment interruption
Time frame: At week 16
Variation in cerebellar syndrome assessed by the CCFS score at week 16, i.e. 4 weeks after treatment interruption
Time frame: At week 16
Variation in extracerebellar signs assessed by the Inventory of Non-Ataxia Signs (INAS) at week 16, i.e. 4 weeks after treatment interruption
Time frame: At week 16
Variation on Timed 25-foot walk (T25FW) at week 16, i.e. 4 weeks after treatment interruption
Time frame: At week 16
Variation in oculomotor signs assessed by the Scale for Ocular motor Disorders in Ataxia (SODA) at week 16, i.e. 4 weeks after treatment interruption
Time frame: At week 16
Variation in oculomotor signs assessed by oculomotor recording (OMR) at week 16, i.e. 4 weeks after treatment interruption
Time frame: At week 16
Variation in daily frequency of diplopia assessed on a Numerical Diplopia Rating Scale (NDRS) at week 16, i.e. 4 weeks after treatment interruption
Time frame: At week 16
Variation in daily living activities evaluated by the FARS -Activities of Daily Living scale at week 16, i.e. 4 weeks after treatment interruption
Time frame: At week 16
Quality of life evaluated by the PROM-ATAXIA questionnaire at week 16, i.e. 4 weeks after treatment interruption
Time frame: At week 16
Quality of life evaluated by the 36-Item Short Form Health Survey (SF-36) questionnaire at week 16, i.e. 4 weeks after treatment interruption
Time frame: At week 16
Patient's impression evaluated by the Patient Global Impression of change (PGI-C) at week 16, i.e. 4 weeks after treatment interruption
Time frame: At week 16
Clinician's impression evaluated by the Clinician Global Impression of Change (CGI-C) at week 16, i.e. 4 weeks after treatment interruption
Time frame: At week 16
Plan to share: Yes — Data are available upon reasonable request The procedures carried out with the French data privacy authority (CNIL, Commission nationale de l'informatique et des libertés) do not provide for the transmission of the database, nor do the information and consent documents signed by the patients. Consultation by the editorial board or interested researchers of individual participant data that underlie the results reported in the article after deidentification may nevertheless be considered, subject to prior determination of the terms and conditions of such consultation and in respect for compliance with the applicable regulations.
Supporting information: Study protocol, Statistical analysis plan, Informed consent form
Time frame: Beginning 3 months and ending 3 years following article publication. Requests out of these time frame can also be submitted to the sponsor
Access criteria: Researchers who provide a methodologically sound proposal.
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Studies of the drug tested here: 4-Aminopyridine
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