Spinocerebellar Ataxia 27B (SCA27B)Phase 3TREAT-FGF14NCT07185347
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
A Phase 3 study testing Fampridine 10 mg prolonged-release tablet (per os) in people with Spinocerebellar Ataxia 27B (SCA27B), run by Assistance Publique - Hôpitaux de Paris at 9 sites in France.
Trial 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 form often presenting paroxysmal episodes of ataxia and/or diplopia. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Spinocerebellar Ataxia 27B (SCA27B)
- What is being tested?
- Fampridine 10 mg prolonged-release tablet (per os)
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 70 participants
- Where
- 9 sitesFrance
- Study dates
- 21 Oct 2025to 21 May 2027
- Phase
- Phase 3
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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.
Show all 17Show fewer
- 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
Trial locations and contacts
9 sites in 1 country. Contact the study team or a site near you to ask about taking part.
Alexandra DURR contact
9 sites in 1 country
France9 sites, 9 recruiting
- Not yet recruiting
Neurology Department, CHU d'Angers
Angers
Christophe VERNY
- Recruiting
Genetics Department, CHU de Bordeaux
Bordeaux
Cyril GOIZET
- Recruiting
Neurology and Gentics Department, CHU de Dijon
Dijon
Quentin THOMAS
- Not yet recruiting
Neurology Department, Hôpital Pierre Wertheimer Hospital
Lyon
Caroline FROMENT
Stéphane THOBOIS
- Recruiting
Neurology Department, Gui De Chauliac Hospital
Montpellier
Cecilia MARELLI TOSI
- Recruiting
Genetics Department, Pitié-Salpêtrière University Hospital
Paris
Giulia COARELLI, + 33 (0)1 57 27 46 82, giulia.coarelli@aphp.fr
- Not yet recruiting
Genetics Department, CHU de Rouen
Rouen
Gael NICOLAS
- Not yet recruiting
Neurology Department, CHRU de Strasbourg
Strasbourg
Mathieu ANHEIM
- Not yet recruiting
Neurology Department, CHU de Toulouse
Toulouse
Fabienne ORY-MAGNE
About this study
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.
- Conditions
- Spinocerebellar Ataxia 27B (SCA27B)
- Being tested
- Fampridine 10 mg prolonged-release tablet (per os), Placebo (tablets per os)
- Keywords
- SCA27B, Fampridine, Ataxia, Diplopia, FARS-Functional Staging
- Medical subject headings
- Ataxia, Diplopia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Fampridine 10 mg prolonged-release tablet (per os)Drug
Patients randomized in the experimental arm will take 1 tablet twice a day, 12 hours apart, on an empty stomach, for 12 weeks.
Placebo (tablets per os)Drug
Patients randomized in the control arm will take 1 tablet twice a day, 12 hours apart, on an empty stomach, for 12 weeks.
| Study group | Role | What they receive |
|---|---|---|
| Fampridine | Experimental | Not described Receives: Drug: Fampridine 10 mg prolonged-release tablet (per os) |
| Placebo | Placebo comparator | Not described Receives: Drug: Placebo (tablets per os) |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: At week 12
Secondary outcomes (30)
Improvement of at least 0.5 point on the Friedreich's Ataxia Rating Scale (FARS) -Functional Staging at week 2
Measured: 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
Measured: 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
Measured: 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
Measured: At week 2 and week 12
Variation in extracerebellar signs assessed by the Inventory of Non-Ataxia Signs (INAS) at week 12
Measured: At week 12
Variation on Timed 25-foot walk (T25FW) at week 2 and week 12
Measured: 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
Measured: At week 2 and week 12
Variation in oculomotor signs assessed by oculomotor recording (OMR) at week 2 and week 12
Measured: 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
Measured: 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
Measured: At week 12
Quality of life evaluated by the Patient Reported Outcome Measure of Ataxia (PROM-ATAXIA) questionnaire at week 12
Measured: At week 12
Quality of life evaluated by the 36-Item Short Form Health Survey (SF-36) questionnaire at week 12
Measured: At week 12
Patient's impression evaluated by the Patient Global Impression of change (PGI-C) at week 2 and week 12
Measured: 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
Measured: 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.
Measured: At week 2 and week 12
Clinical assessments at week 16, i.e. 4 weeks after treatment interruption
Measured: At week 16
Variation in cerebellar syndrome assessed by the FARS-Functional Staging at week 16, i.e. 4 weeks after treatment interruption
Measured: At week 16
Variation in cerebellar syndrome assessed by the SARA scale at week 16, i.e. 4 weeks after treatment interruption
Measured: At week 16
Variation in cerebellar syndrome assessed by the mFARS scale at week 16, i.e. 4 weeks after treatment interruption
Measured: At week 16
Variation in cerebellar syndrome assessed by the CCFS score at week 16, i.e. 4 weeks after treatment interruption
Measured: 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
Measured: At week 16
Variation on Timed 25-foot walk (T25FW) at week 16, i.e. 4 weeks after treatment interruption
Measured: 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
Measured: At week 16
Variation in oculomotor signs assessed by oculomotor recording (OMR) at week 16, i.e. 4 weeks after treatment interruption
Measured: 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
Measured: 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
Measured: At week 16
Quality of life evaluated by the PROM-ATAXIA questionnaire at week 16, i.e. 4 weeks after treatment interruption
Measured: 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
Measured: 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
Measured: 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
Measured: At week 16
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Sep 2025
- Study start21 Oct 2025
- Primary completion21 May 2027 (estimated)
- Study completion21 May 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Spinocerebellar Ataxia 27B (SCA27B)Studies of the same condition, open and under way.
- More trials run by Assistance Publique - Hôpitaux de ParisThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 70 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Assistance Publique - Hôpitaux de Paris
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Giulia COARELLI, Principal investigator, Assistance Publique - Hôpitaux de Paris (AP-HP)
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07185347
- Sponsor's protocol ID
- APHP240921
- Other IDs
- 2024-520413-53-00
- Acronym
- TREAT-FGF14
- Status last verified
- Dec 2025
- First submitted
- 3 Sep 2025
- Last updated
- 10 Dec 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- 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.
Study at a glance
From the registry record, last updated by the sponsor on 10 Dec 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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
- ClinicalTrials.gov ID
- NCT07185347
- Acronym / sponsor ID
- TREAT-FGF14 / APHP240921
- Conditions
- Spinocerebellar Ataxia 27B (SCA27B)
- Keywords
- SCA27B, Fampridine, Ataxia, Diplopia, FARS-Functional Staging
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 70 participants (estimated)
- Lead sponsor
- Assistance Publique - Hôpitaux de ParisUniversity, hospital or other
- Study start
- 21 Oct 2025 (actual)
- Primary completion
- 21 May 2027 (estimated)
- Study completion
- 21 May 2027 (estimated)
- First posted
- 22 Sep 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Fampridine | Experimental | Drug: Fampridine 10 mg prolonged-release tablet (per os) |
| Placebo | Placebo comparator | Drug: Placebo (tablets per os) |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- 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
Key exclusion criteria (17 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Fampridine 10 mg prolonged-release tablet (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. | Drug | Fampridine |
| Placebo (tablets per os)Patients randomized in the control arm will take 1 tablet twice a day, 12 hours apart, on an empty stomach, for 12 weeks. | Drug | Placebo |
Primary outcome
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
30 secondary outcomes
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
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
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 10 Dec 2025.
- Individual participant dataSharing planned: 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.
Related trials for Spinocerebellar Ataxia 27B (SCA27B)
RecruitingObservational study
Spinocerebellar Ataxia Type 27B Natural History Study (SCA27B-NHS)
Spinocerebellar Ataxia Type 27B
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Alexandra DURR contact
Giulia COARELLI principal investigator
Assistance Publique - Hôpitaux de Paris (AP-HP)
9 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Fampridine 10 mg prolonged-release tablet (per os) in people with Spinocerebellar Ataxia 27B (SCA27B), run by Assistance Publique - Hôpitaux de Paris at 9 sites in France.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 17 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 10 Dec 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 9 sites in France. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Giulia COARELLI (+ 33 (0)1 57 27 46 82, giulia.coarelli@aphp.fr), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 10 Dec 2025.