BridgeMD
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.(TREAT-FGF14)
EU CTIS ID: 2024-520413-53-00
What this study is testing
The main objective of this randomized, double-blind, placebo-controlled clinical trial is to demonstrate the efficacy of a 12-week treatment with fampridine 10 mg bid in SCA27B patients.
- Therapeutic confirmatory (Phase III)
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- Genetic diagnosis of cerebellar 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.
- Signature of informed consent
- Covered by social security
- Physically able and expected to complete the trial as designed and having the ability to take oral medication
You likely can't join if
- Hypersensitivity to fampridine
- Previous treatment with fampridine
- 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)
- Hypersensitivity to any excipients present in fampridine
- 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.
See the full eligibility criteria
Who can join
- Genetic diagnosis of cerebellar 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.
- Signature of informed consent
- Covered by social security
- Physically able and expected to complete the trial as designed and having the ability to take oral medication
What rules you out
- Hypersensitivity to fampridine
- Previous treatment with fampridine
- 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)
- Hypersensitivity to any excipients present in fampridine
- 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 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.
- Legally incapacitated adults (e.g., individuals under legal protection such as guardianship or curatorship)
- Inability to understand information about the protocol
- Persons deprived of their liberty by judicial decision
- Other ataxic syndromes than SCA27B
- 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)
- 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
The study team makes the final eligibility decision.
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 65+ years, 18-64 years. The study team makes the final eligibility decision.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.