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Tests treatment safety and results for Spinocerebellar Ataxia 27B (SCA27B)
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
What this study is testing
What is Fampridine 10 mg prolonged-release tablet (per os)?
Fampridine 10 mg prolonged-release tablet (per os) is an investigational medicine, given as a twice-daily pill taken by mouth, being studied as a potential treatment for spinocerebellar ataxia 27b (sca27b).
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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.
- Phase 3: a large, late-stage study
- You might receive a placebo (an inactive treatment) instead of the study drug, decided by chance. You may not know which one you got.
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 18 and older
You may be able to join if
- 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
You likely can't join if
- 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 \ 480 msec in 3 consecutive...
- Unstable, clinically significant neurologic (other than the disease being studied; eg, recurrent strokes), psychiatric, cardiovascular (eg, pulmonary...
- Patients with known recurrent, active, or chronic infections.
- Patients with prior history of seizure.
See the full eligibility 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
- 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 \ 480 msec in 3 consecutive ECG recordings taken at least 5 minutes apart, uncompensated...
- Unstable, clinically significant neurologic (other than the disease being studied; eg, recurrent strokes), psychiatric, cardiovascular (eg, pulmonary arterial hypertension, cardiac valvulopathy, orthostatic...
- 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...
- 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...
- 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
The study team makes the final eligibility decision.
Where it's taking place
- Angers, France
- Bordeaux, France
- Dijon, France
- Lyon, France
- Montpellier, France
- Paris, France
- Rouen, France
- Strasbourg, France
- Toulouse, France
Compensation & support
A stipend or compensation may be offered.
Compensation mentioned.
ClinicalTrials.gov doesn't provide a reliable structured field for payment or travel support - confirm details with the study team.
Questions & answers
Do participants get paid in this trial?
This study's listing includes signals that participants may be compensated or receive a stipend. Amounts vary and are set by the study team - confirm the details with them.
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, 18 years and older. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Angers, France; Bordeaux, France; Dijon, France; Lyon, France; Montpellier, France; Paris, France and 3 more location(s). Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.