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

Recruiting Phase 3 Interventional Study
Spinocerebellar Ataxia 27B (SCA27B)
Pivotal Trial This treatment is in the last trial phase before FDA approval. Prior Safety Data This treatment has already been tested in at least one earlier human trial.
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At a Glance
Age
18 and older
Sex
Any
Trial phase
Phase 3
Study type
Interventional
Purpose
Treatment
Participants needed
70 (estimated)
Sponsor
Assistance Publique - Hôpitaux de Paris · Other
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About This Trial
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) - Function…
Trial Locations
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Eligibility Criteria
Inclusion 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…
Contacts

Giulia COARELLI

+ 33 (0)1 57 27 46 82

giulia.coarelli@aphp.fr

CONTACT

Alexandra DURR

alexandra.durr@icm-institute.org

CONTACT