Clinical trials: Spinocerebellar Ataxia 27B (SCA27B)
Spinocerebellar Ataxia 27B (SCA27B)
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
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).
- Trial name
- TREAT-FGF14 (NCT07185347)
- Testing
- Fampridine 10 mg prolonged-release tablet (per os)
- Where
- 9 sites in France
- Volunteers
- 70 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Spinocerebellar Ataxia Type 27B
Spinocerebellar Ataxia Type 27B Natural History Study (SCA27B-NHS)
This international, multi-center, multi-modal, and prospective observational cohort study aims to validate trial outcomes for capturing disease progression in Spinocerebellar Ataxia Type 27B (SCA27B), with combined multi-modal…
- Trial name
- NCT06472557
- Where
- 5 sites in Czechia, Germany and 2 more
- Volunteers
- 300 people, healthy volunteers welcome
- Sponsor
- University Hospital Tuebingen