Machado-Joseph Disease / Spinocerebellar Ataxia 3 Clinical Trial
Official title:
A Single-Center, Randomized, Double-Blind, Parallel-Group, Dose-Controlled Study, to Assess Safety, Tolerability and Efficacy of Intravenous Cabaletta® in Patients With Machado-Joseph Disease
- This is an exploratory, randomized, parallel-group, dose escalation and dose-controlled
study without a placebo arm.
- Eligible patients will be randomized in a 1:1 ratio (double-blind) to receive Cabaletta
in 2 doses, once weekly for 22 weeks (total of 24 weeks of treatment).
n/a
Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Investigator, Outcomes Assessor), Primary Purpose: Treatment