Clinical Trial: Study To Assess Safety, Tolerability and Efficacy of Intravenous Cabaletta in Patients With Machado-Joseph Disease

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

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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

Brief Summary:

• 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).