Study To Assess Safety, Tolerability and Efficacy of Intravenous Cabaletta in Patients With Machado-Joseph Disease
Status:
Completed
Trial end date:
2016-11-01
Target enrollment:
Participant gender:
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).