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

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).

Timeline

Start

2014-07

Primary completion

2016-10

Completion

2016-11

Drugs

Indications