NCT02147886

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

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

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 15 (actual)
• Sponsor: Bioblast Pharma Ltd. · Industry
• Sex: All
• Age: 18 Years – 75 Years
• Healthy volunteers: Not accepted

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).

Conditions

• Machado-Joseph Disease / Spinocerebellar Ataxia 3

Interventions

Type	Name	Description
DRUG	Cabaletta for IV infusion once weekly during 24 weeks	Cabaletta for IV infusion once weekly
DRUG	Cabaletta for IV infusion once weekly during 24 weeks

Timeline

• Start date: 2014-07-01
• Primary completion: 2016-10-01
• Completion: 2016-11-01
• First posted: 2014-05-28
• Last updated: 2016-11-22

Locations

1 site across 1 country: Israel