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

NCT02147886 · Status: COMPLETED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 15

Last updated 2016-11-22

No results posted yet for this study

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

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

Sponsors & Collaborators

  • Bioblast Pharma Ltd.

    lead INDUSTRY

Principal Investigators

  • Carlos Gordon, Prof. · Meir Medical Center Kfar Saba Israel

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
TRIPLE
Model
PARALLEL

Eligibility

Min Age
18 Years
Max Age
75 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2014-07-31
Primary Completion
2016-10-31
Completion
2016-11-30

Countries

  • Israel

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT02147886 on ClinicalTrials.gov