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

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 Mach

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).

Detailed Summary:
Sponsor: Bioblast Pharma Ltd.

Current Primary Outcome:

  • Adverse events [ Time Frame: 28 weeks ]

    Safety will be evaluated on the basis of the following assessments:

    Adverse events , physical examination, 12-lead ECG, vital signs, safety laboratory evaluations

  • Physical examination [ Time Frame: 28 weeks ]

    Safety will be evaluated on the basis of the following assessments:

    Adverse events , physical examination, 12-lead ECG, vital signs, safety laboratory evaluations

  • Vital signs [ Time Frame: 28weeks ]

    Safety will be evaluated on the basis of the following assessments:

    Adverse events , physical examination, 12-lead ECG, vital signs, safety laboratory evaluations

  • 12-lead ECG [ Time Frame: 28weeks ]

    Safety will be evaluated on the basis of the following assessments:

    Adverse events , physical examination, 12-lead ECG, vital signs, safety laboratory evaluations

  • Safety laboratory tests [ Time Frame: 28weeks ]

    Safety will be evaluated on the basis of the following assessments:

    Adverse events , physica

    Original Primary Outcome: Same as current

    Current Secondary Outcome: Disease markers [ Time Frame: 27 weeks ]

    Changes in disease markers will be assessed based on the following assessments:

    Scale for the Assessment and Rating of Ataxia (SARA); Neurological Examination Score for Spinocerebellar Ataxia (NESSCA); Change in BMI - screening, spinocerebellar Ataxia Functional Tests; quality of life



    Original Secondary Outcome: Same as current

    Information By: Bioblast Pharma Ltd.

    Dates:
    Date Received: May 21, 2014
    Date Started: July 2014
    Date Completion:
    Last Updated: November 21, 2016
    Last Verified: November 2016