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