This trial has been completed.

Condition machado-joseph disease / spinocerebellar ataxia 3
Treatments cabaletta for iv infusion once weekly during 24 weeks
Phase phase 2
Sponsor Bioblast Pharma Ltd.
Start date July 2014
End date October 2016
Trial size 15 participants
Trial identifier NCT02147886, BB-MJD-201


- 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).

United States No locations recruiting
Other countries No locations recruiting

Study Design

Allocation randomized
Endpoint classification safety/efficacy study
Intervention model parallel assignment
Masking double blind (subject, investigator, outcomes assessor)
Primary purpose treatment
Cabaletta 15gr
cabaletta for iv infusion once weekly during 24 weeks
Cabaletta for IV infusion once weekly
Cabaletta 30gr
cabaletta for iv infusion once weekly during 24 weeks

Primary Outcomes

Adverse events
time frame: 28 weeks
Physical examination
time frame: 28 weeks
Vital signs
time frame: 28weeks
12-lead ECG
time frame: 28weeks
Safety laboratory tests
time frame: 28weeks

Secondary Outcomes

Disease markers
time frame: 27 weeks

Eligibility Criteria

Male or female participants from 18 years up to 75 years old.

Inclusion Criteria: 1. Men and women, 18 - 75 years 2. Clinically diagnosed as Machado-Joseph disease/Spinocerebellar ataxia 3 confirmed by genetic testing 3. With disease stage 2 or less 4. Stable doses of all medications for 30 days prior to study entry and for the duration of the study. 5. Body Mass Index (BMI) ≤32 kg/m2. 6. Ability to ambulate with or without assistance Exclusion Criteria: 1. Diabetes mellitus type 1 or 2 2. Other major diseases 3. Uncontrolled heart disease, chronic heart failure (CHF). 4. Other neurological diseases. 5. Ataxia derived from any other cause than genetically-confirmed spinocerebellar ataxia 6. Presence of psychosis, bipolar disorder, untreated depression 7. History of malignancy (except non-invasive skin malignancy).

Additional Information

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 Machado-Joseph Disease
Principal investigator Carlos Gordon, Prof.
Trial information was received from ClinicalTrials.gov and was last updated in November 2016.
Information provided to ClinicalTrials.gov by Bioblast Pharma Ltd..