Machado-Joseph Disease / Spinocerebellar Ataxia 3
Conditions
Keywords
Bioblast, Randomized, parallel-group, and dose-controlled
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).
Interventions
DRUGCabaletta for IV infusion once weekly during 24 weeks
Cabaletta for IV infusion once weekly
Sponsors
Bioblast Pharma Ltd.
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
TRIPLE (Subject, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
18 Years to 75 Years
Healthy volunteers
No
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).
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Physical examination | 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 |
| 12-lead ECG | 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 | 28weeks | Safety will be evaluated on the basis of the following assessments: Adverse events , physical examination, 12-lead ECG, vital signs, safety laboratory evaluations |
| Adverse events | 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 | 28weeks | Safety will be evaluated on the basis of the following assessments: Adverse events , physical examination, 12-lead ECG, vital signs, safety laboratory evaluations |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Disease markers | 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 |
Other
| Measure | Time frame | Description |
|---|---|---|
| Biochemical marker | 27 weeks | Assessment of disease biochemical marker neuron specific enolase (NSE) and protein S 100 B (S100B) |
Countries
Israel
Outcome results
None listed