Machado-Joseph Disease / Spinocerebellar Ataxia 3 Clinical Trial
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
| Verified date | November 2016 |
| Source | Bioblast Pharma Ltd. |
| Contact | n/a |
| Is FDA regulated | No |
| Health authority | Israel: Ministry of Health |
| Study type | Interventional |
- 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).
| Status | Completed |
| Enrollment | 15 |
| Est. completion date | November 2016 |
| Est. primary completion date | October 2016 |
| Accepts healthy volunteers | No |
| Gender | Both |
| Age group | 18 Years to 75 Years |
| Eligibility |
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). |
Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Investigator, Outcomes Assessor), Primary Purpose: Treatment
| Country | Name | City | State |
|---|---|---|---|
| Israel | Meir Medical Center | Kfar Saba |
| Lead Sponsor | Collaborator |
|---|---|
| Bioblast Pharma Ltd. |
Israel,
| Type | Measure | Description | Time frame | Safety issue |
|---|---|---|---|---|
| Other | Biochemical marker | Assessment of disease biochemical marker neuron specific enolase (NSE) and protein S 100 B (S100B) | 27 weeks | No |
| Primary | Adverse events | Safety will be evaluated on the basis of the following assessments: Adverse events , physical examination, 12-lead ECG, vital signs, safety laboratory evaluations |
28 weeks | Yes |
| Primary | Physical examination | Safety will be evaluated on the basis of the following assessments: Adverse events , physical examination, 12-lead ECG, vital signs, safety laboratory evaluations |
28 weeks | Yes |
| Primary | Vital signs | Safety will be evaluated on the basis of the following assessments: Adverse events , physical examination, 12-lead ECG, vital signs, safety laboratory evaluations |
28weeks | Yes |
| Primary | 12-lead ECG | Safety will be evaluated on the basis of the following assessments: Adverse events , physical examination, 12-lead ECG, vital signs, safety laboratory evaluations |
28weeks | Yes |
| Primary | Safety laboratory tests | Safety will be evaluated on the basis of the following assessments: Adverse events , physical examination, 12-lead ECG, vital signs, safety laboratory evaluations |
28weeks | Yes |
| Secondary | Disease markers | 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 |
27 weeks | No |