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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Cabaletta 15gr | Experimental | Cabaletta 15gr |
|
| Cabaletta 30gr | Experimental | Cabaletta 30gr |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Cabaletta for IV infusion once weekly during 24 weeks | Drug | Cabaletta for IV infusion once weekly |
|
| Measure | Description | Time Frame |
|---|---|---|
| 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 |
| 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 |
| 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 |
| 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 |
| 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 |
| Measure | Description | Time Frame |
|---|---|---|
| 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 |
| Measure | Description | Time Frame |
|---|---|---|
| Biochemical marker | Assessment of disease biochemical marker neuron specific enolase (NSE) and protein S 100 B (S100B) | 27 weeks |
Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Carlos Gordon, Prof. | Meir Medical Center Kfar Saba Israel | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Meir Medical Center | Kfar Saba | Israel |
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| Cabaletta for IV infusion once weekly during 24 weeks | Drug |
|
| ID | Term |
|---|---|
| D017827 | Machado-Joseph Disease |
| ID | Term |
|---|---|
| D020754 | Spinocerebellar Ataxias |
| D002524 | Cerebellar Ataxia |
| D002526 | Cerebellar Diseases |
| D001927 | Brain Diseases |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
| D013132 | Spinocerebellar Degenerations |
| D013118 | Spinal Cord Diseases |
| D020271 | Heredodegenerative Disorders, Nervous System |
| D019636 | Neurodegenerative Diseases |
| D001259 | Ataxia |
| D020820 | Dyskinesias |
| D009461 | Neurologic Manifestations |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
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