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| ID | Type | Description | Link |
|---|---|---|---|
| EUDRACT N°2009-016317-20 |
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The purpose of this study is to determine safety and tolerability of the treatment with lithium in Spinocerebellar Ataxia 2. Moreover, clinical symptoms, neuronal loss, quality of life and depressive symptoms, will be considered to further investigate the effect of lithium therapy.
Patients will be progressively enrolled in the study and undergo a screening visit to test for inclusion/exclusion criteria. Patients will then be randomized to receive either Lithium carbonate or placebo. Patients will visit study center at 2, 4, 8, 12, 24, 36 and 48 weeks, for endpoint and laboratory assessments.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| LITHIUM CARBONATE 150 and/or 300 mg | Experimental |
| |
| PLACEBO | Placebo Comparator |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| LITHIUM CARBONATE | Drug | Lithium Carbonate will be dosed based on lithiemy, which will be in the range 0.9-1.2 mEq/L. Maximum allowed dose will be 1500mg/day. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Primary endpoint of the study will be the difference in the number and frequency of Severe Adverse Events (SAE) and Non Severe Adverse Events (nSAE) recorded during the study, between treatment and placebo group. | Adverse events and Severe Adverse events will be recorded during the trial at each visit starting from Baseline to Visit 8 at 48 weeks of treatment. | the endpoint will be recorded at all visits |
| Measure | Description | Time Frame |
|---|---|---|
| Secondary outcome will be the Scale for the Assessment and Rating of Ataxia (SARA). Statistical analysis will be performed to compare the effect of treatment on both groups. | 0 weeks | |
| Micro- and macrostructural Magnetic Resonance parameters will be compared before and after treatment. This will include Voxel Based Morphometry, resting functional MRI, Diffusion tensor imaging and MRI spectroscopy. |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Alessandro Filla, MD | University Federico II | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Dipartimento di Scienze Neurologiche | Naples | 80131 | Italy |
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| Label | URL |
|---|---|
| University Federico II | View source |
| AOU Policlinico "Federico II" | View source |
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| ID | Term |
|---|---|
| D020754 | Spinocerebellar Ataxias |
| ID | Term |
|---|---|
| D002524 | Cerebellar Ataxia |
| D002526 | Cerebellar Diseases |
| D001927 | Brain Diseases |
| D002493 | Central Nervous System Diseases |
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| ID | Term |
|---|---|
| D016651 | Lithium Carbonate |
| ID | Term |
|---|---|
| D002254 | Carbonates |
| D000468 | Alkalies |
| D007287 | Inorganic Chemicals |
| D002255 | Carbonic Acid |
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| 0 weeks |
| The effect of Lithium on mood will be explored with the Beck depression inventory. | 0 weeks |
| Effect of Lithium on quality of life will be assessed with the EQ-5D scale. | 0 weeks |
| Secondary outcome will be the Scale for the Assessment and Rating of Ataxia (SARA). Statistical analysis will be performed to compare the effect of treatment on both groups. | 24 weeks |
| Secondary outcome will be the Scale for the Assessment and Rating of Ataxia (SARA). Statistical analysis will be performed to compare the effect of treatment on both groups. | 48 weeks |
| Micro- and macrostructural Magnetic Resonance parameters will be compared before and after treatment. This will include Voxel Based Morphometry, resting functional MRI, Diffusion tensor imaging and MRI spectroscopy. | 48 weeks |
| The effect of Lithium on mood will be explored with the Beck depression inventory. | 24 weeks |
| The effect of Lithium on mood will be explored with the Beck depression inventory. | 48 weeks |
| Effect of Lithium on quality of life will be assessed with the EQ-5D scale. | 24 weeks |
| Effect of Lithium on quality of life will be assessed with the EQ-5D scale. | 48 weeks |
| 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 |
| D017554 |
| Carbon Compounds, Inorganic |
| D018020 | Lithium Compounds |