SPINOCEREBELLAR ATAXIA 2 Clinical Trial
— LISCA2Official title:
Randomized, Placebo-controlled Trial to Test Safety, Tolerability and Efficacy of Lithium Carbonate in Spinocerebellar Ataxia 2
| Verified date | January 2013 |
| Source | Federico II University |
| Contact | n/a |
| Is FDA regulated | No |
| Health authority | Italy: Ethics Committee |
| Study type | Interventional |
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.
| Status | Completed |
| Enrollment | 20 |
| Est. completion date | November 2012 |
| Est. primary completion date | November 2012 |
| Accepts healthy volunteers | No |
| Gender | Both |
| Age group | 18 Years to 80 Years |
| Eligibility |
Inclusion Criteria: - Molecular diagnosis of SCA2 (=34 CAG in the ataxin-2 gene) - Age =18, <80 - SARA score =8 Exclusion Criteria: - SARA score >32 - Heart failure - Liver disease - Kidney failure - Thyroid disease - Sick sinus syndrome and/or significant ECG alterations - Hyposodemia - Treatment with diuretics - Treatment with haloperidol and/or other antipsychotics - Treatment with NSAIDs or corticosteroids - Treatment with ACE inhibitors - Treatment with aminophyllines - Treatment with mannitol - Pregnancy and/or breastfeeding - Acute diseases that might interfere with the trial |
Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Primary Purpose: Treatment
| Country | Name | City | State |
|---|---|---|---|
| Italy | Dipartimento di Scienze Neurologiche | Napoli |
| Lead Sponsor | Collaborator |
|---|---|
| Federico II University |
Italy,
| Type | Measure | Description | Time frame | Safety issue |
|---|---|---|---|---|
| Primary | 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 | Yes |
| Secondary | 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 | No | |
| Secondary | 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. | 0 weeks | No | |
| Secondary | The effect of Lithium on mood will be explored with the Beck depression inventory. | 0 weeks | No | |
| Secondary | Effect of Lithium on quality of life will be assessed with the EQ-5D scale. | 0 weeks | No | |
| Secondary | 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 | No | |
| Secondary | 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 | No | |
| Secondary | 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 | No | |
| Secondary | The effect of Lithium on mood will be explored with the Beck depression inventory. | 24 weeks | No | |
| Secondary | The effect of Lithium on mood will be explored with the Beck depression inventory. | 48 weeks | No | |
| Secondary | Effect of Lithium on quality of life will be assessed with the EQ-5D scale. | 24 weeks | No | |
| Secondary | Effect of Lithium on quality of life will be assessed with the EQ-5D scale. | 48 weeks | No |