Clinical trial NCT00998634: Safety and Tolerability of Lithium in Spinocerebellar Ataxia 2 (SCA2)
Official title: Randomized, Placebo-controlled Trial to Test Safety, Tolerability and Efficacy of Lithium Carbonate in Spinocerebellar Ataxia 2
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: SPINOCEREBELLAR ATAXIA 2
Keywords: SCA2
Lithium
Lead sponsor: Federico II University
Sponsor class: OTHER
First posted: 2009-10-20
Enrollment: 20
Sex: ALL
Minimum age (years): 18
Maximum age (years): 80
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: 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.

Detailed description: 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.

Eligibility criteria: 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

Primary outcomes:
measure: 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.
timeFrame: the endpoint will be recorded at all visits
Secondary outcomes:
measure: 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.
timeFrame: 0 weeks
measure: 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.
timeFrame: 0 weeks
measure: The effect of Lithium on mood will be explored with the Beck depression inventory.
timeFrame: 0 weeks
measure: Effect of Lithium on quality of life will be assessed with the EQ-5D scale.
timeFrame: 0 weeks
measure: 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.
timeFrame: 24 weeks
measure: 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.
timeFrame: 48 weeks
measure: 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.
timeFrame: 48 weeks
measure: The effect of Lithium on mood will be explored with the Beck depression inventory.
timeFrame: 24 weeks
measure: The effect of Lithium on mood will be explored with the Beck depression inventory.
timeFrame: 48 weeks
measure: Effect of Lithium on quality of life will be assessed with the EQ-5D scale.
timeFrame: 24 weeks
measure: Effect of Lithium on quality of life will be assessed with the EQ-5D scale.
timeFrame: 48 weeks
Other outcomes:
Locations:
city: Napoli
country: Italy
facility: Dipartimento di Scienze Neurologiche
Study officials:
affiliation: University Federico II
name: Alessandro Filla, MD
role: PRINCIPAL_INVESTIGATOR
References: