Safety and Tolerability of Lithium in Spinocerebellar Ataxia 2 (SCA2)
Information source: Federico II University
Information obtained from ClinicalTrials.gov on October 04, 2010
Link to the current ClinicalTrials.gov record.
Condition(s) targeted: Spinocerebellar Ataxia 2
Intervention: LITHIUM CARBONATE (Drug)
Phase: Phase 2
Status: Recruiting
Sponsored by: Federico II University Official(s) and/or principal investigator(s):
Alessandro Filla, MD, Principal Investigator, Affiliation: University Federico II
Overall contact:
Francesco Saccà, MD, Phone: 0817462671, Ext: +39, Email: francesco.sacca@unina.it
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.
Clinical Details
Official title: Randomized, Placebo-controlled Trial to Test Safety, Tolerability and Efficacy of Lithium Carbonate in Spinocerebellar Ataxia 2
Study design: Allocation: Randomized, Control: Placebo Control, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Primary Purpose: Treatment
Primary outcome: 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.
Secondary outcome: 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.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. The effect of Lithium on mood will be explored with the Beck depression inventory. Effect of Lithium on quality of life will be assessed with the EQ-5D scale. 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. 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. 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. The effect of Lithium on mood will be explored with the Beck depression inventory. The effect of Lithium on mood will be explored with the Beck depression inventory. Effect of Lithium on quality of life will be assessed with the EQ-5D scale. Effect of Lithium on quality of life will be assessed with the EQ-5D scale.
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
Minimum age: 18 Years.
Maximum age: 80 Years.
Gender(s): Both.
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
Locations and Contacts
Francesco Saccà, MD, Phone: 0817462671, Ext: +39, Email: francesco.sacca@unina.it
Dipartimento di Scienze Neurologiche, Napoli 80131, Italy; Recruiting
Francesco Saccà, Phone: 081 7462671, Email: francesco.sacca@unina.it
Francesco Saccà, MD, Sub-Investigator
Additional Information
University Federico II AOU Policlinico "Federico II" AISA sez. Campania
Starting date: October 2009
Last updated: April 12, 2010
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