Safety and Tolerability of Lithium in Spinocerebellar Ataxia 2 (SCA2) (LISCA2)

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Alessandro Filla, Federico II University
ClinicalTrials.gov Identifier:
NCT00998634
First received: October 16, 2009
Last updated: January 7, 2013
Last verified: January 2013

October 16, 2009
January 7, 2013
October 2009
November 2012   (final data collection date for primary outcome 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. [ Time Frame: the endpoint will be recorded at all visits ] [ Designated as safety issue: Yes ]
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.
Primary endpoint of the study will be the difference in Severe Adverse Events (SAE) and Non Severe Adverse Events (nSAE) recorded during the study, between treatment and placebo group. [ Time Frame: 0, 2, 4, 8, 12, 24, 36 and 48 weeks ] [ Designated as safety issue: Yes ]
Complete list of historical versions of study NCT00998634 on ClinicalTrials.gov Archive Site
  • 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. [ Time Frame: 0 weeks ] [ Designated as safety issue: No ]
  • 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. [ Time Frame: 0 weeks ] [ Designated as safety issue: No ]
  • The effect of Lithium on mood will be explored with the Beck depression inventory. [ Time Frame: 0 weeks ] [ Designated as safety issue: No ]
  • Effect of Lithium on quality of life will be assessed with the EQ-5D scale. [ Time Frame: 0 weeks ] [ Designated as safety issue: No ]
  • 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. [ Time Frame: 24 weeks ] [ Designated as safety issue: No ]
  • 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. [ Time Frame: 48 weeks ] [ Designated as safety issue: No ]
  • 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. [ Time Frame: 48 weeks ] [ Designated as safety issue: No ]
  • The effect of Lithium on mood will be explored with the Beck depression inventory. [ Time Frame: 24 weeks ] [ Designated as safety issue: No ]
  • The effect of Lithium on mood will be explored with the Beck depression inventory. [ Time Frame: 48 weeks ] [ Designated as safety issue: No ]
  • Effect of Lithium on quality of life will be assessed with the EQ-5D scale. [ Time Frame: 24 weeks ] [ Designated as safety issue: No ]
  • Effect of Lithium on quality of life will be assessed with the EQ-5D scale. [ Time Frame: 48 weeks ] [ Designated as safety issue: No ]
Same as current
Not Provided
Not Provided
 
Safety and Tolerability of Lithium in Spinocerebellar Ataxia 2 (SCA2)
Randomized, Placebo-controlled Trial to Test Safety, Tolerability and Efficacy of Lithium Carbonate in Spinocerebellar Ataxia 2

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.

Interventional
Phase 2
Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
SPINOCEREBELLAR ATAXIA 2
Drug: LITHIUM CARBONATE
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.
Other Name: Lithium Carbonate
  • Experimental: LITHIUM CARBONATE 150 and/or 300 mg
    Intervention: Drug: LITHIUM CARBONATE
  • Placebo Comparator: PLACEBO
    Intervention: Drug: LITHIUM CARBONATE
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Completed
20
November 2012
November 2012   (final data collection date for primary outcome measure)

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
Both
18 Years to 80 Years
No
Contact information is only displayed when the study is recruiting subjects
Italy
 
NCT00998634
SCA_LITIO_12, EUDRACT N°2009−016317−20
No
Alessandro Filla, Federico II University
Federico II University
Not Provided
Principal Investigator: Alessandro Filla, MD University Federico II
Federico II University
January 2013

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP