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

Primary Purpose

SPINOCEREBELLAR ATAXIA 2

Status
Completed
Phase
Phase 2
Locations
Italy
Study Type
Interventional
Intervention
LITHIUM CARBONATE
Sponsored by
Federico II University
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for SPINOCEREBELLAR ATAXIA 2 focused on measuring SCA2, Lithium

Eligibility Criteria

18 Years - 80 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • Dipartimento di Scienze Neurologiche

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Placebo Comparator

Arm Label

LITHIUM CARBONATE 150 and/or 300 mg

PLACEBO

Arm Description

Outcomes

Primary Outcome Measures

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.

Secondary Outcome Measures

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.

Full Information

First Posted
October 16, 2009
Last Updated
January 7, 2013
Sponsor
Federico II University
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1. Study Identification

Unique Protocol Identification Number
NCT00998634
Brief Title
Safety and Tolerability of Lithium in Spinocerebellar Ataxia 2 (SCA2)
Acronym
LISCA2
Official Title
Randomized, Placebo-controlled Trial to Test Safety, Tolerability and Efficacy of Lithium Carbonate in Spinocerebellar Ataxia 2
Study Type
Interventional

2. Study Status

Record Verification Date
January 2013
Overall Recruitment Status
Completed
Study Start Date
October 2009 (undefined)
Primary Completion Date
November 2012 (Actual)
Study Completion Date
November 2012 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
Federico II University

4. Oversight

Data Monitoring Committee
No

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
SPINOCEREBELLAR ATAXIA 2
Keywords
SCA2, Lithium

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
20 (Actual)

8. Arms, Groups, and Interventions

Arm Title
LITHIUM CARBONATE 150 and/or 300 mg
Arm Type
Experimental
Arm Title
PLACEBO
Arm Type
Placebo Comparator
Intervention Type
Drug
Intervention Name(s)
LITHIUM CARBONATE
Intervention Description
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.
Primary Outcome Measure Information:
Title
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.
Description
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.
Time Frame
the endpoint will be recorded at all visits
Secondary Outcome Measure Information:
Title
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
Title
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
Title
The effect of Lithium on mood will be explored with the Beck depression inventory.
Time Frame
0 weeks
Title
Effect of Lithium on quality of life will be assessed with the EQ-5D scale.
Time Frame
0 weeks
Title
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
Title
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
Title
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
Title
The effect of Lithium on mood will be explored with the Beck depression inventory.
Time Frame
24 weeks
Title
The effect of Lithium on mood will be explored with the Beck depression inventory.
Time Frame
48 weeks
Title
Effect of Lithium on quality of life will be assessed with the EQ-5D scale.
Time Frame
24 weeks
Title
Effect of Lithium on quality of life will be assessed with the EQ-5D scale.
Time Frame
48 weeks

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
80 Years
Accepts Healthy Volunteers
No
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Alessandro Filla, MD
Organizational Affiliation
University Federico II
Official's Role
Principal Investigator
Facility Information:
Facility Name
Dipartimento di Scienze Neurologiche
City
Napoli
ZIP/Postal Code
80131
Country
Italy

12. IPD Sharing Statement

Links:
URL
http://www.unina.it
Description
University Federico II
URL
http://www.policlinico.unina.it
Description
AOU Policlinico "Federico II"

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

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