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
  Purpose

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.


Condition Intervention Phase
SPINOCEREBELLAR ATAXIA 2
Drug: LITHIUM CARBONATE
Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: Randomized, Placebo-controlled Trial to Test Safety, Tolerability and Efficacy of Lithium Carbonate in Spinocerebellar Ataxia 2

Resource links provided by NLM:


Further study details as provided by Federico II University:

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. [ 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.


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. [ 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 ]

Enrollment: 20
Study Start Date: October 2009
Study Completion Date: November 2012
Primary Completion Date: November 2012 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: LITHIUM CARBONATE 150 and/or 300 mg 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
Placebo Comparator: PLACEBO 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

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

Ages Eligible for Study:   18 Years to 80 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00998634

Locations
Italy
Dipartimento di Scienze Neurologiche
Napoli, Italy, 80131
Sponsors and Collaborators
Federico II University
Investigators
Principal Investigator: Alessandro Filla, MD University Federico II
  More Information

Additional Information:
No publications provided

Responsible Party: Alessandro Filla, Principal Investigator, Federico II University
ClinicalTrials.gov Identifier: NCT00998634     History of Changes
Other Study ID Numbers: SCA_LITIO_12, EUDRACT N°2009−016317−20
Study First Received: October 16, 2009
Last Updated: January 7, 2013
Health Authority: Italy: Ethics Committee

Keywords provided by Federico II University:
SCA2
Lithium

Additional relevant MeSH terms:
Ataxia
Spinocerebellar Ataxias
Spinocerebellar Degenerations
Cerebellar Ataxia
Cerebellar Diseases
Brain Diseases
Central Nervous System Diseases
Dyskinesias
Genetic Diseases, Inborn
Heredodegenerative Disorders, Nervous System
Nervous System Diseases
Neurodegenerative Diseases
Neurologic Manifestations
Signs and Symptoms
Spinal Cord Diseases
Lithium
Lithium Carbonate
Antidepressive Agents
Antimanic Agents
Antipsychotic Agents
Central Nervous System Agents
Central Nervous System Depressants
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action
Pharmacologic Actions
Physiological Effects of Drugs
Psychotropic Drugs
Therapeutic Uses
Tranquilizing Agents

ClinicalTrials.gov processed this record on October 22, 2014