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Completed

NCT Number: NCT00998634

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

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.

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Key information

Conditions

SPINOCEREBELLAR ATAXIA 2 Ataxia Brain Diseases Central Nervous System Diseases Cerebellar Ataxia Cerebellar Diseases Congenital, Hereditary, and Neonatal Diseases and Abnormalities Dyskinesias Genetic Diseases, Inborn Heredodegenerative Disorders, Nervous System Nervous System Diseases Neurodegenerative Diseases Neurologic Manifestations Spinal Cord Diseases Spinocerebellar Ataxias Spinocerebellar Degenerations

Age range

18 year–80 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Dipartimento di Scienze Neurologiche

Naples, 80131, Italy

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Lithium Carbonate

Drug

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 outcomes

  1. 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

    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 outcomes

  1. 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

  2. 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

  3. The effect of Lithium on mood will be explored with the Beck depression inventory.

    Time frame: 0 weeks

  4. Effect of Lithium on quality of life will be assessed with the EQ-5D scale.

    Time frame: 0 weeks

  5. 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

  6. 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

  7. 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

  8. The effect of Lithium on mood will be explored with the Beck depression inventory.

    Time frame: 24 weeks

  9. The effect of Lithium on mood will be explored with the Beck depression inventory.

    Time frame: 48 weeks

  10. Effect of Lithium on quality of life will be assessed with the EQ-5D scale.

    Time frame: 24 weeks

  11. Effect of Lithium on quality of life will be assessed with the EQ-5D scale.

    Time frame: 48 weeks

Sponsors and collaborators

Lead sponsor

Federico II University

Other

Registry information

Official study title

Randomized, Placebo-controlled Trial to Test Safety, Tolerability and Efficacy of Lithium Carbonate in Spinocerebellar Ataxia 2

Acronym: LISCA2

Important dates

Study start
2009
Primary completion
2012
Study completion
2012
First posted
Oct 20, 2009
Registry last updated
Jan 8, 2013

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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