NCT00998634

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.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
20

participants targeted

Target at below P25 for phase_2

Timeline
Completed

Started Oct 2009

Typical duration for phase_2

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

October 1, 2009

Completed
15 days until next milestone

First Submitted

Initial submission to the registry

October 16, 2009

Completed
4 days until next milestone

First Posted

Study publicly available on registry

October 20, 2009

Completed
3 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

November 1, 2012

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

November 1, 2012

Completed
Last Updated

January 8, 2013

Status Verified

January 1, 2013

Enrollment Period

3.1 years

First QC Date

October 16, 2009

Last Update Submit

January 7, 2013

Conditions

SPINOCEREBELLAR ATAXIA 2

Keywords

SCA2Lithium

Outcome Measures

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.

    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.

    the endpoint will be recorded at all visits

Secondary Outcomes (11)

  • 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.

    0 weeks

  • 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.

    0 weeks

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

    0 weeks

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

    0 weeks

  • 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.

    24 weeks

  • +6 more secondary outcomes

Study Arms (2)

LITHIUM CARBONATE 150 and/or 300 mg

EXPERIMENTAL
Drug: LITHIUM CARBONATE

PLACEBO

PLACEBO COMPARATOR
Drug: LITHIUM CARBONATE

Interventions

LITHIUM CARBONATEDRUG

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.

LITHIUM CARBONATE 150 and/or 300 mgPLACEBO

Eligibility Criteria

Age18 Years - 80 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • Molecular diagnosis of SCA2 (≥34 CAG in the ataxin-2 gene)
  • Age ≥18, \<80
  • SARA score ≥8

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Dipartimento di Scienze Neurologiche

Napoli, 80131, Italy

Location

Related Links

MeSH Terms

Conditions

Spinocerebellar Ataxias

Interventions

Lithium Carbonate

Condition Hierarchy (Ancestors)

Cerebellar AtaxiaCerebellar DiseasesBrain DiseasesCentral Nervous System DiseasesNervous System DiseasesSpinocerebellar DegenerationsSpinal Cord DiseasesHeredodegenerative Disorders, Nervous SystemNeurodegenerative DiseasesAtaxiaDyskinesiasNeurologic ManifestationsGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

CarbonatesAlkaliesInorganic ChemicalsCarbonic AcidCarbon Compounds, InorganicLithium Compounds

Study Officials

  • Alessandro Filla, MD

    University Federico II

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
phase 2
Allocation
RANDOMIZED
Masking
QUADRUPLE
Who Masked
PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
Principal Investigator

Study Record Dates

First Submitted

October 16, 2009

First Posted

October 20, 2009

Study Start

October 1, 2009

Primary Completion

November 1, 2012

Study Completion

November 1, 2012

Last Updated

January 8, 2013

Record last verified: 2013-01

Locations

IT(1)