SPINOCEREBELLAR ATAXIA 2Phase 2LISCA2NCT00998634
Safety and Tolerability of Lithium in Spinocerebellar Ataxia 2 (SCA2)
A Phase 2 study testing LITHIUM CARBONATE in people with SPINOCEREBELLAR ATAXIA 2, run by Federico II University at 1 site in Italy.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 80 years
- Conditions the study is for
- SPINOCEREBELLAR ATAXIA 2
- What is being tested?
- LITHIUM CARBONATE
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 20 participants
- Where
- 1 siteItaly
- Study dates
- Oct 2009to Nov 2012
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 80 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Molecular diagnosis of SCA2 (≥34 CAG in the ataxin-2 gene)
- Age ≥18, <80
- SARA score ≥8
Who cannot join
- SARA score >32
- Heart failure
- Liver disease
- Kidney failure
- Thyroid disease
- Sick sinus syndrome and/or significant ECG alterations
Show all 15Show fewer
- 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
Trial locations and contacts
1 site in 1 country.
About this study
Official title: 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.
Read the 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.
- Conditions
- SPINOCEREBELLAR ATAXIA 2
- Being tested
- LITHIUM CARBONATE
- Keywords
- SCA2, Lithium
- Medical subject headings
- Spinocerebellar Ataxias
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
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.
| Study group | Role | What they receive |
|---|---|---|
| LITHIUM CARBONATE 150 and/or 300 mg | Experimental | Not described Receives: Drug: LITHIUM CARBONATE |
| PLACEBO | Placebo comparator | Not described Receives: Drug: LITHIUM CARBONATE |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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.
Measured: 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.
Measured: 0 weeks
The effect of Lithium on mood will be explored with the Beck depression inventory.
Measured: 0 weeks
Effect of Lithium on quality of life will be assessed with the EQ-5D scale.
Measured: 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.
Measured: 24 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.
Measured: 48 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.
Measured: 48 weeks
The effect of Lithium on mood will be explored with the Beck depression inventory.
Measured: 24 weeks
The effect of Lithium on mood will be explored with the Beck depression inventory.
Measured: 48 weeks
Effect of Lithium on quality of life will be assessed with the EQ-5D scale.
Measured: 24 weeks
Effect of Lithium on quality of life will be assessed with the EQ-5D scale.
Measured: 48 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Oct 2009 (estimated)
- Study startOct 2009
- Primary completionNov 2012
- Study completionNov 2012
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for SPINOCEREBELLAR ATAXIA 2Studies of the same condition, open and under way.
- More trials run by Federico II UniversityThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 20 participants (actual)
Sponsor and investigators
- Lead sponsor
- Federico II University
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Alessandro Filla, Federico II University
- Investigators
- Alessandro Filla, MD, Principal investigator, University Federico II
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00998634
- Sponsor's protocol ID
- SCA_LITIO_12
- Other IDs
- EUDRACT N°2009-016317-20
- Acronym
- LISCA2
- Status last verified
- Jan 2013
- First submitted
- 16 Oct 2009
- Last updated
- 8 Jan 2013
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 8 Jan 2013. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Randomized, Placebo-controlled Trial to Test Safety, Tolerability and Efficacy of Lithium Carbonate in Spinocerebellar Ataxia 2
- ClinicalTrials.gov ID
- NCT00998634
- Acronym / sponsor ID
- LISCA2 / SCA_LITIO_12
- Conditions
- SPINOCEREBELLAR ATAXIA 2
- Keywords
- SCA2, Lithium
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 20 participants (actual)
- Lead sponsor
- Federico II UniversityUniversity, hospital or other
- Study start
- Oct 2009
- Primary completion
- Nov 2012 (actual)
- Study completion
- Nov 2012 (actual)
- First posted
- 20 Oct 2009 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| LITHIUM CARBONATE 150 and/or 300 mg | Experimental | Drug: LITHIUM CARBONATE |
| PLACEBO | Placebo comparator | Drug: LITHIUM CARBONATE |
Who may qualify
- Ages
- 18 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (3 listed)
- Molecular diagnosis of SCA2 (≥34 CAG in the ataxin-2 gene)
- Age ≥18, <80
- SARA score ≥8
Key exclusion criteria (15 listed)
- SARA score >32
- Heart failure
- Liver disease
- Kidney failure
- Thyroid disease
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| LITHIUM CARBONATELithium 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. | Drug | LITHIUM CARBONATE 150 and/or 300 mg, PLACEBO |
Primary outcome
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.
11 secondary outcomes
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
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
The effect of Lithium on mood will be explored with the Beck depression inventory.
Time frame: 0 weeks
Effect of Lithium on quality of life will be assessed with the EQ-5D scale.
Time frame: 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.
Time frame: 24 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.
Time frame: 48 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.
Time frame: 48 weeks
The effect of Lithium on mood will be explored with the Beck depression inventory.
Time frame: 24 weeks
The effect of Lithium on mood will be explored with the Beck depression inventory.
Time frame: 48 weeks
Effect of Lithium on quality of life will be assessed with the EQ-5D scale.
Time frame: 24 weeks
Effect of Lithium on quality of life will be assessed with the EQ-5D scale.
Time frame: 48 weeks
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Jan 2013.
- University Federico IILink provided by the sponsor.
- AOU Policlinico "Federico II"Link provided by the sponsor.
Related trials for SPINOCEREBELLAR ATAXIA 2
Active, not recruitingPhase 3
Pivotal Study of N-acetyl-L-leucine for CACNA1A
CACNA1A, Spinocerebellar Ataxia Type 6
RecruitingObservational study
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
Rare Disorders, Undiagnosed Disorders
Active, not recruitingPhase 3
A Study to Learn More About the Effects and Long-Term Safety of Omaveloxolone (BIIB141) in Children and Teens With Friedreich's Ataxia
Friedreich Ataxia
Active, not recruitingPhase 1
A Study to Find Out How BIIB141 (Omaveloxolone) is Processed in the Body and to Learn More About Its Safety in Participants With Friedreich's Ataxia Aged 2 to 15 Years Old
Friedreich Ataxia
RecruitingPhase 2
Study of LX2006 Gene Therapy in Friedreich Ataxia Cardiomyopathy
Friedreich Ataxia, Cardiomyopathy, Secondary
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Alessandro Filla, MD principal investigator
University Federico II
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing LITHIUM CARBONATE in people with SPINOCEREBELLAR ATAXIA 2, run by Federico II University at 1 site in Italy.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 15 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 8 Jan 2013. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Italy. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the 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). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 8 Jan 2013.