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Sponsored by: |
National Institute of Neurological Disorders and Stroke (NINDS) |
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Information provided by: | National Institutes of Health Clinical Center (CC) |
ClinicalTrials.gov Identifier: | NCT00683943 |
This study will evaluate the side effects and tolerability of the drug lithium in patients with spinocerebellar ataxia type I (SCA1) an inherited disorder caused by loss of nerve cells in parts of the brain. Symptoms include ataxia (difficulty walking) and loss of muscle coordination and strength. Recent studies suggest that lithium may be helpful in treating some SCA1 symptoms.
People between 18 and 65 years of age with SCA1 who have only difficulty walking or who have difficulty walking as well as tremor, hand incoordination or speech problems, may be eligible for this study. Participation requires three hospital admissions at the NIH Clinical Center and one outpatient visit.
Participants undergo the following tests and procedures:
Admission 1 (2-6 weeks)
Admission 2 (2-4 days, 4 weeks after hospital discharge).
Admission 3 (2-4 days, 8 weeks after Admission 2).
Outpatient Visit (4 weeks after Admission 3)
Condition | Intervention | Phase |
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Spinocerebellar Ataxia Type I |
Drug: Lithium Carbonate |
Phase I |
Study Type: | Interventional |
Study Design: | Treatment, Non-Randomized, Open Label, Uncontrolled, Single Group Assignment, Safety Study |
Official Title: | Pilot Study of Tolerability of Lithium Therapy in Patients With Spinocerebellar Ataxia Type I (SCA1) |
Estimated Enrollment: | 15 |
Study Start Date: | May 2008 |
Objective
Evaluation of tolerability and safety of lithium in SCA1 patients.
Study Population
A total of 10 molecularly diagnosed SCA1 patients divided in two groups. One group would be composed of 5 patients with gait difficulty only and 5 patients with gait difficulty and tremor, hand incoordination or speech difficulties.
Design
Patients will be admitted to the NIH Clinical Center for initiation and titration of Lithium until high therapeutic levels currently accepted for the treatment of Bipolar affective disorder are achieved (1.0-1.2 mmol/L) or a maximum tolerated dose. During their admissions, they will have neurological evaluations, ataxia and tremor evaluations, timed-up-and-go testing, quality of life assessment, questionnaires evaluating sleep, balance confidence and depression, memory testing, alertness assessment and quantitative balance/sway evaluations. Parameters and side effect profiles will be assessed at baseline, 1 month (plus or minus 5 days) and 3 months (plus or minus 5 days) after achievement of therapeutic levels. They will also have an exit evaluation one month (plus or minus 5 days) after discontinuation of Lithium also at the NIH. For their baseline evaluation and titration, patients will be admitted to the NIH for 4 weeks plus or minus 2 weeks. Evaluation at 1 month (plus or minus 5 days) and 3 months (plus or minus 5 days)
Outcome Measures
Primary Outcomes
Incidence of side effects
Side effect profile
Secondary Outcomes
Timed-up-and-go test
ICARS score
SARA score
Modified Falls Efficacy Scale
Quantitative Sway/Balance assessment
Tremor Rating Scale:
Part A - Tremor location/severity rating
Part B -Handwriting and drawings
Part C - Functional disabilities resulting from tremor
Questionnaires related to sleep, depression (Beck Depression Inventory) (BDI), alertness (Computerized Continuous Performing Tasks) and memory (Mini Mental State Examination)
Dexterity evaluation - At-Home Testing Program device
Ages Eligible for Study: | 18 Years to 65 Years |
Genders Eligible for Study: | Both |
Accepts Healthy Volunteers: | No |
EXCLUSION CRITERIA:
Contact: Patient Recruitment and Public Liaison Office | (800) 411-1222 | prpl@mail.cc.nih.gov |
Contact: TTY | 1-866-411-1010 |
United States, Maryland | |
National Institutes of Health Clinical Center, 9000 Rockville Pike | Recruiting |
Bethesda, Maryland, United States, 20892 |
Study ID Numbers: | 080134, 08-N-0134 |
Study First Received: | May 22, 2008 |
Last Updated: | September 3, 2008 |
ClinicalTrials.gov Identifier: | NCT00683943 |
Health Authority: | United States: Federal Government |
Spinocerebellar Ataxia Lithium Spinocerebellar Ataxia |
Spinal Cord Diseases Lithium Carbonate Central Nervous System Diseases Brain Diseases Neurodegenerative Diseases Dyskinesias Signs and Symptoms Cerebellar Ataxia Heredodegenerative Disorders, Nervous System |
Genetic Diseases, Inborn Ataxia Neurologic Manifestations Cerebellar Diseases Cerebellar ataxia Spinocerebellar Degenerations Lithium Spinocerebellar Ataxias |
Tranquilizing Agents Molecular Mechanisms of Pharmacological Action Nervous System Diseases Physiological Effects of Drugs Psychotropic Drugs Central Nervous System Depressants Enzyme Inhibitors |
Antimanic Agents Antipsychotic Agents Pharmacologic Actions Therapeutic Uses Central Nervous System Agents Antidepressive Agents |