Lithium Treatment for Patients With Spinocerebellar Ataxia Type I

December 14, 2019 updated by: National Institute of Neurological Disorders and Stroke (NINDS)

Pilot Study of Tolerability of Lithium Therapy in Patients With Spinocerebellar Ataxia Type I (SCA1)

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)

  • Medical history, physical examination, blood and urine tests, electrocardiogram.
  • Evaluation of SCA1 symptoms (balance, walking, dexterity, tremor, memory, mood and concentration).
  • Monitoring of liquid intake and output (urine) and weight changes.
  • Lithium treatment Start treatment and remain in hospital until the blood level of the drug is stabilized; continue treatment at home after hospital discharge.

Admission 2 (2-4 days, 4 weeks after hospital discharge).

  • Repeat of some or all of the procedures done at the first admission.
  • Continue lithium in hospital and at home after discharge, with local physician checking laboratory values as needed.

Admission 3 (2-4 days, 8 weeks after Admission 2).

  • Repeat of some or all of the procedures done at other admissions.
  • Stop lithium.

Outpatient Visit (4 weeks after Admission 3)

  • Evaluation of SCA1 symptoms.
  • Blood and urine tests.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Detailed Description

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

  1. Incidence of side effects
  2. Side effect profile

Secondary Outcomes

  1. Timed-up-and-go test
  2. ICARS score
  3. SARA score
  4. Modified Falls Efficacy Scale
  5. Quantitative Sway/Balance assessment

  6. Tremor Rating Scale:

    • Part A Tremor location/severity rating
    • Part B Handwriting and drawings
    • Part C Functional disabilities resulting from tremor
  7. Questionnaires related to sleep, depression (Beck Depression Inventory) (BDI), alertness (Computerized Continuous Performing Tasks) and memory (Mini Mental State Examination)
  8. Dexterity evaluation At-Home Testing Program device

Study Type

Interventional

Enrollment (Actual)

13

Phase

  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • Maryland
      • Bethesda, Maryland, United States, 20892
        • National Institutes of Health Clinical Center, 9000 Rockville Pike

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years to 65 years (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

  • INCLUSION CRITERIA:
  • 18-65 years of age
  • Molecularly diagnosed SCA1
  • Gait disturbance only or gait disturbance and tremor and/or hand incoordination and/or speech difficulty.
  • Women of childbearing age who agree to use contraceptive methods for the duration of the study.

EXCLUSION CRITERIA:

  • Children
  • Pregnant or nursing women
  • Anyone on a low-sodium diet
  • Cognitively impaired individuals
  • Schizophrenics
  • Family history of thyroid diseases
  • Patients with medical conditions that can be affected by Lithium such as diabetes mellitus, difficulty urinating, infections (severe occurring with fever, prolonged sweating, diarrhea, or vomiting), kidney disease, epilepsy, thyroid disease, heart disease, pulmonary disease, bipolar affective disorder, cardiac arrhythmia, diabetes insipidus, Parkinson disease, psoriasis and leukemia.
  • Anyone taking the following medications: diuretics, nonsteroidal anti-inflammatory agents, methyldopa, tetracycline, antipsychotic, neuromuscular blocking agents, digoxin, phenytoin, acetazolamide, calcium iodide, iodinated glycerol, potassium iodide, calcium channel blockers and/or metronidazole. Patients on these medications may be enrolled and their lithium levels will be followed very closely. If levels are consistently maintained, the subject may remain in the study.
  • Age greater than 65 years

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Non-Randomized
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Incidence of side effects, side effect profile, and number of falls.

Secondary Outcome Measures

Outcome Measure
ICARS scores, SARA scores, Quantitative Sway/Balance assessment, Tremor Rating Scale scores, dexterity evaluation, sleep questionnaire, Computerized Continuous Performing Tasks assessment

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

National Institute of Neurological Disorders and Stroke (NINDS)

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

May 18, 2008

Primary Completion (Actual)

February 25, 2010

Study Completion (Actual)

February 25, 2010

Study Registration Dates

First Submitted

May 22, 2008

First Submitted That Met QC Criteria

May 22, 2008

First Posted (Estimate)

May 26, 2008

Study Record Updates

Last Update Posted (Actual)

December 17, 2019

Last Update Submitted That Met QC Criteria

December 14, 2019

Last Verified

February 27, 2014

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 080134
  • 08-N-0134

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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