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Lithium Treatment for Patients With Spinocerebellar Ataxia Type I

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT00683943
Recruitment Status : Completed
First Posted : May 26, 2008
Last Update Posted : December 17, 2019
Sponsor:
Information provided by:
National Institutes of Health Clinical Center (CC)

Tracking Information
First Submitted Date  ICMJE May 22, 2008
First Posted Date  ICMJE May 26, 2008
Last Update Posted Date December 17, 2019
Study Start Date  ICMJE May 18, 2008
Actual Primary Completion Date February 25, 2010   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: May 29, 2008)
Incidence of side effects, side effect profile, and number of falls.
Original Primary Outcome Measures  ICMJE
 (submitted: May 22, 2008)
Incidence of side effects
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: May 22, 2008)
ICARS scores, SARA scores, Quantitative Sway/Balance assessment, Tremor Rating Scale scores, dexterity evaluation, sleep questionnaire, Computerized Continuous Performing Tasks assessment
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Lithium Treatment for Patients With Spinocerebellar Ataxia Type I
Official Title  ICMJE Pilot Study of Tolerability of Lithium Therapy in Patients With Spinocerebellar Ataxia Type I (SCA1)
Brief Summary

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.
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  ICMJE Interventional
Study Phase  ICMJE Phase 1
Study Design  ICMJE Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Spinocerebellar Ataxia Type I
Intervention  ICMJE Drug: Lithium Carbonate
Study Arms  ICMJE Not Provided
Publications *

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: February 26, 2010)
13
Original Enrollment  ICMJE
 (submitted: May 22, 2008)
15
Actual Study Completion Date  ICMJE February 25, 2010
Actual Primary Completion Date February 25, 2010   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE
  • 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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years to 65 Years   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT00683943
Other Study ID Numbers  ICMJE 080134
08-N-0134
Has Data Monitoring Committee Not Provided
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Not Provided
Study Sponsor  ICMJE National Institute of Neurological Disorders and Stroke (NINDS)
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Principal Investigator: Grisel J Lopez, M.D. National Human Genome Research Institute (NHGRI)
PRS Account National Institutes of Health Clinical Center (CC)
Verification Date February 27, 2014

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP