Spinocerebellar Ataxia Type I Clinical Trial
Official title:
Pilot Study of Tolerability of Lithium Therapy in Patients With Spinocerebellar Ataxia Type I (SCA1)
| Verified date | February 27, 2014 |
| Source | National Institutes of Health Clinical Center (CC) |
| Contact | n/a |
| Is FDA regulated | No |
| Health authority | |
| Study type | Interventional |
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.
| Status | Completed |
| Enrollment | 13 |
| Est. completion date | February 25, 2010 |
| Est. primary completion date | February 25, 2010 |
| Accepts healthy volunteers | No |
| Gender | All |
| Age group | 18 Years to 65 Years |
| Eligibility |
- 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 |
| Country | Name | City | State |
|---|---|---|---|
| United States | National Institutes of Health Clinical Center, 9000 Rockville Pike | Bethesda | Maryland |
| Lead Sponsor | Collaborator |
|---|---|
| National Institute of Neurological Disorders and Stroke (NINDS) |
United States,
Allagui MS, Vincent C, El feki A, Gaubin Y, Croute F. Lithium toxicity and expression of stress-related genes or proteins in A549 cells. Biochim Biophys Acta. 2007 Jul;1773(7):1107-15. Epub 2007 Apr 21. — View Citation
Cummings CJ, Sun Y, Opal P, Antalffy B, Mestril R, Orr HT, Dillmann WH, Zoghbi HY. Over-expression of inducible HSP70 chaperone suppresses neuropathology and improves motor function in SCA1 mice. Hum Mol Genet. 2001 Jul 1;10(14):1511-8. — View Citation
de Chiara C, Menon RP, Dal Piaz F, Calder L, Pastore A. Polyglutamine is not all: the functional role of the AXH domain in the ataxin-1 protein. J Mol Biol. 2005 Dec 9;354(4):883-93. Epub 2005 Oct 18. — View Citation
| Type | Measure | Description | Time frame | Safety issue |
|---|---|---|---|---|
| Primary | Incidence of side effects, side effect profile, and number of falls. | |||
| Secondary | ICARS scores, SARA scores, Quantitative Sway/Balance assessment, Tremor Rating Scale scores, dexterity evaluation, sleep questionnaire, Computerized Continuous Performing Tasks assessment |