This study will determine the optimal dose of 1-octanol that will safely reduce tremors in
patients with essential tremor-a disorder in which the hands, and sometimes the head, shake
involuntarily. Current treatments may be ineffective or produce unwanted side effects.
Ethanol (the chemical in beer and wine that causes intoxication) reduces tremor in many
patients, but patients generally don't use it regularly because it interferes with daily
activities. Laboratory studies show that 1-octanol, a drug that is similar to ethanol, may
have the same beneficial effect on tremors with less likelihood of intoxication.
Patients 21 years of age and older with essential tremor may be eligible for this 10-day
study. Candidates will be evaluated with a neurological examination, blood tests, urinalysis
and electrocardiogram (EKG). Those enrolled will be admitted to the hospital for 4 days for
1-octanol administration and monitoring. On day 1, patients will have a medical history and
physical examination. A catheter (a thin plastic tube) will be placed in a vein of the
forearm for sampling blood. Patients will take one 1-octanol capsule (at one of seven doses)
by mouth and will be monitored for tremors and drug side effects. Blood will be sampled
periodically in the first 3 hours to determine 1-octanol blood levels. On days 2 and 3,
patients will be monitored for additional side effects. On days 3 and 4, laboratory tests
(blood and urine) will be done to evaluate liver and kidney function. On day 4, the catheter
will be removed and the patient will be discharged from the hospital. A follow-up visit will
be scheduled 1 week after discharge for a physical examination and blood, urine and EKG
tests.
Phase:
Phase 1
Details
Lead Sponsor:
National Institute of Neurological Disorders and Stroke (NINDS)