This study will evaluate the safety and effectiveness of the food additive 1-octanol for
treating essential tremor. This disorder, which is an involuntary shaking, usually of the
hands, has no satisfactory treatment. It affects more than one of every 100 people in the
general population, with the figure climbing to nearly 4 in every hundred among people over
40 years old. In animal studies, 1-octanol reduced chemically induced tremors in rats. This
study will test the effects of the accepted daily intake of 1-octanol (1 milligram per
kilogram of body weight) on essential tremor in humans.
Patients with essential tremor 21 years old and older who wish to enroll in this study will
undergo eligibility screening with a medical history and physical examination that includes
tests for thyroid, liver and kidney problems. Participants will be randomly assigned to
receive either 1-octanol or a placebo (an inactive substance). Patients in both groups will
have an intravenous catheter (a thin, plastic tube) placed in an arm vein for collecting
blood samples during the study. Those in the 1-octanol group will be given a 1-octanol
capsule; the placebo group will receive a look-alike capsule containing no active ingredient.
Neither the patient nor the doctor will know which patients are taking 1-octanol or placebo
until the end of the study.
Tremors will be measured once before the catheter is placed, every 15 minutes during the
first 2 hours after taking the capsule, twice during the third hour (30 minutes apart), and
once again after 5 hours. The tremors are measured using procedures called accelerometry and
surface electromyography. For these procedures, electrodes are taped to the skin; needles are
not used. Blood samples will be collected once before taking the capsule, every 15 minutes
for the first hour and a half after taking the capsule and again at 2 hours, 4 hours and 5
hours after taking the capsule. Vital signs (blood pressure, pulse, and respiratory rate)
will be measured every 15 minutes during the first 2 hours of taking the capsule, every 30
minutes during the third hour, and again at 4 hours and 5 hours.
Participants will stay in the hospital overnight for observation and return after 3 days for
a follow-up physical examination, including a blood test.
Phase:
Phase 1
Details
Lead Sponsor:
National Institute of Neurological Disorders and Stroke (NINDS)