Essential TremorPhase 2NCT00080366
Octanol to Treat Essential Tremor
A Phase 2 study testing 1-Octanol in people with Essential Tremor, run by National Institute of Neurological Disorders and Stroke (NINDS) at 1 site in United States.
Trial summary
This study will evaluate the effectiveness of 1-octanol, a substance similar to alcohol but less intoxicating, for treating essential tremor. Essential tremor is an involuntary shaking, usually of the hands, for which there is no satisfactory treatment. It affects about 1.4 percent of the general U.S. Read more about this study
- Who is it for?
- Ages 21 years and older
- Conditions the study is for
- Essential Tremor
- What is being tested?
- 1-Octanol
- How many people
- 14 participants
- Where
- 1 siteUnited States
- Study dates
- Mar 2004to Mar 2007
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 21 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 14 patients with a clinical diagnosis of essential tremor will participate in the study. Selection criterion is essential tremor with a history of ethanol responsiveness. Informed consent will be obtained by any of the co-investigators.
- Patients must be off any medications used to treat essential tremor such as mysoline or propranolol for at least 2 weeks. Patients must withhold ethanol and caffeine 24 hours prior to starting the treatment periods through the end of treatment periods, including alcohol or caffeine containing over the counter medications. Ethanol and caffeine consumption is allowed in the washout period.
Who cannot join
- Patients with abnormalities on neurologic exam other than tremor.
- Patients with a history of chronic alcohol dependence.
- Patients with chronic medical conditions such as renal failure, hepatic failure and chronic lung disease.
- Patients on other medications that cannot be temporarily discontinued for the length of the study.
- Patients, who, for moral or religious reasons, do not wish to take a potentially intoxicating drug.
- Patients with abnormalities on their baseline screening laboratory tests.
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- Women who are pregnant or lactating.
- Patients under the age of 21.
- Asians and Pacific Islanders.
Trial locations and contacts
1 site in 1 country.
United States1 site
National Institutes of Health Clinical Center, 9000 Rockville Pike
Bethesda, Maryland, 20892
About this study
Official title: Treatment Efficacy of 1-Octanol Compared to Placebo in Adults With Essential Tremor
This study will evaluate the effectiveness of 1-octanol, a substance similar to alcohol but less intoxicating, for treating essential tremor. Essential tremor is an involuntary shaking, usually of the hands, for which there is no satisfactory treatment. It affects about 1.4 percent of the general U.S. population, with the figure climbing to nearly 4 percent among people over 40. Results of two previous NIH studies have shown 1-octanol to be promising as a potential new treatment. This study will test the effectiveness of 1-octanol on essential tremor at doses lower than those given previously.
Patients 21 years old and older with essential tremor may be eligible for this study. Participants are admitted to the NIH Clinical Center for two treatment periods of 1 week each, with a 1-week break at home between treatments. Before beginning treatment, participants undergo a medical history, physical examination, blood and urine tests, and an electrocardiogram (EKG). In addition, tremors are measured using accelerometry, a procedure in which a small device, mounted on a piece of cardboard, is taped to the patient's hand for about 30 minutes.
Patients are randomly assigned to one of two groups. One group takes 2 to 4 capsules of 1-octanol 3 times a day for 1 week, followed by a 1-week "washout" period (no treatment), and then 2 to 4 capsules of placebo 3 times a day for 1 week. Following the same dosage schedule, the second group takes placebo the first week, followed by the washout period and then 1-octanol treatment. Blood pressure and pulse are measured at 15, 30, and 60 minutes after the first dose of the day and then 3 times a day each day of hospitalization, EKG and blood draws are done every other day during hospitalization, and blood is drawn again 1 week after the end of the study. Patients evaluate their tremor daily according to a tremor scale and are also rated according to an alcohol intoxication scale.
Read the detailed description
Essential tremor is a very common movement disorder affecting approximately 1.4% of the population. Response to medications such as beta blockers and mysoline may be only partial or be accompanied by intolerable side effects. Roughly 80% of patients have significant tremor reduction from? ethanol although daily use of this as a treatment has potentially serious social and legal consequences. The leading hypothesis for the pathophysiology of essential tremor is the unmasking of spontaneous oscillation of neurons in the inferior olive. Both ethanol and 1-octanol have been shown to reduce these spontaneous oscillations in a animal model of essential tremor; however, 1-octanol does this at a dose much lower than an intoxicating dose suggesting that it may be useful in the treatment of essential tremor. Two pilot studies with 1-octanol in patients with essential tremor suggested it was both efficacious and safe.
Objectives: The aim of the present study is to further assess efficacy of 1-octanol in essential tremor in a double-blinded, placebo-controlled study.
Study population: Patients with alcohol-responsive essential tremor.
Design: Double-blind, placebo-controlled two-period cross-over design.
Outcome parameters: Primary outcome parameter is tremor as assessed by accelerometry, secondary outcome measures are spiral drawing, subjective and clinical scoring.
Estimate of the outcome: 1-octanol at the dose of 32mg/kg three times a day will significantly improve tremor in patients with alcohol-responsive essential tremor.
Anticipated risks and benefits: Besides the unusual taste, there are no anticipated risks associated with 1-octanol ingestion. Patients may experience an improvement of their tremor.
Potential meaning for the field: Better treatment of essential tremor.
- Conditions
- Essential Tremor
- Being tested
- 1-Octanol
- Keywords
- Essential Tremor, Cross over Design, Accelerometry, Placebo, Alcohol Responsive, Double Blind, 1-Octanol
- Medical subject headings
- Essential Tremor
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
1-OctanolDrug
What is being measured
The primary outcome is the main question the study is designed to answer.
Tremor as assessed by accelerometry.
Secondary outcomes (1)
Spiral drawing, subjective and clinical scoring.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Mar 2004 (estimated)
- Study startMar 2004
- Study completionMar 2007
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Essential TremorStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Enrolment
- 14 participants
Sponsor and investigators
- Lead sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
- Sponsor type
- US National Institutes of Health
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00080366
- Sponsor's protocol ID
- 040147
- Other IDs
- 04-N-0147
- Status last verified
- Mar 2007
- First submitted
- 27 Mar 2004
- Last updated
- 4 Apr 2007
- Results posted
- No
Publications
Bikson M, Ghai RS, Baraban SC, Durand DM. Modulation of burst frequency, duration, and amplitude in the zero-Ca(2+) model of epileptiform activity. J Neurophysiol. 1999 Nov;82(5):2262-70. doi: 10.1152/jn.1999.82.5.2262.
PubMed 10561404
Study at a glance
From the registry record, last updated by the sponsor on 4 Apr 2007. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Treatment Efficacy of 1-Octanol Compared to Placebo in Adults With Essential Tremor
- ClinicalTrials.gov ID
- NCT00080366
- Acronym / sponsor ID
- 040147
- Conditions
- Essential Tremor
- Keywords
- Essential Tremor, Cross over Design, Accelerometry, Placebo, Alcohol Responsive, Double Blind, 1-Octanol
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 14 participants
- Lead sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)US National Institutes of Health
- Study start
- Mar 2004
- Study completion
- Mar 2007
- First posted
- 29 Mar 2004 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This study will evaluate the effectiveness of 1-octanol, a substance similar to alcohol but less intoxicating, for treating essential tremor. Essential tremor is an involuntary shaking, usually of the hands, for which there is no satisfactory treatment. It affects about 1.4 percent of the general U.S. population, with the figure climbing to nearly 4 percent among people over 40. Results of two previous NIH studies have shown 1-octanol to be promising as a potential new treatment. This study will test the effectiveness of 1-octanol on essential tremor at doses lower than those given previously.
Patients 21 years old and older with essential tremor may be eligible for this study. Participants are admitted to the NIH Clinical Center for two treatment periods of 1 week each, with a 1-week break at home between treatments. Before beginning treatment, participants undergo a medical history, physical examination, blood and urine tests, and an electrocardiogram (EKG). In addition, tremors are measured using accelerometry, a procedure in which a small device, mounted on a piece of cardboard, is taped to the patient's hand for about 30 minutes.
Patients are randomly assigned to one of two groups. One group takes 2 to 4 capsules of 1-octanol 3 times a day for 1 week, followed by a 1-week "washout" period (no treatment), and then 2 to 4 capsules of placebo 3 times a day for 1 week. Following the same dosage schedule, the second group takes placebo the first week, followed by the washout period and then 1-octanol treatment. Blood pressure and pulse are measured at 15, 30, and 60 minutes after the first dose of the day and then 3 times a day each day of hospitalization, EKG and blood draws are done every other day during hospitalization, and blood is drawn again 1 week after the end of the study. Patients evaluate their tremor daily according to a tremor scale and are also rated according to an alcohol intoxication scale.
Detailed description
Essential tremor is a very common movement disorder affecting approximately 1.4% of the population. Response to medications such as beta blockers and mysoline may be only partial or be accompanied by intolerable side effects. Roughly 80% of patients have significant tremor reduction from? ethanol although daily use of this as a treatment has potentially serious social and legal consequences. The leading hypothesis for the pathophysiology of essential tremor is the unmasking of spontaneous oscillation of neurons in the inferior olive. Both ethanol and 1-octanol have been shown to reduce these spontaneous oscillations in a animal model of essential tremor; however, 1-octanol does this at a dose much lower than an intoxicating dose suggesting that it may be useful in the treatment of essential tremor. Two pilot studies with 1-octanol in patients with essential tremor suggested it was both efficacious and safe.
Objectives: The aim of the present study is to further assess efficacy of 1-octanol in essential tremor in a double-blinded, placebo-controlled study.
Study population: Patients with alcohol-responsive essential tremor.
Design: Double-blind, placebo-controlled two-period cross-over design.
Outcome parameters: Primary outcome parameter is tremor as assessed by accelerometry, secondary outcome measures are spiral drawing, subjective and clinical scoring.
Estimate of the outcome: 1-octanol at the dose of 32mg/kg three times a day will significantly improve tremor in patients with alcohol-responsive essential tremor.
Anticipated risks and benefits: Besides the unusual taste, there are no anticipated risks associated with 1-octanol ingestion. Patients may experience an improvement of their tremor.
Potential meaning for the field: Better treatment of essential tremor.
Study design
- Primary purpose
- Treatment
Who may qualify
- Ages
- 21 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (2 listed)
- 14 patients with a clinical diagnosis of essential tremor will participate in the study. Selection criterion is essential tremor with a history of ethanol responsiveness. Informed consent will be obtained by any of the co-investigators.
- Patients must be off any medications used to treat essential tremor such as mysoline or propranolol for at least 2 weeks. Patients must withhold ethanol and caffeine 24 hours prior to starting the treatment periods through the end of treatment periods, including alcohol or caffeine containing over the counter medications. Ethanol and caffeine consumption is allowed in the washout period.
Key exclusion criteria (9 listed)
- Patients with abnormalities on neurologic exam other than tremor.
- Patients with a history of chronic alcohol dependence.
- Patients with chronic medical conditions such as renal failure, hepatic failure and chronic lung disease.
- Patients on other medications that cannot be temporarily discontinued for the length of the study.
- Patients, who, for moral or religious reasons, do not wish to take a potentially intoxicating drug.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| 1-Octanol | Drug | – |
Primary outcome
Tremor as assessed by accelerometry.
1 secondary outcome
Spiral drawing, subjective and clinical scoring.
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 4 Apr 2007.
- Background: PubMed 10561404Bikson M, Ghai RS, Baraban SC, Durand DM. Modulation of burst frequency, duration, and amplitude in the zero-Ca(2+) model of epileptiform activity. J Neurophysiol. 1999 Nov;82(5):2262-70. doi: 10.1152/jn.1999.82.5.2262.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing 1-Octanol in people with Essential Tremor, run by National Institute of Neurological Disorders and Stroke (NINDS) at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 4 Apr 2007. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Tremor as assessed by accelerometry.. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00080366, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 4 Apr 2007.