NCT01468948

Octanoic Acid for Essential Tremor

Completed Phase 1 Last updated 16 December 2019

What this trial tests

Phase 1 trial testing Octanoic Acid in Essential Tremor in 18 participants. Completed in 18 July 2012.

Timeline

28 October 2011

Primary endpoint
18 July 2012

18 July 2012

Quick facts

Lead sponsor	National Institute of Neurological Disorders and Stroke (NINDS)
Phase	Phase 1
Status	Completed
Study type	INTERVENTIONAL
Allocation	non randomized
Design	single group
Masking	none
Primary purpose	treatment
Enrollment	18
Start date	28 October 2011
Primary completion	18 July 2012
Estimated completion	18 July 2012
Sites	1 location across United States

Drugs / interventions tested

• Octanoic Acid (OCTANOIC ACID) — full drug profile →

Conditions studied

• Essential Tremor — all drugs for Essential Tremor →

Sponsor

National Institute of Neurological Disorders and Stroke (NINDS)

Who can join

21 and older, any sex, with Essential Tremor. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

Background:

\- Essential tremor (ET) is a condition of out-of-control shaking. Several drugs are used to treat ET. However, they are often only partly helpful and can have side effects. Many people with ET get some relief from drinking alcohol. Octanol, a food additive similar to alcohol, can improve tremor in animals and is less likely to make people feel drunk. One form of octanol, called 1-octanol, has been shown to improve tremor in some people and had few side effects. 1-octanol is converted to octanoic acid, and research suggests that octanoic acid itself might suppress ET with no significant side effects such as drunkenness. Researchers want to see what dose of octanoic acid is most useful in reducing ET.

Objectives:

\- To test different doses of octanoic acid to treat essential tremor.

Eligibility:

* Individuals at least 21 years of age who have ET that responds to treatment with alcohol.
* Participants must be able to stop taking certain ET medications during the study.

Design:

* This study requires three visits. Visit 1 is a screening visit that will take up to 5 hours. Visit 2 is a 2- to 3-day inpatient admission to the National Institutes of Health Clinical Center. Visit 3 is a followup outpatient visit 1 to 2 weeks after the hospital admission.
* At the screening visit, participants will have a physical exam, neurological exam, and medical history. Blood and urine samples will be collected. Participants will also have an alcohol dose test to measure the tremor s response to alcohol.
* For the study visit, participants will enter the hospital for testing. Participants will have the study drug and test the tremor's response to it. Frequent blood samples will be collected.
* One to two weeks after leaving the hospital, participants will have a final followup study visit. Blood samples will be collected.

Publications & conference data

4 peer-reviewed publications reference this trial (live from Europe PMC):

1. Dose-escalation study of octanoic acid in patients with essential tremor.
   Voller B, Lines E, McCrossin G, Tinaz S, et al · · 2016 · cited 18× · PMID 26927672 · DOI 10.1172/jci83621
2. Alcohol challenge and sensitivity to change of the Essential Tremor Rating Assessment Scale.
   Voller B, Lines E, McCrossin G, Artiles A, et al · · 2014 · cited 17× · PMID 24123358 · DOI 10.1002/mds.25667
3. Mechanisms and Pharmacotherapy for Ethanol-Responsive Movement Disorders.
   Wu J, Tang H, Chen S, Cao L. · · 2020 · cited 8× · PMID 32982923 · DOI 10.3389/fneur.2020.00892
4. Treatment of essential tremor with long-chain alcohols: still experimental or ready for prime time?
   Haubenberger D, Nahab FB, Voller B, Hallett M. · · 2014 · cited 8× · PMID 24587968 · DOI 10.7916/d8rx991r