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Essential TremorPhase 1NCT01468948

Octanoic Acid for Essential Tremor

A Phase 1 study testing Octanoic Acid in people with Essential Tremor, run by National Institute of Neurological Disorders and Stroke (NINDS) at 1 site in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

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. 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?
Octanoic Acid
How many people
18 participants
Where
1 siteUnited States
Study dates
28 Oct 2011to 18 Jul 2012
Phase
Phase 1
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

  • Diagnosis of essential tremor with bilateral hand tremor as the predominant feature, which is known to be responsive to ethanol.
  • Unequivocal accelerometric tremor of both hands on screening examination (bilateral central tremor component during postural tremor accelerometry, consistent with ET)
  • Reduction of accelerometric tremor power of at least 35% following a formal ethanol challenge during the screening visit.
  • Subjects must be willing and safely able to abstain from any medication for the treatment of tremor for a period of at least 5 plasma half-lives of the individual drug prior to study participation. (For Propranolol/Inderal , Gabapentin/Neurontin this will be 1 day; for Primidone/Mysoline : 26 days).
  • Subjects must be willing to refrain from alcohol and drinks or food containing caffeine starting 48 hours prior to the study visits

Who cannot join

  • Patients with any other significant pathological finding in the neurological examination other than typical symptoms of ET
  • Acute or chronic severe medical conditions which would preclude the subject from participating (e.g., severe heart disease NYHA grade 3 or 4, renal failure, hepatic failure, lung disease, uncontrolled hyperthyroidism)
  • Subjects with concomitant therapy with warfarin or NSAIDs (other than aspirin), when taken on a regular basis and cannot be discontinued at least 14 days prior to study participation, because of potential interactions with octanoic acid (displacement of albumin binding in human serum)(Noctor et al. 1992)
  • Established diagnosis of diabetes mellitus, as fasting-periods of up to 12 hours are required in the protocol.
  • Subjects with active or past alcohol abuse or dependence (AUDIT score greater than or equal to 8)
  • Elevated liver function parameters (AST, ALT, GGT), higher than the 1.5 fold upper limit of the normal range (as defined by the NIH Clinical Center Laboratory Medicine Department), or any other clinically significant abnormalities on their baseline laboratory tests. The limit for AST therefore will be 51 U/l, for ALT 62 U/L, and GGT 128 U/l.
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  • Female subjects who are pregnant or breastfeeding
  • Subjects aged < 21 years
  • Known flushing symptoms after alcohol intake or allergy to alcohol (any yes answer in the standardized Alcohol Flushing Questionnaire)

Trial locations and contacts

1 site in 1 country.

United States1 site

  • National Institutes of Health Clinical Center, 9000 Rockville Pike

    Bethesda, Maryland, 20892

Other trials in United States

About this study

Official title: Dose Escalation Study of Oral Octanoic Acid in Patients With Essential Tremor

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.
Read more

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.
Read the detailed description

OBJECTIVE:

To determine the maximum tolerated dose of oral octanoic acid (OA) in patients with essential tremor. Further study objectives include the evaluation of the efficacy and tolerability of octanoic acid with escalation doses, as well as the pharmacokinetic and pharmacodynamic profile.

STUDY POPULATION:

Up to 30 subjects with ethanol-responsive essential tremor (ET) will be included in the study. Subjects will be recruited in groups of 6 per dose level.

DESIGN:

The study objectives will be tested using a 3+3 dose escalation design. Per dose level, 3 subjects will be recruited, and dose levels will be 8, 16, 32, 64, and 128 mg/kg, with additional 3 subjects at the same level if one of the three subjects exhibits dose limit toxicity. Subjects will undergo a screening visit, followed by a 2 to 3-day inpatient admission during which the study drug OA will be administered. An outpatient follow-up visit will conclude the study.

OUTCOME MEASURES:

The primary outcome will be measured by evaluating dose-limiting toxicity, which will be reached once 2 or more subjects exhibit a grade 2 adverse event (CTCAE) on the same dose-level, which is related to OA. The dose below the level at which 2 or more grade 2 OA-related adverse events have been observed, will then be defined as maximum tolerated dose (MTD) and the study stopped. Toxicity for the primary outcome will be monitored by an unblinded independent data safety monitoring board (DSMB), who will determine when the primary outcome is reached.

Secondary measures will include safety measures such as routine laboratory parameters, EKG measures, vital signs as well as a standardized assessment for signs of intoxication. Additional secondary outcome measures will include efficacy measures such as tremor accelerometry and digital spiral analysis, as well as a standardized clinical tremor rating scale. Furthermore, pharmacokinetic sampling will be performed.

Being tested
Octanoic Acid
Keywords
Essential Tremor, Tremor, Octanol, Octanoic Acid
Medical subject headings
Essential Tremor, Tremor

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Octanoic AcidDrug

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Dose-limiting toxicity

    Measured: 2 years

Secondary outcomes (1)

  1. Effect on tremor

    Measured: 2 years

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted10 Nov 2011 (estimated)
  2. Study start28 Oct 2011
  3. Primary completion18 Jul 2012
  4. Study completion18 Jul 2012

Resources

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 1
Primary purpose
Treatment
Allocation
Non-randomised
Intervention model
Single group
Masking
None (open label)
Enrolment
18 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT01468948
Sponsor's protocol ID
120024
Other IDs
12-N-0024
Status last verified
18 Jul 2012
First submitted
8 Nov 2011
Last updated
16 Dec 2019
Results posted
No

Publications

  • Bain P, Brin M, Deuschl G, Elble R, Jankovic J, Findley L, Koller WC, Pahwa R. Criteria for the diagnosis of essential tremor. Neurology. 2000;54(11 Suppl 4):S7. No abstract available.

    PubMed 10854345

  • Ashitani J, Matsumoto N, Nakazato M. Effect of octanoic acid-rich formula on plasma ghrelin levels in cachectic patients with chronic respiratory disease. Nutr J. 2009 Jun 16;8:25. doi: 10.1186/1475-2891-8-25.

    PubMed 19527531

  • Bach AC, Babayan VK. Medium-chain triglycerides: an update. Am J Clin Nutr. 1982 Nov;36(5):950-62. doi: 10.1093/ajcn/36.5.950.

    PubMed 6814231

  • Voller B, Lines E, McCrossin G, Tinaz S, Lungu C, Grimes G, Starling J, Potti G, Buchwald P, Haubenberger D, Hallett M. Dose-escalation study of octanoic acid in patients with essential tremor. J Clin Invest. 2016 Apr 1;126(4):1451-7. doi: 10.1172/JCI83621. Epub 2016 Feb 29.

    PubMed 26927672

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing Octanoic Acid 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 5 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 16 Dec 2019. 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 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

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?

Dose-limiting toxicity (time frame: 2 years). 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 NCT01468948, which also lists 4 linked publications.

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 16 Dec 2019.

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Octanoic Acid for Essential Tremor (NCT01468948) | AzTrials