Trial reportThe Journal of clinical investigation2016
Dose-escalation study of octanoic acid in patients with essential tremor.
Trial report in The Journal of clinical investigation, 2016. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 13 papers, 1 of them a synthesis that pooled it.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
13 citing papers in PubMed, 1 synthesis or guideline pooled it, 35 citations in OpenAlex.
- Gastrointestinal issues predominate among side effects of medium-chain triglyceride consumption in humans.Frontiers in nutrition · 2026Pooled it
- The Effect of Octanoic Acid on Essential Voice Tremor: A Double-Blind, Placebo-Controlled Study.The Laryngoscope · 2019Trial
- The response of the central and peripheral tremor component to octanoic acid in patients with essential tremor.Clinical neurophysiology : official journal of the International Federation of Clinical Neurophysiology · 2018Trial
- Postmortem Fatty Acid Abnormalities in the Cerebellum of Patients with Essential Tremor.Cerebellum (London, England) · 2024Article
- Tips and tricks in tremor treatment.Journal of neural transmission (Vienna, Austria : 1996) · 2024Review
- Octanoic acid mitigates busulfan-induced blood-testis barrier damage by alleviating oxidative stress and autophagy.Lipids in health and disease · 2024Article
- T-type calcium channels as therapeutic targets in essential tremor and Parkinson's disease.Annals of clinical and translational neurology · 2023Review
- Essential tremor.Nature reviews. Disease primers · 2021Review
- Local Vibrational Therapy for Essential Tremor Reduction: A Clinical Study.Medicina (Kaunas, Lithuania) · 2020Article
- Effect of Gold Electronic State on the Catalytic Performance of Nano Gold Catalysts inNanomaterials (Basel, Switzerland) · 2020Article
- Mechanisms and Pharmacotherapy for Ethanol-Responsive Movement Disorders.Frontiers in neurology · 2020Review
- Current and Future Neuropharmacological Options for the Treatment of Essential Tremor.Current neuropharmacology · 2020Review
- Emerging strategies in the management of essential tremor.Therapeutic advances in neurological disorders · 2017Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
11 authors at 3 institutions in 2 countries.
Funding
Abstract
backgroundRecently, 1-octanol has been shown to have efficacy in treating patients with essential tremor (ET). The primary metabolite of 1-octanol is octanoic acid (OA), which is now thought to be the active substance that mediates tremor suppression. Our aim was to describe the maximum tolerated dose (MTD) of oral OA in patients with ET and assess the pharmacokinetics (PK) and pharmacodynamics (PD) profile of OA.
methodsThe MTD was studied using an open-label, single-ascending 3 + 3 dose-escalation design. Predefined single doses ranged from 8 to 128 mg/kg, with grade 2 adverse events (AEs) defined as dose-limiting toxicity. Tremor was assessed using accelerometry, digital spiral analysis, and a standard clinical rating scale at baseline and up to 600 minutes after intake. Safety assessments and PK sampling were also performed.
resultsDose-limiting toxicity was not reached. The most frequent AE was mild abdominal discomfort. Exposure (AUC) increased linearly with the dose. Secondary efficacy measures suggested a dose-dependent reduction of tremor. Accordingly, a single unified PK/PD model with an effect compartment and sigmoid maximum effect (Emax) response could be built that accounted well for the time profiles of plasma concentrations as well as effects on tremor severity across the 5 dose levels.
conclusionAlthough our trial did not reach an MTD, a dose-dependent effect was demonstrated in the PK/PD model as well as in secondary efficacy outcomes. Future studies are needed to explore the safety in higher dose ranges and to confirm dose-dependent efficacy in a placebo-controlled design.
trial registrationClinicaltrials.gov NCT01468948FUNDING. NINDS Intramural Research Program; TG Therapeutics Inc.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.