Evidence map›Paper›PMID 39754397›Full record

Trial reportMovement disorders clinical practice2025

An Open-Label Pilot Study to Examine the Safety, Tolerability and Efficacy of Deutetrabenazine in Isolated Dystonia.

Andres Deik, Whitley Aamodt, Christina Cadet, Aaron Lasker, Alexandria Oliver, Meredith Spindler, Thomas F Tropea, Pavan Vaswani, Andrew Siderowf

Abstract readClinical Trial
PubMed Full text at PMC Publisher
In one paragraph

Trial report in Movement disorders clinical practice, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 2 papers.

0numbers the graph read from it
0cells of the map it votes in
2citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

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.

2 · The registry

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.

3 · Its place in the literature

Who cites it

2 citing papers in PubMed.

  1. Review
  2. Review
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

9 authors.

Andres DeikParkinson Disease and Movement Disorders Center, University of Pennsylvania, Philadelphia, Pennsylvania, USA.
Whitley AamodtParkinson Disease and Movement Disorders Center, University of Pennsylvania, Philadelphia, Pennsylvania, USA.ORCID https://orcid.org/0000-0001-8130-2809
Christina CadetParkinson Disease and Movement Disorders Center, University of Pennsylvania, Philadelphia, Pennsylvania, USA.
Aaron LaskerParkinson Disease and Movement Disorders Center, University of Pennsylvania, Philadelphia, Pennsylvania, USA.
Alexandria OliverParkinson Disease and Movement Disorders Center, University of Pennsylvania, Philadelphia, Pennsylvania, USA.
Meredith SpindlerParkinson Disease and Movement Disorders Center, University of Pennsylvania, Philadelphia, Pennsylvania, USA.
Thomas F TropeaParkinson Disease and Movement Disorders Center, University of Pennsylvania, Philadelphia, Pennsylvania, USA.
Pavan VaswaniParkinson Disease and Movement Disorders Center, University of Pennsylvania, Philadelphia, Pennsylvania, USA.ORCID https://orcid.org/0000-0002-1826-6275
Andrew SiderowfParkinson Disease and Movement Disorders Center, University of Pennsylvania, Philadelphia, Pennsylvania, USA.

Funding

Teva Pharmaceutical Industries TV50717-NDG-60301
6 · The paper itself

Abstract

backgroundDystonia may respond to VMAT2 inhibition.

objectivesProviding pilot data on the safety, tolerability, and efficacy of deutetrabenazine in non dopa-responsive dystonia.

methodsDeutetrabenazine was titrated by adults with isolated dystonia. Primary study endpoints included the proportion who maintained the individual, maximum tolerated dose for 6 weeks, and how many titrated to 48 mg/day. Secondary endpoints included rates of QTc prolongation/arrhythmias, suicidality, excessive daytime sleepiness, cognitive decline, and drug-induced parkinsonism. Exploratory endpoints for clinical efficacy were assessed.

resultsAmong 15 participants, four (26.7%) withdrew early and six (40%) titrated to 48 mg/day. Common adverse events included fatigue and diarrhea. Secondary safety endpoints did not change significantly, but MDS-UPDRS III scores worsened by ≥3 points in seven participants (46.7%). PGI-C and the blinded CGI-C and GDS improved in three women with blepharospasm.

conclusionsMost participants tolerated deutetrabenazine for 6 weeks, and those with blepharospasm may have benefitted from its use.

Indexed as

Adrenergic Uptake InhibitorsDystoniaTetrabenazineAdultAgedFemaleHumansMaleMiddle AgedPilot ProjectsTreatment OutcomeAdrenergic Uptake InhibitorsdeutetrabenazineTetrabenazineclinical trialdeutetrabenazineisolated dystoniaVMAT2 inhibitor

Identifiers

PMID39754397
PMCPMC11998685

What OpenQuestion holds

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Registered trials

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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.