Evidence map›Paper›PMID 39740803›Full record

Trial reportJournal of medical genetics2025

Clonazepam repurposing in

Pleuntje J van der Sluijs, Koshar Safai Pour, Cécile L Berends, Matthijs D Kruizinga, Annelieke R Müller, Agnies M van Eeghen, Mar Rodríguez-Girondo, Maria J Juachon, Duco Steenbeek, Adam F Cohen and 2 more

Abstract readRandomized Controlled Trial
In one paragraph

Trial report in Journal of medical genetics, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper.

0numbers the graph read from it
0cells of the map it votes in
1citing 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

1 citing paper in PubMed.

  1. Article
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

12 authors.

Pleuntje J van der Sluijs *Department of Clinical Genetics, Leiden University Medical Center, Leiden, the Netherlands.ORCID 0000-0002-1174-2806
Koshar Safai Pour *Centre for Human Drug Research, Leiden, the Netherlands.
Cécile L BerendsCentre for Human Drug Research, Leiden, the Netherlands.
Matthijs D KruizingaCentre for Human Drug Research, Leiden, the Netherlands.
Annelieke R MüllerDepartment of Pediatrics, Amsterdam University Medical Center, Amsterdam, the Netherlands.
Agnies M van EeghenDepartment of Pediatrics, Amsterdam University Medical Center, Amsterdam, the Netherlands.
Mar Rodríguez-GirondoDepartment of Medical Statistics and Bioinformatics, Leiden University Medical Center, Leiden, the Netherlands.
Maria J JuachonCentre for Human Drug Research, Leiden, the Netherlands.
Duco SteenbeekDepartment of Rehabilitation Medicine, Maastricht University Medical Center, Maastricht, the Netherlands.
Adam F CohenCentre for Human Drug Research, Leiden, the Netherlands.
Rob G J A ZuikerCentre for Human Drug Research, Leiden, the Netherlands.
Gijs W E SantenDepartment of Clinical Genetics, Leiden University Medical Center, Leiden, the Netherlands santen@lumc.nl.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

backgroundClinical trials for rare disorders have unique challenges due to low prevalence, patient phenotype variability and high expectations. These challenges are highlighted by our study on clonazepam in

methodsThis study used a randomised, double-blinded, placebo-controlled, two-way crossover study (RCT), followed by an N-of-1 design. In the crossover study,

resultsIn the clonazepam group (

conclusionsOur approach shows the feasibility and strength of combining conventional RCT and N-of-1 studies for therapeutic studies in populations with intellectual disabilities, distinguishing real treatment effects from expectation bias. Our findings suggest that clonazepam has no additional therapeutic value in TRIAL REGISTRATION NUMBER: EUCTR2019-003558-98, ISRCTN11225608.

Indexed as

ClonazepamDNA-Binding ProteinsDrug RepositioningRare DiseasesTranscription FactorsAdolescentAdultChildCross-Over StudiesDouble-Blind MethodFemaleHumansMaleTreatment OutcomeYoung AdultClonazepamDNA-Binding ProteinsTranscription FactorsMental DisordersTherapeutics

Identifiers

PMID39740803
PMCPMC11877031

What OpenQuestion holds

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Registered trials

None linked

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.