Evidence map›Paper›PMID 35799144›Full record

ArticleBMC psychiatry2022

Single-case experimental designs for bumetanide across neurodevelopmental disorders: BUDDI protocol.

Lisa Geertjens, Gianina Cristian, Eva Haspels, Jennifer Ramautar, Gert Jan van der Wilt, Matthijs Verhage, Hilgo Bruining

Open access · goldAbstract readClinical Trial Protocol
In one paragraph

Article in BMC psychiatry, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 3 papers.

0numbers the graph read from it
0cells of the map it votes in
3citing papers in PubMed
0.4field-weighted citation impact, top 33% of its field
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

3 citing papers in PubMed, 4 citations in OpenAlex.

  1. Trial
  2. Article
  3. 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

7 authors at 5 institutions in 2 countries.

Lisa GeertjensN=You Neurodevelopmental Precision Center, Amsterdam Neuroscience, Amsterdam Reproduction and Development, Amsterdam UMC, Meibergdreef 5, 1105, AZ, Amsterdam, The Netherlands.
Gianina CristianN=You Neurodevelopmental Precision Center, Amsterdam Neuroscience, Amsterdam Reproduction and Development, Amsterdam UMC, Meibergdreef 5, 1105, AZ, Amsterdam, The Netherlands.
Eva HaspelsN=You Neurodevelopmental Precision Center, Amsterdam Neuroscience, Amsterdam Reproduction and Development, Amsterdam UMC, Meibergdreef 5, 1105, AZ, Amsterdam, The Netherlands.
Jennifer RamautarN=You Neurodevelopmental Precision Center, Amsterdam Neuroscience, Amsterdam Reproduction and Development, Amsterdam UMC, Meibergdreef 5, 1105, AZ, Amsterdam, The Netherlands.
Gert Jan van der WiltDepartment of Health Evidence, Radboud University Medical Center, 6500, HB, Nijmegen, The Netherlands.
Matthijs VerhageDepartment of Human Genetics, Center for Neurogenomics and Cognitive Research (CNCR), Amsterdam UMC-location VUmc, De Boelelaan 1105, 1081, HV, Amsterdam, The Netherlands.
Hilgo BruiningN=You Neurodevelopmental Precision Center, Amsterdam Neuroscience, Amsterdam Reproduction and Development, Amsterdam UMC, Meibergdreef 5, 1105, AZ, Amsterdam, The Netherlands. h.bruining@amsterdamumc.nl.
Amsterdam Neuroscience · NLAmsterdam University Medical Centers · NLEmma Kinderziekenhuis · NLRadboud University Medical Center · NLRadboud University Nijmegen · NL

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

backgroundBumetanide is a selective NKCC1 chloride importer antagonist which is being repurposed as a mechanism-based treatment for neurodevelopmental disorders (NDDs). Due to their specific actions, these kinds of interventions will only be effective in particular subsets of patients. To anticipate stratified application, we recently completed three bumetanide trials each focusing on different stratification strategies with the additional objective of deriving the most optimal endpoints. Here we publish the protocol of the post-trial access combined cohort study to confirm previous effects and stratification strategies in the trial cohorts and in new participants. METHOD/

designParticipants of the three previous cohorts and a new cohort will be subjected to 6 months bumetanide treatment using multiple baseline Single Case Experimental Designs. The primary outcome is the change, relative to baseline, in a set of patient reported outcome measures focused on direct and indirect effects of sensory processing difficulties. Secondary outcome measures include the conventional questionnaires 'social responsiveness scale', 'repetitive behavior scale', 'sensory profile' and 'aberrant behavior scale'. Resting-state EEG measurements will be performed at several time-points including at Tmax after the first administration. Assessment of cognitive endpoints will be conducted using the novel Emma Tool box, an in-house designed battery of computerized tests to measure neurocognitive functions in children. DISCUSSION: This study aims to replicate previously shown effects of bumetanide in NDD subpopulations, validate a recently proposed treatment prediction effect methodology and refine endpoint measurements.

trial registrationEudraCT: 2020-002196-35, registered 16 November 2020, https://www.clinicaltrialsregister.eu/ctr-search/trial/2020-002196-35/NL.

Indexed as

BumetanideNeurodevelopmental DisordersChildCohort StudiesHumansResearch DesignTreatment OutcomeBumetanideAutism spectrum disorderBumetanideChildrenNeurodevelopmental disorders

Identifiers

PMID35799144
PMCPMC9260985
OpenAlexW4284895985

What OpenQuestion holds

Textmetadata
LicenceCC BY
Read underepoch 390

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.