Single-case experimental designs for bumetanide across neurodevelopmental disorders: BUDDI protocol.
BMC Psychiatry
; 22(1): 452, 2022 07 07.
Article
em En
| MEDLINE
| ID: mdl-35799144
ABSTRACT
BACKGROUND:
Bumetanide 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/DESIGN:
Participants 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 REGISTRATION EudraCT 2020-002196-35, registered 16 November 2020, https//www.clinicaltrialsregister.eu/ctr-search/trial/2020-002196-35/NL.Palavras-chave
Texto completo:
1
Coleções:
01-internacional
Base de dados:
MEDLINE
Assunto principal:
Bumetanida
/
Transtornos do Neurodesenvolvimento
Tipo de estudo:
Etiology_studies
/
Guideline
/
Incidence_studies
/
Observational_studies
/
Prognostic_studies
/
Risk_factors_studies
Aspecto:
Patient_preference
Limite:
Child
/
Humans
Idioma:
En
Revista:
BMC Psychiatry
Assunto da revista:
PSIQUIATRIA
Ano de publicação:
2022
Tipo de documento:
Article
País de afiliação:
Holanda