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

Area of Science:

• Neuroscience
• Pharmacology
• Clinical Trials

Background:

• Bumetanide, a NKCC1 chloride importer antagonist, is being explored for neurodevelopmental disorders (NDDs).
• Treatment efficacy is expected in specific patient subsets, necessitating stratified approaches.
• Previous trials focused on stratification and optimal endpoints for bumetanide in NDDs.

Purpose of the Study:

• To confirm the efficacy of bumetanide in NDD subpopulations.
• To validate treatment prediction effect methodologies.
• To refine outcome measurements for clinical trials.

Main Methods:

• A combined cohort study including previous participants and new individuals.
• Six-month bumetanide treatment using multiple baseline Single Case Experimental Designs.
• Primary outcomes: patient-reported measures of sensory processing difficulties; Secondary outcomes: standardized NDD questionnaires, resting-state EEG, and cognitive assessments via the Emma Toolbox.

Main Results:

• This section is not available in the provided abstract.

Conclusions:

• This study protocol outlines a plan to replicate bumetanide's effects in NDD subpopulations.
• The research will validate predictive treatment methodologies and refine endpoint measurements for future NDD studies.