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

ABSTRACT: 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. Participants of the three previous cohorts and a new cohort will be subjected to 6 months bumetanide treatment using multiple baseline S

SUBMITTER: Geertjens L 

PROVIDER: S-EPMC9260985 | biostudies-literature | 2022 Jul

REPOSITORIES: biostudies-literature