Table 5 Trial registration data set

Primary registry and trial identifying number

EudraCT database: 2020–002196-35

Date of registration in primary registry

16th November 2020

Secondary identifying numbers

NA

Source(s) of monetary or material support

Dutch National research agenda, the Netherlands

Primary sponsor

Amsterdam UMC, location VUmc

Secondary sponsor(s)

Na

Contact for public queries

H. Bruining (MD, phd) n.is.you@amsterdamumc.nl

Contact for scientific queries

H. Bruining (MD, phd) n.is.you@amsterdamumc.nl

Public title

Bumetanide for developmental disorders

Scientific title

Post trial access cohort bumetanide for developmental disorders

Countries of recruitment

Netherlands

Health condition(s) or problem(s) studied

Neurodevelopmental disorders, autism spectrum disorder, attention deficit hyperactivity disorder (ADHD), learning disorders

Intervention(s)

Bumetanide bidaily 0.5–1.5 mg

Key inclusion and exclusion criteria

Participation in previous bumetanide trials by H. Bruining (BAMBI, BASCET, BATSCH)

or

Ages eligible for study: 7–17

Sexes eligible for study: both

Accepts healthy volunteers: no

Inclusion criteria:

One of the following: 1) Above clinical cut-off scores of altered sensory reactivity on the Sensory Profile and either a clinical ASD or ADHD diagnosis based on DSM-5 (or DSM-IV) or an epilepsy diagnosis, 2) Criteria met for autism on DSM-IV or V and Social Responsiveness Scale (SRS) 3) A history of behavioral problems combined with a definite diagnosis of TSC: either meeting criteria for clinical definite TSC, or a mutation identified in the TSC1 or TSC2 gene;

Exclusion criteria: 1) inability to comply with study protocol, 2) presence of severe medical or genetic disorder other than related to ASD, TSC or epilepsy, 3) renal insufficiency, 4) start of behavioral treatment during study, 5) treatment with methylphenidate, NSAIDs, aminoglycoside, digitalis, antihypertensive agents, indomethacin, probenecid, acetazolamide, lithium or other diuretics, 6) history of hypersensitivity to sulfonamide derivatives 7) body weight < 30 kg

Study type

Intervention model: open label post-trial access

Primary purpose: treatment

Phase II

Date of first enrolment

December 2020

Target sample size

115

Recruitment status

Recruiting

Primary outcome(s)

Set of parent proxy PROMIS questionnaires: physical stress experience, psychological stress experience, sleep disturbances, sleep-related impairment, cognitive function, anxiety, fatigue, peer relationships, life satisfaction, depressive symptoms

Key secondary outcomes

Conventional questionnaires: SRS, RBS, ABC, SP-NL

Resting-state EEG

Neurocognitive testing (using in-house Emma Toolbox)