Bumetanide treatment for autism in clinical practice trial

Phase 1

• Conditions: Autism Spectrum Disorder, Attention Deficit Hyperactivity Disorder, Epilepsy; MedDRA version: 20.0Level: LLTClassification code 10008520Term: Childhood autismSystem Organ Class: 10037175 - Psychiatric disorders; MedDRA version: 20.0Level: PTClassification code 10063844Term: Autism spectrum disorderSystem Organ Class: 10037175 - Psychiatric disorders; MedDRA version: 20.0Level: PTClassification code 10015037Term: EpilepsySystem Organ Class: 10029205 - Nervous system disorders; MedDRA version: 20.0Level: LLTClassification code 10061345Term: Pervasive developmental disorderSystem Organ Class: 10037175 - Psychiatric disorders; MedDRA version: 20.0Level: LLTClassification code 10003805Term: AutismSystem Organ Class: 10037175 - Psychiatric disorders; MedDRA version: 20.0Level: LLTClassification code 10034739Term: Pervasive developmental disorder NOSSystem Organ Class: 10037175 - Psychiatric disorders; MedDRA version: 20.0Level: LLTClassification code 10064104Term: ADHDSystem Organ Class: 10037175 - Psychiatric disorders; Therapeutic area: Psychiatry and Psychology [F] - Mental Disorders [F03]

• Registration Number: EUCTR2016-002875-81-NL
• Lead Sponsor: MC Utrecht

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2016-10-25
• Last Update Posted: 30 November 2020

Recruitment & Eligibility

• Status: Authorised-recruitment may be ongoing or finished
• Sex: All
• Target Recruitment: 172

Inclusion Criteria

1.Males or females aged =5 years to =15 years;
2.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;
3.Written informed consent.
Are the trial subjects under 18? yes
Number of subjects for this age range: 172
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

1.Total IQ < 55 (WISC or WPPSI) and/or inability to comply with the protocol-specified procedures for the duration of the study, including treatment, blood sampling to control diuretic effects;
2.Presence of a severe medical or genetic disorder other than related to ASD or epilepsy;
3.Serious, unstable illnesses including, gastroenterological, respiratory, cardiovascular (arrhythmias, QT interval lengthening), endocrinologic, immunologic, hematologic disease, dehydration or hypotension, electrolyte disturbances (Na <133 mmol/L, K <3.5 mmol/L or Ca <2.17 mmol/L (<13y) or <2.2 mmol/L (>13y);
4.Renal insufficiency (CKD st2-5; estimated glomerular filtration rate < 90 ml/min/1.73m2), congenital or acquired renal disease with decreased concentration capacity (tubulopathy, diabetes insipidus) and liver insufficiency interfering with excretion or metabolism of bumetanide;
5.Start of behavioural treatment during study;
6.Treatment with psychoactive medications, including antipsychotics, psychostimulant drugs and AEDs, except methylphenidate, is allowed albeit on a stable regime in terms of types and dosage from 2 months prior to the study to the end of the study;
7.Treatment with NSAIDS, aminoglycosides, digitals, antihypertensive agents, indomethacin, probenecid, acetazolamide, Lithium, other diuretics (e.g., furosemide, hydrochlorothiazide), drugs known to have a nephrotoxic potential;
8.Documented history of hypersensitivity reaction to sulfonamide derivatives;
9. Body weight <17 kg.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: Improve behavioral and cognitive dysfunctioning;Secondary Objective: Reduce neuronal hyperexcitability;Primary end point(s): Aberrant Behavior Checklist (ABC) irritability subscale;Timepoint(s) of evaluation of this end point: 3 months

Secondary Outcome Measures

Name	Time	Method
Secondary end point(s): Other behavioral and quality of life parameters; seizure frequency.;Timepoint(s) of evaluation of this end point: 3 months