Bumetanide to treat behavioral problems in TSC

Phase 1

Conditions: Tuberous Sclerosis Complex
MedDRA version: 20.0Level: PTClassification code 10045138Term: Tuberous sclerosisSystem Organ Class: 10010331 - Congenital, familial and genetic disorders
Therapeutic area: Psychiatry and Psychology [F] - Mental Disorders [F03]

Registration Number: EUCTR2016-002408-13-NL

Lead Sponsor: MC Utrecht

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: Authorised-recruitment may be ongoing or finished

Sex: All

Target Recruitment: Not specified

Inclusion Criteria

1.Males or females aged =8 years;
2.Definite diagnosis of TSC: either meeting criteria for clinical definite TSC, or a mutation identified in the TSC1 or TSC2 gene;
3.History of behavioural problems;
4.Written informed consent.

Are the trial subjects under 18? yes
Number of subjects for this age range: 20
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 4
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

1.Inability to comply with the protocol-specified procedures for the duration of the study, including treatment and blood sampling to control diuretic effects;
2.Presence of a severe medical or genetic disorder other than related to TSC or epilepsy;
3.Serious, unstable illnesses including, gastroenterological, respiratory, cardiovascular (arrhythmias, QT interval lengthening), endocrinologic, immunologic, hematologic disease, dehydration or hypotension, electrolyte disurbances (Na, K or Ca);

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: Reduce behavioral symptoms;Secondary Objective: Improve seizure control;Primary end point(s): ABC-I scale;Timepoint(s) of evaluation of this end point: 3 months

Secondary Outcome Measures