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Bumetanide to Ameliorate Tuberous Sclerosis Complex Hyperexcitable Behaviors


- candidate number26476
- NTR NumberNTR6328
- ISRCTNISRCTN no longer applicable
- Date ISRCTN created
- date ISRCTN requested
- Date Registered NTR13-jan-2017
- Secondary IDsNL58183.041.16 METC 16-463
- Public TitleBumetanide to Ameliorate Tuberous Sclerosis Complex Hyperexcitable Behaviors
- Scientific TitleBumetanide to Ameliorate Tuberous Sclerosis Complex Hyperexcitable Behaviors
- ACRONYMBATSCH
- hypothesisWe propose to conduct a pilot study to confirm the efficacy of bumetanide as add-on treatment to reduce irritability and other behavioral symptoms typical in children and adolescents with TSC, with and without mental retardation.
- Healt Condition(s) or Problem(s) studiedTuberous sclerosis complex
- Inclusion criteriaGeneral inclusion criteria:
1. Males and females aged 8-15 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 behavioral problems
4. Written informed consent
Specific inclusion criteria Group 1:
- No intellectual disability (TIQ>70)
Specific inclusion criteria Group 2:
- Intellectual disability (TIQ≤70)
- Exclusion criteria1. Inability to comply with the protocol-specified procedures
2. Presence of a severe medical or genetic disorder other than related to TSC or epilepsy
3. Serious, unstable illnesses
4. Renal insufficiency, congenital or acquired renal disease with decreased concentration capacity and liver insufficiency interfering with excretion or metabolism of bumetanide
5. Behavioral treatment
6. Treatment with psychoactive medications, including antipsychotics, antidepressants, anxiolytic drugs, psychostimulant drugs, except melatonin. If clinically feasible, then it is allowed to stop psychoactive medication to allow enrolment in the study after a 4 week washout period of their psychoactive medication. Notably, an exception is treatment with AEDs, which are allowed albeit on a stable regime in terms of types of AEDs 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, drugs known to have a nephrotoxic potential
8. Documented history of hypersensitivity reaction to sulfonamide derivatives
9. Body weight < 30 kg
- mec approval receivedno
- multicenter trialno
- randomisedno
- groupParallel
- TypeSingle arm
- Studytypeintervention
- planned startdate 1-mrt-2017
- planned closingdate1-mrt-2019
- Target number of participants20
- InterventionsThe investigational product will consist of bumetanide, which will be provided for 91 days as an add-on treatment, supplementary to the regular use of AEDs or other (allowed) co-medications. Dose reductions to manage side effects will be allowed at any time. Based upon the expected chance of frequent mild to moderate hypokalemia, all subjects will receive standard potassium supplementation during the 91 days of treatment. The treatment period will be followed by a wash-out period to evaluate return of symptomatology and reversibility of treatment effect.
- Primary outcomeAberrant Behavior Checklist (ABC) irritability subscale
- Secondary outcomeQuestionnaires
- PedsQL
- WHOQOL-BREF, EQ-5D-5L and EQ5D-Youth
- TRF, BRIEF
- ABC subscales
- RBS-V
- SRS
- SP-NL and SP-SC
- TAND
Neurocognitive measurements
- Cognitive test battery
Epilepsy related variables
- Seizure frequency
- Number of occasions rescue medication is necessary Electroencephalography
- Resting state EEG and ERP assessments
- Timepoints Pre-treatment and screening
o Screening for eligibility
o Baseline measurements
Treatment (D0 to D91)
o Blood analysis at D4, D7, D14, D28, D56
o End of treatment outcome measurements D91
Washout (D91 to D119)
o End of washout outcome measurements D119
- Trial web site
- statusplanned
- CONTACT FOR PUBLIC QUERIES D.M. van Andel
- CONTACT for SCIENTIFIC QUERIES D.M. van Andel
- Sponsor/Initiator University Medical Center Utrecht (UMCU)
- Funding
(Source(s) of Monetary or Material Support)
Michelle Foundation
- Publications
- Brief summaryPilot open label study confirming the applicability and efficacy of bumetanide as add-on treatment for behavioural problems and seizures in 2x10 patients with TSC and a history of behavioural problems between 8 and 15 years of age. After the baseline screening, included subjects receive bumetanide for 91 days, followed by a 28 days discontinuation phase, after which endpoint monitoring will be repeated to evaluate the persistence of treatment effect. Test battery assessment will take place at baseline, at the end of bumetanide treatment (Day 91) and after the discontinuation phase (Day 119). The primary endpoint of the study is the ABC-I subscale at Day 91. In addition, a number of cognitive and neurophysiological markers as well as measures of seizure control will be tested.
- Main changes (audit trail)
- RECORD13-jan-2017 - 28-mei-2017


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