Down Syndrome
Conditions
Interventions
Trade Name: Burinex
Product Name: Bumetanide
Pharmaceutical Form: Tablet
Trade Name: Burinex
Product Name: Bumetanide
Pharmaceutical Form: Tablet
Trade Name: Burinex
Product Name: Bumetanide
Pharmac
Sponsors
IRCCS Bambino Gesù Children's Hospital
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1)The presence of a free trisomy 21 documented by karyotyping 2)Adolescents from 10 to 16 years old (included) 3)Informed consent from each child and their parents. Are the trial subjects under 18? yes Number of subjects for this age range: 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
Exclusion criteria: 1) The presence of any neurosensory deficits, such as hypoacusis or serious visual impairments 2) The presence of epilepsy 3) The presence of disorders electrolytes 4) The presence of a hypersensibility known about sulpha drugs 5) The presence of contraindications relative to the treatment by bumetanide 6) Patients already treated by diuretics 7) Any of the following abnormal laboratory values at screening: - Hemoglobin 450 msec at Screening
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: The aim of the study is to investigate the potential of 3 months of treatment with Bumetanide to rescue cognitive functions and associated psychopathological aspects in DS of Bumetanide treatment in children and adolescents with DS aged 10-16 years. The overreaching goal of our project is to test the ability of a treatment with Bumetanide to rescue cognitive functions and associated psychopathological aspects in children and adolescents with DS. As Bumetanide has been extensively used in the past in humans with little side effects, it is orally active, and it is very economical, we will test here the potentials of our therapeutic approach to be translated directly in a clinical trial on a cohort of DS patients. ;Secondary Objective: ;Primary end point(s): The primary end-point will be a clinical improvement in the total score on visual-spatial episodic memory task (Promea). At month three we expect for the Bumetanide treatment group an improvement on the primary outcome measure comparable with the improvement in the normative sample, considering the test-retest reliability at three months (Vicari et al., 2007). According to the test re-test reliability of the memory tasks for 3 months’ time interval in normative population (Promea - Vicari et al., 2007) and the correlation between the measures (r = 0.6), it has been considered clinically significant an increment of at least 5.1 of total scores, since it is comparable to typical developing controls improvement (Vicari et al., 2007). ;Timepoint(s) of evaluation of this end point: 3 months | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Secondary end-points will be clinical improvement after 3 months bumetanide treatment in the total scores of other episodic memory tasks (verbal and visual long term memory, associative memory) and in psychopathological measures as: standardized questionnaires and interviews as the Kiddie-Sads-Present and Lifetime Version (K-SADS-PL) (Kaufman et al., 2004), the Multidimensional Anxiety Scale For Children – MASC (March, 1997) and the The Conners 3rd Edition–Parent Parent Rating Scale–Revised: Long Version (CPRS-R:L;. Conners, 1997; Nobile, Alberti, & Zuddas, 2007).;Timepoint(s) of evaluation of this end point: After 3 months | — |
Countries
Italy
Contacts
Public ContactDr. Stefano Vicari
IRCCS Bambino Gesù Children's Hospital
Outcome results
None listed