Efficacy of Bumetanide to Improve Cognitive Functions in Down Syndrome
The aim of the study is to evaluate the clinical efficacy of a known diuretic drug, Bumetanide, in terms of improvement of memory and psychological functioning in children and adolescents with Down syndrome (DS), in order to develop therapeutic strategies for cognitive and psychopathology aspects associated with the sy
| Condition(s) | Down Syndrome |
|---|---|
| Status | Recruiting |
| Phase | Phase 2 |
| Study type | Interventional |
| Summary | The aim of the study is to evaluate the clinical efficacy of a known diuretic drug, Bumetanide, in terms of improvement of memory and psychological functioning in children and adolescents with Down syndrome (DS), in order to develop therapeutic strategies for cognitive and psychopathology aspects associated with the syndrome. The study also aims to identify possible predictors and biological and genetic markers related to the efficacy of the treatment. Recently, preliminary studies conducted on the animal model of Down syndrome have proven the efficacy of the drug Bumetanide in counteracting some brain anomalies related to communication between nerve cells (synaptic transmission) typical of the syndrome, with the effect of improving memory skills. Behaviour-enhancing effects have also been |
| Who can participate | Principal inclusion criteria 1. The presence of a free trisomy 21 documented by karyotyping 2. Adolescents from 10 to 17 years old (included) 3. 3 4.5 ≥ Mental age ≤ 8.5 (as assessed by Leiter-3 at visit 1 or by assessment with Leiter-3 within 6 months of the first visit (Visit 1) 4. Informed consent from their parents and assent from child/adolescent Principal 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 electrolyte disorders; 4. The presence of clinically and/or hemodynamically significant congenital heart defects, defined as patients with congenital heart disease who already underwent or are awaiting surgical/percutaneous correction (including palliative cardiac surgery |
| Ages | 10 Years to 17 Years |
| Sex | All |
| Lead sponsor | Stefano Vicari |
| Locations | Rome, Italy |
| Start date | 2023-01-11 |
| NCT ID | NCT06465823 |
| Official listing | https://clinicaltrials.gov/study/NCT06465823 |