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Assessing the efficacy of Bumetanide for the improvement of cognitive functions in children and adolescents with Down syndrome
EU CTIS ID: 2024-519342-71-00
What this study is testing
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.
- Therapeutic exploratory (Phase II)
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- 1) The presence of a free trisomy 21 documented by karyotyping 2) Adolescents from 10 to 17 years old (included) 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 each child and their parents.
You likely can't join if
- 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 as Glenn and/or Fontan) or who are under current treatment with cardiac medications. 5) The presence of a hypersensibility known about sulpha drugs 6) The presence of contraindications relative to the treatment by bumetanide 7) Patients already treated by diuretics 8) Any of the following abnormal laboratory values at screening: - Hemoglobin <10 g/dL - Abnormal liver function defined as any 2 or more of the following: ≥3 × upper limit of normal (ULN) aspartate aminotransferase (AST), ≥3 × ULN alanine aminotransferase (ALT), ≥3 × ULN gamma-glutamyl transpeptidase (GGT), ≥3 × ULN alkaline phosphatase (ALP), or ≥2 × ULN total bilirubin - Abnormal liver function defined as any increase of ≥5 × ULN AST or ALT - Estimated glomerular filtration rate ≤80 mL/min/1.73 m2 (calculated by the Schwartz equation) - Plasma HCO3 > 32 9) A 12-lead ECG demonstrating QTc >450 msec at Screening 10) Subject’s weight less than 25 Kg 11) Pregnancy as assessed by urine beta HCG
The study team makes the final eligibility decision.
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 0-17 years. The study team makes the final eligibility decision.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.