Efficacy of Bumetanide to Improve Cognitive Functions in Down Syndrome

Sponsor
Stefano Vicari
Study ID
NCT06465823
Phase
PHASE2
Status
Recruiting
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Conditions

  • Down Syndrome

Eligibility Criteria

Sex
ALL
Age
10 Years - 17 Years
Healthy Volunteers
Not accepted

Interventions

  • Bumetanide — DRUG
    Patients in the Bumetanide group will be treated for 3 months with a dose of 0.02 mg/kg twice a day, oral administration. It will be labeled (pre-printed and indistinguishable) with the randomization number and site number and will be delivered in separate blocks during the first, second and fourth appointment. The patients will start the treatment with half of the full target dose during the first week: * If the target dose is 0.5mg BID, only the morning dose will be administrated. * If the target dose is 1.0 mg BID the dose will be 0.5 mg BID. * If the target dose is 1.5mg BID, the morning dose will be 1.0 mg, the evening dose will be 0.5 mg. * If the target dose is 2.0 mg BID the dose will be 1.0 mg BID The patient will continue with the full dose starting from Visit 2 after 1 week of dosing.
  • Placebo — DRUG
    Patients in the control (placebo) group will be given placebo for 3 months twice a day, oral administration. The Placebo tablets will be visually indistinguishable from Bumetanide and packaged as Bumetanide. The placebo will be labeled (pre-printed and indistinguishable) with the randomisation number and site number and will be delivered in separate blocks during the first, second and fourth visits.

Study Details

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 found in preliminary studies in humans with other neurodevelopmental disorders (e.g., autism spectrum disorders). The drug Bumetanide could therefore be useful in counteracting the biological mechanisms that cause some cognitive deficits associated with Down syndrome. The potential of this therapeutic approach will be tested through a clinical trial in a population of children and adolescent patients with DS, in a randomized placebo-controlled trial with a three-month treatment with Bumetanide. Participants will be randomly assigned to the experimental group that will receive the treatment (Bumetanide) vs the control/comparison group that will receive the placebo. Bumetanide is a diuretic drug that has been widely used in humans in the past with few side effects, is orally active, and is very inexpensive. 64 participants will be recruited.

Key Dates

First listed
Jun 20, 2024
Start date
Jan 11, 2023
Status verified
Jun 2024
Primary completion
Dec 30, 2025
Completion
Sep 30, 2026

Study Design

Enrollment
64 participants (estimated)
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT

Arms

  • Experimental: Bumetanide
    Patients in the experimental group will receive the pharmacological treatment with Bumetanide
  • Placebo Comparator: Placebo
    Patients in the control group will receive the placebo

Primary Outcome Measure

Visual-Object long-term memory measures [ Time Frame: Change from baseline (day 1) to 3 month (day 90 ± 3), day 150 ± 4 ]

Central Contacts

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