A phase 2 double blind placebo controlled study on the efficacy of Bumetanide for cognitive improvement in children and adolescents with Down syndrome

Conditions

Down Syndrome

Brief summary

The primary end-point will be the improvement at the Visual-Object learning test, defined as the increase of 22% in the mean total score occurred after three months of treatment. This improvement is considered clinically significant since the value is similar to the improvement in the healthy population (22,6%), considering the test-retest reliability at three months (Vicari et al., 2007).

Detailed description

Secondary end-points will be significant higher improvement in the bumetanide group compared to the placebo group after 3 months in the total scores and subdomain scores of the other episodic memory tasks (Verbal and Visual-Spatial long-term memory, associative memory); the executive function measures (Behavior Rating Inventory Executive Function Second Edition questionnaire – parent report form - BRIEF2), the psychopathological measures, the adaptive level and quality of life

Costanzo F, Mongiovì VM, Alfieri P, Pontrelli G, Abbate S, De Palma L, Chinali M, Massella L, Scorpecci A, Rocchi F, Fucà E, Averna R, Rava L, Rossetti S, Franco L, Contestabile A, Cancedda L, Vicari S.

2026-02-27

10.1136/bmjopen-2025-112826

Interventions

DRUGPlacebo of Burinex tablet

DRUGBurinex 1 mg tabletter

Eligibility

Sex/Gender

All

Age

0 Years to 17 Years

Design outcomes

Primary

Measure	Time frame
The primary end-point will be the improvement at the Visual-Object learning test, defined as the increase of 22% in the mean total score occurred after three months of treatment. This improvement is considered clinically significant since the value is similar to the improvement in the healthy population (22,6%), considering the test-retest reliability at three months (Vicari et al., 2007).	—

Secondary

Measure	Time frame
Secondary end-points will be significant higher improvement in the bumetanide group compared to the placebo group after 3 months in the total scores and subdomain scores of the other episodic memory tasks (Verbal and Visual-Spatial long-term memory, associative memory); the executive function measures (Behavior Rating Inventory Executive Function Second Edition questionnaire – parent report form - BRIEF2), the psychopathological measures, the adaptive level and quality of life	—

Measure

Time frame

Secondary end-points will be significant higher improvement in the bumetanide group compared to the placebo group after 3 months in the total scores and subdomain scores of the other episodic memory tasks (Verbal and Visual-Spatial long-term memory, associative memory); the executive function measures (Behavior Rating Inventory Executive Function Second Edition questionnaire – parent report form - BRIEF2), the psychopathological measures, the adaptive level and quality of life

—

Countries

Italy

Outcome results

None listed