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TERMINATED
NCT05307679
PHASE2

A Study to Evaluate the Safety and Efficacy of Basmisanil Treatment in Children Aged 2-14 Years With Dup15q Syndrome

Sponsor: Hoffmann-La Roche

View on ClinicalTrials.gov

Summary

This study consists of two parts. Part 1 will evaluate the safety, efficacy, and pharmacodynamics of 52-weeks of basmisanil treatment in children and adolescents (aged 2-14 years) with Dup15q syndrome. Part 1 will test the hypothesis that negative allosteric modulation of a GABAA receptor subtype can address excessive receptor function and positively impact core neurodevelopmental disease feature in individuals with Dup15q syndrome. Part 2 is an optional 2-year open-label extension to evaluate long-term safety, tolerability, and to provide supportive evidence of benefit of continued treatment with basmisanil in selected efficacy outcomes.

Official title: A Phase II, Randomized, Double-Blind, Placebo-Controlled, Parallel Group Study to Evaluate the Safety, Efficacy, and Pharmacodynamics of 52 Weeks of Treatment With Basmisanil in Participants Aged 2 to 14 Years Old With Dup15q Syndrome Followed by a 2-Year Optional Open-Label Extension

Key Details

Gender

All

Age Range

2 Years - 14 Years

Study Type

INTERVENTIONAL

Enrollment

7

Start Date

2022-12-16

Completion Date

2024-03-04

Last Updated

2026-08-18

Healthy Volunteers

No

Conditions

Interventions

DRUG

Basmisanil

Participants will receive oral basmisanil at age-appropriate dosages

DRUG

Placebo

Participants will receive oral placebo

Locations (5)

Children's Hospital Los Angeles

Los Angeles, California, United States

Rush University Medical Center

Chicago, Illinois, United States

Hospital Sant Joan De Deu

Esplugues de Llobregas, Barcelona, Spain

Hospital Universitario La Paz

Madrid, Spain

Evelina London Children's Hospital

London, United Kingdom