NCT05307679

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

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

• Status: Terminated
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 7 (actual)
• Sponsor: Hoffmann-La Roche · Industry
• Sex: All
• Age: 2 Years – 14 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Dup15q Syndrome

Interventions

Type	Name	Description
DRUG	Basmisanil	Participants will receive oral basmisanil at age-appropriate dosages
DRUG	Placebo	Participants will receive oral placebo

Timeline

• Start date: 2022-12-16
• Primary completion: 2024-03-04
• Completion: 2024-03-04
• First posted: 2022-04-01
• Last updated: 2025-12-12
• Results posted: 2025-12-12

Locations

5 sites across 3 countries: United States, Spain, United Kingdom

Regulatory

• FDA-regulated drug study