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

NCT05307679 · Status: TERMINATED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 7

Last updated 2025-12-12

Study results available
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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

DRUG

Basmisanil

Participants will receive oral basmisanil at age-appropriate dosages

DRUG

Placebo

Participants will receive oral placebo

Sponsors & Collaborators

Principal Investigators

  • Clinical Trials · Hoffmann-La Roche

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
DOUBLE
Model
PARALLEL

Eligibility

Min Age
2 Years
Max Age
14 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2022-12-16
Primary Completion
2024-03-04
Completion
2024-03-04
FDA Drug
Yes

Countries

  • United States
  • Spain
  • United Kingdom

Study Locations

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Entities

Drugs
Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT05307679 on ClinicalTrials.gov