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GABAA Receptor Modulator

Basmisanil for Dup15q Syndrome

Phase 2

Waitlist Available

Research Sponsored by Hoffmann-La Roche

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Documented maternal duplication (3 copies) or triplication (4 copies) of the chromosome 15q11.2-q13.1 region that includes the Prader Willi/Angelman critical region defined as [BP2-BP3] segment

Body weight equal to or above the third percentile for age

Must not have

Significant risk for suicidal behavior, as assessed through the suicidal behavior question adapted from the Columbia Classification Algorithm for Suicide Assessment (C-CASA) (participants ≥ 6 years of age only)

Concomitant use of prohibited medications

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 5 years

Summary

This trial will test if a medicine can help improve the symptoms of Dup15q syndrome in children aged 2-11.

Who is the study for?

This trial is for children aged 2-14 with Dup15q syndrome, who are at least moderately ill. They must have a specific genetic duplication or triplication and weigh in the normal range for their age. A caregiver must consent, be involved in treatment compliance, and use electronic devices for home assessments.

What is being tested?

The study tests Basmisanil's safety and effectiveness over 52 weeks against placebo in treating Dup15q syndrome by modulating GABAA receptor function. It includes an optional two-year extension to assess long-term benefits.

What are the potential side effects?

While not specified here, potential side effects may include reactions related to the central nervous system due to GABAA receptor modulation such as changes in mood or behavior, sleep disturbances, gastrointestinal issues, or allergic reactions.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I have a genetic condition with extra copies of a specific chromosome segment.

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My weight is at or above the third percentile for my age.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I am at significant risk for suicidal behavior.

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I am not taking any medications that are not allowed in the study.

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My epilepsy is not under control.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 5 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to 5 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 5 years for reporting.

Treatment Details

Side effects data

From 2019 Phase 2 trial • 214 Patients • NCT02953639

Headache

Arthralgia

Diarrhoea

Fatigue

100%

80%

60%

40%

20%

Study treatment Arm

Placebo

Basmisanil 80mg BID

Basmisanil 240mg BID

Trial Design

2Treatment groups

Experimental Treatment

Placebo Group

Group I: BasmisanilExperimental Treatment1 Intervention

Participants will receive oral basmisanil twice daily (BID) on the first day of treatment, then three times per day (TID) until the end of Part 1 of the trial (Day 365) or the end of Part 2 (Day 1095)

Group II: PlaceboPlacebo Group1 Intervention

Participants will receive oral placebo BID on the first day of treatment, then TID until the end of Part 1 of the trial (Day 365).

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Basmisanil

2015

Completed Phase 2

~220

0 / 5Eligibility criteria met