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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
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to 52 weeks
Awards & highlights

Study 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.Check my eligibility
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.See study design
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
Select...
I have a genetic condition with extra copies of a specific chromosome segment.
Select...
My weight is at or above the third percentile for my age.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to 52 weeks
This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 52 weeks for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Vineland-3 adaptive behavior composite scores
Secondary outcome measures
Aberrant Behavior Checklist - Second Edition - Community Version (ABC-2-C) domain scores
Abnormal changes in EEG recordings compared to baseline with a focus on treatment-emergent epileptiform abnormalities
Apparent clearance (CL/F) of basmisanil
+31 more

Side effects data

From 2019 Phase 2 trial • 214 Patients • NCT02953639
6%
Headache
6%
Diarrhoea
6%
Arthralgia
2%
Fatigue
100%
80%
60%
40%
20%
0%
Study treatment Arm
Basmisanil 80mg BID
Basmisanil 240mg BID
Placebo

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
2022
Completed Phase 2
~230

Find a Location

Who is running the clinical trial?

Hoffmann-La RocheLead Sponsor
2,428 Previous Clinical Trials
1,089,047 Total Patients Enrolled
Clinical TrialsStudy DirectorHoffmann-La Roche
2,199 Previous Clinical Trials
888,495 Total Patients Enrolled

Media Library

Basmisanil (GABAA Receptor Modulator) Clinical Trial Eligibility Overview. Trial Name: NCT05307679 — Phase 2
Dup15q Syndrome Research Study Groups: Placebo, Basmisanil
Dup15q Syndrome Clinical Trial 2023: Basmisanil Highlights & Side Effects. Trial Name: NCT05307679 — Phase 2
Basmisanil (GABAA Receptor Modulator) 2023 Treatment Timeline for Medical Study. Trial Name: NCT05307679 — Phase 2

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Are there currently any open spots in this experimental research?

"Per the clinicaltrials.gov listing, this trial is actively searching for test subjects. This research project was first posted on December 16th 2022 and has undergone its most recent revision as of June 12th 2023."

Answered by AI

How many individuals have participated in this clinical research?

"Indeed, the information hosted on clinicaltrials.gov reveals that this trial is currently recruiting participants. Originally posted on December 16th 2022 and last updated June 12th 2023, 90 individuals need to be recruited from 10 centres for this medical study."

Answered by AI

Is the age limit for participating in this research experiment above 25 years old?

"The specified age range for this trial is between 2 and 11 years old. Currently, 380 other trials are looking to recruit minors while an additional 1008 clinical studies are recruiting seniors over 65."

Answered by AI

Is there a possibility for me to participate in this medical experiment?

"This research seeks 90 young people, aged 2-11, with a maternal duplication or triplication of chromosome 15q11.2-Q13.1 that marks them as having Dup15q syndrome. The participants must have an overall severity score on the Dup15q Clinical Global Impression Severity scale (CGI-S) of at least 4 and be above the third percentile for their age in terms of body weight. Moreover, they need to come accompanied by a legal guardian who is over 18 years old, speaks the local language fluently, can reliably assess the subject’s condition and regularly attend all clinic visits;"

Answered by AI

What are the potential risks associated with Basmisanil use?

"According to our experts at Power, Basmisanil is considered a moderate safety risk with a rating of 2. This assessment takes into account the fact that Phase 2 trials have yielded some evidence for its security but there are no studies which demonstrate efficacy."

Answered by AI

Are there numerous facility sites engaging in this research within city limits?

"IRCCS Eugenio Medea, U.O. di Epilessia e Neurofisiologia clinica in Conegliano Veneto (TV), Hospital Sant Joan De Deu in Esplugues De Llobregas, and Evelina London Children's Hospital are among the 10 sites currently running this trial."

Answered by AI
Recent research and studies
clinicaltrials.govStudy
A Study to Evaluate the Safety and Efficacy of Basmisanil Treatment in Children Aged 2-14 Years With Dup15q Syndrome
2022. "A Study to Evaluate the Safety and Efficacy of Basmisanil Treatment in Children Aged 2-14 Years With Dup15q Syndrome". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT05307679.
~3 spots leftby Apr 2025
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