Your session is about to expire
← Back to Search
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 belowSelect...
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 weeks3 visits
Treatment ~ Varies
Follow Up ~ up to 52 weeks
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to 52 weeks
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 moreSide effects data
From 2019 Phase 2 trial • 214 Patients • NCT029536396%
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
2015
Completed Phase 2
~220
Find a Location
Who is running the clinical trial?
Hoffmann-La RocheLead Sponsor
2,434 Previous Clinical Trials
1,091,385 Total Patients Enrolled
Clinical TrialsStudy DirectorHoffmann-La Roche
2,202 Previous Clinical Trials
890,029 Total Patients Enrolled
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- I am at significant risk for suicidal behavior.I am not allergic to the study's drugs or severely lactose intolerant.I am not taking any medications that are not allowed in the study.I have a genetic condition with extra copies of a specific chromosome segment.My weight is at or above the third percentile for my age.I haven't had cancer, except for certain skin cancers, in the last 5 years.My current medications have been stable for at least 6 weeks, and if I'm on anti-epileptic treatment, it's been stable for 8 weeks.My epilepsy is not under control.I haven't changed my non-drug therapies in the last 6 weeks and won't change them during the study.I haven't been in a drug study recently.I haven't had a serious infection or been hospitalized for one in the last 6 weeks.My caregiver lives with me and can help with my treatment and clinic visits.
Research Study Groups:
This trial has the following groups:- Group 1: Placebo
- Group 2: Basmisanil
Awards:
This trial has 0 awards, including:Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
Other Trials to Consider
Popular Categories
Share this study with friends
Copy Link
Messenger