← Back to Search

GABA-B Agonist

Arbaclofen for Deletion Syndrome

Phase 2
Recruiting
Research Sponsored by Clinical Research Associates, LLC
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Diagnosis of 16p11.2 BP4-BP5 deletion
All medication regimens must be stable for 30 days prior to Screening
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 12 weeks
Awards & highlights

Study Summary

This trial is testing a medication to see if it is safe and effective for children with a certain gene deletion.

Who is the study for?
This trial is for children aged 5-17 with a genetic condition called 16p11.2 deletion syndrome, who need educational or therapeutic support and have stable medication regimens. They must not be planning to change treatments during the study, can't be on certain medications like antipsychotics or more than two psychoactive drugs, and should not have had seizures recently.Check my eligibility
What is being tested?
The trial tests arbaclofen against a placebo in kids with deletion syndrome to see if it's safe and works well. It's randomized and double-blind, meaning neither the researchers nor participants know who gets the real drug versus placebo until after the results are collected.See study design
What are the potential side effects?
While specific side effects of arbaclofen aren't listed here, similar muscle relaxants can cause drowsiness, dizziness, weakness, trouble sleeping or breathing issues. The severity varies from person to person.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
Select...
I have a 16p11.2 BP4-BP5 deletion diagnosis.
Select...
My medications have not changed in the last 30 days.
Select...
I am between 5 and 17 years old.
Select...
I have been seizure-free for 6 months on stable medication or 3 years without medication.
Select...
I am not pregnant and will use birth control during and after the trial.
Select...
I haven't changed my treatment or diet for my condition in the last 2 months.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~12 weeks
This trial's timeline: 3 weeks for screening, Varies for treatment, and 12 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
Goldman-Fristoe Test of Articulation, 3rd edition (GFTA-3), Sounds-in-Words
Secondary outcome measures
Bruininks-Oseretsky Test - 2nd edition (BOT-2), Fine Motor Control and Body Coordination subtests
Differential Ability Scale, 2nd edition (DAS-II)
Wide Range Assessment of Memory and Learning - 2nd edition (WRAML2)

Side effects data

From 2019 Phase 3 trial • 536 Patients • NCT03290131
31%
Urinary Tract Disorder
24%
Muscular Weakness
22%
Nausea
15%
Dizziness
13%
Asthenia
11%
Somnolence
8%
Vomiting
3%
Vertigo
2%
Headache
2%
Fatigue
1%
Contusion
1%
Hip Fracture
1%
Pneumothorax
1%
Restless Leg Syndrome
1%
Status Epilepticus
1%
Delirium
1%
Pulmonary Embolism
1%
Gait Disturbance
1%
Multiple Sclerosis Relapse
100%
80%
60%
40%
20%
0%
Study treatment Arm
AERT 40 mg
AERT 80 mg
Placebo

Trial Design

2Treatment groups
Experimental Treatment
Placebo Group
Group I: ArbaclofenExperimental Treatment1 Intervention
Arbaclofen will be dosed flexibly, with maximum permissible dose depending on age.
Group II: PlaceboPlacebo Group1 Intervention
The placebo tablet is manufactured to match arbaclofen in shape, size, color, and taste, and will be administered in the same manner as arbaclofen.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Arbaclofen
Not yet FDA approved

Find a Location

Who is running the clinical trial?

Clinical Research Associates, LLCLead Sponsor
1 Previous Clinical Trials
25 Total Patients Enrolled
Paul Wang, MDStudy DirectorClinical Research Associates, LLC

Media Library

Arbaclofen (GABA-B Agonist) Clinical Trial Eligibility Overview. Trial Name: NCT04271332 — Phase 2
Deletion Syndrome Research Study Groups: Arbaclofen, Placebo
Deletion Syndrome Clinical Trial 2023: Arbaclofen Highlights & Side Effects. Trial Name: NCT04271332 — Phase 2
Arbaclofen (GABA-B Agonist) 2023 Treatment Timeline for Medical Study. Trial Name: NCT04271332 — Phase 2

Frequently Asked Questions

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

To whom does this experimental procedure extend?

"This study is for 5-17 year olds that have 16p11.2 deletion syndrome. They are looking for a maximum of 60 patients in total."

Answered by AI

Are recruitment efforts for this trial still ongoing?

"That is correct, the online information does show that this study is looking for more participants. The 60 individuals needed for the trial will be recruited from 3 different locations, and recruitment started on September 1st, 2020 according to the most recent update on August 25th, 2020."

Answered by AI

Are there any harmful side-effects associated with Arbaclofen?

"Arbaclofen is a Phase 2 trial medication, meaning that while there is data suggesting it is safe, there is no evidence yet of the drug's efficacy. Our team rates its safety at a 2."

Answered by AI

Are octogenarians welcome in this trial?

"Children aged 5 to 17 can be enrolled in this trial, as long as they meet the other entrance requirements. There are a total of 4 different trials: 3 for patients under 18 and 1 for those over 65."

Answered by AI

Is Arbaclofen a common medication in research trials?

"As of now, there are 3 ongoing clinical trials for Arbaclofen with 1 trial in Phase 3. While many of the trials for Arbaclofen are located in Houston, Texas, there are 10 locations operating clinical trials for Arbaclofen globally."

Answered by AI

How many people are subjects in this experiment?

"That is accurate. The clinicaltrials.gov website contains information suggesting that this study, which was originally published on September 1st 2022, is still recruiting patients. They are looking for a total of 60 individuals from 3 different locations."

Answered by AI
~18 spots leftby Dec 2024