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GABA-B Agonist

Arbaclofen for Deletion Syndrome

Phase 2
Waitlist Available
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
Male or female subjects, 5 through 17 years of age, at Screening
Must not have
Subjects currently treated with more than 2 psychoactive medications, including antiepileptics used as an anti-seizure treatment, but not including sleep aids used on an as-needed basis
Subjects who are not able or willing to take oral disintegrating tablets
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 12 weeks

Summary

This trial tests arbaclofen, a medication, in children and teenagers aged 5 to 17 with the 16p11.2 genetic deletion. The goal is to see if it can improve speech and other brain functions by enhancing communication between brain cells.

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.
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.
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
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I have a 16p11.2 BP4-BP5 deletion diagnosis.
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I am between 5 and 17 years old.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
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I am currently taking more than 2 medications for mental health, not counting as-needed sleep aids.
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I cannot or do not want to take medicine that dissolves in the mouth.
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I am currently taking vigabatrin, tiagabine, or riluzole.
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I have a genetic disorder in addition to the 16p11.2 BP4-BP5 deletion.
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I am currently taking medication for a psychiatric condition.
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I haven't had seizures in the last 6 months and am not on antiepileptics.

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

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Goldman-Fristoe Test of Articulation, 3rd edition (GFTA-3), Sounds-in-Words
Secondary study objectives
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%
Fatigue
2%
Headache
1%
Gait Disturbance
1%
Delirium
1%
Pulmonary Embolism
1%
Pneumothorax
1%
Contusion
1%
Multiple Sclerosis Relapse
1%
Status Epilepticus
1%
Hip Fracture
1%
Restless Leg Syndrome
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
~2 spots leftby Dec 2024
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