Arbaclofen for Deletion Syndrome
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial tests a medication called arbaclofen to determine its safety and effectiveness for children and teens with 16p11.2 deletion syndrome, a genetic condition that often affects development and behavior. The trial aims to improve symptoms by comparing arbaclofen to a placebo (a pill with no active medication). Participants must have this genetic condition, receive educational or therapeutic support, and not have experienced seizures in the past 6 months. As a Phase 2 trial, the research focuses on measuring the treatment's effectiveness in an initial, smaller group of people.
Will I have to stop taking my current medications?
The trial requires that your current medication regimens be stable for 30 days before starting. You cannot start new medications or change your current ones during the study. Some specific medications, like antipsychotics and certain drugs with anxiolytic properties, are not allowed.
Is there any evidence suggesting that arbaclofen is likely to be safe for humans?
Research shows that people usually tolerate arbaclofen well. The FDA has already approved it for treating muscle stiffness, indicating its safety for that use. In studies examining its use for 16p11.2 deletion syndrome, no serious side effects were reported. However, some individuals might experience mild side effects like dizziness, tiredness, or nausea. Discuss any concerns with the trial team and your doctor.12345
Why do researchers think this study treatment might be promising?
Unlike the standard treatment options for Deletion Syndrome, which primarily focus on managing symptoms with behavioral therapy and medications like antipsychotics, arbaclofen introduces a novel approach by targeting the GABA-B receptor. This mechanism of action aims to improve social and communication difficulties by enhancing inhibitory neurotransmission in the brain. Researchers are excited about arbaclofen because it offers a more targeted way to address core symptoms of Deletion Syndrome, potentially leading to more effective and specific symptom relief compared to existing therapies.
What evidence suggests that arbaclofen might be an effective treatment for deletion syndrome?
Research has shown that arbaclofen, which participants in this trial may receive, may help treat conditions caused by certain genetic deletions. In earlier studies, arbaclofen improved thinking and memory problems in mice with 16p11.2 deletion syndrome. Other studies have found that arbaclofen can help with social challenges in conditions like autism and Fragile X syndrome, suggesting it might also benefit people with 16p11.2 deletions. The drug balances certain brain signals, potentially leading to better behavior and thinking. While more research is needed, these early results suggest arbaclofen could be helpful for those with 16p11.2 deletion syndrome.56789
Who Is on the Research Team?
Paul Wang, MD
Principal Investigator
Clinical Research Associates, LLC
Are You a Good Fit for This Trial?
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.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive either placebo or arbaclofen for 16 weeks with flexible dosing
Tapering
Participants taper off the study drug after the treatment period
Follow-up
Participants are monitored for safety and effectiveness after treatment
Open-label extension (optional)
Participants may opt into continuation of arbaclofen treatment long-term
What Are the Treatments Tested in This Trial?
Interventions
- Arbaclofen
- Placebo
Trial Overview
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.
How Is the Trial Designed?
2
Treatment groups
Experimental Treatment
Placebo Group
Arbaclofen will be dosed flexibly, with maximum permissible dose depending on age.
The placebo tablet is manufactured to match arbaclofen in shape, size, color, and taste, and will be administered in the same manner as arbaclofen.
Find a Clinic Near You
Who Is Running the Clinical Trial?
Clinical Research Associates, LLC
Lead Sponsor
Citations
1.
allospharma.com
allospharma.com/allos-pharma-acquires-global-rights-to-arbaclofen-reports-positive-asd-trial-results-and-grant-award/Allos Pharma Acquires Global Rights to Arbaclofen ...
2 deletion syndrome. It has been administered to over 700 children, adolescents, and adults with ASD and FXS in previous trials and demonstrated ...
Towards Preclinical Validation of Arbaclofen (R-baclofen) ...
Arbaclofen rescued cognitive deficits seen in two 16p11.2 deletion mouse lines in traditional recognition memory paradigms. Using an ...
Neurodevelopmental outcome in 22q11.2 deletion ...
The 22q11.2 deletion syndrome (22q11.2 DS) places affected individuals at an increased risk for neurodevelopmental/cognitive, behavioral and social–emotional ...
4.
thetransmitter.org
thetransmitter.org/spectrum/going-on-trial-arbaclofen-reboot-cell-implants-psilocybin-microdoses/Going on Trial: Arbaclofen reboot; cell implants; psilocybin ...
Children with 22q11.2 deletion syndrome who received the transdermal cannabidiol gel Zygel showed significant improvement on multiple ...
5.
researchgate.net
researchgate.net/publication/309217763_Arbaclofen_in_Children_and_Adolescents_with_Autism_Spectrum_Disorder_A_Randomized_Controlled_Phase_2_TrialArbaclofen in Children and Adolescents with Autism ...
A phase 2 randomized, placebo-controlled, crossover trial found that the GABA-B agonist arbaclofen improved social avoidance symptoms in FXS. A ...
22q11.2 Deletion Syndrome - GeneReviews® - NCBI - NIH
Individuals with 22q11.2 deletion syndrome (22q11.2DS) can present with a wide range of features that are highly variable, even within families.
DiGeorge syndrome (22q11.2 deletion syndrome)
This condition is due to missing part of chromosome 22. It may cause heart issues, thyroid problems, cleft palate, lower immunity and other ...
Safety, Tolerability, and Efficacy of Arbaclofen in 16p11.2 ...
This Phase 2 study examines the safety, tolerability, and efficacy of arbaclofen in pediatric subjects with 16p11.2 deletion. Male or female subjects aged 5 ...
DiGeorge Syndrome - StatPearls - NCBI Bookshelf
DiGeorge syndrome (DGS) is a congenital disorder with a broad phenotypic presentation, which results predominantly from the microdeletion of chromosome 22.
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