Arbaclofen for Deletion Syndrome

Recruiting at 3 trial locations

Overseen By Karen Walton-Bowen

Age: < 18

Sex: Any

Trial Phase: Phase 2

Sponsor: Clinical Research Associates, LLC

Must be taking: Antiepileptics

Must not be taking: Antipsychotics, Anxiolytics

Disqualifiers: Genetic disorders, Neurologic conditions, others

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

Duration: 16 weeks

60 Participants Needed

Trial Summary

What is the purpose of this trial?

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.

Research Team

Paul Wang, MD

Principal Investigator

Clinical Research Associates, LLC

Eligibility Criteria

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

I have a 16p11.2 BP4-BP5 deletion diagnosis.

My medications have not changed in the last 30 days.

The subject's parent/caregiver/LAR must be able to speak and understand English sufficiently to understand the nature of the study and to allow for the completion of all study assessments. The parent/caregiver/LAR should be capable of providing reliable information about the subject's condition, agree to oversee the administration of the study drug, and accompany the subject to all clinic visits. The same parent/caregiver/LAR should accompany the subject to each visit

Exclusion Criteria

I am currently taking more than 2 medications for mental health, not counting as-needed sleep aids.

I have been treated with baclofen in the last 30 days.

I cannot or do not want to take medicine that dissolves in the mouth.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive either placebo or arbaclofen for 16 weeks with flexible dosing

16 weeks

Multiple visits (in-person), multiple phone communications

Tapering

Participants taper off the study drug after the treatment period

2-4 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Open-label extension (optional)

Participants may opt into continuation of arbaclofen treatment long-term

Treatment Details

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.

Participant Groups

Group I: Arbaclofen Experimental Treatment 1 Intervention

Arbaclofen will be dosed flexibly, with maximum permissible dose depending on age.

Group II: Placebo Placebo Group 1 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.

Who Is Running the Clinical Trial?

Clinical Research Associates, LLC

Lead Sponsor

Trials

2

Recruited

90+