Safety, Tolerability, and Efficacy of Arbaclofen in 16p11.2 Deletion

Summary

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 to 17 years of age will be randomized to receive either placebo or arbaclofen in a double-blind study design. If the subject completes all study requirements through Visit 4 (Close-out Visit), he/she may be eligible for an optional open-label study with arbaclofen.

Timeline

Start

2022-09-01

Primary completion

2024-12-22

Completion

2025-03-23

Publications

• Rea HM, Mattson JT, Arutiunian V, Verdes A, Bhattachan S, Santhosh M, Garcia A, Ajwani A, Bowen M, Faja S, Hanson E, Pawlowski K, Kochel RP, Prock L, Veenstra-VanderWeele J, Walton-Bowen K, Vogel A, Morgan A, Webb SJ, Wang PP. Rationale and study design for the first precision medicine randomized placebo-controlled trial in the 16p11.2 deletion syndrome. Expert Rev Neurother. 2026 Jun;26(6):597-607. doi: 10.1080/14737175.2026.2655767. Epub 2026 May 4.

Drugs

Indications