Placebo-Controlled, Single-Dose Challenge Study of Gaboxadol in Adult Males With Fragile X Syndrome (FXS)

Summary

This is a single dose, placebo-controlled study. Male subjects aged 18 to 40 years (inclusive) with a diagnosis of FXS. Eligible subjects may enroll in this study comprised of two in home and two in clinic visits each 14 days apart, for a total of four visits. Subjects will be given single dose gaboxadol (10 mg) or matched placebo at each of these visits to take orally. Thus, all enrolled subjects will receive placebo at home and in clinic and receive gaboxadol at home and in clinic in a blinded fashion.

Timeline

Start

2024-01-29

Primary completion

2025-05-13

Completion

2025-06-24

Publications

• De Stefano LA, Kim H, Erickson CA, Schmitt LM, Dominick KC, Pedapati EV, Huckle R, Wilson R, McKinney WS, Reisinger DL, Nelson MN, Dapore AE, Horn PS, Miyakoshi M. Gaboxadol increases resting theta and alpha power without affecting evoked responses in fragile X syndrome in a home-based setting. J Neurodev Disord. 2026 May 8;18(1):36. doi: 10.1186/s11689-026-09700-5.

Drugs

Indications