AFQ056 for Language Learning in Children With FXS

NCT02920892 ↗

Phase 2 Completed 110 enrolled Elizabeth Berry-Kravis National Institute of Neurological Disorders and Stroke (NINDS) · collab Novartis Pharmaceuticals · collab

RandomizedParallel-groupDouble-blindTreatment

Summary

The purpose of this clinical trial is to investigate the impact of AFQ056 on language learning in 3-6 year old children with Fragile X Syndrome (FXS).

Timeline

Start: 2017-08-17
Primary completion: 2022-05-17
Completion: 2022-05-17

Outcome

Missed primary endpoint

registry p = 0.1587; Slope -0.1 (90% CI -0.22 to 0.02); Mixed Models Analysis NCT02920892 ↗

paper These findings indicate that AFQ056 does not provide benefits as assessed by primary or secondary endpoints. PMID 39483619 ↗

Publications

Protic D, Breeze E, Mendoza G, Zafarullah M, Abbeduto L, Hagerman R, Coffey C, Cudkowicz M, Durbin-Johnson B, Ashwood P, Berry-Kravis E, Erickson CA, Filipink R, Gropman A, Lehwald L, Maxwell-Horn A, Morris S, Bennett AP, Prock L, Talboy A, Tartaglia N, Veenstra-VanderWeele J, Tassone F. Negative effect of treatment with mGluR5 negative allosteric modulator AFQ056 on blood biomarkers in young individuals with Fragile X syndrome. SAGE Open Med. 2024 Sep 29;12:20503121241282401. doi: 10.1177/20503121241282401. eCollection 2024.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Mavoglurant	Small molecule	12.5 mg	Oral
Subject	Mavoglurant	Small molecule	100 mg	Oral