Efficacy, Safety, Tolerability, and Pharmacokinetics of NBI-827104 in Pediatric Subjects With Epileptic Encephalopathy With Continuous Spike-and-Wave During Sleep

Summary

This is a phase 2, double-blind study to assess the efficacy, safety, tolerability, and pharmacokinetics of NBI-827104 when administered once daily for 13 weeks in pediatric subjects with Epileptic Encephalopathy with Continuous Spike-and-Wave During Sleep (EECSWS).

Timeline

Start

2021-04-26

Primary completion

2022-08-08

Completion

2022-10-11

Outcome

Missed primary endpoint

registry analysis (superiority test); NBI-827104 vs Placebo; p = 0.4326; ANCOVA NCT04625101 ↗

release “While the blinded study did not meet the primary endpoint, ACT-709478 was generally well tolerated” idorsia.com ↗

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	APINOCALTAMIDE	Small molecule	—	Oral

Indications