A Study to Investigate the Pharmacokinetics (PK) and Safety and to Provide Proof of Mechanism of Alogabat in Children and Adolescents Aged 5-17 Years With Angelman Syndrome (AS) With Deletion Genotype.

Phase 2 Completed 48 enrolled Hoffmann-La Roche

Non-randomizedSequentialOpen-labelTreatment

Summary

This is a two-part, Phase IIa, multicenter, 12-week, open-label study. Up to 56 participants with deletion AS aged 5-17 years (inclusive) will be enrolled in the study.

Timeline

Start: 2023-07-27
Primary completion: 2025-12-02
Completion: 2025-12-02

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Alogabat	Small molecule	—	—

A Study to Investigate the Pharmacokinetics (PK) and Safety and to Provide Proof of Mechanism of Alogabat in Children and Adolescents Aged 5-17 Years With Angelman Syndrome (AS) With Deletion Genotype.

Summary

Timeline

Drugs

Indications