NCT05630066

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.

A Phase IIa Multicenter, Open-label, 12-week Study to Investigate the Pharmacokinetics 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

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 48 (actual)
• Sponsor: Hoffmann-La Roche · Industry
• Sex: All
• Age: 5 Years – 17 Years
• Healthy volunteers: Not accepted

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.

Detailed description

The study will have Part 1-dose confirmations and Part 2 with dose levels to be decided based on the cumulative PK, electroencephalography (EEG), and safety data emerging from Part 1. The dose levels for the first cohort of Part 2 will be decided based on the cumulative PK, EEG, and safety data emerging from Part 1. Part 2 will explore the change in EEG beta-band power relative to baseline at Week 2, Week 4 (i.e., approximately 2 weeks after the start of the Dose B), and at the end of the 12-week treatment period after daily administration of alogabat.

Conditions

• Angelman Syndrome

Interventions

Type	Name	Description
DRUG	Alogabat	Alogabat will be administered QD with dose depending on cohort and age of the participant.

Timeline

• Start date: 2023-07-27
• Primary completion: 2025-12-02
• Completion: 2025-12-02
• First posted: 2022-11-29
• Last updated: 2025-12-04

Locations

19 sites across 6 countries: United States, Australia, France, Germany, Italy, Spain

Regulatory

• FDA-regulated drug study