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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. — Aldebaran  

Angelman Syndrome Clinical Trial

Official title:
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

Clinical Trial Summary

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

Clinical Trial Description

The study will have Part 1-dose confirmations and Part 2 with dose levels to be decided based on the cumulative PK, 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 second dose), and at the end of the 12-week treatment period after daily administration of Alogabat. ;

Study Design

Related Conditions & MeSH terms

Clinical Trial Details
NCT number NCT05630066
Study type Interventional
Source Hoffmann-La Roche
Contact Reference Study ID Number: BP41315 https://forpatients.roche.com
Phone 888-662-6728 (U.S. Only)
Email global-roche-genentech-trials@gene.com
Status Recruiting
Phase Phase 2
Start date July 27, 2023
Completion date September 12, 2025
View Details
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