CompletedPhase 2

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.

United States48 participantsStarted 2023-07-27

Plain-language 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.

Who can participate

Age range5 Years – 17 Years

SexALL

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Inclusion criteria

✓Male participants: Male contraception is not required in this study because of the minimal seminal dose transmitted through sexual intercourse

What they're measuring

Part 1: Age-group Based Ratio of Plasma PK Parameter, Area Under the Concentration-time Curve (AUC)

Timeframe: Up to 12 Weeks

Part 1: Age-group Based Ratio of Plasma PK Parameter, Apparent Clearance (CL/F)

Timeframe: Up to 12 Weeks

Part 2: Change From Baseline to Week 2, 4, and 12 in Resting State EEG Power in the Beta Band

Timeframe: Week 2, 4, and 12

Trial details

NCT IDNCT05630066

SponsorHoffmann-La Roche

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2025-12-02

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