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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.

NCT05630066 · Status: COMPLETED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 48

Last updated 2025-12-04

No results posted yet for this study

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.

Conditions

Interventions

DRUG

Alogabat

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

Sponsors & Collaborators

Principal Investigators

  • Clinical Trials · Hoffmann-La Roche

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SEQUENTIAL

Eligibility

Min Age
5 Years
Max Age
17 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2023-07-27
Primary Completion
2025-12-02
Completion
2025-12-02
FDA Drug
Yes

Countries

  • United States
  • Australia
  • France
  • Germany
  • Italy
  • Spain

Study Locations

More Related Trials

Entities

Diseases
Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT05630066 on ClinicalTrials.gov