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.
Angelman Syndrome
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.
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.
-
Rady Children's Hospital - San Diego, San Diego, California, United States, 92123
Rush Medical Center, Chicago, Illinois, United States, 60612
Boston Children's Hospital, Boston, Massachusetts, United States, 02115
Columbia University Medical Center, New York, New York, United States, 10032
Carolina Institute for Development DisabilitiesUniversity of North Carolina/School of Medicine, Carrboro, North Carolina, United States, 27510
Vanderbilt Children's Hospital, Nashville, Tennessee, United States, 37232-9119
Multicare Institute for Research and Innovation, Tacoma, Washington, United States, 98405
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
For general information about clinical research, read Learn About Studies.
5 Years to 17 Years
ALL
No
Hoffmann-La Roche,
Clinical Trials, STUDY_DIRECTOR, Hoffmann-La Roche
2025-11-04