Ultragenyx Pharmaceutical Inc. today announced that the Phase 3 Aspire study evaluating GTX-102 (apazunersen) as a treatment for Angelman Syndrome is fully enrolled, with approximately 129 participants ages four to 17 with a genetically confirmed diagnosis of full maternal UBE3A gene deletion.
“The accelerated enrollment of the Phase 3 Aspire study underscores the urgent need and strong desire for an effective treatment for these patients. Support from the Angelman syndrome community was critical to the achievement of this important milestone for GTX-102 with completion of enrollment in seven months. We are grateful to the study site teams, investigators, and families for their dedication and support,” said Eric Crombez, M.D., chief medical officer at Ultragenyx.
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