First patient dosed in Ultragenyx Phase 3 Aspire study of GTX-102 for Angelman syndrome

Ultragenyx Pharmaceutical Inc.

Global Phase 3 Aspire study will enroll ~120 children ages 4-17 with full maternal UBE3A deletion.
Patients randomized 1:1 to GTX-102 intrathecal injections or sham comparator for 48-week primary analysis.
Primary endpoint: cognition improvement (Bayley-4 cognitive raw score); key secondary: Multi-domain Responder Index.
Active group receives three monthly 8 mg loading doses then quarterly maintenance up to 14 mg; sham crossover after Week 48.

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