Ionis Pharmaceuticals, Inc.
The primary purpose of the study is to evaluate efficacy of obudanersen in participants with AS due to uniparental disomy or imprinting defects (UPD/ID) as measured through expressive communication.
This is a Phase 3, open-label, single treatment arm, multi-center study in participants with Angelman syndrome due to paternal uniparental disomy or imprinting defects. The study will consist of 4 periods: a screening period of up to 28 days, an approximate 60-week Treatment Evaluation Period, followed by 101 weeks of Long-Term Extension (LTE) period, and an 8-month Post-Treatment Follow-up Period. There will be two age-based Cohorts enrolled in this study: Cohort 1 (pediatric participants (aged 2 to \<18 years old)) and Cohort 2 (adult participants (aged 18 to ≤50 years old). More individuals…
Key Inclusion Criteria: 1. Clinical diagnosis of AS with molecular result indicating either paternal UPD of 15q11.2-q13 or ID of the maternal 15q11.2-q13 region, provided by the Investigator and confirmed by either a qualified central vendor or a qualified local geneticist at the site. 2. The participant's caregiver(s)/legally-authorized representative (LAR) must have given written informed consent and any authorizations required by local law and be able and willing to comply with all study requirements. 3. Medically stable and can undergo sedation and/or general anesthesia without intubation…
ION582 will be administered by IT injection.
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