A Phase 3 interventional study of obudanersen in Angelman Syndrome, sponsored by Ionis Pharmaceuticals, Inc.. Not yet recruiting. Open to participants aged 2 Years to 50 Years. Per ClinicalTrials.gov, last updated 2026-08-24.
Sponsored by Ionis Pharmaceuticals, Inc. · Phase 3, Interventional, and Treatment
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 will be enrolled in Cohort 1 than in Cohort 2.
Key Inclusion Criteria:
Key Exclusion Criteria:
Other inclusion/exclusion criteria may apply
Participants in Cohorts 1 and 2 will be administered obudanersen via intrathecal (IT) bolus injection every 12 weeks during the Treatment Evaluation and LTE periods.
Drug: obudanersen
ION582 will be administered by IT injection.
Also known as: ION582
Change From Baseline in Performance on the Expressive Communication Domain Raw Score Without Caregiver Input of the Bayley Scales for Infant and Toddler Development-4 (Bayley-4) in Cohort 1
Time frame: Baseline and Week 52
Change From Baseline in Bayley-4: Cognition Scale Raw Score Without Caregiver Input
Time frame: Baseline and Week 52
Change From Baseline in Aberrant Behavior Checklist - Second Edition - Community Version (ABC-2-C): Hyperactivity
Time frame: Baseline and Week 52
Change From Baseline in (ABC-2-C): Irritability
Time frame: Baseline and Week 52
Change From Baseline in Vineland Adaptive Behavior Scale-3 (Vineland-3): Receptive Communication Domain Raw Score
Time frame: Baseline and Week 52
Change From Baseline in Vineland-3: Daily Living Skills, Personal Domain Raw Score
Time frame: Baseline and Week 52
Change From Baseline in (Bayley-4): Fine Motor Domain Raw Score Without Caregiver Input
Time frame: Baseline and Week 52
Change From Baseline in Observer-Reported Communication Ability (ORCA): Overall Emerging and Mastery T Score
Time frame: Baseline and Week 52
No study locations are listed for this record.
Plan to share: Yes — Ionis may share anonymized individual participant data, aggregated clinical data, and other types of data that support the results in this study. Data requests from qualified researchers will be considered once all three of the following criteria are met: (1) 12 months from marketing approval of the study drug in both the United States and European Union; (2) 18 months from conclusion of the study; and (3) 6 months from publication of study article. Access would be via a secure environment and is contingent upon approval of a research proposal and entry into an appropriate data use agreement. Requests to access data can be submitted via the website https://vivli.org/ourmember/ionis/.
No publications or documents are linked to this record.
This study is not yet recruiting, as verified in Aug 2026. You cannot join it, but the record below documents what was studied.
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Ionis Pharmaceuticals, Inc.