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.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
30
ION582 will be administered by IT injection.
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
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