Phase 3 Study of the Efficacy and Safety of ION582 in Children and Adults With Angelman Syndrome
Ionis Pharmaceuticals, Inc.
Summary
The purpose of this study is to evaluate the efficacy and safety of ION582 in children and adults with Angelman syndrome caused by a deletion or mutation of the UBE3A gene.
Description
This is a Phase 3, randomized, double-blind, placebo-controlled study in people with Angelman syndrome. The study will consist of 4 periods: a screening period of up to 28 days, an approximate 60-week double blind, placebo-controlled treatment period, followed by an approximate 25-month Long-Term Extension (LTE) treatment period, and an approximate 8-month Post-LTE follow-up period. The study will be comprised of 2 cohorts. Cohort 1 will include pediatric participants, aged 2 to less than (\<)18 years old and serve as the population for evaluation of primary and secondary outcome measures; Coh…
Eligibility
- Age range
- 2–50 years
- Sex
- All
- Healthy volunteers
- No
Key Inclusion Criteria: 1. The participants caregiver(s)/ legally authorized representative must have given written informed consent and any authorizations required by local law and be able to comply with all study requirements. 2. Medically stable and can undergo sedation and/or general anesthesia without intubation. 3. Male or female between 2 and lesser than or equal to (≤)50 years of age, depending on specific cohort, at the time of the in-clinic Screening visit. 4. Participant has a clinical diagnosis of Angelman syndrome (AS) with molecular confirmation of either Ubiquitin-protein ligas…
Interventions
- DrugION582
ION582 will be administered by IT injection.
- DrugPlacebo
ION582 matching placebo will be administered by IT injection.
Locations (38)
- Cedars-Sinai Medical CenterLos Angeles, California
- Rady Children's HospitalSan Diego, California
- Colorado Children's Hospital Research InstituteAurora, Colorado
- Children's National HospitalWashington D.C., District of Columbia
- Nicklaus Children's HospitalMiami, Florida
- Children's Healthcare of AtlantaAtlanta, Georgia