Status: Not yet recruiting
BEACON - Phase III Clinical Study of Rugonersen in Angelman Syndrome.
Purpose of the study is to evaluate the efficacy and safety of intrathecally administered rugonersen in pediatric and adult participants with Angelman syndrome.
Participants needed: 165
Trial details
Phase: Phase 3Age: 1-50Biological sex: AllType: InterventionalSponsor: OHB Pediatrics Ltd.Updated: Jun 4, 2026
Eligibility criteria
Male or female and ≥ 1 year to ≤ 50 years of age at signing of the informed cons... [+16]
Uniparental paternal disomy (UPD) of 15q11-q13, [+30]