A randomised, multi-center, double-blind, sham-controlled, parallel-group, phase III clinical study to evaluate the efficacy and safety of intrathecally administered rugonersen in pediatric and adult participants with Angelman syndrome.

Condition studied

Angelman syndrome

Investigational medicinal product(s)

Eligibility

Primary endpoint

Change from baseline in the Bayley-4 cognition and/or expressive communication raw scores without caregiver input at Week 56.

Endpoint detail

1. Symptoms of Angelman Syndrome - Clinician Global Impression of Change (SAS-CGI-C) overall at Week 56., 2. Change from baseline in electroencephalogram (EEG) deltaband power at Week 56., 3. Change from baseline in the Bayley-4 fine motor raw score without caregiver input at Week 56., 4. Change from baseline in the Bayley-4 receptive communication raw score without caregiver input at Week 56., 5. Change from baseline in Vineland-3 raw score for the gross motor domain at Week 56., 6. Incidence of serious adverse events (SAEs)., 7.Incidence of neurological AEs of National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) v5.0 grade 3, 4, or 5., 8. Incidence of fever of National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) v5.0 grade 3, 4, or 5.