A Randomized, Multi-center, Double-blind, Sham-controlled, Phase III Clinical Study to Evaluate the Efficacy and Safety of Intrathecally Administered Rugonersen in Pediatric and Adult Participants With Angelman Syndrome
OHB Pediatrics Ltd.
Summary
Purpose of the study is to evaluate the efficacy and safety of intrathecally administered rugonersen in pediatric and adult participants with Angelman syndrome.
Description
This study is a Phase III, randomized, double-blind, sham-controlled, multi center study designed to evaluate the efficacy and safety of intrathecally (IT) administered rugonersen compared with sham in up to 165 participants with AS (Part 1), followed by an open-label extension (OLE) of approximately 116 weeks (2 years) for long-term evaluation of the efficacy and safety of IT administered rugonersen in participants with AS (Part 2).
Eligibility
- Age range
- 1–50 years
- Sex
- All
- Healthy volunteers
- No
Inclusion Criteria: * Male or female and ≥ 1 year to ≤ 50 years of age at signing of the informed consent form. * Independent of the age of the participant, the participant has a parent, caregiver or legal representative (herein after referred to as caregiver) who is reliable and competent in the Investigator's judgement. The caregiver is: * Able to consent for the participant according to ICH and local regulations, * At least 18 years of age, * Willing and able to accompany the participant to clinic visits and be available to the investigational site by telephone, email, or other elec…
Interventions
- Drugrugonersen
Study Drug
- ProcedureSham procedure
Sham Procedure
Locations (2)
- Rady Children's HospitalSan Diego, California
- University of North Carolina at Chapel Hill School of MedicineChapel Hill, North Carolina