BEACON - Phase III Clinical Study of Rugonersen in Angelman Syndrome.
ConditionAngelman Syndrome
Trial statusNot yet recruiting
Trial phasePhase 3
Trial typeInterventional
Biological sexAll
Age1-50
SponsorOHB Pediatrics Ltd.
About this trial
Purpose of the study is to evaluate the efficacy and safety of intrathecally administered rugonersen in pediatric and adult participants with Angelman syndrome.
Eligibility criteria
Qualifiers
Male or female and ≥ 1 year to ≤ 50 years of age at signing of the informed consent form.
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 electronic form as needed,
Disqualifiers
Uniparental paternal disomy (UPD) of 15q11-q13,
Imprinting center defect (ICD) within 15q11-q13,
A partial molecular diagnosis of AS, that cannot exclude UPD or ICD despite appropriate genetic testing.
Presence of clinically relevant electrocardiogram (ECG) abnormalities prior to dosing such as QT interval corrected for heart rate using Fredericia's formula (QTcF) > 460 ms, personal or family history of congenital long QT syndrome indicating safety risk in the Investigator's opinion. First-degree atrioventricular block or isolated right bundle branch block is allowed.
Trial design
Treatments tested in this trial
- rugonersen
- Sham procedure
Treatment groups
165 Participants
are divided into 2 treatment groupsLocations
Sponsors and collaborators
OHB Pediatrics Ltd.
Lead sponsor
Medpace, Inc.
Collaborator
Source ClinicalTrials.gov