RecruitingAngelman Syndrome
BEACON - Phase III Clinical Study of Rugonersen in Angelman Syndrome.
Eligible age
1–50 yrs
Accepts
All genders
Locations
3 states
Healthy volunteers
No
See if you qualify for this study
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About this study
Purpose of the study is to evaluate the efficacy and safety of intrathecally administered rugonersen in pediatric and adult participants with Angelman syndrome.
Sponsor: OHB Pediatrics Ltd.
You may qualify if…
- ✓ 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 electronic form as needed,
- ✓ Is, and will likely remain, sufficiently knowledgeable of participant's condition throughout the study to be able to respond to queries, and is willing and able to complete caregiver assessments and inform the site personnel about the participant's condition as requested.
- ✓ Clinical diagnosis of Angelman syndrome.
- ✓ Pre-existing medical records confirm the clinical diagnosis of AS and the molecular diagnosis with genotypic classification of either:
You may not qualify if…
- ✕ Molecular diagnosis of AS with genotypic classification of:
- ✕ 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.
- ✕ Clinically significant vital signs or laboratory abnormalities during screening, including:
- ✕ o Abnormal coagulation profile demonstrated by platelet count at or below lower limit of normal (140 × 109/L), or by abnormal international normalized ratio (INR) and/or prothrombin time (PT), or activated partial thromboplastin time (aPTT).
- ✕ 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.
- ✕ Clinically relevant disease or condition, including hematological, hepatic, cardiac or renal disease or abnormality, that would, in the judgement of the Investigator, pose an unacceptable risk to the participant or interfere with the conduct of the study
Where it's recruiting
Source: ClinicalTrials.gov · NCT07605429 · last updated 2026-09-10