🏛️ Verified NIH Record • Updated 2026-07-27

BEACON - Phase III Clinical Study of Rugonersen in Angelman Syndrome. (NCT07605429)

🟢 Recruiting Phase3 💊 Investigational Drug / Biologic Sponsor: OHB Pediatrics Ltd.

🩺 Plain-English Summary (8th-Grade Level)

Purpose of the study is to evaluate the efficacy and safety of intrathecally administered rugonersen in pediatric and adult participants with Angelman syndrome.

Who can join: To join this study, participants generally need to meet the listed inclusion criteria (19 items) and avoid the listed exclusions (35 items).

Location: Rady Children's Hospital — San Diego, California

📋 "Do I Qualify?" — 1-Minute Self Screener

Instant Checklist

Check every box below. If you can check all of them, you may meet the study's basic screening requirements (final eligibility is confirmed by the site team).

Usually NOT eligible if any of the following apply:

  • Exclusion Criteria:
  • 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
  • Any concomitant condition that might interfere with the clinical evaluation of AS and that is not related to AS.
  • Known history of human immunodeficiency virus (HIV), hepatitis B, C, or E virus.
  • Any condition that increases the risk of meningitis.
  • History of bleeding diathesis or coagulopathy.

🔍 Extracted verbatim from the official NIH eligibility criteria. Always confirm with the study coordinator.

📍 All Participating Trial Locations (2 Sites)

  • Rady Children's Hospital — San Diego, California
  • University of North Carolina at Chapel Hill School of Medicine — Chapel Hill, North Carolina

💰 Cost, Insurance & Patient Rights

  • 100% Free Items: The investigational treatment and protocol-required procedures are supplied at zero cost by the sponsor.
  • Routine Patient Care: Under federal law (ACA § 2709) and many state statutes, routine care costs are covered during participation.
  • Voluntary Participation: You may withdraw at any time without affecting your standard medical care.
🏛️ View Official NIH Record ↗