BEACON - Phase III Clinical Study of Rugonersen in Angelman Syndrome.
Public ClinicalTrials.gov record NCT07605429. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Official title
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
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
Purpose of the study is to evaluate the efficacy and safety of intrathecally administered rugonersen in pediatric and adult participants with Angelman syndrome.
Study identification
- NCT ID
- NCT07605429
- Recruitment status
- Recruiting
- Study type
- Interventional
- Phase
- Phase 3
- Enrollment
- 165 participants
Conditions and interventions
Conditions
Eligibility (public fields only)
- Age range
- 1 Year to 50 Years
- Sex
- All
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Jun 23, 2026 (Actual)
- Primary completion
- Mar 2029 (Estimated)
- Completion
- Mar 2031 (Estimated)
- Last update posted
- Oct 20, 2026
2026 – 2031
United States locations
- U.S. sites
- 5
- U.S. states
- 5
- U.S. cities
- 5
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| Rady Children's Hospital | San Diego | California | 92123 | Recruiting |
| Nicklaus Children's Hospital | Miami | Florida | 33155 | Recruiting |
| Rush University | Chicago | Illinois | 60612 | Recruiting |
| University of North Carolina at Chapel Hill School of Medicine | Chapel Hill | North Carolina | 27510 | Recruiting |
| Vanderbilt University Medical Center - Monroe Carell Jr. Children's Hospital | Nashville | Tennessee | 37232 | Recruiting |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
Non-U.S. locations
This page focuses on the U.S. directory. The official record also lists 1 non-U.S. site.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT07605429, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Oct 20, 2026 · Synced Oct 28, 2026
Related: full search, browse by condition, browse by drug or therapy, browse by sponsor, browse by U.S. city.
Open the official record
The complete public registry record, listed eligibility criteria, and available contact information for NCT07605429 live on ClinicalTrials.gov.