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

Part of paid clinical trials in San Diego, California.

Sponsor
OHB Pediatrics Ltd.
Study ID
NCT07605429
Phase
PHASE3
Status
Recruiting
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Conditions

  • Angelman Syndrome

Eligibility Criteria

Sex
ALL
Age
1 Year - 50 Years
Healthy Volunteers
Not accepted

Interventions

  • rugonersen — DRUG
    Study Drug
  • Sham procedure — PROCEDURE
    Sham Procedure

Study Details

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

Key Dates

First listed
May 26, 2026
Start date
Jun 30, 2026
Status verified
Jul 2026
Primary completion
Mar 31, 2029
Completion
Mar 31, 2031

Study Design

Enrollment
165 participants (estimated)
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT

Arms

  • Experimental: rugonersen
    Study Drug
  • Sham Comparator: Sham
    Sham Procedure

Primary Outcome Measure

Change from baseline in the Bayley-4 cognition and/or expressive communication raw scores without caregiver input at Week 56. [ Time Frame: Baseline to week 56 ]

Central Contacts

Locations (2)

FacilityCityStateZIPSite coordinators
Rady Children's HospitalSan DiegoCalifornia92123
Lynne Bird (PRINCIPAL_INVESTIGATOR)
University of North Carolina at Chapel Hill School of MedicineChapel HillNorth Carolina27510
Elizabeth Jalazo (PRINCIPAL_INVESTIGATOR)

Find similar trials in San Diego, CA

By research site
Rady Children's Hospital· San Diego, CAUniversity of North Carolina at Chapel Hill School of Medicine· Chapel Hill, NC

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