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rugonersen

Phase 3

Angelman Syndrome | Small molecule | Other |Medpace Holdings, Inc. Common Stock|Last Updated: Sep 10, 2026

Success Probability

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Market & Valuation

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Trial Design

RandomizedDouble-BlindSHAM_CONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment165

FDA Designations

No designations recorded

Clinical trial landscape

rugonersen · 1 trial · 1 indication

Phase 3 1
NCT07605429BEACON - Phase III Clinical Study of Rugonersen in Angelman Syndrome.Angelman Syndrome
RECRUITING165 Analytics
PHASE3RECRUITING
BEACON - Phase III Clinical Study of Rugonersen in Angelman Syndrome.
Angelman Syndrome
NCT07605429165
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Study Endpoints

Primary Endpoints

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

The Bayley-4 is a performance-based assessment of developmental functioning of five core battery scales: cognitive, language (two subtests: expressive and receptive communication), motor (two subtests: gross and fine motor), social-emotional, and adaptive behavior. Change from baseline in the raw scores of the cognition and/or expressive communication scales of the Bayley-4, without caregiver input, higher change reflects a better outcome of the core scales.

Secondary Endpoints

Symptoms of Angelman Syndrome - Clinician Global Impression of Change (SAS-CGI-C) overall at Week 56.
Week 56
Change from baseline in electroencephalogram (EEG) delta-band power at Week 56.
Week 56
Incidence of serious adverse events (SAEs).
Week 60
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Study Design & Arms

AllocationRANDOMIZED
MaskingQUADRUPLE
ModelPARALLEL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
rugonersenEXPERIMENTALStudy Drug
ShamSHAM_COMPARATORSham Procedure

Interventions

NameTypeDescription
rugonersenDRUGStudy Drug
Sham procedurePROCEDURESham Procedure
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Eligibility Criteria

Age Range1 Year to 50 Years
SexALL
Healthy VolunteersNo
Study Sites3

Inclusion Criteria: * 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 I...

Countries:United States
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