CHAMPION: A Study to Evaluate the Efficacy and Safety of Obudanersen (ION582) in Children and Adults With Angelman Syndrome (AS)

Part of paid clinical trials in Carrboro, North Carolina.

Sponsor
Ionis Pharmaceuticals, Inc.
Study ID
NCT07782827
Phase
PHASE3
Status
Recruiting
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Conditions

  • Angelman Syndrome

Eligibility Criteria

Sex
ALL
Age
2 Years - 50 Years
Healthy Volunteers
Not accepted

Interventions

  • obudanersen — DRUG
    ION582 will be administered by IT injection.

Study Details

The primary purpose of the study is to evaluate efficacy of obudanersen in participants with AS due to uniparental disomy or imprinting defects (UPD/ID) as measured through expressive communication.

Key Dates

First listed
Aug 24, 2026
Start date
Sep 30, 2026
Status verified
Sep 2026
Primary completion
May 31, 2028
Completion
May 31, 2031

Study Design

Enrollment
30 participants (estimated)
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Experimental: obudanersen 80 mg
    Participants in Cohorts 1 and 2 will be administered obudanersen via intrathecal (IT) bolus injection every 12 weeks during the Treatment Evaluation and LTE periods.

Primary Outcome Measure

Change From Baseline in Performance on the Expressive Communication Domain Raw Score Without Caregiver Input of the Bayley Scales for Infant and Toddler Development-4 (Bayley-4) in Cohort 1 [ Time Frame: Baseline and Week 52 ]

Central Contacts

Locations (1)

FacilityCityStateZIPSite coordinators
University of North Carolina at Chapel Hill School of MedicineCarrboroNorth Carolina27510-

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By research site
University of North Carolina at Chapel Hill School of Medicine· Carrboro, NC

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