REVEAL: A Phase 3 Study of Obudanersen (ION582) in Angelman Syndrome

Part of paid clinical trials in Los Angeles, California.

Sponsor
Ionis Pharmaceuticals, Inc.
Study ID
NCT06914609
Phase
PHASE3
Status
Recruiting
Apply to this trial

Conditions

  • Angelman Syndrome

Eligibility Criteria

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

Interventions

  • obudanersen — DRUG
    obudanersen will be administered by IT injection.
  • Placebo — DRUG
    obudanersen matching placebo will be administered by IT injection.

Study Details

The purpose of this study is to evaluate the efficacy and safety of obudanersen in children and adults with Angelman syndrome caused by a deletion or mutation of the UBE3A gene.

Key Dates

First listed
Apr 6, 2025
Start date
Jun 10, 2025
Status verified
Aug 2026
Primary completion
Aug 31, 2027
Completion
Apr 30, 2030

Study Design

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

Arms

  • Experimental: Cohort 1 obudanersen 80 mg
    Participants (aged 2 to \<18 years old) will be administered obudanersen 80 mg via intrathecal (IT) injection every 12 weeks during the double blind and LTE treatment periods.
  • Placebo Comparator: Cohort 1 Placebo
    Participants (aged 2 to \<18 years old) will be administered obudanersen matching placebo via IT injection every 12 weeks during the double-blind treatment period and then administered obudanersen 40 mg or 80 mg every 12 weeks during the LTE treatment period.
  • Experimental: Cohort 2 obudanersen 80 mg
    Participants (aged 18 to ≤50 years old) will be administered obudanersen 80 mg via IT injection every 12 weeks during the double blind and LTE treatment periods.
  • Placebo Comparator: Cohort 2 Placebo
    Participants (aged 18 to ≤50 years old) will be administered obudanersen matching placebo via IT injection every 12 weeks during the double-blind treatment period and then randomized to obudanersen 40 mg or 80 mg every 12 weeks during the LTE treatment period.

Primary Outcome Measure

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

Central Contacts

Locations (18)

FacilityCityStateZIPSite coordinators
Cedars-Sinai Medical CenterLos AngelesCalifornia90048-
UCLA Clinical & Translational Research Center (CTRC)Los AngelesCalifornia90095-
Rady Children's HospitalSan DiegoCalifornia92123-
Colorado Children's Hospital Research InstituteAuroraColorado80045-
Children's National HospitalWashington D.C.District of Columbia20011-
Nicklaus Children's HospitalMiamiFlorida33155-
Children's Healthcare of AtlantaAtlantaGeorgia30329-
Rush University Medical CenterChicagoIllinois60612-
Boston Children's HospitalBostonMassachusetts02115-
Children's MercyKansas CityMissouri64108-
Columbia University Irving Medical CenterNew YorkNew York10032-
Ichan School of Medicine at Mount SinaiNew YorkNew York10029-
University of North Carolina at Chapel Hill School of MedicineCarrboroNorth Carolina27510-
Cincinnati Children's HospitalCincinnatiOhio45229-
Nationwide Children's HospitalColumbusOhio43205-
Children's Hospital of PhiladelphiaPhiladelphiaPennsylvania19104-
Vanderbilt Clinical Research CenterNashvilleTennessee37232-
Texas Children's HospitalHoustonTexas77030-

Find similar trials in Los Angeles, CA

By research site
Cedars-Sinai Medical Center· Los Angeles, CAUCLA Clinical & Translational Research Center (CTRC)· Los Angeles, CARady Children's Hospital· San Diego, CAColorado Children's Hospital Research Institute· Aurora, COChildren's National Hospital· Washington D.C., DCNicklaus Children's Hospital· Miami, FL

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