REVEAL: A Phase 3 Study of 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

  • ION582 — DRUG
    ION582 will be administered by IT injection.
  • Placebo — DRUG
    ION582 matching placebo will be administered by IT injection.

Study Details

The purpose of this study is to evaluate the efficacy and safety of ION582 in children and adults with Angelman syndrome caused by a deletion or mutation of the UBE3A gene.

Key Dates

Start date
Jun 10, 2025
Status verified
May 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 ION582 80 mg
    Participants (aged 2 to \<18 years old) will be administered ION582 80 mg via IT injection Q12W during the double blind and LTE treatment periods.
  • Placebo Comparator: Cohort 1 Placebo
    Participants (aged 2 to \<18 years old) will be administered ION582 matching placebo via IT injection Q12W during the double-blind treatment period and then administered ION582 40 mg or 80 mg Q12W during the LTE treatment period.
  • Experimental: Cohort 2 ION582 80 mg
    Participants (aged 18 to ≤50 years old) will be administered ION582 80 mg via IT injection Q12W during the double blind and LTE treatment periods.
  • Placebo Comparator: Cohort 2 Placebo
    Participants (aged 18 to ≤50 years old) will be administered ION582 matching placebo via IT injection Q12W during the double-blind treatment period and then randomized to ION582 40 mg or 80 mg Q12W 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 (15)

FacilityCityStateZIPSite coordinators
Cedars-Sinai Medical CenterLos AngelesCalifornia90048-
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-
Ichan School of Medicine at Mount SinaiNew YorkNew York10029-
University of North Carolina at Chapel Hill School of MedicineCarrboroNorth Carolina27510-
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, 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, FLChildren's Healthcare of Atlanta· Atlanta, GA

Related Studies