CHAMPION: A Study to Evaluate the Efficacy and Safety of Obudanersen (ION582) in Children and Adults With Angelman Syndrome (AS)
NCT07782827 · Status: NOT_YET_RECRUITING · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 30
Last updated 2026-08-24
Summary
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.
Conditions
Interventions
- DRUG
-
obudanersen
ION582 will be administered by IT injection.
Sponsors & Collaborators
-
Ionis Pharmaceuticals, Inc.
lead INDUSTRY
Study Design
- Allocation
- NA
- Purpose
- TREATMENT
- Masking
- NONE
- Model
- SINGLE_GROUP
Eligibility
- Min Age
- 2 Years
- Max Age
- 50 Years
- Sex
- ALL
- Healthy Volunteers
- No
Timeline & Regulatory
- Start
- 2026-09-30
- Primary Completion
- 2028-05-31
- Completion
- 2031-05-31
- FDA Drug
- Yes
More Related Trials
-
Clinical Procedures to Support Research in ALS
NCT03489278 ·Status: RECRUITING
-
Valproic Acid in Ambulant Adults With Spinal Muscular Atrophy
NCT00481013 ·Status: COMPLETED ·Phase: PHASE2
-
Clinical Trial of Sodium Phenylbutyrate in Children With Spinal Muscular Atrophy Type I
NCT00439218 ·Status: TERMINATED ·Phase: PHASE1/PHASE2
-
Clinical Trial of Sodium Phenylbutyrate in Children With Spinal Muscular Atrophy Types II or III
NCT00439569 ·Status: TERMINATED ·Phase: PHASE1/PHASE2
-
A Phase III Trial of Lorenzo's Oil in Adrenomyeloneuropathy
NCT00545597 ·Status: TERMINATED ·Phase: PHASE3
-
Phase II Clinical Trial of Clenbuterol in Adult Patients With Pompe Disease
NCT04094948 ·Status: WITHDRAWN ·Phase: PHASE2
-
Long-term Safety, Tolerability and Efficacy of Idebenone in Duchenne Muscular Dystrophy (DELPHI Extension)
NCT00758225 ·Status: COMPLETED ·Phase: PHASE2
-
Adults With SMA Treated With Nusinersen
NCT04591678 ·Status: COMPLETED
-
A Multi-Center Controlled Screening Trial of Safety and Efficacy of Lithium Carbonate in Subjects With Amyotrophic Lateral Sclerosis (ALS)
NCT00790582 ·Status: COMPLETED ·Phase: PHASE2
-
Investigation of Individualised Antisense Oligonucleotides (ASOs) in People With Unique Genetic Variants Causing Severely Debilitating, Life Threatening (SDLT) Central Nervous System (CNS) Conditions
NCT07410143 ·Status: ACTIVE_NOT_RECRUITING ·Phase: PHASE1/PHASE2
-
Long-term Follow-up Study of Patients Receiving Onasemnogene Abeparvovec-xioi
NCT04042025 ·Status: ACTIVE_NOT_RECRUITING ·Phase: PHASE3
-
Exploratory Study of NS-065/NCNP-01 in DMD
NCT02081625 ·Status: COMPLETED ·Phase: PHASE1
-
Clenbuterol to Target DUX4 in FSHD
NCT06721299 ·Status: RECRUITING ·Phase: PHASE1
-
Study of Safety and Dosing Effect on SMN Levels of Valproic Acid (VPA) in Patients With Spinal Muscular Atrophy
NCT00374075 ·Status: COMPLETED ·Phase: PHASE1
-
Clinical Trial of SB-509 in Subjects With Amyotrophic Lateral Sclerosis (ALS)
NCT00748501 ·Status: COMPLETED ·Phase: PHASE2
-
A Study to Learn About the Effect of Nusinersen (BIIB058) Given as Injections to Children With Spinal Muscular Atrophy (SMA) Who Were Previously Treated With Onasemnogene Abeparvovec (RESPOND)
NCT04488133 ·Status: COMPLETED ·Phase: PHASE4
-
Study to Evaluate Sodium Phenylbutyrate in Pre-symptomatic Infants With Spinal Muscular Atrophy
NCT00528268 ·Status: COMPLETED ·Phase: PHASE1/PHASE2
-
Phase1 First in Human Ascending Dose Study to Evaluate the Safety and Tolerability of FC-12738 in Health Adults
NCT05978908 ·Status: COMPLETED ·Phase: PHASE1
-
Study to Assess the Safety, Tolerability, and Efficacy of Viltolarsen in Ambulant and Non-Ambulant Boys With DMD (Galactic53)
NCT04956289 ·Status: COMPLETED ·Phase: PHASE2
-
Phase IIIb, Open-label, Multi-center Study to Evaluate Safety, Tolerability and Efficacy of OAV101 Administered Intrathecally to Participants With SMA Who Discontinued Treatment With Nusinersen or Risdiplam
NCT05386680 ·Status: COMPLETED ·Phase: PHASE3
-
A Study of the Safety, Tolerability & Efficacy of Long-term Administration of Drisapersen in US & Canadian Subjects
NCT01803412 ·Status: TERMINATED ·Phase: PHASE3
-
Study to Determine the Safety and Tolerability of Varenicline (Chantix®) in Treating Spinocerebellar Ataxia Type 3
NCT00992771 ·Status: COMPLETED ·Phase: PHASE2
-
Metabolomics of Children With SMA
NCT04587492 ·Status: COMPLETED
-
A Real-World Medical Chart Review of Spinal Muscular Atrophy Patients Treated With Onasemnogene Abeparvovec in Saudi Arabia
NCT07737756 ·Status: NOT_YET_RECRUITING
-
A Study to Learn About Salanersen's (BIIB115) Effects on Movement and Its Safety When Given Before Symptoms Appear in Babies With Genetically Diagnosed Spinal Muscular Atrophy (SMA)
NCT07221669 ·Status: RECRUITING ·Phase: PHASE3