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Not yet recruitingNCT07782827Updated Aug 24, 2026

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

A Phase 3 interventional study of obudanersen in Angelman Syndrome, sponsored by Ionis Pharmaceuticals, Inc.. Not yet recruiting. Open to participants aged 2 Years to 50 Years. Per ClinicalTrials.gov, last updated 2026-08-24.

Sponsored by Ionis Pharmaceuticals, Inc. · Phase 3, Interventional, and Treatment

Phase
Phase 3
Study type
Interventional
Enrollment
30
Allocation
Not applicable
Ages
2 Years to 50 Years
Sex
All
01

Study 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.

Read the detailed description

This is a Phase 3, open-label, single treatment arm, multi-center study in participants with Angelman syndrome due to paternal uniparental disomy or imprinting defects. The study will consist of 4 periods: a screening period of up to 28 days, an approximate 60-week Treatment Evaluation Period, followed by 101 weeks of Long-Term Extension (LTE) period, and an 8-month Post-Treatment Follow-up Period. There will be two age-based Cohorts enrolled in this study: Cohort 1 (pediatric participants (aged 2 to \<18 years old)) and Cohort 2 (adult participants (aged 18 to ≤50 years old). More individuals will be enrolled in Cohort 1 than in Cohort 2.

02

Conditions studied

  • Angelman Syndrome

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Keywords

  • Paternal Uniparental Disomy
  • ION582
  • imprinting defects
03

Who can participate

Ages eligible
2 Years to 50 Years
Sexes eligible
All
Accepts healthy volunteers
No

Eligibility criteria

Key Inclusion Criteria:

  1. Clinical diagnosis of AS with molecular result indicating either paternal UPD of 15q11.2-q13 or ID of the maternal 15q11.2-q13 region, provided by the Investigator and confirmed by either a qualified central vendor or a qualified local geneticist at the site.
  2. The participant's caregiver(s)/legally-authorized representative (LAR) must have given written informed consent and any authorizations required by local law and be able and willing to comply with all study requirements.
  3. Medically stable and can undergo sedation and/or general anesthesia without intubation.
  4. Male or female between 2 and ≤ 50 years of age, depending on the specific cohort, at the time of the in-clinic Screening visit.
  5. If applicable, is currently receiving stable doses of concomitant medications typically prescribed for AS, such as anti-epileptic medication, behavioral management medications, sleep medications, gabapentin, cannabidiol, and special diets, supplements, or nutritional support for at least 8 weeks prior to the Baseline visit. If recent changes (\< 8 weeks stable) in medications, the participant may be allowed per Investigator judgment if the change is not expected to have an impact on the signs and symptoms of AS.
  6. LAR/caregiver(s) agree(s) not to post any of the participant's personal medical data or information related to the study on any website or social media site (e.g., Facebook, Instagram, X, YouTube, TikTok, WhatsApp) from the time of enrollment until they are notified that the study is completed.

Key Exclusion Criteria:

  1. Participant has a clinical diagnosis of AS with molecular confirmation of a UBE3A deletion or UBE3A mutation.
  2. Any clinically significant abnormalities in medical history (e.g., major surgery within 3 months of Screening), or on physical examination for which treatment with an antisense oligonucleotide (ASO) would be contraindicated or which, in the opinion of the Investigator, could confound the results of this study.
  3. Known brain or spinal disease that would interfere with the lumbar puncture (LP) procedure, cerebrospinal fluid (CSF) circulation, or presence of other factors that would affect the safety of the LP procedure, including tumors or abnormalities by MRI or computed tomography (CT), subarachnoid hemorrhage, suggestion of raised intracranial pressure (ICP) on magnetic resonance imaging (MRI) or ophthalmic examination, Chiari malformation, obstructive hydrocephalus, syringomyelia, tethered spinal cord syndrome, or connective tissue disorders such as Ehlers-Danlos syndrome and Marfan syndrome.
  4. Any laboratory abnormalities or any other clinically significant abnormalities that would, as assessed by the Investigator, at Screening or Baseline, render a participant unsuitable for inclusion.
  5. Previous treatment with an oligonucleotide (including small interfering ribonucleic acid [siRNA] and ASOs) or gene therapy or gene editing. This exclusion criterion does not apply to approved nucleic acid-based vaccines, including messenger Ribonucleic Acid (mRNA) vaccines, which are allowed.

Other inclusion/exclusion criteria may apply

04

Study design

Phase
Phase 3
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrollment
30 participants (estimated)

Study 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.

    Drug: obudanersen

Interventions

  • Drugobudanersen

    ION582 will be administered by IT injection.

    Also known as: ION582

05

What researchers measure

Primary outcomes

  1. 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

Secondary outcomes

  1. Change From Baseline in Bayley-4: Cognition Scale Raw Score Without Caregiver Input

    Time frame: Baseline and Week 52

  2. Change From Baseline in Aberrant Behavior Checklist - Second Edition - Community Version (ABC-2-C): Hyperactivity

    Time frame: Baseline and Week 52

  3. Change From Baseline in (ABC-2-C): Irritability

    Time frame: Baseline and Week 52

  4. Change From Baseline in Vineland Adaptive Behavior Scale-3 (Vineland-3): Receptive Communication Domain Raw Score

    Time frame: Baseline and Week 52

  5. Change From Baseline in Vineland-3: Daily Living Skills, Personal Domain Raw Score

    Time frame: Baseline and Week 52

  6. Change From Baseline in (Bayley-4): Fine Motor Domain Raw Score Without Caregiver Input

    Time frame: Baseline and Week 52

  7. Change From Baseline in Observer-Reported Communication Ability (ORCA): Overall Emerging and Mastery T Score

    Time frame: Baseline and Week 52

06

Study locations

No study locations are listed for this record.

07

References and documents

Individual participant data

Plan to share: Yes — Ionis may share anonymized individual participant data, aggregated clinical data, and other types of data that support the results in this study. Data requests from qualified researchers will be considered once all three of the following criteria are met: (1) 12 months from marketing approval of the study drug in both the United States and European Union; (2) 18 months from conclusion of the study; and (3) 6 months from publication of study article. Access would be via a secure environment and is contingent upon approval of a research proposal and entry into an appropriate data use agreement. Requests to access data can be submitted via the website https://vivli.org/ourmember/ionis/.

No publications or documents are linked to this record.

08

Registry details

Key details

Study ID
NCT07782827
Lead sponsor
Ionis Pharmaceuticals, Inc.
Responsible party
Sponsor
First posted
Aug 24, 2026
Start date
Sep 2026 (estimated)
Primary completion
May 2028 (estimated)
Completion
May 2031 (estimated)
Last update
Aug 24, 2026

Study contacts

Ionis Pharmaceuticals, Inc.
Contact
Champion@clinicaltrialmedia.com
(844) 373-1141

Oversight

Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

Not currently enrolling

This study is not yet recruiting, as verified in Aug 2026. You cannot join it, but the record below documents what was studied.

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