At a glance
- What the study gets you
- Access to the study treatment being tested
- Type of study
- Interventional (receives a drug or procedure)
- Time in hospital
- In-person visits at study sites — visit count not specified by the sponsor
- Drug or intervention
- obudanersen (drug), Placebo (drug)
- How long the study runs
- Study runs about 58 months (dates as stated)
- About the drug or intervention
- obudanersen — drug: obudanersen will be administered by IT injection. · Placebo — drug: obudanersen matching placebo will be administered by IT injection.
- Patient visit burden
- Not specified by the sponsor
- Type of study
- Testing a treatment
- Ages
- 2 Years to 50 Years
- Who
- All
- Number of participants
- 156
- Started
- 2025-06-10
- Last checked
- 2026-09
Plain English Summary
What is this study?
- • Testing a new treatment for angelman syndrome
- • Phase3 - 156 participants
- • 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
Who can take part?
- • Ages 2 Years to 50 Years
- • Diagnosed with angelman syndrome
Where?
- • London - Great Ormond Street Hospital for Children - NHS Foundation Trust
- • Oxford - John Radcliffe Hospital
This is a simplified summary. Always discuss with your doctor before making any decisions.
About This Trial
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.
More detail
This is a Phase 3, randomized, double-blind, placebo-controlled study in people with Angelman syndrome. The study will consist of 4 periods: a screening period of up to 28 days, an approximate 60-week double blind, placebo-controlled treatment period, followed by an approximate 25-month Long-Term Extension (LTE) treatment period, and an approximate 8-month Post-LTE follow-up period. The study will be comprised of 2 cohorts. Cohort 1 will include pediatric participants, aged 2 to less than (\<)18 years old and serve as the population for evaluation of primary and secondary outcome measures; Cohort 2 will include adult participants, aged 18 to ≤50 years old. Participants will be randomized 1:1 to 80 mg obudanersen or placebo during the double-blind placebo-controlled treatment period. Participants from both cohorts completing the placebo-controlled treatment period will be eligible to transition into the LTE Treatment Period wherein all trial participants will receive obudanersen. Participant, Caregiver, Investigator and Sponsor will remain blinded to the obudanersen dose administered to participants during the LTE. The study initiated in 2024 with three dosing groups (40 mg obudanersen, 80 mg obudanersen, and placebo). Following additional review of data from the ongoing Phase 1/2 trial of obudanersen (HALOS), REVEAL was amended in December 2025 to its current form as a two-arm study: 80 mg obudanersen and placebo. Following the amendment, participants who were randomized to receive 40 mg obudanersen will be transitioned to receive 80 mg obudanersen.
How this trial compares with your answers
Answer 2 more questions to improve match
What we know so far
Still need:
- • Tell us your age for better matching
- • Tell us your sex for better matching
Preliminary match based on your answers. Full eligibility requires on-site assessment including medical history, physical exam, and lab tests. This does not guarantee enrolment.
Eligibility at a Glance
Key info
- Age: 2 Years - 50 Years
- Who can join: All genders
What the study is looking for
- ✓Medically stable and can undergo sedation and/or general anesthesia without intubation.
- ✓Male or female between 2 and lesser than or equal to (≤)50 years of age, depending on specific cohort, at the time...
- ✓Participant has a clinical diagnosis of Angelman syndrome (AS) with molecular confirmation of either...
Who cannot take part
- ✗Has molecular confirmation of AS due to paternal uniparental disomy, imprinting center defect, or mosaic findings.
- ✗Other inclusion/exclusion criteria may apply.
See the full criteria
Where Is This Study? (2 UK sites)
Great Ormond Street Hospital for Children - NHS Foundation Trust
London WC1N 3JH, United Kingdom
Main Email: Research.Governance@gosh.nhs.uk
Research.Governance@gosh.nhs.uk0207 905 2700John Radcliffe Hospital
Oxford OX3 9DU, United Kingdom
