🇮🇪Ireland
16°C Partly Cloudy · Dublin
Live Updates
--:--:-- IST
Writer Login
Latest
Clinical Trials in the UK / NCT06914609
Recruiting Phase 3

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

NCT06914609 · tracked via the Priya Life Science UK tracker
Sponsor
Ionis Pharmaceuticals, Inc.
Phase
Phase 3
Started
2025-06-10
Last updated
2026-08-28

Condition(s) studied

Angelman Syndrome

Investigational drug(s) / intervention(s)

obudanersenPlacebo

obudanersen: obudanersen will be administered by IT injection.

Placebo: obudanersen matching placebo will be administered by IT injection.

Study summary

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.

Eligibility

Sex
ALL
Min age
2 Years
Max age
50 Years
Healthy volunteers
No
Key Inclusion Criteria: 1. The participants caregiver(s)/ legally authorized representative must have given written informed consent and any authorizations required by local law and be able to comply with all study requirements. 2. Medically stable and can undergo sedation and/or general anesthesia without intubation. 3. Male or female between 2 and lesser than or equal to (≤)50 years of age, depending on specific cohort, at the time of the in-clinic Screening visit. 4. Participant has a clinical diagnosis of Angelman syndrome (AS) with molecular confirmation of either Ubiquitin-protein ligase E3A (UBE3A) deletion or UBE3A mutation. 5. 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. 6. Legally authorized representative/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 (formerly Twitter), YouTube, TikTok, etc.) from the time of enrollment until they are notified that the study is completed. Key Exclusion Criteria: 1. Must not have 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 Principal Investigator (PI), could confound the results of this study. 2. Known brain or spinal disease that would interfere with the lumbar puncture (LP) procedure, cerebrospinal fluid (CSF) circulation, or presence of other factors would affect the safety of the LP procedure. 3. Must not have any other conditions, which, in the opinion of the Investigator, would make the participant unsuitable for inclusion or could interfere with the participant participating in or completing the study. 4. Must not have 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 (RNA) \[siRNA\], ASOs) gene therapy or gene editing. This exclusion criterion does not apply to approved nucleic acid-based vaccines, including mRNA vaccines, which are allowed. 6. Has molecular confirmation of AS due to paternal uniparental disomy, imprinting center defect, or mosaic findings. Other inclusion/exclusion criteria may apply.

Primary outcome measure(s)

Trial sites (41)

FacilityCityRegionStatus
Cedars-Sinai Medical Center Los Angeles California Recruiting
UCLA Clinical & Translational Research Center (CTRC) Los Angeles California Recruiting
Rady Children's Hospital San Diego California Recruiting
Colorado Children's Hospital Research Institute Aurora Colorado Recruiting
Children's National Hospital Washington D.C. District of Columbia Recruiting
Nicklaus Children's Hospital Miami Florida Recruiting
Children's Healthcare of Atlanta Atlanta Georgia Recruiting
Rush University Medical Center Chicago Illinois Recruiting
Boston Children's Hospital Boston Massachusetts Recruiting
Children's Mercy Kansas City Missouri Recruiting
Ichan School of Medicine at Mount Sinai New York New York Recruiting
Columbia University Irving Medical Center New York New York Recruiting
University of North Carolina at Chapel Hill School of Medicine Carrboro North Carolina Recruiting
Cincinnati Children's Hospital Cincinnati Ohio Recruiting
Nationwide Children's Hospital Columbus Ohio Recruiting
Children's Hospital of Philadelphia Philadelphia Pennsylvania Recruiting
Vanderbilt Clinical Research Center Nashville Tennessee Recruiting
Texas Children's Hospital Houston Texas Recruiting
Queensland Children's Hospital South Brisbane Queensland Recruiting
Austin Hospital Heidelberg Australia Recruiting
Perth Children's Hospital Nedlands Australia Recruiting
Sydney Children's Hospital Randwick Australia Recruiting
London Health Science Centre - Children's Hospital London Ontario Recruiting
McGill University Health Centre Montreal Quebec Recruiting
University of Alberta Hospital Edmonton Canada Recruiting
British Columbia Children's Hospital Vancouver Canada Recruiting
Klinikum der Ludwig-Maximilians-Universitaet Muenchen München Germany Recruiting
Sheba Medical Center Ramat Gan Israel Recruiting
Fondazione IRCCS Istituto Neurologico Carlo Besta Milan Italy Recruiting
Azienda Ospedaliero Universitaria Pisana Pisa Italy Recruiting
Ospedale Pediatrico Bambino Gesù Roma Italy Recruiting
Osaka Women's and Children's Hospital Izumi Osaka Recruiting
National Center of Neurology and Psychiatry Kodaira Tokyo Recruiting
Uniwersyteckie Centrum Kliniczne Gdansk Poland Recruiting
KK Women's and Children's Hospital Singapore Singapore Recruiting
Seoul National University Hospital Seoul South Korea Recruiting
Samsung Medical Center Seoul South Korea Recruiting
Hospital Sant Joan de Deu Barcelona Spain Recruiting
Corporacio Sanitaria Parc Tauli - Hospital de Sabadell Sabadell Spain Recruiting
Great Ormond Street Hospital for Children - NHS Foundation Trust London United Kingdom Recruiting

+ 1 more sites — see the full list on the official registry below.

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT06914609 on ClinicalTrials.gov ↗ ← All trials in the UK