Active Not Recruiting PHASE3
REVEAL: A Phase 3 Study of Obudanersen (ION582) in Angelman Syndrome
About this study
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.
Drug
- obudanersen
Placebo or sham
Placebo
Study locations
Australia
Queensland
South Brisbane (1)
- Queensland Children's Hospital — Site status not reported
State / region not reported
Heidelberg (1)
- Austin Hospital — Site status not reported
Nedlands (1)
- Perth Children's Hospital — Site status not reported
Randwick (1)
- Sydney Children's Hospital — Site status not reported
Canada
Ontario
London (1)
- London Health Science Centre - Children's Hospital — Site status not reported
Quebec
Montreal (1)
- McGill University Health Centre — Site status not reported
State / region not reported
Edmonton (1)
- University of Alberta Hospital — Site status not reported
Vancouver (1)
- British Columbia Children's Hospital — Site status not reported
Germany
State / region not reported
München (1)
- Klinikum der Ludwig-Maximilians-Universitaet Muenchen — Site status not reported
Israel
State / region not reported
Ramat Gan (1)
- Sheba Medical Center — Site status not reported
Italy
State / region not reported
Milan (1)
- Fondazione IRCCS Istituto Neurologico Carlo Besta — Site status not reported
Pisa (1)
- Azienda Ospedaliero Universitaria Pisana — Site status not reported
Roma (1)
- Ospedale Pediatrico Bambino Gesù — Site status not reported
Japan
Osaka
Izumi (1)
- Osaka Women's and Children's Hospital — Site status not reported
Tokyo
Kodaira (1)
- National Center of Neurology and Psychiatry — Site status not reported
Poland
State / region not reported
Gdansk (1)
- Uniwersyteckie Centrum Kliniczne — Site status not reported
Singapore
State / region not reported
Singapore (1)
- KK Women's and Children's Hospital — Site status not reported
South Korea
State / region not reported
Seoul (2)
- Seoul National University Hospital — Site status not reported
- Samsung Medical Center — Site status not reported
Spain
State / region not reported
Barcelona (1)
- Hospital Sant Joan de Deu — Site status not reported
Sabadell (1)
- Corporacio Sanitaria Parc Tauli - Hospital de Sabadell — Site status not reported
United Kingdom
State / region not reported
London (1)
- Great Ormond Street Hospital for Children - NHS Foundation Trust — Site status not reported
Oxford (1)
- John Radcliffe Hospital — Site status not reported
United States
California
Los Angeles (2)
- Cedars-Sinai Medical Center — Site status not reported
- UCLA Clinical & Translational Research Center (CTRC) — Site status not reported
San Diego (1)
- Rady Children's Hospital — Site status not reported
Colorado
Aurora (1)
- Colorado Children's Hospital Research Institute — Site status not reported
District of Columbia
Washington D.C. (1)
- Children's National Hospital — Site status not reported
Florida
Miami (1)
- Nicklaus Children's Hospital — Site status not reported
Georgia
Atlanta (1)
- Children's Healthcare of Atlanta — Site status not reported
Illinois
Chicago (1)
- Rush University Medical Center — Site status not reported
Massachusetts
Boston (1)
- Boston Children's Hospital — Site status not reported
Missouri
Kansas City (1)
- Children's Mercy — Site status not reported
New York
New York (2)
- Ichan School of Medicine at Mount Sinai — Site status not reported
- Columbia University Irving Medical Center — Site status not reported
North Carolina
Carrboro (1)
- University of North Carolina at Chapel Hill School of Medicine — Site status not reported
Ohio
Cincinnati (1)
- Cincinnati Children's Hospital — Site status not reported
Columbus (1)
- Nationwide Children's Hospital — Site status not reported
Pennsylvania
Philadelphia (1)
- Children's Hospital of Philadelphia — Site status not reported
Tennessee
Nashville (1)
- Vanderbilt Clinical Research Center — Site status not reported
Texas
Houston (1)
- Texas Children's Hospital — Site status not reported
Full registry eligibility criteria
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.
Important information
Registry information may change. FAST context and classifications are editorial additions. Inclusion does not confirm eligibility or availability at a specific site. Review the official record and contact the study team for participation requirements.