REVEAL: A Phase 3 Study of Obudanersen (ION582) in Angelman Syndrome
Condition(s) studied
Investigational drug(s) / intervention(s)
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
Primary outcome measure(s)
- Change in Performance on the Expressive Communication Subdomain Raw Score of the Bayley Scales for Infant and Toddler Development-4 (Bayley-4) Without Caregiver Input in Cohort 1 — Baseline and Week 52
The Bayley-4 is a performance-based assessment of developmental functioning across communication, cognition, and motor skills. The expressive communication subdomain of communication measures preverbal and verbal communication. The total raw score reflects the sum of all the item scores within the expressive communication subdomain, with higher scores reflecting greater expressive communication ability.
Trial sites (41)
| Facility | City | Region | Status |
|---|---|---|---|
| Cedars-Sinai Medical Center | Los Angeles | California | |
| UCLA Clinical & Translational Research Center (CTRC) | Los Angeles | California | |
| Rady Children's Hospital | San Diego | California | |
| Colorado Children's Hospital Research Institute | Aurora | Colorado | |
| Children's National Hospital | Washington D.C. | District of Columbia | |
| Nicklaus Children's Hospital | Miami | Florida | |
| Children's Healthcare of Atlanta | Atlanta | Georgia | |
| Rush University Medical Center | Chicago | Illinois | |
| Boston Children's Hospital | Boston | Massachusetts | |
| Children's Mercy | Kansas City | Missouri | |
| Ichan School of Medicine at Mount Sinai | New York | New York | |
| Columbia University Irving Medical Center | New York | New York | |
| University of North Carolina at Chapel Hill School of Medicine | Carrboro | North Carolina | |
| Cincinnati Children's Hospital | Cincinnati | Ohio | |
| Nationwide Children's Hospital | Columbus | Ohio | |
| Children's Hospital of Philadelphia | Philadelphia | Pennsylvania | |
| Vanderbilt Clinical Research Center | Nashville | Tennessee | |
| Texas Children's Hospital | Houston | Texas | |
| Queensland Children's Hospital | South Brisbane | Queensland | |
| Austin Hospital | Heidelberg | Australia | |
| Perth Children's Hospital | Nedlands | Australia | |
| Sydney Children's Hospital | Randwick | Australia | |
| London Health Science Centre - Children's Hospital | London | Ontario | |
| McGill University Health Centre | Montreal | Quebec | |
| University of Alberta Hospital | Edmonton | Canada | |
| British Columbia Children's Hospital | Vancouver | Canada | |
| Klinikum der Ludwig-Maximilians-Universitaet Muenchen | München | Germany | |
| Sheba Medical Center | Ramat Gan | Israel | |
| Fondazione IRCCS Istituto Neurologico Carlo Besta | Milan | Italy | |
| Azienda Ospedaliero Universitaria Pisana | Pisa | Italy | |
| Ospedale Pediatrico Bambino Gesù | Roma | Italy | |
| Osaka Women's and Children's Hospital | Izumi | Osaka | |
| National Center of Neurology and Psychiatry | Kodaira | Tokyo | |
| Uniwersyteckie Centrum Kliniczne | Gdansk | Poland | |
| KK Women's and Children's Hospital | Singapore | Singapore | |
| Seoul National University Hospital | Seoul | South Korea | |
| Samsung Medical Center | Seoul | South Korea | |
| Hospital Sant Joan de Deu | Barcelona | Spain | |
| Corporacio Sanitaria Parc Tauli - Hospital de Sabadell | Sabadell | Spain | |
| Great Ormond Street Hospital for Children - NHS Foundation Trust | London | United Kingdom |
+ 1 more sites — see the full list on the official registry below.
More Ionis Pharmaceuticals, Inc. trials in Poland
Other trials for the same condition
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 Poland