150
Participants
Start Date
June 4, 2025
Primary Completion Date
March 31, 2027
Study Completion Date
August 31, 2027
zorevunersen
Zorevunersen group will receive study drug by intrathecal (IT) administration on Day 1 (after the 8-week Baseline Period), Day 57 (Week 8), Day 169 (Week 24), and Day 281 (Week 40) at a dose level of 70 mg on Day 1 and Day 57, and 45 mg on Day 169 and Day 281.
Sham Comparator
Sham group will not have drug administered. Sham group will have a lumbar puncture with CSF withdrawal.
NOT_YET_RECRUITING
NYU Langone Health, New York
RECRUITING
Weill Cornell Medicine, New York
NOT_YET_RECRUITING
University of Rochester Medical Center, Rochester
NOT_YET_RECRUITING
Children's Hospital of Philadelphia, Philadelphia
NOT_YET_RECRUITING
Children's National Medical Center, Washington D.C.
NOT_YET_RECRUITING
UVA Health, Charlottesville
RECRUITING
University of North Carolina at Chapel Hill, Chapel Hill
RECRUITING
Duke University Health System, Durham
NOT_YET_RECRUITING
Nemours Children's Health, Jacksonville
NOT_YET_RECRUITING
Advent Health Neuroscience Research Institute, Orlando
RECRUITING
Nicklaus Children's Hospital, Miami
RECRUITING
LeBonheur Children's Hospital, Memphis
NOT_YET_RECRUITING
Nationwide Children's Hospital, Columbus
NOT_YET_RECRUITING
Cleveland Clinic, Cleveland
NOT_YET_RECRUITING
Cincinnati Children's Hospital Medical Center, Cincinnati
RECRUITING
CS Mott Children's Hospital, Ann Arbor
NOT_YET_RECRUITING
University of Iowa Hospital and Clinics, Iowa City
RECRUITING
Mayo Clinic, Rochester
NOT_YET_RECRUITING
Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago
NOT_YET_RECRUITING
Washington University in St. Louis School of Medicine, St Louis
RECRUITING
Arkansas Children's Hospital, Little Rock
RECRUITING
Cook Children's Medical Center, Fort Worth
NOT_YET_RECRUITING
Texas Children's Hospital, Houston
RECRUITING
Children's Hospital Colorado, Aurora
NOT_YET_RECRUITING
Phoenix Children's Hospital, Phoenix
NOT_YET_RECRUITING
Cedars Sinai Medical Center, Los Angeles
NOT_YET_RECRUITING
Children's Hospital of Orange County, Orange
RECRUITING
USCF Medical Center, San Francisco
RECRUITING
Oregon Health & Science University (OHSU), Portland
NOT_YET_RECRUITING
Seattle Children's Hospital, Seattle
NOT_YET_RECRUITING
Massachusetts General Hospital, Boston
NOT_YET_RECRUITING
Boston Children's Hospital, Boston
RECRUITING
Fukuoka Children's Hospital, Fukuoka
NOT_YET_RECRUITING
Hokkaido University Hospital, Hokkaido
NOT_YET_RECRUITING
Kyoto University Hospital, Kyoto
NOT_YET_RECRUITING
Nagoya University Hospital, Nagoya
NOT_YET_RECRUITING
National Hospital Organization Nishi Niigata Central Hospital, Niigata
NOT_YET_RECRUITING
Okayama University Hospital, Okayama
NOT_YET_RECRUITING
Jichi Medical University Hospital, Shimotsuke
NOT_YET_RECRUITING
National Center of Neurology and Psychiatry, Tokyo
NOT_YET_RECRUITING
Yokohama City University Medical Center, Yokohama
NOT_YET_RECRUITING
Royal Hospital for Children, Glasgow
NOT_YET_RECRUITING
Great Ormond Street Hospital for Children, London
RECRUITING
Sheffield Children's Hospital, Sheffield
Stoke Therapeutics, Inc
INDUSTRY