Study Summary
The study will evaluate the safety and efficacy of gene transfer therapy in boys with DMD. It is a randomized, double-blind, placebo-controlled study. The participants who are randomized to the placebo arm will have an opportunity for treatment with gene transfer therapy at the beginning of the second year.
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Request More InfoInterventions
delandistrogene moxeparvovecGENETIC
Single IV infusion of delandistrogene moxeparvovec.
placeboGENETIC
Single IV infusion of matching placebo.
Study Locations
| Facility | City | State | Country |
|---|---|---|---|
| Arkansas Children's | Little Rock | Arkansas | United States |
| UC San Diego Altman Clinical and Translational Research Institute | La Jolla | California | United States |
| UCLA Medical Center | Los Angeles | California | United States |
| Lucile Packard Children's Hospital at Stanford | Palo Alto | California | United States |
| University of California, Davis | Sacramento | California | United States |
| Children's Hospital Colorado | Aurora | Colorado | United States |
| University of Florida | Gainesville | Florida | United States |
| Lurie Children's Hospital of Chicago | Chicago | Illinois | United States |
| University of Iowa Stead Family Children's Hospital | Iowa City | Iowa | United States |
| The Johns Hopkins Hospital | Baltimore | Maryland | United States |
| Boston Children's Hospital | Boston | Massachusetts | United States |
| Washington University of St. Louis | St Louis | Missouri | United States |
| Columbia University/NYPH | New York | New York | United States |
| University of Rochester | Rochester | New York | United States |
| Duke University Medical Center, Lenox Baker Children's Hospital | Durham | North Carolina | United States |
| Nationwide Children's Hospital | Columbus | Ohio | United States |
| Oregon Health & Science University | Portland | Oregon | United States |
| The Children's Hospital of Philadelphia | Philadelphia | Pennsylvania | United States |
| Vanderbilt University Medical Center | Nashville | Tennessee | United States |
| University of Texas Southwestern | Dallas | Texas | United States |
| University of Utah Hospital | Salt Lake City | Utah | United States |
| Children's Hospital of the King's Daughters | Norfolk | Virginia | United States |
| Children's Wisconsin | Milwaukee | Wisconsin | United States |
| University Hospital Ghent | Ghent | Belgium | |
| LMU - Klinikum der Universitaet Muenchen - Kinderklinik und | Bayern | Germany | |
| Universitätsklinikum Essen - Klinik für Kinderheilkunde I | Essen | Germany | |
| University Hospital Hamburg- Eppendorf | Hamburg | Germany | |
| Hong Kong Children's Hospital | Kowloon | Hong Kong | |
| IRCCS Istituto G.Gaslini, U.O. | Genoa | Italy | |
| UOC Neurologia, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico | Milan | Italy | |
| UOC Neuropsichiatria Infantile, Area Salute del Bambino, Fondazione Policlinico Universitario A. Gamelli IRCCS | Roma | Italy | |
| Kobe University Hospital | Kobe | Japan | |
| National Center for Child Health and Development | Tokyo | Japan | |
| Tokyo Women's Medical University Hospital - Pediatrics | Tokyo | Japan | |
| National Center of Neurology and Psychiatry | Tokyo | Japan | |
| Hospital Universitari i Politécnico La Fe | Valencia | Comunidad Valencia | Spain |
| Hospital Sant Joan de Déu | Barcelona | Spain | |
| Kaohsiung Medical University Chung-Ho Memorial Hospital | Kaohsiung City | Taiwan | |
| National Taiwan University Hospital | Taipei | Taiwan | |
| Oxford University Hospitals NHS Foundation Trust | Headington | Oxford | United Kingdom |
| Great Ormond Street Hospital for Children NHS Foundation Trust | London | United Kingdom | |
| The Newcastle Upon Tyne NHS Hospital NHS Foundation Trust, Royal Victoria Infirmary | Newcastle upon Tyne | United Kingdom |