Gene therapy trial offers hope for boys with devastating muscle disease
NCT ID NCT04281485
Summary
This study is testing whether a one-time gene therapy is safe and can help boys with Duchenne muscular dystrophy (DMD) maintain their ability to walk and move. About 99 boys, aged 4 to 7, will receive either the gene therapy or a placebo infusion. The main goal is to see if the treatment improves scores on a walking and movement test after one year, and all participants will be followed for safety for 15 years.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes NO responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Contacts and locations
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Locations
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Alberta Children's Hospital
Calgary, Alberta, T3B 6A8, Canada
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Alder Hey Children's NHS Foundation Trust
Liverpool, Merseyside, L12 2AP, United Kingdom
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Arkansas Children's
Little Rock, Arkansas, 72202, United States
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Arkansas Children's Hospital
Little Rock, Arkansas, 72202, United States
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CHU de Nantes- Hotel Dieu
Nantes, 44093, France
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Charité - Universitätsmedizin Berlin
Berlin, 13353, Germany
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Children's Hospital - London Health Sciences Centre
London, Ontario, N6A 4G5, Canada
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Childrens Hospital of Eastern Ontario
Ottawa, Ontario, K1H8L1, Canada
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Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Roma, 00168, Italy
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Great Ormond Street Institute of Child Health
London, WCIN 1EH, United Kingdom
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Hadassah University Medical Center, Ein Kerem
Jerusalem, 91120, Israel
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Hopital Necker
Paris, 75015, France
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Hospital Sant Joan de Déu
Esplugues de Llobregat, Barcelona, 08950, Spain
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Hospital Universitari i Politecnic La Fe de Valencia
Valencia, 46026, Spain
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Hospital Universitario Vall d'Hebron
Barcelona, 08035, Spain
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Hyogo College of Medicine College Hospital
Nishinomiya, Hyōgo, 663-8501, Japan
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IRCCS Ospedale Pediatrico Bambino Gesù
Rome, 00165, Italy
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Inselspital, University Children's Hospital Berne
Bern, 3010, Switzerland
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KU Clinical Research Center - Clinical and Translational Science Unit (CTSU) - Fairway
Fairway, Kansas, 66205, United States
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KU Clinical Research Center - Clinical and Translational Science Unit (CTSU) - Rainbow
Kansas City, Kansas, 66160, United States
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Kaohsiung Medical University Chung-Ho Memorial Hospital
Kaohsiung City, 807, Taiwan
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Lenox Baker Children's Hospital
Durham, North Carolina, 27705, United States
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Nagoya City University Hospital
Nagoya, Aichi-ken, 467-8602, Japan
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National Center of Neurology and Psychiatry
Tokyo, 187-8551, Japan
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National Taiwan University Hospital
Taipei, 100, Taiwan
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Pediatric Cardiology
Prairie Village, Kansas, 66208, United States
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Perth Children's Hospital
Nedlands, Western Australia, 6009, Australia
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Primary Childrens Hospital
Salt Lake City, Utah, 84113, United States
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Pusan National University Yangsan Hospital
Yangsan, Gyeongsangnam-do, 50612, South Korea
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Saint Petersburg State Paediatric Medical University
Saint Petersburg, 194100, Russia
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Samsung Medical Center
Seoul, 06351, South Korea
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Schneider Children's Medical Center of Israel
Petach Tikvah, 4920235, Israel
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Seattle Children's
Seattle, Washington, 98105, United States
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Seoul National University Hospital
Seoul, 03080, South Korea
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State Autonomous Healthcare Institution of Sverdlovsk Region Children's City Clinical Hospital No 9
Yekaterinburg, 620134, Russia
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Taipei Veterans General Hospital
Taipei, 11217, Taiwan
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The Children's Hospital at Westmead
Westmead, New South Wales, 2145, Australia
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The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
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The Hospital For Sick Children
Toronto, Ontario, M5G 1X8, Canada
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The Newcastle upon Tyne Hospitals NHS Foundation Trust, Royal Victoria Infirmary
Newcastle upon Tyne, England, NE1 4LP, United Kingdom
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The Royal Children's Hospital Melbourne
Parkville, Victoria, 3052, Australia
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UCLA Medical Center
Los Angeles, California, 90095, United States
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UZ Gent
Ghent, 9000, Belgium
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UZ leuven
Leuven, 3000, Belgium
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Universitaets-Kinderspital Zuerich
Zurich, 8008, Switzerland
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Universitatsklinikum Essen
Essen, 45147, Germany
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University of Florida
Gainesville, Florida, 32610, United States
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University of Iowa Hospitals and Clinics
Iowa City, Iowa, 52242, United States
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University of Kansas Hospital - Investigational Pharmacy
Kansas City, Kansas, 66160, United States
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University of Kansas Hospital - Pediatric and Pediatric ICU - Operating Room
Kansas City, Kansas, 66160, United States
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University of Kansas Medical Center
Kansas City, Kansas, 66160, United States
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University of Utah Clinical Neurosciences Center
Salt Lake City, Utah, 84132, United States
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University of Utah Hospital
Salt Lake City, Utah, 84132, United States
Conditions
Explore the condition pages connected to this study.