Study tracks Long-Term safety of DMD drug in boys Who've used it before
NCT ID NCT06713135
Summary
This study is monitoring the long-term safety and effectiveness of the drug vamorolone in boys with Duchenne muscular dystrophy (DMD). It includes 80 boys who have already been taking vamorolone through special access programs. The main goal is to check for side effects like bone fractures and cataracts over a long period, while also seeing how well the drug continues to help with muscle function.
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Contacts and locations
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Locations
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Alder Hey Children's Hospital
Liverpool, Merseyside, L14 5AB, United Kingdom
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Children's Health Ireland at Tallaght, Tallaght University Hospital
Dublin, D24TN3C, Ireland
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Children's Hospital Agia Sofia
Athens, 115 27, Greece
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Fakultni Nemocnice Motol
Prague, 150 06, Czechia
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Great Ormond Street Hospital for Children NHS Foundation Trust
London, WC1N 3JH, United Kingdom
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Hospital Universitario Puerta de Hierro Majadahonda
Madrid, 28222, Spain
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Hospital Universitario y Politecnico de La Fe
Valencia, 46026, Spain
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Leeds Teaching Hospitals NHS Trust
Leeds, West Yorkshire, LS1 3EX, United Kingdom
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Leiden University Medical Center
Leiden, 2333 ZA, Netherlands
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Queen Elizabeth University Hospital
Glasgow, Lanarkshire, United Kingdom
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Radboud University Nijmegen
Nijmegen, 6525 HB, Netherlands
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Schneider Children's Medical Center
Petah Tikva, 4920235, Israel
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Te Wao Nui - Child Health Service, Wellington Hospital
Wellington, 6021, New Zealand
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The John Walton Muscular Dystrophy Research Centre
Newcastle, NE1 3BZ, United Kingdom
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UZ Gent (Universitair Ziekenhuis Gent)
Ghent, 9000, Belgium
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UZ Leuven (Universitair Ziekenhuis Leuven)
Leuven, 3000, Belgium
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University Hospital Brno
Brno, Czechia
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University Hospitals Birmingham NHS Foundation Trust
Birmingham, B9 5SS, United Kingdom
Conditions
Explore the condition pages connected to this study.