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A Study on Safety and Effectiveness of Long-term Treatment With Vamorolone in Boys With Duchenne Muscular Dystrophy
Sponsor: Santhera Pharmaceuticals
Summary
This study aims to assess safety and effectivness of long-term treatment with vamorolone in boys with Duchenne Muscular Dystrophy (DMD) who have completed prior studies with vamorolone.
Official title: An Open-label Study to Collect Safety and Effectiveness Information on Long-term Treatment With Vamorolone in Boys With Duchenne Muscular Dystrophy Who Have Completed Prior Studies With Vamorolone
Key Details
Gender
MALE
Age Range
Any - Any
Study Type
INTERVENTIONAL
Enrollment
80
Start Date
2024-11-10
Completion Date
2028-09
Last Updated
2026-01-09
Healthy Volunteers
No
Conditions
Interventions
vamorolone 40 mg/mL oral suspension
Vamorolone is administered at a dose range between 2 mg/kg/day and 6 mg/kg/day for boys weighing \<40 kg. For boys weighing 40 kg or above, the dose range will be 80 mg to 240 mg once daily. Doses can be adjusted within the dose range as determined by the Investigator based on tolerability. The highest tolerated dose should be used.
Locations (18)
UZ Gent (Universitair Ziekenhuis Gent)
Ghent, Belgium
UZ Leuven (Universitair Ziekenhuis Leuven)
Leuven, Belgium
University Hospital Brno
Brno, Czechia
Fakultni Nemocnice Motol
Prague, Czechia
Children's Hospital Agia Sofia
Athens, Greece
Children's Health Ireland at Tallaght, Tallaght University Hospital
Dublin, Ireland
Schneider Children's Medical Center
Petah Tikva, Israel
Leiden University Medical Center
Leiden, Netherlands
Radboud University Nijmegen
Nijmegen, Netherlands
Te Wao Nui - Child Health Service, Wellington Hospital
Wellington, New Zealand
Hospital Universitario Puerta de Hierro Majadahonda
Madrid, Spain
Hospital Universitario y Politecnico de La Fe
Valencia, Spain
Queen Elizabeth University Hospital
Glasgow, Lanarkshire, United Kingdom
Alder Hey Children's Hospital
Liverpool, Merseyside, United Kingdom
Leeds Teaching Hospitals NHS Trust
Leeds, West Yorkshire, United Kingdom
University Hospitals Birmingham NHS Foundation Trust
Birmingham, United Kingdom
Great Ormond Street Hospital for Children NHS Foundation Trust
London, United Kingdom
The John Walton Muscular Dystrophy Research Centre
Newcastle, United Kingdom