Santhera GUARDIAN

All subjects in this study have completed previous studies with vamorolone and continued to receive vamorolone under special programs: Compassionate Use Program [CUP], Named Patient Program [NPP] or Expanded Access Protocol [EAP]. All subjects will continue treatment with vamorolone under Guardian protocol instead. The primary objective of this study is to evaluate the safety of long-term treatment with vamorolone in boys with Duchenne Muscular Dystrophy regarding vertebral fractures. Secondary study objectives will evaluate the safety of long-term treatment with vamorolone on non-vertebral fractures, cataracts, delayed puberty, overall safety as well as ambulatory and non-ambulatory function.

 
Primary Outcome Measures

• Number of vertebral fractures per 1000 person-years based on X-ray central reading.

 
Secondary Outcome Measures

• Time to first vertebral fractures (cumulative incidence)
• Number of non-vertebral fractures per 1000 person-years based on investigator reporting
• Time to first non-vertebral fractures (cumulative incidence)
• Number of cataracts per 1000 person-years based on ophthalmologist assessment
• Number of subjects not reaching Tanner stage 2 by 15 years of age
• Frequency of adverse events (AEs) and serious adverse events (SAEs)
• Change from baseline in body weight
• Number of subjects with clinically relevant laboratory abnormalities
• Change from baseline in Time to Stand (TTSTAND) velocity
• Six-minute Walk Test (6MWT)
• Change from baseline in 6MWT distance
• Age at ambulatory and non-ambulatory milestones
• North Star Ambulatory Assessment (NSAA) scores
• Change from baseline in body height
• Change from baseline in Body Mass Index (BMI)

 

 

 

 

 

 
Can I take part?

 
Inclusion Criteria

 
Exclusion Criteria