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 (The GUARDIAN Study)

Conditions

Duchenne Muscular Dystrophy

Brief summary

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

Detailed description

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, height and body mass index (BMI), Number of subjects with clinically relevant laboratory abnormalities including glycosylated haemoglobin (HbA1c), and morning cortisol, Change from baseline in Time to Stand Test (TTSTAND) velocity, 6-Minute Walk Test (6MWT) distance, Change from baseline in 6MWT distance, NorthStar Ambulatory Assessment (NSAA) scores, Age at ambulatory and non-ambulatory milestones

Related papers

No related papers found yet.

Interventions

DRUGAGAMREE 40 mg/ml oral suspension

Eligibility

Sex/Gender

Male

Age

0 Years to 17 Years

Design outcomes

Primary

Measure	Time frame
Number of vertebral fractures per 1000 person-years based on X- ray central reading	—

Secondary

Measure	Time frame
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, height and body mass index (BMI), Number of subjects with clinically relevant laboratory abnormalities including glycosylated haemoglobin (HbA1c), and morning cortisol, Change from baseline in Time to Stand Test (TTSTAND) velocity, 6-Minute Walk Test (6MWT) distance, Change from baseline in 6MWT distance, NorthStar Ambulatory Assessment (NSAA) scores, Age at ambulatory and non-ambulatory milestones	—

Measure

Time frame

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, height and body mass index (BMI), Number of subjects with clinically relevant laboratory abnormalities including glycosylated haemoglobin (HbA1c), and morning cortisol, Change from baseline in Time to Stand Test (TTSTAND) velocity, 6-Minute Walk Test (6MWT) distance, Change from baseline in 6MWT distance, NorthStar Ambulatory Assessment (NSAA) scores, Age at ambulatory and non-ambulatory milestones

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Countries

Belgium, Czechia, Greece, Ireland, Netherlands, Spain

Outcome results

None listed