This long-term extension study is an open-label, multiple-dose study to evaluate the long-term safety, tolerability, efficacy and PD of vamorolone administered once daily by liquid oral suspension over a Treatment Period of 24 months to young boys with DMD who participated in the VBP15-002 Phase IIa and VBP15-003 Phase IIa extension core studies.
This study will evaluate if it is safe to use a new medication called vamorolone for more than two weeks in children with DMD, if boys with DMD who take the study medication have improved muscle function compared to boys with DMD in other studies who did not take any type of steroid, and to see if boys with DMD who take the study medication gain less weight compared to boys with DMD in a prior study who took another type of steroid called prednisone. Enrolled participants will take the study medication for 24 months.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
46
Oral administration of 0.25 mg/kg/day daily for 24 months.
Oral administration of 0.75 mg/kg/day daily for 24 months.
Oral administration of 2.0 mg/kg/day daily for 24 months.
University of California Davis
Davis, California, United States
University of Florida
Gainesville, Florida, United States
Nemours Children's Hospital
Orlando, Florida, United States
Number of Participants With Treatment-related Adverse Events as Assessed by CTCAE Version 4.03
To evaluate the long-term safety and tolerability of vamorolone, administered orally at daily doses up to 6.0 mg/kg/day over a 24- month Treatment Period, in boys ages 4-7 years with DMD; Treatment-emergent adverse events (TEAEs) are defined as any adverse event or worsening of an existing conditions after initiation of the investigational product and through the subject's last study visit (study completion or early termination);
Time frame: 24 months
Total Number of Adverse Events as Assessed by CTCAE Version 4.03
To evaluate the long-term safety and tolerability of vamorolone, administered orally at daily doses up to 6.0 mg/kg/day over a 24-month Treatment Period, in boys ages 4-7 years with DMD. Treatment-emergent adverse events (TEAEs) are defined as any adverse event or worsening of an existing conditions after initiation of the investigational product and through the subject's last study visit (study completion or early termination).
Time frame: 24 Months
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Oral administration of 6.0 mg/kg/day daily for 24 months.
Ann & Robert H. Lurie Children's Hospital
Chicago, Illinois, United States
Duke University
Durham, North Carolina, United States
University of Texas Southwestern Medical Center
Dallas, Texas, United States
Royal Children's Hospital
Melbourne, Australia
Sydney Children's Hospital
Westmead, Australia
Alberta Children's Hospital
Calgary, Alberta, Canada
Schneider Children's Medical Center
Petah Tikva, Israel
...and 2 more locations