A 24-month Phase II Open-label, Multicenter Long-term Extension Study to Assess the Long-Term Safety and Efficacy of Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD)
Status: Completed
Location: See all (12) locations...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 2
SUMMARY
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.
Eligibility
Participation Requirements
Sex: Male
Minimum Age: 4
Maximum Age: 7
Healthy Volunteers: f
View:
• Subject's parent or legal guardian has provided written informed consent and HIPAA authorization (if applicable) prior to any VBP15-LTE long-term extension study-specific procedures;
• Subject has previously completed study VBP15-003 up to and including the Week 24 Final assessments, prior to enrolling in the VBP15-LTE study at the conclusion of the VBP15-003 Week 24 Visit \[Note: if entering the dose-tapering period, subject is enrolling within 8 weeks after the VBP15-003 final visit following dose-tapering\]; and
• Subject and parent/guardian are willing and able to comply with scheduled visits, study drug administration plan, and study procedures.
Locations
United States
California
University of California Davis
Davis
Florida
University of Florida
Gainesville
Nemours Children's Hospital
Orlando
Illinois
Ann & Robert H. Lurie Children's Hospital
Chicago
North Carolina
Duke University
Durham
Texas
University of Texas Southwestern Medical Center
Dallas
Other Locations
Australia
Royal Children's Hospital
Melbourne
Sydney Children's Hospital
Westmead
Canada
Alberta Children's Hospital
Calgary
Israel
Schneider Children's Medical Center
Petah Tikwah
Sweden
Queen Silvia Children's Hospital
Gothenburg
United Kingdom
Newcastle upon Tyne Hospitals NHS Foundation Trust
Newcastle Upon Tyne
Time Frame
Start Date:2017-02-02
Completion Date:2020-04-30
Participants
Target number of participants:46
Treatments
Experimental: Dose Level Group 1
Participants enrolled in Dose Level Group 1 will receive vamorolone 0.25 mg/kg/day.
Experimental: Dose Level Group 2
Participants enrolled in Dose Level Group 2 will receive vamorolone 0.75 mg/kg/day.
Experimental: Dose Level Group 3
Participants enrolled in Dose Level Group 3 will receive vamorolone 2.0 mg/kg/day.
Experimental: Dose Level Group 4
Participants enrolled in Dose Level Group 4 will receive vamorolone 6.0 mg/kg/day.