Expanded Access Protocol for Boys With Duchenne Muscular Dystrophy
NCT ID: NCT03863119
Last Updated: 2026-01-20
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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AVAILABLE
EXPANDED_ACCESS
Brief Summary
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Detailed Description
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VBP15-EAP (under IND 118942) has been closed as vamaorolone is approved for marketing in the US VBP15-EAP CANADA has been approved by Health Canada on 11 July 2019 and is currently on-going,but not recruiting
Conditions
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Interventions
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Vamorolone
2.0 mg/kg/day, 4.0 mg/kg/day, or 6.0 mg/kg/day at physician discretion
Eligibility Criteria
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Inclusion Criteria
* Subject has previously completed at a participating US or Canada study site VBP15-LTE up to and including the Month 24 assessments, OR VBP15-004 up to and including the Week 48 assessments, VBP15-006 up to and including the Week 12 assessment
* Subject and parent/guardian are willing and able to comply with recommended study drug administration plan, and standard of care follow-up and monitoring as recommended by their Treating Physician
Exclusion Criteria
* Subject and/or parent/guardian are unable and/or unwilling to comply with regular medical care and follow-up as recommended by their Treating Physician throughout participation in the VBP15-EAP
MALE
No
Sponsors
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Santhera Pharmaceuticals
INDUSTRY
Responsible Party
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Locations
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University of California Davis
Davis, California, United States
Nemours Children's Hospital
Orlando, Florida, United States
urie Children's Hospital of Chicago
Chicago, Illinois, United States
Duke University
Durham, North Carolina, United States
University of Texas Southwestern Medical Center
Dallas, Texas, United States
Seattle Children's
Seattle, Washington, United States
Alberta's Children Hospital
Calgary, Alberta, Canada
British Columbia Children's Hospital
Vancouver, British Columbia, Canada
Children's Hospital of Eastern Ontario
Ottawa, Ontario, Canada
The Hospital for Sick Children
Toronto, Ontario, Canada
Montreal Childrens Hospital
Montreal, Quebec, Canada
Schneider Chidlren's Medical Center
Petah Tikva, , Israel
Countries
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Central Contacts
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Facility Contacts
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Julie Dao
Role: primary
Related Links
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Vamorolone Phase IIa trial in Duchenne Muscular Dystrophy
Other Identifiers
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VBP15-EAP
Identifier Type: -
Identifier Source: org_study_id
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