Expanded Access Protocol for Boys With Duchenne Muscular Dystrophy
An Open-Label, Expanded Access Protocol for Boys With Duchenne Muscular Dystrophy Who Have Completed the Long-Term Extension (VBP15-LTE) or VBP15-004 or VBP15-006 Studies
1 other identifier
expanded_access
N/A
3 countries
12
Brief Summary
The intent of this protocol is to provide continued access to vamorolone for subjects in the United States and Canada who have completed the VBP15-LTE, VBP15- 004, or VBP15-006 protocols (and are thereby ineligible to enroll in another trial of vamorolone therapy), during the time a new drug application for vamorolone is under preparation and review.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
12 active sites
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Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
February 21, 2019
CompletedFirst Posted
Study publicly available on registry
March 5, 2019
CompletedJanuary 20, 2026
January 1, 2026
February 21, 2019
January 16, 2026
Conditions
Keywords
Interventions
2.0 mg/kg/day, 4.0 mg/kg/day, or 6.0 mg/kg/day at physician discretion
Eligibility Criteria
You may qualify if:
- Subject's parent or legal guardian has provided written informed consent/HIPAA authorization
- 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
You may not qualify if:
- Subject had a serious or severe adverse event in study VBP15-LTE or VBP15-004 or VBP15-006 that, in the opinion of the Treating Physician and Sponsor, was probably or definitely related to vamorolone use and precludes safe use of vamorolone for the subject in this expanded access program
- 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
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (12)
University of California Davis
Davis, California, 95616, United States
Nemours Children's Hospital
Orlando, Florida, 32827, United States
urie Children's Hospital of Chicago
Chicago, Illinois, 60611, United States
Duke University
Durham, North Carolina, 27710, United States
University of Texas Southwestern Medical Center
Dallas, Texas, 75207, United States
Seattle Children's
Seattle, Washington, 98105, United States
Alberta's Children Hospital
Calgary, Alberta, T3B 6A8, Canada
British Columbia Children's Hospital
Vancouver, British Columbia, V6H 3N1, Canada
Children's Hospital of Eastern Ontario
Ottawa, Ontario, K1H 8L1, Canada
The Hospital for Sick Children
Toronto, Ontario, M5G 1X8, Canada
Montreal Childrens Hospital
Montreal, Quebec, H4A 3J1, Canada
Schneider Chidlren's Medical Center
Petah Tikva, 4920435, Israel
Related Publications (1)
Lochmuller H, Gonorazky H, Nigro E, Mah JK, Aleman A, Yaworski A, Oskoui M, Sbrocchi AM, Selby K, de Vera A, McAdam L, Gresko E, Linden A, Dutreix C, Hoffman EP. Results of a phase II open-label, multiple-dose study of vamorolone (VBP15-006) in 7- to < 18-year-old boys with duchenne muscular dystrophy. J Neurol. 2026 Mar 3;273(3):177. doi: 10.1007/s00415-026-13711-6.
PMID: 41774261DERIVED