Expanded Access Protocol for Boys With Duchenne Muscular Dystrophy

Available (expanded access)

Conditions studied: Duchenne Muscular Dystrophy

In brief

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.

Key facts

Study ID

NCT03863119

Run by

Santhera Pharmaceuticals

Last updated by the study team

2026-01-20

Who can join

Age: any. Sex: male. Healthy volunteers: not accepted.

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

Where it is running

• 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

Full record on ClinicalTrials.gov

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