Expanded Access Protocol for Boys With Duchenne Muscular Dystrophy
ConditionDuchenne Muscular Dystrophy
Trial statusAvailable
Trial phaseNot listed
Trial typeExpanded Access
Biological sexMale
AgeNot listed
SponsorSanthera Pharmaceuticals
About this trial
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.
Eligibility criteria
Qualifiers
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
Disqualifiers
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
Trial design
Treatments tested in this trial
- Vamorolone
Treatment groups
No treatment groups listed
Sponsors and collaborators
Source ClinicalTrials.gov