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Expanded Access Protocol for Boys With Duchenne Muscular Dystrophy

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ClinicalTrials.gov Identifier: NCT03863119
Expanded Access Status : Available
First Posted : March 5, 2019
Last Update Posted : April 8, 2022
Information provided by (Responsible Party):
ReveraGen BioPharma, Inc.

Tracking Information
First Submitted Date February 21, 2019
First Posted Date March 5, 2019
Last Update Posted Date April 8, 2022
Descriptive Information
Brief Title Expanded Access Protocol for Boys With Duchenne Muscular Dystrophy
Brief Summary The intent of this protocol is to provide continued access to vamorolone for subjects in the United States who Have Completed the VBP15-LTE, VBP15- 004, or VBP15-006 protocols (and are thereby ineligible to enroll in another trial ofvamorolone therapy), during the time a new drug application for vamorolone is under preparation and review.
Detailed Description Vamorolone will be shipped to the subject's family by the study site. The patient will receive standard of care treatment and procedures for management of DMD. Treating Physicians participating in the expanded access program are required to collect and document any physician, patient, or caregiver reported safety events and report to the Sponsor. The subject's dose of vamorolone may be increased or decreased within a range of 2.0 to 6.0 mg/kg/day (only doses of 2 mg/kg, 4 mg/kg and 6 mg/kg are allowed), given once daily. Administration of vamorolone (taken with an 8 ounce (240 ml) glass of full fat milk, or equivalent high-fat food portion) will be unchanged from the VBP15-LTE, VBP15-004, or VBP15-006 studies. In the absence of safety concerns, and while this Expanded Access protocol isactive, vamorolone may be provided indefinitely or until approval, providedthat the Treating Physician and family agrees that continued administrationof vamorolone is in the best interest of the chil.d
Study Type Expanded Access
Expanded Access Type Intermediate-size Population
Intervention Drug: Vamorolone
2.0 mg/kg/day, 4.0 mg/kg/day, or 6.0 mg/kg/day at physician discretion
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
Recruitment Information
Expanded Access Status Available
Contact: Eric Hoffman, PhD 202-390-3225 eric.hoffman@reveragen.com
Listed Location Countries Canada,   Israel,   United States
Removed Location Countries  
Administrative Information
NCT Number NCT03863119
Current Responsible Party ReveraGen BioPharma, Inc.
Original Responsible Party Same as current
Current Study Sponsor ReveraGen BioPharma, Inc.
Original Study Sponsor Same as current
Collaborators Not Provided
Investigators Not Provided
PRS Account ReveraGen BioPharma, Inc.
Verification Date March 2022