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Expanded Access Protocol for Boys With Duchenne Muscular Dystrophy

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. Identifier: NCT03863119
Expanded Access Status : Available
First Posted : March 5, 2019
Last Update Posted : March 5, 2019
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 March 5, 2019
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 or VBP15- 004 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
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 or VBP15-004 studies. In the absence of safety concerns, and while this Expaneded Access protocol is active, vamorolone may be provided indefinitely or until approval, provided that the Treating Physician and family agrees that continued administration of vamorolone is in the best interest of the child
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 Identifier (NCT Number) in Medline.
Recruitment Information
Expanded Access Status Available
Contact: Laurie Conklin, M.D. 2404085347
Listed Location Countries United States
Removed Location Countries  
Administrative Information
NCT Number NCT03863119
Responsible Party ReveraGen BioPharma, Inc.
Study Sponsor ReveraGen BioPharma, Inc.
Collaborators Not Provided
Investigators Not Provided
PRS Account ReveraGen BioPharma, Inc.
Verification Date February 2019