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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 : November 9, 2023
Information provided by (Responsible Party):
Santhera Pharmaceuticals

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 of vamorolone therapy), during the time a new drug application for vamorolone is under preparation and review.

Condition or disease Intervention/treatment
Duchenne Muscular Dystrophy Drug: Vamorolone

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 child.

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Study Type : Expanded Access
Expanded Access Type : Intermediate-size Population
  See clinical trials of the intervention/treatment in this expanded access record.
Official Title: 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

Intervention Details:
  • Drug: Vamorolone
    2.0 mg/kg/day, 4.0 mg/kg/day, or 6.0 mg/kg/day at physician discretion

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   Male
Gender Based Eligibility:   Yes

Inclusion Criteria:

  • 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

Exclusion Criteria:

  • 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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT03863119

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Contact: Ana de Vera, MD +41619068 ext 985

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United States, California
University of California Davis Available
Davis, California, United States, 95616
Principal Investigator: Craig McDonald, MD         
United States, Florida
Nemours Children's Hospital Available
Orlando, Florida, United States, 32827
Principal Investigator: Omer Abdul Hamid, MD         
United States, Illinois
urie Children's Hospital of Chicago Available
Chicago, Illinois, United States, 60611
Principal Investigator: Nancy L. Kuntz, MD         
United States, North Carolina
Duke University Available
Durham, North Carolina, United States, 27710
Principal Investigator: Mai K. ElMallah, M.D.         
United States, Texas
University of Texas Southwestern Medical Center Available
Dallas, Texas, United States, 75207
Principal Investigator: Kaitlin Batley, MD         
United States, Washington
Seattle Children's Available
Seattle, Washington, United States, 98105
Principal Investigator: Seth J. Perlman, MD         
Canada, Alberta
Alberta's Children Hospital Available
Calgary, Alberta, Canada, AB T3B 6A8
Principal Investigator: Jean Mah, MD         
Canada, British Columbia
British Columbia Children's Hospital Available
Vancouver, British Columbia, Canada, V6H 3N1
Principal Investigator: Kathryn Selby, MD         
Canada, Ontario
Children's Hospital of Eastern Ontario Available
Ottawa, Ontario, Canada, K1H 8L1
Principal Investigator: Hanns Lochmüller, MD         
The Hospital for Sick Children Available
Toronto, Ontario, Canada, M5G 1X8
Principal Investigator: Hernan Gonorazky, MD         
Canada, Quebec
The Montreal Children's Hospital Available
Montréal, Quebec, Canada, H4A 3J1
Principal Investigator: Maryam Oskoui, MD         
Schneider Children's Medical Center Available
Petah Tikvah, Israel, 49202
Principal Investigator: Yoram Nevo, MD         
Sponsors and Collaborators
Santhera Pharmaceuticals
Additional Information:
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Responsible Party: Santhera Pharmaceuticals Identifier: NCT03863119    
Other Study ID Numbers: VBP15-EAP
First Posted: March 5, 2019    Key Record Dates
Last Update Posted: November 9, 2023
Last Verified: September 2023
Keywords provided by Santhera Pharmaceuticals:
Duchenne Muscular Dystrophy
Additional relevant MeSH terms:
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Muscular Dystrophies
Muscular Dystrophy, Duchenne
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, Inborn
Genetic Diseases, X-Linked