Expanded Access Protocol for Boys With Duchenne Muscular Dystrophy

• ClinicalTrials.gov Identifier: NCT03863119
• Expanded Access Status: Available
• First Posted: March 5, 2019
• Last Update Posted: November 9, 2023
• Sponsor: Santhera Pharmaceuticals
• Information provided by (Responsible Party): Santhera Pharmaceuticals

Study Description

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.

Study Design

• 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

Interventions

Intervention Details:

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

Eligibility Criteria

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

Criteria

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

Contacts and Locations

Contacts

Contact: Ana de Vera, MD; +41619068 ext 985; ana.devera@santhera.com

Locations

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

Israel

Schneider Children's Medical Center; Available

Petah Tikvah, Israel, 49202

Principal Investigator: Yoram Nevo, MD

Sponsors and Collaborators

Santhera Pharmaceuticals

More Information

Additional Information:

ReveraGen BioPharma 

Vamorolone Phase IIa trial in Duchenne Muscular Dystrophy 

• Responsible Party: Santhera Pharmaceuticals
• ClinicalTrials.gov Identifier: NCT03863119
• Other Study ID Numbers: VBP15-EAP
• First Posted: March 5, 2019
• Last Update Posted: November 9, 2023
• Last Verified: September 2023

Keywords provided by Santhera Pharmaceuticals:

Duchenne Muscular Dystrophy; Vamorolone; VBP-15; DMD

Additional relevant MeSH terms:

Muscular Dystrophies; Muscular Dystrophy, Duchenne; Muscular Disorders, Atrophic; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Genetic Diseases, Inborn; Genetic Diseases, X-Linked