Expanded Access Protocol for Boys With Duchenne Muscular Dystrophy

November 7, 2023 updated by: Santhera Pharmaceuticals

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

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.

Study Overview

Status

Available

Conditions

Intervention / Treatment

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 Type

Expanded Access

Expanded Access Type

  • Intermediate-size Population

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

  • Canada
    • Alberta
      • Calgary, Alberta, Canada, AB T3B 6A8
        • Available
        • Alberta's Children Hospital
        • Principal Investigator:
          • Jean Mah, MD
    • British Columbia
      • Vancouver, British Columbia, Canada, V6H 3N1
        • Available
        • British Columbia Children's Hospital
        • Principal Investigator:
          • Kathryn Selby, MD
    • Ontario
      • Ottawa, Ontario, Canada, K1H 8L1
        • Available
        • Children's Hospital of Eastern Ontario
        • Principal Investigator:
          • Hanns Lochmuller, MD
      • Toronto, Ontario, Canada, M5G 1X8
        • Available
        • The Hospital for Sick Children
        • Principal Investigator:
          • Hernan Gonorazky, MD
    • Quebec
      • Montréal, Quebec, Canada, H4A 3J1
        • Available
        • The Montreal Children's Hospital
        • Principal Investigator:
          • Maryam Oskoui, MD
  • Israel
      • Petah Tikvah, Israel, 49202
        • Available
        • Schneider Children's Medical Center
        • Principal Investigator:
          • Yoram Nevo, MD
  • United States
    • California
      • Davis, California, United States, 95616
        • Available
        • University of California Davis
        • Principal Investigator:
          • Craig McDonald, MD
    • Florida
      • Orlando, Florida, United States, 32827
        • Available
        • Nemours Children's Hospital
        • Principal Investigator:
          • Omer Abdul Hamid, MD
    • Illinois
      • Chicago, Illinois, United States, 60611
        • Available
        • urie Children's Hospital of Chicago
        • Principal Investigator:
          • Nancy L. Kuntz, MD
    • North Carolina
      • Durham, North Carolina, United States, 27710
        • Available
        • Duke University
        • Principal Investigator:
          • Mai K. ElMallah, M.D.
    • Texas
      • Dallas, Texas, United States, 75207
        • Available
        • University of Texas Southwestern Medical Center
        • Principal Investigator:
          • Kaitlin Batley, MD
    • Washington
      • Seattle, Washington, United States, 98105
        • Available
        • Seattle Children's
        • Principal Investigator:
          • Seth J. Perlman, MD

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

N/A

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Santhera Pharmaceuticals

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

First Submitted

February 21, 2019

First Submitted That Met QC Criteria

March 4, 2019

First Posted (Actual)

March 5, 2019

Study Record Updates

Last Update Posted (Actual)

November 9, 2023

Last Update Submitted That Met QC Criteria

November 7, 2023

Last Verified

September 1, 2023

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • VBP15-EAP

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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