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Expanded Access Protocol for Boys With Duchenne Muscular Dystrophy

Primary Purpose

Duchenne Muscular Dystrophy

Status
Available
Phase
Locations
International
Study Type
Expanded Access
Intervention
Vamorolone
Sponsored by
ReveraGen BioPharma, Inc.
About
Eligibility
Locations
Outcomes
Full info

About this trial

This is an expanded access trial for Duchenne Muscular Dystrophy focused on measuring Duchenne Muscular Dystrophy, Vamorolone, VBP-15, DMD

Eligibility Criteria

undefined - undefined (Child, Adult, Older Adult)Male

Inclusion Criteria:

  • Subject's parent or legal guardian has provided written informed consent/HIPAA authorization
  • Subject has previously completed at a participating US study site VBP15-LTE up to and including the Month 24 assessments, OR VBP15-004 up to and including the Week 48 assessments, within 30 days prior to participation in the VBP15-EAP
  • 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 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

Sites / Locations

  • University of California Davis
  • Nemours Children's Hospital
  • Duke University
  • University of Texas Southwestern Medical Center
  • Alberta's Children Hospital
  • British Columbia Children's Hospital
  • Children's Hospital of Eastern Ontario
  • Schneider Children's Medical Center

Outcomes

Primary Outcome Measures

Secondary Outcome Measures

Full Information

First Posted
February 21, 2019
Last Updated
March 31, 2022
Sponsor
ReveraGen BioPharma, Inc.
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1. Study Identification

Unique Protocol Identification Number
NCT03863119
Brief Title
Expanded Access Protocol for Boys With Duchenne Muscular Dystrophy
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 Studies
Study Type
Expanded Access

2. Study Status

Record Verification Date
March 2022
Overall Recruitment Status
Available
Study Start Date
undefined (undefined)
Primary Completion Date
undefined (undefined)
Study Completion Date
undefined (undefined)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
ReveraGen BioPharma, Inc.

4. Oversight

5. 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 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

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Duchenne Muscular Dystrophy
Keywords
Duchenne Muscular Dystrophy, Vamorolone, VBP-15, DMD

7. Study Design

8. Arms, Groups, and Interventions

Intervention Type
Drug
Intervention Name(s)
Vamorolone
Intervention Description
2.0 mg/kg/day, 4.0 mg/kg/day, or 6.0 mg/kg/day at physician discretion

10. Eligibility

Sex
Male
Gender Based
Yes
Eligibility Criteria
Inclusion Criteria: Subject's parent or legal guardian has provided written informed consent/HIPAA authorization Subject has previously completed at a participating US study site VBP15-LTE up to and including the Month 24 assessments, OR VBP15-004 up to and including the Week 48 assessments, within 30 days prior to participation in the VBP15-EAP 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 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
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Eric Hoffman, PhD
Phone
202-390-3225
Email
eric.hoffman@reveragen.com
Facility Information:
Facility Name
University of California Davis
City
Davis
State/Province
California
ZIP/Postal Code
95616
Country
United States
Individual Site Status
Available
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Craig McDonald, MD
Facility Name
Nemours Children's Hospital
City
Orlando
State/Province
Florida
ZIP/Postal Code
32827
Country
United States
Individual Site Status
Available
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Richard Finkel, MD
Facility Name
Duke University
City
Durham
State/Province
North Carolina
ZIP/Postal Code
27710
Country
United States
Individual Site Status
Available
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Debra Heydt
Email
debra.heydt@duke.edu
First Name & Middle Initial & Last Name & Degree
Edward Smith, M.D.
Facility Name
University of Texas Southwestern Medical Center
City
Dallas
State/Province
Texas
ZIP/Postal Code
75207
Country
United States
Individual Site Status
Available
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Diana Castro, MD
Facility Name
Alberta's Children Hospital
City
Calgary
State/Province
Alberta
ZIP/Postal Code
AB T3B 6A8
Country
Canada
Individual Site Status
Available
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Jean Mah, MD
Facility Name
British Columbia Children's Hospital
City
Vancouver
State/Province
British Columbia
ZIP/Postal Code
V6H 3N1
Country
Canada
Individual Site Status
Available
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Kathryn Selby, MD
Facility Name
Children's Hospital of Eastern Ontario
City
Ottawa
State/Province
Ontario
ZIP/Postal Code
K1H 8L1
Country
Canada
Individual Site Status
Available
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Hugh McMillan, MD
Facility Name
Schneider Children's Medical Center
City
Petah Tikvah
ZIP/Postal Code
49202
Country
Israel
Individual Site Status
Available
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Yoram Nevo, MD

12. IPD Sharing Statement

Links:
URL
http://www.reveragen.com
Description
ReveraGen BioPharma
URL
http://www.ncbi.nlm.nih.gov/pubmed/30219580
Description
Vamorolone Phase IIa trial in Duchenne Muscular Dystrophy

Learn more about this trial

Expanded Access Protocol for Boys With Duchenne Muscular Dystrophy

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