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Trial record 26 of 993 for:    BMD

Expanded Access Protocol for Boys With Duchenne Muscular Dystrophy

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ClinicalTrials.gov Identifier: NCT03863119
Expanded Access Status : Available
First Posted : March 5, 2019
Last Update Posted : March 5, 2019
Sponsor:
Information provided by (Responsible Party):
ReveraGen BioPharma, Inc.

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

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 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

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Study Type : Expanded Access
Expanded Access Type : Intermediate-size Population
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



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

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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 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

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 ClinicalTrials.gov identifier (NCT number): NCT03863119


Contacts
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Contact: Laurie Conklin, M.D. 2404085347 laurie.conklin@reveragen.com

Locations
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United States, North Carolina
Duke University
Durham, North Carolina, United States, 27710
Contact: Debra Heydt       debra.heydt@duke.edu   
Principal Investigator: Edward Smith, M.D.         
Sponsors and Collaborators
ReveraGen BioPharma, Inc.

Additional Information:
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Responsible Party: ReveraGen BioPharma, Inc.
ClinicalTrials.gov Identifier: NCT03863119     History of Changes
Other Study ID Numbers: VBP15-EAP
First Posted: March 5, 2019    Key Record Dates
Last Update Posted: March 5, 2019
Last Verified: February 2019

Keywords provided by ReveraGen BioPharma, Inc.:
Duchenne Muscular Dystrophy
Vamorolone
VBP-15
DMD

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