Long-term Extension Study to Assess Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD)

April 28, 2021 updated by: ReveraGen BioPharma, Inc.

A 24-month Phase II Open-label, Multicenter Long-term Extension Study to Assess the Long-Term Safety and Efficacy of Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD)

This long-term extension study is an open-label, multiple-dose study to evaluate the long-term safety, tolerability, efficacy and PD of vamorolone administered once daily by liquid oral suspension over a Treatment Period of 24 months to young boys with DMD who participated in the VBP15-002 Phase IIa and VBP15-003 Phase IIa extension core studies.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Detailed Description

This study will evaluate if it is safe to use a new medication called vamorolone for more than two weeks in children with DMD, if boys with DMD who take the study medication have improved muscle function compared to boys with DMD in other studies who did not take any type of steroid, and to see if boys with DMD who take the study medication gain less weight compared to boys with DMD in a prior study who took another type of steroid called prednisone. Enrolled participants will take the study medication for 24 months.

Study Type

Interventional

Enrollment (Actual)

46

Phase

  • Phase 2

Contacts and Locations

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

Study Locations

  • Australia
      • Melbourne, Australia
        • Royal Children's Hospital
      • Westmead, Australia
        • Sydney Children's Hospital
  • Canada
    • Alberta
      • Calgary, Alberta, Canada, T3B 6A8
        • Alberta Children's Hospital
  • Israel
      • Petah Tikwah, Israel, 49202
        • Schneider Children's Medical Center
  • Sweden
      • Gothenburg, Sweden, 41685
        • Queen Silvia Children's Hospital
  • United Kingdom
      • Newcastle upon Tyne, United Kingdom, NE7 7DN
        • Newcastle upon Tyne Hospitals NHS Foundation Trust
  • United States
    • California
      • Davis, California, United States, 95616
        • University of California Davis
    • Florida
      • Gainesville, Florida, United States, 32611
        • University of Florida
      • Orlando, Florida, United States, 32827
        • Nemours Children's Hospital
    • Illinois
      • Chicago, Illinois, United States, 60611
        • Ann & Robert H. Lurie Children's Hospital
    • North Carolina
      • Durham, North Carolina, United States, 27710
        • Duke University
    • Texas
      • Dallas, Texas, United States, 75207
        • University of Texas Southwestern Medical Center

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

6 months to 3 years (Child)

Accepts Healthy Volunteers

No

Genders Eligible for Study

Male

Description

Inclusion Criteria:

  1. Subject's parent or legal guardian has provided written informed consent and HIPAA authorization (if applicable) prior to any VBP15-LTE long-term extension study-specific procedures;
  2. Subject has previously completed study VBP15-003 up to and including the Week 24 Final assessments, prior to enrolling in the VBP15-LTE study at the conclusion of the VBP15-003 Week 24 Visit [Note: if entering the dose-tapering period, subject is enrolling within 8 weeks after the VBP15-003 final visit following dose-tapering]; and
  3. Subject and parent/guardian are willing and able to comply with scheduled visits, study drug administration plan, and study procedures.

Exclusion Criteria:

  1. Subject had a serious or severe adverse event in study VBP15-003 that, in the opinion of the Investigator, was probably or definitely related to vamorolone use and precludes safe use of vamorolone for the subject in this long-term extension study;
  2. Subject has current or history of major renal or hepatic impairment, diabetes mellitus or immunosuppression;
  3. Subject has current or history of chronic systemic fungal or viral infections;
  4. Subject has used mineralocorticoid receptor agents, such as spironolactone, eplerenone, canrenone (canrenoate potassium), prorenone (prorenoate potassium), mexrenone (mexrenoate potassium) within 4 weeks prior to the first dose of study medication;
  5. Subject has evidence of symptomatic cardiomyopathy. [Note: Asymptomatic cardiac abnormality on investigation would not be exclusionary];
  6. Subject is currently being treated or has received previous treatment with oral glucocorticoids or other immunosuppressive agents [Notes: Past transient use of oral glucocorticoids or other oral immunosuppressive agents for no longer than 3 months cumulative, with last use at least 3 months prior to first dose of study medication, will be considered for eligibility on a case-by-case basis. Inhaled and/or topical glucocorticoids prescribed for an indication other than DMD are permitted but must be administered at stable dose for at least 3 months prior to study drug administration];
  7. Subject has used idebenone within 4 weeks prior to the first dose of study medication;
  8. Subject has an allergy or hypersensitivity to the study medication or to any of its constituents;
  9. Subject has severe behavioral or cognitive problems that preclude participation in the study, in the opinion of the Investigator;
  10. Subject has previous or ongoing medical condition, medical history, physical findings or laboratory abnormalities that could affect safety, make it unlikely that treatment and follow-up will be correctly completed or impair the assessment of study results, in the opinion of the Investigator
  11. Subject is currently taking any investigational drug, or has taken any investigational drug other than vamorolone within 3 months prior to the start of study treatment.

Note: Subjects may be re-evaluated if ineligible due to a transient condition which would prevent the subject from participating.

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?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Non-Randomized
  • Interventional Model: Parallel Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Dose Level Group 1
Participants enrolled in Dose Level Group 1 will receive vamorolone 0.25 mg/kg/day.
Drug: Vamorolone 0.25 mg/day/day
Oral administration of 0.25 mg/kg/day daily for 24 months.
Other Names:
  • VBP15
Experimental: Dose Level Group 2
Participants enrolled in Dose Level Group 2 will receive vamorolone 0.75 mg/kg/day.
Drug: Vamorolone 0.75 mg/day/day
Oral administration of 0.75 mg/kg/day daily for 24 months.
Other Names:
  • VBP15
Experimental: Dose Level Group 3
Participants enrolled in Dose Level Group 3 will receive vamorolone 2.0 mg/kg/day.
Drug: Vamorolone 2.0 mg/day/day
Oral administration of 2.0 mg/kg/day daily for 24 months.
Other Names:
  • VBP15
Experimental: Dose Level Group 4
Participants enrolled in Dose Level Group 4 will receive vamorolone 6.0 mg/kg/day.
Drug: Vamorolone 6.0 mg/day/day
Oral administration of 6.0 mg/kg/day daily for 24 months.
Other Names:
  • VBP15

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Number of Participants With Treatment-related Adverse Events as Assessed by CTCAE Version 4.03
Time Frame: 24 months
To evaluate the long-term safety and tolerability of vamorolone, administered orally at daily doses up to 6.0 mg/kg/day over a 24- month Treatment Period, in boys ages 4-7 years with DMD; Treatment-emergent adverse events (TEAEs) are defined as any adverse event or worsening of an existing conditions after initiation of the investigational product and through the subject's last study visit (study completion or early termination);
24 months
Total Number of Adverse Events as Assessed by CTCAE Version 4.03
Time Frame: 24 Months
To evaluate the long-term safety and tolerability of vamorolone, administered orally at daily doses up to 6.0 mg/kg/day over a 24-month Treatment Period, in boys ages 4-7 years with DMD. Treatment-emergent adverse events (TEAEs) are defined as any adverse event or worsening of an existing conditions after initiation of the investigational product and through the subject's last study visit (study completion or early termination).
24 Months

Collaborators and Investigators

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

Sponsor

ReveraGen BioPharma, Inc.

Collaborators

University of Pittsburgh
Cooperative International Neuromuscular Research Group

Investigators

  • Study Chair: Paula R Clemens, MD, University of Pittsburgh

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.

General Publications

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 Major Dates

Study Start (Actual)

February 2, 2017

Primary Completion (Actual)

April 30, 2020

Study Completion (Actual)

April 30, 2020

Study Registration Dates

First Submitted

January 30, 2017

First Submitted That Met QC Criteria

January 30, 2017

First Posted (Estimate)

January 31, 2017

Study Record Updates

Last Update Posted (Actual)

May 20, 2021

Last Update Submitted That Met QC Criteria

April 28, 2021

Last Verified

April 1, 2021

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • VBP15-LTE

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

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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