An Extension Study to Assess Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD)
A Phase II Open-label, Multicenter Extension Study to Assess the Long-term Safety and Efficacy of Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD)
3 other identifiers
interventional
48
6 countries
12
Brief Summary
The main purposes of this study are to see if it is safe to use a new medication called vamorolone for more than two weeks in children with Duchenne muscular dystrophy (DMD), to see if vamorolone works for the treatment for DMD, and to see how any potential side effects compare to those seen in boys using steroids.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P25-P50 for phase_2
Started Jul 2016
12 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
April 28, 2016
CompletedFirst Posted
Study publicly available on registry
May 3, 2016
CompletedStudy Start
First participant enrolled
July 28, 2016
CompletedPrimary Completion
Last participant's last visit for primary outcome
April 26, 2018
CompletedStudy Completion
Last participant's last visit for all outcomes
April 26, 2018
CompletedResults Posted
Study results publicly available
July 23, 2019
CompletedJuly 23, 2019
July 1, 2019
1.7 years
April 28, 2016
February 25, 2019
July 19, 2019
Conditions
Keywords
Outcome Measures
Primary Outcomes (4)
Number of Participants With Adverse Events as Assessed by CTCAE Version 4.03
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). Serious adverse events were recorded for up to 30 days after the final administration of study drug; 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- week Treatment Period, in boys ages 4-7 years with DMD.
24 weeks
Total Number of Adverse Events as Assessed by CTCAE Version 4.03
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). Serious adverse events were recorded for up to 30 days after the final administration of study drug; 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- week Treatment Period, in boys ages 4-7 years with DMD.
24 weeks
Muscle Function Measured by Time to Stand Test (TTSTAND)- Velocity
To compare the efficacy, as measured by the Time to Stand Test (TTSTAND), of vamorolone administered orally at daily doses up to 6.0 mg/kg over a 24-week Treatment Period vs. untreated DMD historical controls in boys ages 4-7 years with DMD
002 Baseline, 003 Baseline, 003 Week 12, Week 24 (Note: 002 Baseline is from VBP15-002 4 week study (NCT02760264), previous to VBP15-003)
BMI Z-score
Summary of BMI Z-score of Safety Population. Please note 0 is the mean. A negative result indicates a response that is many standard deviations below the mean, and a positive result indicates a response that is many standard deviations above the mean. In this case, the closer the group mean BMI Z-score is to 0 is more favorable.
002 Baseline, 003 Week 12, Week 24
Secondary Outcomes (11)
Serum Pharmacodynamics Biomarkers Measured by Levels of HbA1c
002 Baseline, 003 Week 8, 003 Week 16, 003 Week 24, 003 Week 26-29
Serum Pharmacodynamics Biomarkers Measured by Levels of ACTH
002 Baseline, 003 Baseline, 003 Week 8, 003 Week 16, 003 Week 24, 003 Week 26-29 (Note: 002 Baseline is from VBP15-002 4 week study (NCT02760264), previous to VBP15-003)
Serum Pharmacodynamics Biomarkers Measured by Levels of Fasting Glucose
002 Baseline, 003 Week 12, 003 Week 24
Serum Pharmacodynamics Biomarkers Measured by Levels of Fasting Insulin
002 Baseline, 003 Week 12, 003 Week 24
Serum Pharmacodynamics Biomarkers Measured by Levels of Osteocalcin
002 Baseline, 003 Baseline, 003 Week 8, 003 Week 16, 003 Week 24, 003 Week 26-29 (Note: 002 Baseline is from VBP15-002 4 week study (NCT02760264), previous to VBP15-003)
- +6 more secondary outcomes
Study Arms (4)
Dose Level Group 1
EXPERIMENTALParticipants enrolled in Dose Level Group 1 will receive vamorolone 0.25 mg/kg/day.
Dose Level Group 2
EXPERIMENTALParticipants enrolled in Dose Level Group 2 will receive vamorolone 0.75 mg/kg/day.
Dose Level Group 3
EXPERIMENTALParticipants enrolled in Dose Level Group 3 will receive vamorolone 2.0 mg/kg/day.
Dose Level Group 4
EXPERIMENTALParticipants enrolled in Dose Level Group 4 will receive vamorolone 6.0 mg/kg/day.
Interventions
Oral administration of 0.25 mg/kg/day daily for 24 weeks.
Oral administration of 0.75 mg/kg/day daily for 24 weeks.
Oral administration of 2.0 mg/kg/day daily for 24 weeks.
Oral administration of 6.0 mg/kg/day daily for 24 weeks.
Eligibility Criteria
You may qualify if:
- Participant's parent or legal guardian has provided written informed consent/HIPAA authorization prior to any extension study-specific procedures;
- Participant has previously completed study VBP15-002 up to and including the Week 4 Follow-up assessments within 8 weeks prior to enrollment; and
- Participant and parent/guardian are willing and able to comply with scheduled visits, study drug administration plan, and study procedures.
You may not qualify if:
- Participant had a serious or severe adverse event in study VBP15-002 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 study;
- Participant has current or history of major renal or hepatic impairment, diabetes mellitus or immunosuppression;
- Participant has current or history of chronic systemic fungal or viral infections;
- Participant 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;
- Participant is currently being treated or has received previous treatment with oral glucocorticoids or other immunosuppressive agents. \[Notes: Past transient use of oral glucocorticoids or other oral immunosuppressive agents for no longer than 3 months cumulative, with last use at least 3 months prior to first dose of study medication, will be considered for eligibility on a case-by-case basis. Inhaled and/or topical corticosteroids prescribed for an indication other than DMD are permitted but must be administered at stable dose for at least 3 months prior to study drug administration\];
- Subject has used idebenone within 4 weeks prior to the first dose of study medication;
- Participant has an allergy or hypersensitivity to the study medication or to any of its constituents;
- Participant has severe behavioral or cognitive problems that preclude participation in the study, in the opinion of the Investigator;
- Participant 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; or
- Participant 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: Participants may be re-evaluated if ineligible due to a transient condition which would prevent the subject from participating
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (12)
University of California Davis
Davis, California, 95616, United States
University of Florida
Gainesville, Florida, 32611, United States
Nemours Children's Hospital
Orlando, Florida, 32827, United States
Ann & Robert H. Lurie Children's Hospital
Chicago, Illinois, 60611, United States
Duke University
Durham, North Carolina, 27710, United States
University of Texas Southwestern Medical Center
Dallas, Texas, 75207, United States
Royal Children's Hospital
Melbourne, Australia
Sydney Children's Hospital
Westmead, Australia
Alberta Children's Hospital
Calgary, Alberta, T3B 6A8, Canada
Schneider Children's Medical Center
Petah Tikva, 49202, Israel
Queen Silvia Children's Hospital
Gothenburg, 41685, Sweden
Newcastle upon Tyne Hospitals NHS Foundation Trust
Newcastle upon Tyne, NE7 7DN, United Kingdom
Related Publications (3)
Hoffman E, Gaglianone S, Ketema R, Tu W, Peay H, Clemens P, Dang U, Conklin L. Return of participant-level clinical trial results to participants: pilot of a simplified centralised approach. BMJ Open. 2024 Mar 23;14(3):e080097. doi: 10.1136/bmjopen-2023-080097.
PMID: 38521535DERIVEDSmith EC, Conklin LS, Hoffman EP, Clemens PR, Mah JK, Finkel RS, Guglieri M, Tulinius M, Nevo Y, Ryan MM, Webster R, Castro D, Kuntz NL, Kerchner L, Morgenroth LP, Arrieta A, Shimony M, Jaros M, Shale P, Gordish-Dressman H, Hagerty L, Dang UJ, Damsker JM, Schwartz BD, Mengle-Gaw LJ, McDonald CM; CINRG VBP15 and DNHS Investigators. Efficacy and safety of vamorolone in Duchenne muscular dystrophy: An 18-month interim analysis of a non-randomized open-label extension study. PLoS Med. 2020 Sep 21;17(9):e1003222. doi: 10.1371/journal.pmed.1003222. eCollection 2020 Sep.
PMID: 32956407DERIVEDLi X, Conklin LS, van den Anker J, Hoffman EP, Clemens PR, Jusko WJ. Exposure-Response Analysis of Vamorolone (VBP15) in Boys With Duchenne Muscular Dystrophy. J Clin Pharmacol. 2020 Oct;60(10):1385-1396. doi: 10.1002/jcph.1632. Epub 2020 May 20.
PMID: 32434278DERIVED
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Results Point of Contact
- Title
- Dr. Eric P. Hoffman
- Organization
- ReveraGen Biopharma Inc.
Study Officials
- STUDY CHAIR
Paula R Clemens, MD
University of Pittsburgh
Publication Agreements
- PI is Sponsor Employee
- No
- Restrictive Agreement
- Yes
Study Design
- Study Type
- interventional
- Phase
- phase 2
- Allocation
- NON RANDOMIZED
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SEQUENTIAL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
April 28, 2016
First Posted
May 3, 2016
Study Start
July 28, 2016
Primary Completion
April 26, 2018
Study Completion
April 26, 2018
Last Updated
July 23, 2019
Results First Posted
July 23, 2019
Record last verified: 2019-07