Long-term Extension Study to Assess Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD)
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)
1 other identifier
interventional
46
6 countries
12
Brief Summary
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.
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 Feb 2017
Typical duration for phase_2
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
January 30, 2017
CompletedFirst Posted
Study publicly available on registry
January 31, 2017
CompletedStudy Start
First participant enrolled
February 2, 2017
CompletedPrimary Completion
Last participant's last visit for primary outcome
April 30, 2020
CompletedStudy Completion
Last participant's last visit for all outcomes
April 30, 2020
CompletedResults Posted
Study results publicly available
May 20, 2021
CompletedMay 20, 2021
April 1, 2021
3.2 years
January 30, 2017
April 28, 2021
April 28, 2021
Conditions
Keywords
Outcome Measures
Primary Outcomes (2)
Number of Participants With Treatment-related Adverse Events as Assessed by CTCAE Version 4.03
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
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
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 months.
Oral administration of 0.75 mg/kg/day daily for 24 months.
Oral administration of 2.0 mg/kg/day daily for 24 months.
Oral administration of 6.0 mg/kg/day daily for 24 months.
Eligibility Criteria
You may qualify if:
- 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;
- 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
- Subject and parent/guardian are willing and able to comply with scheduled visits, study drug administration plan, and study procedures.
You may not qualify if:
- 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;
- Subject has current or history of major renal or hepatic impairment, diabetes mellitus or immunosuppression;
- Subject has current or history of chronic systemic fungal or viral infections;
- 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;
- Subject is currently being treated or has received previous treatment with oral glucocorticoids or other immunosuppressive agents \[Notes: Past transient use of oral glucocorticoids or other oral immunosuppressive agents for no longer than 3 months cumulative, with last use at least 3 months prior to first dose of study medication, will be considered for eligibility on a case-by-case basis. Inhaled and/or topical glucocorticoids prescribed for an indication other than DMD are permitted but must be administered at stable dose for at least 3 months prior to study drug administration\];
- Subject has used idebenone within 4 weeks prior to the first dose of study medication;
- Subject has an allergy or hypersensitivity to the study medication or to any of its constituents;
- Subject has severe behavioral or cognitive problems that preclude participation in the study, in the opinion of the Investigator;
- 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
- 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.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- ReveraGen BioPharma, Inc.lead
- University of Pittsburghcollaborator
- Cooperative International Neuromuscular Research Groupcollaborator
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 (2)
Smith 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: 32956407RESULTMah JK, Clemens PR, Guglieri M, Smith EC, Finkel RS, Tulinius M, Nevo Y, Ryan MM, Webster R, Castro D, Kuntz NL, McDonald CM, Damsker JM, Schwartz BD, Mengle-Gaw LJ, Jackowski S, Stimpson G, Ridout DA, Ayyar-Gupta V, Baranello G, Manzur AY, Muntoni F, Gordish-Dressman H, Leinonen M, Ward LM, Hoffman EP, Dang UJ; NorthStar UK Network and CINRG DNHS Investigators. Efficacy and Safety of Vamorolone in Duchenne Muscular Dystrophy: A 30-Month Nonrandomized Controlled Open-Label Extension Trial. JAMA Netw Open. 2022 Jan 4;5(1):e2144178. doi: 10.1001/jamanetworkopen.2021.44178.
PMID: 35076703DERIVED
Related Links
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Results Point of Contact
- Title
- Dr. Eric 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
- PARALLEL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
January 30, 2017
First Posted
January 31, 2017
Study Start
February 2, 2017
Primary Completion
April 30, 2020
Study Completion
April 30, 2020
Last Updated
May 20, 2021
Results First Posted
May 20, 2021
Record last verified: 2021-04