Duchenne Muscular DystrophyPhase 2NCT02760277
An Extension Study to Assess Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD)
A Phase 2 study testing Vamorolone 0.25 mg/day/day, Vamorolone 0.75 mg/day/day and Vamorolone 2.0 mg/day/day in people with Duchenne Muscular Dystrophy, run by ReveraGen BioPharma, Inc. at 12 sites in 6 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 4 to 7 years, men only
- Conditions the study is for
- Duchenne Muscular Dystrophy
- What is being tested?
- Vamorolone 0.25 mg/day/day, Vamorolone 0.75 mg/day/day and Vamorolone 2.0 mg/day/day
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 48 participants
- Where
- 12 sitesin 6 countries
- Study dates
- 28 Jul 2016to 26 Apr 2018
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 4 to 7 years
- Men only
- No healthy volunteers
- Child
Who may be able to join
- 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.
Who cannot join
- 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 has evidence of symptomatic cardiomyopathy. [Note: Asymptomatic cardiac abnormality on investigation would not be exclusionary];
- 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];
Show all 12Show fewer
- 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
Trial locations and contacts
12 sites in 6 countries.
12 sites in 6 countries
United States6 sites
Ann & Robert H. Lurie Children's Hospital
Chicago, Illinois, 60611
University of Texas Southwestern Medical Center
Dallas, Texas, 75207
University of California Davis
Davis, California, 95616
Duke University
Durham, North Carolina, 27710
University of Florida
Gainesville, Florida, 32611
Nemours Children's Hospital
Orlando, Florida, 32827
Australia2 sites
Royal Children's Hospital
Melbourne
Sydney Children's Hospital
Westmead
United Kingdom1 site
Newcastle upon Tyne Hospitals NHS Foundation Trust
Newcastle upon Tyne, NE7 7DN
About this study
Official title: 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)
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.
Read the 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 weeks.
- Conditions
- Duchenne Muscular Dystrophy
- Being tested
- Vamorolone 0.25 mg/day/day, Vamorolone 0.75 mg/day/day, Vamorolone 2.0 mg/day/day, Vamorolone 6.0 mg/day/day
- Keywords
- Duchenne muscular dystrophy, vamorolone
- Medical subject headings
- Muscular Dystrophy, Duchenne
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Vamorolone 0.25 mg/day/dayDrug
Also called VBP15
Oral administration of 0.25 mg/kg/day daily for 24 weeks.
Vamorolone 0.75 mg/day/dayDrug
Also called VBP15
Oral administration of 0.75 mg/kg/day daily for 24 weeks.
Vamorolone 2.0 mg/day/dayDrug
Also called VBP15
Oral administration of 2.0 mg/kg/day daily for 24 weeks.
Vamorolone 6.0 mg/day/dayDrug
Also called VBP15
Oral administration of 6.0 mg/kg/day daily for 24 weeks.
| Study group | Role | What they receive |
|---|---|---|
| Dose Level Group 1 | Experimental | Participants enrolled in Dose Level Group 1 will receive vamorolone 0.25 mg/kg/day. Receives: Drug: Vamorolone 0.25 mg/day/day |
| Dose Level Group 2 | Experimental | Participants enrolled in Dose Level Group 2 will receive vamorolone 0.75 mg/kg/day. Receives: Drug: Vamorolone 0.75 mg/day/day |
| Dose Level Group 3 | Experimental | Participants enrolled in Dose Level Group 3 will receive vamorolone 2.0 mg/kg/day. Receives: Drug: Vamorolone 2.0 mg/day/day |
| Dose Level Group 4 | Experimental | Participants enrolled in Dose Level Group 4 will receive vamorolone 6.0 mg/kg/day. Receives: Drug: Vamorolone 6.0 mg/day/day |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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.
Measured: 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
Measured: 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.
Measured: 002 Baseline, 003 Week 12, Week 24
Secondary outcomes (11)
Serum Pharmacodynamics Biomarkers Measured by Levels of HbA1c
To investigate the effects of vamorolone, administered orally at daily doses up to 6.0 mg/kg over a 24-week Treatment Period vs. prednisone-treated historical controls, on serum pharmacodynamic (PD) biomarkers of safety (insulin resistance, adrenal axis suppression, and bone turnover). SomaScan aptamer panels testing 1,200 serum proteins were used to discover a candidate set of prednisone-responsive biomarkers, with a subset of these validating in a longitudinal sample set (individual DMD patients pre/post steroid treatment). These PD biomarkers were assigned to a safety panel or efficacy panel based on comparison to normal controls and information concerning the function of each protein.
Measured: 002 Baseline, 003 Week 8, 003 Week 16, 003 Week 24, 003 Week 26-29
Serum Pharmacodynamics Biomarkers Measured by Levels of ACTH
To investigate the effects of vamorolone, administered orally at daily doses up to 6.0 mg/kg over a 24-week Treatment Period vs. prednisone-treated historical controls, on serum pharmacodynamic (PD) biomarkers of safety (insulin resistance, adrenal axis suppression, and bone turnover). SomaScan aptamer panels testing 1,200 serum proteins were used to discover a candidate set of prednisone-responsive biomarkers, with a subset of these validating in a longitudinal sample set (individual DMD patients pre/post steroid treatment). These PD biomarkers were assigned to a safety panel or efficacy panel based on comparison to normal controls and information concerning the function of each protein.
Measured: 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
To investigate the effects of vamorolone, administered orally at daily doses up to 6.0 mg/kg over a 24-week Treatment Period vs. prednisone-treated historical controls, on serum pharmacodynamic (PD) biomarkers of safety (insulin resistance, adrenal axis suppression, and bone turnover). SomaScan aptamer panels testing 1,200 serum proteins were used to discover a candidate set of prednisone-responsive biomarkers, with a subset of these validating in a longitudinal sample set (individual DMD patients pre/post steroid treatment). These PD biomarkers were assigned to a safety panel or efficacy panel based on comparison to normal controls and information concerning the function of each protein.
Measured: 002 Baseline, 003 Week 12, 003 Week 24
Serum Pharmacodynamics Biomarkers Measured by Levels of Fasting Insulin
To investigate the effects of vamorolone, administered orally at daily doses up to 6.0 mg/kg over a 24-week Treatment Period vs. prednisone-treated historical controls, on serum pharmacodynamic (PD) biomarkers of safety (insulin resistance, adrenal axis suppression, and bone turnover). SomaScan aptamer panels testing 1,200 serum proteins were used to discover a candidate set of prednisone-responsive biomarkers, with a subset of these validating in a longitudinal sample set (individual DMD patients pre/post steroid treatment). These PD biomarkers were assigned to a safety panel or efficacy panel based on comparison to normal controls and information concerning the function of each protein.
Measured: 002 Baseline, 003 Week 12, 003 Week 24
Serum Pharmacodynamics Biomarkers Measured by Levels of Osteocalcin
To investigate the effects of vamorolone, administered orally at daily doses up to 6.0 mg/kg over a 24-week Treatment Period vs. prednisone-treated historical controls, on serum pharmacodynamic (PD) biomarkers of safety (insulin resistance, adrenal axis suppression, and bone turnover). SomaScan aptamer panels testing 1,200 serum proteins were used to discover a candidate set of prednisone-responsive biomarkers, with a subset of these validating in a longitudinal sample set (individual DMD patients pre/post steroid treatment). These PD biomarkers were assigned to a safety panel or efficacy panel based on comparison to normal controls and information concerning the function of each protein.
Measured: 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 P1NP
To investigate the effects of vamorolone, administered orally at daily doses up to 6.0 mg/kg over a 24-week Treatment Period vs. prednisone-treated historical controls, on serum pharmacodynamic (PD) biomarkers of safety (insulin resistance, adrenal axis suppression, and bone turnover). SomaScan aptamer panels testing 1,200 serum proteins were used to discover a candidate set of prednisone-responsive biomarkers, with a subset of these validating in a longitudinal sample set (individual DMD patients pre/post steroid treatment). These PD biomarkers were assigned to a safety panel or efficacy panel based on comparison to normal controls and information concerning the function of each protein.
Measured: 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 CTX
To investigate the effects of vamorolone, administered orally at daily doses up to 6.0 mg/kg over a 24-week Treatment Period vs. prednisone-treated historical controls, on serum pharmacodynamic (PD) biomarkers of safety (insulin resistance, adrenal axis suppression, and bone turnover). SomaScan aptamer panels testing 1,200 serum proteins were used to discover a candidate set of prednisone-responsive biomarkers, with a subset of these validating in a longitudinal sample set (individual DMD patients pre/post steroid treatment). These PD biomarkers were assigned to a safety panel or efficacy panel based on comparison to normal controls and information concerning the function of each protein.
Measured: 002 Baseline, 003 Baseline, 003 Week 8, 003 Week 16, Week 24, 003 Week 26-29 (Note: 002 Baseline is from VBP15-002 4 week study (NCT02760264), previous to VBP15-003)
Muscle Strength, Mobility, and Functional Exercise Capacity as Measured by Time to Climb Test (TTCLIMB)- Velocity
To investigate the effects of vamorolone, administered orally at daily doses up to 6.0 mg/kg over a 24-week Treatment Period, on muscle strength, mobility and functional exercise capacity vs. historical controls as measured by Time to Climb Test (TTCLIMB) in boys ages 4-7 years with DMD.
Measured: 002 Baseline, 003 Baseline, 003 Week 12, 003 Week 24 (Note: 002 Baseline is from VBP15-002 4 week study (NCT02760264), previous to VBP15-003)
Muscle Strength, Mobility, and Functional Exercise Capacity as Measured by Time to Run/Walk 10 Meters Test (TTRW)- Velocity
To investigate the effects of vamorolone, administered orally at daily doses up to 6.0 mg/kg over a 24-week Treatment Period, on muscle strength, mobility and functional exercise capacity vs. historical controls as measured by Time to Run/Walk Test (TTRW) in boys ages 4-7 years with DMD.
Measured: 002 Baseline, 003 Baseline, 003 Week 12, 003 Week 24 (Note: 002 Baseline is from VBP15-002 4 week study (NCT02760264), previous to VBP15-003)
Muscle Strength, Mobility, and Functional Exercise Capacity as Measured by North Star Ambulatory Assessment (NSAA)
To investigate the effects of vamorolone, administered orally at daily doses up to 6.0 mg/kg over a 24-week Treatment Period, on muscle strength, mobility and functional exercise capacity vs. historical controls as measured by North Star Ambulatory Assessment (NSAA) in boys ages 4-7 years with DMD. ***Total NSAA score is being reported. The score can range from 0 to 32. Higher scores (approaching 32) indicate a better outcome assessing functional mobility.
Measured: 002 Baseline, 003 Baseline, 003 Week 12, 003 Week 24 (Note: 002 Baseline is from VBP15-002 4 week study (NCT02760264), previous to VBP15-003)
Muscle Strength, Mobility, and Functional Exercise Capacity vs. Historical Controls as Measured by 6-minute Walk Test (6MWT) Meters
To investigate the effects of vamorolone, administered orally at daily doses up to 6.0 mg/kg over a 24-week Treatment Period, on muscle strength, mobility and functional exercise capacity vs. historical controls as measured by 6-minute Walk Test (6MWT) in boys ages 4-7 years with DMD.
Measured: 002 Baseline, 003 Baseline, 003 Week 12, 003 Week 24 (Note: 002 Baseline is from VBP15-002 4 week study (NCT02760264), previous to VBP15-003)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 May 2016 (estimated)
- Study start28 Jul 2016
- Primary completion26 Apr 2018
- Study completion26 Apr 2018
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (4)Papers the sponsor has linked to this study.
- Other trials for Duchenne Muscular DystrophyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 48 participants (actual)
Sponsor and investigators
- Lead sponsor
- ReveraGen BioPharma, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- University of Pittsburgh, National Institute of Neurological Disorders and Stroke (NINDS), Cooperative International Neuromuscular Research Group, National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)
- Responsible party
- Sponsor
- Investigators
- Paula R Clemens, MD, Study chair, University of Pittsburgh
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02760277
- Sponsor's protocol ID
- VBP15-003
- Other IDs
- 1R44NS095423-01, 1U34AR068616-01
- Status last verified
- Jul 2019
- First submitted
- 28 Apr 2016
- Last updated
- 23 Jul 2019
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Publications
Dang UJ, Fang Y, Sabbatini D, Pegoraro E, Bello L, Clemens PR, Guglieri M, van den Anker J, Damsker J, Hagerty L, Hathout Y, Ziemba M, Morgenroth L, Bhattacharya S, Nagaraju K, Jaiswal JK, Hoffman EP. Integrating Genetic Modifier Genotype With Serum Proteomics in Duchenne Muscular Dystrophy Clinical Trials Links LTBP4 Genetic Modifier to IL-23/CD93 Pathways in Muscle. Am J Med Genet A. 2026 Aug 18. doi: 10.1002/ajmg.a.70277. Online ahead of print.
PubMed 42613652Hoffman 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.
PubMed 38521535Smith 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.
PubMed 32956407Li 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.
PubMed 32434278
Study at a glance
From the registry record, last updated by the sponsor on 23 Jul 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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)
- ClinicalTrials.gov ID
- NCT02760277
- Acronym / sponsor ID
- VBP15-003
- Conditions
- Duchenne Muscular Dystrophy
- Keywords
- Duchenne muscular dystrophy, vamorolone
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 48 participants (actual)
- Lead sponsor
- ReveraGen BioPharma, Inc.Industry (pharma, biotech, devices)
- Collaborators
- University of Pittsburgh, National Institute of Neurological Disorders and Stroke (NINDS), Cooperative International Neuromuscular Research Group, National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)
- Study start
- 28 Jul 2016 (actual)
- Primary completion
- 26 Apr 2018 (actual)
- Study completion
- 26 Apr 2018 (actual)
- First posted
- 3 May 2016 (estimated)
- Results
- Posted 23 Jul 2019
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
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 weeks.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| Dose Level Group 1 | Experimental | Drug: Vamorolone 0.25 mg/day/dayParticipants enrolled in Dose Level Group 1 will receive vamorolone 0.25 mg/kg/day. |
| Dose Level Group 2 | Experimental | Drug: Vamorolone 0.75 mg/day/dayParticipants enrolled in Dose Level Group 2 will receive vamorolone 0.75 mg/kg/day. |
| Dose Level Group 3 | Experimental | Drug: Vamorolone 2.0 mg/day/dayParticipants enrolled in Dose Level Group 3 will receive vamorolone 2.0 mg/kg/day. |
| Dose Level Group 4 | Experimental | Drug: Vamorolone 6.0 mg/day/dayParticipants enrolled in Dose Level Group 4 will receive vamorolone 6.0 mg/kg/day. |
Who may qualify
- Ages
- 4 to 7 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (3 listed)
- 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.
Key exclusion criteria (12 listed)
- 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 has evidence of symptomatic cardiomyopathy. [Note: Asymptomatic cardiac abnormality on investigation would not be exclusionary];
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Vamorolone 0.25 mg/day/dayAlso: VBP15Oral administration of 0.25 mg/kg/day daily for 24 weeks. | Drug | Dose Level Group 1 |
| Vamorolone 0.75 mg/day/dayAlso: VBP15Oral administration of 0.75 mg/kg/day daily for 24 weeks. | Drug | Dose Level Group 2 |
| Vamorolone 2.0 mg/day/dayAlso: VBP15Oral administration of 2.0 mg/kg/day daily for 24 weeks. | Drug | Dose Level Group 3 |
| Vamorolone 6.0 mg/day/dayAlso: VBP15Oral administration of 6.0 mg/kg/day daily for 24 weeks. | Drug | Dose Level Group 4 |
Primary outcome
Number of Participants With Adverse Events as Assessed by CTCAE Version 4.03
Time frame: 24 weeks
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.
Total Number of Adverse Events as Assessed by CTCAE Version 4.03
Time frame: 24 weeks
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.
Muscle Function Measured by Time to Stand Test (TTSTAND)- Velocity
Time frame: 002 Baseline, 003 Baseline, 003 Week 12, Week 24 (Note: 002 Baseline is from VBP15-002 4 week study (NCT02760264), previous to VBP15-003)
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
BMI Z-score
Time frame: 002 Baseline, 003 Week 12, Week 24
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.
11 secondary outcomes
Serum Pharmacodynamics Biomarkers Measured by Levels of HbA1c
Time frame: 002 Baseline, 003 Week 8, 003 Week 16, 003 Week 24, 003 Week 26-29
Serum Pharmacodynamics Biomarkers Measured by Levels of ACTH
Time frame: 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
Time frame: 002 Baseline, 003 Week 12, 003 Week 24
Serum Pharmacodynamics Biomarkers Measured by Levels of Fasting Insulin
Time frame: 002 Baseline, 003 Week 12, 003 Week 24
Serum Pharmacodynamics Biomarkers Measured by Levels of Osteocalcin
Time frame: 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 P1NP
Time frame: 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 CTX
Time frame: 002 Baseline, 003 Baseline, 003 Week 8, 003 Week 16, Week 24, 003 Week 26-29 (Note: 002 Baseline is from VBP15-002 4 week study (NCT02760264), previous to VBP15-003)
Muscle Strength, Mobility, and Functional Exercise Capacity as Measured by Time to Climb Test (TTCLIMB)- Velocity
Time frame: 002 Baseline, 003 Baseline, 003 Week 12, 003 Week 24 (Note: 002 Baseline is from VBP15-002 4 week study (NCT02760264), previous to VBP15-003)
Muscle Strength, Mobility, and Functional Exercise Capacity as Measured by Time to Run/Walk 10 Meters Test (TTRW)- Velocity
Time frame: 002 Baseline, 003 Baseline, 003 Week 12, 003 Week 24 (Note: 002 Baseline is from VBP15-002 4 week study (NCT02760264), previous to VBP15-003)
Muscle Strength, Mobility, and Functional Exercise Capacity as Measured by North Star Ambulatory Assessment (NSAA)
Time frame: 002 Baseline, 003 Baseline, 003 Week 12, 003 Week 24 (Note: 002 Baseline is from VBP15-002 4 week study (NCT02760264), previous to VBP15-003)
Muscle Strength, Mobility, and Functional Exercise Capacity vs. Historical Controls as Measured by 6-minute Walk Test (6MWT) Meters
Time frame: 002 Baseline, 003 Baseline, 003 Week 12, 003 Week 24 (Note: 002 Baseline is from VBP15-002 4 week study (NCT02760264), previous to VBP15-003)
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Jul 2019.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 42613652Dang UJ, Fang Y, Sabbatini D, Pegoraro E, Bello L, Clemens PR, Guglieri M, van den Anker J, Damsker J, Hagerty L, Hathout Y, Ziemba M, Morgenroth L, Bhattacharya S, Nagaraju K, Jaiswal JK, Hoffman EP. Integrating Genetic Modifier Genotype With Serum Proteomics in Duchenne Muscular Dystrophy Clinical Trials Links LTBP4 Genetic Modifier to IL-23/CD93 Pathways in Muscle. Am J Med Genet A. 2026 Aug 18. doi: 10.1002/ajmg.a.70277. Online ahead of print.
- Derived from this study: PubMed 38521535Hoffman 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.
- Derived from this study: PubMed 32956407Smith 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.
- Derived from this study: PubMed 32434278Li 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.
Related trials for Duchenne Muscular Dystrophy
RecruitingEarly Phase 1
Safety and Dystrophin Expression of SPOT-03 in Duchenne Muscular Dystrophy (DMD) Patients
Duchenne Muscular Dystrophy (DMD)
RecruitingPhase 2
Safety and Efficacy of KER-065 in Participants With Duchenne Muscular Dystrophy
Duchenne Muscular Dystrophy
Not yet recruitingPhase 2
A Phase 2 Trial to Investigate the Efficacy, Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Tegacorat in Male Participants 4 to <7 Years of Age With Duchenne Muscular Dystrophy
Duchenne Muscular Dystrophy (DMD)
Not yet recruitingObservational study
Multimodal Study About Absence of Dp140 in Becker Muscular Dystrophy: Brain-muscle Imaging, Cognition and Muscle Function
Becker Muscular Dystrophy
Enrolling by invitationObservational study
A Long-term Observational Study Evaluating Eteplirsen, Golodirsen, or Casimersen in Routine Clinical Practice
Duchenne Muscular Dystrophy
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Paula R Clemens, MD study chair
University of Pittsburgh
12 sites in 6 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Vamorolone 0.25 mg/day/day, Vamorolone 0.75 mg/day/day and Vamorolone 2.0 mg/day/day in people with Duchenne Muscular Dystrophy, run by ReveraGen BioPharma, Inc. at 12 sites in 6 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 12 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 23 Jul 2019. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 12 sites in United States, Australia, Canada, Israel, Sweden and 1 more country. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 23 Jul 2019). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Participants With Adverse Events as Assessed by CTCAE Version 4.03 (time frame: 24 weeks). The study lists 4 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02760277, which also lists 4 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 23 Jul 2019.