Duchenne Muscular DystrophyPhase 2NCT02760264
A Study to Assess Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD)
A Phase 2 study testing Vamorolone 0.25 mg/kg/day, Vamorolone 0.75 mg/kg/day and Vamorolone 2.0 mg/kg/day in people with Duchenne Muscular Dystrophy, run by ReveraGen BioPharma, Inc. at 12 sites in 6 countries.
Trial summary
The purpose of this study is to determine whether a new medication called vamorolone is safe and well-tolerated by boys with Duchenne muscular dystrophy (DMD) ages ≥ 4 and < 7 years old. Read more about this study
- Who is it for?
- Ages 4 to 6 years, men only
- Conditions the study is for
- Duchenne Muscular Dystrophy
- What is being tested?
- Vamorolone 0.25 mg/kg/day, Vamorolone 0.75 mg/kg/day and Vamorolone 2.0 mg/kg/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
- Jun 2016to 1 May 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 6 years
- Men only
- No healthy volunteers
- Child
Who may be able to join
- Subject's parent or legal guardian has provided written informed consent/Health Insurance Portability and Accountability Act (HIPAA) authorization prior to any study-related procedures;
- Subject has a confirmed (by Central Genetic Counselor) diagnosis of DMD as defined as:
- Dystrophin immunofluorescence and/or immunoblot showing complete dystrophin deficiency, and clinical picture consistent with typical DMD, OR
- Identifiable mutation within the DMD gene (deletion/duplication of one or more exons) where reading frame can be predicted as 'out-of-frame', and clinical picture consistent with typical DMD, OR
- Complete dystrophin gene sequencing showing an alteration (point mutation, duplication, other) that is expected to preclude production of the dystrophin protein (i.e. nonsense mutation, deletion/duplication leading to a downstream stop codon), with a typical clinical picture of DMD;
- Subject is ≥ 4 years and < 7 years of age at time of enrollment in the study;
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- Subject is able to complete the Time to Stand Test (TTSTAND) without assistance, as assessed at the Screening and Baseline Visits;
- Clinical laboratory test results are within the normal range at the Screening Visit, or if abnormal, are not clinically significant, in the opinion of the Investigator. (Note: Serum gamma glutamyl transferase [GGT], creatinine, and total bilirubin all must be ≤ upper limit of the normal range at the Screening Visit);
- Subject has evidence of chicken pox immunity as determined by presence of IgG antibodies to varicella, as documented by a positive test result from the testing laboratory at the Screening Visit; and
- Subject and parent/guardian are willing and able to comply with scheduled visits, study drug administration plan, and study procedures.
Who cannot join
- 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 had an acute illness within 4 weeks prior to the first dose of study medication;
- 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 has evidence of symptomatic cardiomyopathy. [Note: Asymptomatic cardiac abnormality on investigation would not be exclusionary];
- 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 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];
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- 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 taking any other investigational drug currently or has taken any other investigational drug within 3 months prior to the start of study treatment; or
- Subject has previously been enrolled in the study.
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 IIa Open-Label, Multiple Ascending Dose Study to Assess the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Exploratory Efficacy of Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD)
The purpose of this study is to determine whether a new medication called vamorolone is safe and well-tolerated by boys with Duchenne muscular dystrophy (DMD) ages ≥ 4 and < 7 years old.
Read the detailed description
This study will evaluate the safety and tolerability of a new steroid-like medication called vamorolone in boys with DMD ages ≥ 4 years and < 7 years. Enrolled participants will take the study medication for 14 days followed by a 14 day follow-up period. The potential effectiveness of vamorolone in treating DMD will also be explored.
- Conditions
- Duchenne Muscular Dystrophy
- Being tested
- Vamorolone 0.25 mg/kg/day, Vamorolone 0.75 mg/kg/day, Vamorolone 2.0 mg/kg/day, Vamorolone 6.0 mg/kg/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/kg/dayDrug
Also called VBP15
Oral administration of 0.25 mg/kg/day daily for 14 days.
Vamorolone 0.75 mg/kg/dayDrug
Also called VBP15
Oral administration of 0.75 mg/kg/day daily for 14 days.
Vamorolone 2.0 mg/kg/dayDrug
Also called VBP15
Oral administration of 2.0 mg/kg/day daily for 14 days.
Vamorolone 6.0 mg/kg/dayDrug
Also called VBP15
Oral administration of 6 mg/kg/day daily for 14 days.
| 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/kg/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/kg/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/kg/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/kg/day |
What is being measured
The primary outcome is the main question the study is designed to answer.
Overall Summary 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.
Note: Total Number of Treatment Emergent Adverse Events: The total incidences of TEAEs experienced in study; Any Treatment Emergent Adverse Event: TEAEs reported at least once per dose group
Measured: Adverse events will be recorded from the date of informed consent and through the time of the subject's last study visit. Serious adverse events will be recorded from the date of informed consent and for up to 30 days after final drug administration.
Secondary outcomes (15)
Serum Pharmacodynamic Biomarkers (Insulin Resistance)- Fasting Glucose
Pharmacodynamic biomarkers were measured to investigate the effects of single and multiple oral doses of vamorolone on serum PD biomarkers in ambulant boys ages 4-< 7 years with DMD.
Measured: Baseline, Week 2
Serum Pharmacodynamic Biomarkers (Insulin Resistance) -Fasting Glucose
Pharmacodynamic biomarkers were measured to investigate the effects of single and multiple oral doses of vamorolone on serum PD biomarkers in ambulant boys ages 4-< 7 years with DMD.
Measured: Baseline, Week 2
Serum Pharmacodynamic Biomarkers (Insulin Resistance)- Insulin
Pharmacodynamic biomarkers were measured to investigate the effects of single and multiple oral doses of vamorolone on serum PD biomarkers in ambulant boys ages 4-< 7 years with DMD.
Measured: Baseline , Week 2
Serum Pharmacodynamic Biomarkers (Insulin Resistance)- Insulin
Pharmacodynamic biomarkers were measured to investigate the effects of single and multiple oral doses of vamorolone on serum PD biomarkers in ambulant boys ages 4-< 7 years with DMD.
Measured: Baseline, Week 2
Serum Pharmacodynamic Biomarkers (Adrenal Axis Suppression)- First in Morning Cortisol
Pharmacodynamic biomarkers were measured to investigate the effects of single and multiple oral doses of vamorolone on serum PD biomarkers in ambulant boys ages 4-< 7 years with DMD.
Measured: Week 2 (pre-dose)
Serum Pharmacodynamics Biomarkers (Bone Turnover) -Osteocalcin
Pharmacodynamic biomarkers were measured to investigate the effects of single and multiple oral doses of vamorolone on serum PD biomarkers in ambulant boys ages 4-< 7 years with DMD.
Measured: Baseline, Day 1, Week 2, Week 4
Serum Pharmacodynamic Biomarkers (Bone Turnover)- Procollagen 1 N-Terminal Propeptide
Pharmacodynamic biomarkers were measured to investigate the effects of single and multiple oral doses of vamorolone on serum PD biomarkers in ambulant boys ages 4-< 7 years with DMD.
Measured: Baseline Day 1 Week 2 Week 4
Serum Pharmacodynamic Biomarkers (Bone Turnover)- Procollagen 1 N-Terminal Propeptide
Pharmacodynamic biomarkers were measured to investigate the effects of single and multiple oral doses of vamorolone on serum PD biomarkers in ambulant boys ages 4-< 7 years with DMD.
Measured: Baseline, Day 1, Week 2, Week 4
Serum Pharmacodynamic Biomarkers (Bone Turnover)-Type I Collagen C-Telopeptides
Pharmacodynamic biomarkers were measured to investigate the effects of single and multiple oral doses of vamorolone on serum PD biomarkers in ambulant boys ages 4-< 7 years with DMD.
Measured: Baseline, Day 1, Week 4
Pharmacokinetic (PK) Assessments (Tmax)
Plasma concentrations of vamorolone were measured using a specific and validated liquid chromatography tandem mass spectrometry (LC-MS) assay. tmax= time when plasma concentration is at maximum.
Measured: Day 1, Week 2
Pharmacokinetic (PK) Assessments (AUC Inf)
Plasma concentrations of vamorolone were measured using a specific and validated liquid chromatography tandem mass spectrometry (LC-MS) assay. AUC inf= Area under the concentration vs. time curve to time infinity.
Measured: Day 1, Week 2
Pharmacokinetic (PK) Assessments CL (ml/hr/kg)
Plasma concentrations of vamorolone were measured using a specific and validated liquid chromatography tandem mass spectrometry (LC-MS) assay
Measured: Day 1, Week 2
Pharmacokinetic (PK) Assessments t(1/2)
Plasma concentrations of vamorolone were measured using a specific and validated liquid chromatography tandem mass spectrometry (LC-MS) assay. t1/2= elimination half life.
Measured: Day 1, Week 2
Pharmacokinetic (PK) Assessments (Cmax)
Plasma concentrations of vamorolone were measured using a specific and validated liquid chromatography tandem mass spectrometry (LC-MS) assay
Measured: Day 1, Week 2
Metabolites in Safety Testing (MIST) Assessment
A portion of each blood sample of the Week 2 (Day 14) pharmacokinetic assessment time points for the subjects receiving vamorolone 2 mg/kg/day was used for analysis of vamorolone metabolites.
Measured: Week 2 (Day 14)
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 startJun 2016
- Primary completion1 May 2018
- Study completion1 May 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 Arthritis and Musculoskeletal and Skin Diseases (NIAMS), National Institute of Neurological Disorders and Stroke (NINDS), Cooperative International Neuromuscular Research Group
- Responsible party
- Sponsor
- Investigators
- Paula R Clemens, MD, Study chair, University of Pittsburgh
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02760264
- Sponsor's protocol ID
- VBP15-002
- Other IDs
- 1R44NS095423-01, 1U34AR068616-01
- Status last verified
- Dec 2018
- First submitted
- 28 Apr 2016
- Last updated
- 2 Jan 2019
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- Individual data sharing
- Undecided
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 2 Jan 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase IIa Open-Label, Multiple Ascending Dose Study to Assess the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Exploratory Efficacy of Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD)
- ClinicalTrials.gov ID
- NCT02760264
- Acronym / sponsor ID
- VBP15-002
- 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 Arthritis and Musculoskeletal and Skin Diseases (NIAMS), National Institute of Neurological Disorders and Stroke (NINDS), Cooperative International Neuromuscular Research Group
- Study start
- Jun 2016
- Primary completion
- 1 May 2018 (actual)
- Study completion
- 1 May 2018 (actual)
- First posted
- 3 May 2016 (estimated)
- Results
- Posted 2 Jan 2019
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this study is to determine whether a new medication called vamorolone is safe and well-tolerated by boys with Duchenne muscular dystrophy (DMD) ages ≥ 4 and < 7 years old.
Detailed description
This study will evaluate the safety and tolerability of a new steroid-like medication called vamorolone in boys with DMD ages ≥ 4 years and < 7 years. Enrolled participants will take the study medication for 14 days followed by a 14 day follow-up period. The potential effectiveness of vamorolone in treating DMD will also be explored.
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/kg/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/kg/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/kg/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/kg/dayParticipants enrolled in Dose Level Group 4 will receive vamorolone 6.0 mg/kg/day. |
Who may qualify
- Ages
- 4 to 6 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (10 listed)
- Subject's parent or legal guardian has provided written informed consent/Health Insurance Portability and Accountability Act (HIPAA) authorization prior to any study-related procedures;
- Subject has a confirmed (by Central Genetic Counselor) diagnosis of DMD as defined as:
- Subject is ≥ 4 years and < 7 years of age at time of enrollment in the study;
- Subject is able to complete the Time to Stand Test (TTSTAND) without assistance, as assessed at the Screening and Baseline Visits;
- Clinical laboratory test results are within the normal range at the Screening Visit, or if abnormal, are not clinically significant, in the opinion of the Investigator. (Note: Serum gamma glutamyl transferase [GGT], creatinine, and total bilirubin all must be ≤ upper limit of the normal range at the Screening Visit);
Key exclusion criteria (12 listed)
- 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 had an acute illness within 4 weeks prior to the first dose of study medication;
- 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 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/kg/dayAlso: VBP15Oral administration of 0.25 mg/kg/day daily for 14 days. | Drug | Dose Level Group 1 |
| Vamorolone 0.75 mg/kg/dayAlso: VBP15Oral administration of 0.75 mg/kg/day daily for 14 days. | Drug | Dose Level Group 2 |
| Vamorolone 2.0 mg/kg/dayAlso: VBP15Oral administration of 2.0 mg/kg/day daily for 14 days. | Drug | Dose Level Group 3 |
| Vamorolone 6.0 mg/kg/dayAlso: VBP15Oral administration of 6 mg/kg/day daily for 14 days. | Drug | Dose Level Group 4 |
Primary outcome
Overall Summary of Adverse Events as Assessed by CTCAE Version 4.03
Time frame: Adverse events will be recorded from the date of informed consent and through the time of the subject's last study visit. Serious adverse events will be recorded from the date of informed consent and for up to 30 days after final drug administration.
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. Note: Total Number of Treatment Emergent Adverse Events: The total incidences of TEAEs experienced in study; Any Treatment Emergent Adverse Event: TEAEs reported at least once per dose group
15 secondary outcomes
Serum Pharmacodynamic Biomarkers (Insulin Resistance)- Fasting Glucose
Time frame: Baseline, Week 2
Serum Pharmacodynamic Biomarkers (Insulin Resistance) -Fasting Glucose
Time frame: Baseline, Week 2
Serum Pharmacodynamic Biomarkers (Insulin Resistance)- Insulin
Time frame: Baseline , Week 2
Serum Pharmacodynamic Biomarkers (Insulin Resistance)- Insulin
Time frame: Baseline, Week 2
Serum Pharmacodynamic Biomarkers (Adrenal Axis Suppression)- First in Morning Cortisol
Time frame: Week 2 (pre-dose)
Serum Pharmacodynamics Biomarkers (Bone Turnover) -Osteocalcin
Time frame: Baseline, Day 1, Week 2, Week 4
Serum Pharmacodynamic Biomarkers (Bone Turnover)- Procollagen 1 N-Terminal Propeptide
Time frame: Baseline Day 1 Week 2 Week 4
Serum Pharmacodynamic Biomarkers (Bone Turnover)- Procollagen 1 N-Terminal Propeptide
Time frame: Baseline, Day 1, Week 2, Week 4
Serum Pharmacodynamic Biomarkers (Bone Turnover)-Type I Collagen C-Telopeptides
Time frame: Baseline, Day 1, Week 4
Pharmacokinetic (PK) Assessments (Tmax)
Time frame: Day 1, Week 2
Pharmacokinetic (PK) Assessments (AUC Inf)
Time frame: Day 1, Week 2
Pharmacokinetic (PK) Assessments CL (ml/hr/kg)
Time frame: Day 1, Week 2
Pharmacokinetic (PK) Assessments t(1/2)
Time frame: Day 1, Week 2
Pharmacokinetic (PK) Assessments (Cmax)
Time frame: Day 1, Week 2
Metabolites in Safety Testing (MIST) Assessment
Time frame: Week 2 (Day 14)
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 2 Jan 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.
- Cooperative International Research GroupLink provided by the sponsor.
- ReveraGen BioPharma, IncLink provided by the sponsor.
- Phase IIa trial in Duchenne muscular dystrophy shows vamorolone is a first-in-class dissociative steroidal anti-inflammatory drug.Link provided by the sponsor.
- Individual participant dataSharing planned: Undecided
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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/kg/day, Vamorolone 0.75 mg/kg/day and Vamorolone 2.0 mg/kg/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 10 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 2 Jan 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 2 Jan 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?
Overall Summary of Adverse Events as Assessed by CTCAE Version 4.03 (time frame: Adverse events will be recorded from the date of informed consent and through the time of the subject's last study visit. Serious adverse events will be recorded from the date of informed consent and for up to 30 days after final drug administration.). 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 NCT02760264, 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 2 Jan 2019.