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CompletedNCT02760277Updated Jul 23, 2019Results posted

An Extension Study to Assess Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD)

A Phase 2 interventional study of Vamorolone 0.25 mg/day/day and Vamorolone 0.75 mg/day/day in Duchenne Muscular Dystrophy, sponsored by ReveraGen BioPharma, Inc.. Completed at 12 sites in 6 countries. Open to male participants aged 4 Years to 7 Years. Per ClinicalTrials.gov, last updated 2019-07-23.

Sponsored by ReveraGen BioPharma, Inc. · Phase 2, Interventional, and Treatment

Phase
Phase 2
Study type
Interventional
Enrollment
48
Allocation
Non-randomized
Ages
4 Years to 7 Years
Sex
Male
01

Study 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 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.

02

Conditions studied

  • Duchenne Muscular Dystrophy

Keywords

  • Duchenne muscular dystrophy
  • vamorolone
03

In context

Muscular Dystrophy, Duchenne

480 studies on the registry are indexed under Muscular Dystrophy, Duchenne; 109 are open to participants now.

This study's enrollment of 48 is above the median of 26 across 329 interventional studies indexed under Muscular Dystrophy, Duchenne.

Browse Muscular Dystrophy, Duchenne studies →

Lead sponsor

ReveraGen BioPharma, Inc. is the lead sponsor of 7 studies on the registry; none are open to participants now.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
4 Years to 7 Years
Sexes eligible
Male
Accepts healthy volunteers
No

Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.

Inclusion criteria

  1. Participant's parent or legal guardian has provided written informed consent/HIPAA authorization prior to any extension study-specific procedures;
  2. Participant has previously completed study VBP15-002 up to and including the Week 4 Follow-up assessments within 8 weeks prior to enrollment; and
  3. Participant and parent/guardian are willing and able to comply with scheduled visits, study drug administration plan, and study procedures.

Exclusion criteria

Exclusion Criteria:

  1. 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;
  2. Participant has current or history of major renal or hepatic impairment, diabetes mellitus or immunosuppression;
  3. Participant has current or history of chronic systemic fungal or viral infections;
  4. 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;
  5. Participant has evidence of symptomatic cardiomyopathy. [Note: Asymptomatic cardiac abnormality on investigation would not be exclusionary];
  6. 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];
  7. Subject has used idebenone within 4 weeks prior to the first dose of study medication;
  8. Participant has an allergy or hypersensitivity to the study medication or to any of its constituents;
  9. Participant has severe behavioral or cognitive problems that preclude participation in the study, in the opinion of the Investigator;
  10. 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
  11. 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

05

Study design

Phase
Phase 2
Primary purpose
Treatment
Allocation
Non-randomized
Intervention model
Sequential assignment
Masking
None (open label)
Enrollment
48 participants (actual)

Study arms

  • 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

  • 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

  • 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

  • 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

Interventions

  • DrugVamorolone 0.25 mg/day/day

    Drug page: Vamorolone

    Oral administration of 0.25 mg/kg/day daily for 24 weeks.

    Also known as: VBP15

  • DrugVamorolone 0.75 mg/day/day

    Drug page: Vamorolone

    Oral administration of 0.75 mg/kg/day daily for 24 weeks.

    Also known as: VBP15

  • DrugVamorolone 2.0 mg/day/day

    Drug page: Vamorolone

    Oral administration of 2.0 mg/kg/day daily for 24 weeks.

    Also known as: VBP15

  • DrugVamorolone 6.0 mg/day/day

    Drug page: Vamorolone

    Oral administration of 6.0 mg/kg/day daily for 24 weeks.

    Also known as: VBP15

06

What researchers measure

Primary outcomes

  1. 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.

    Time frame: 24 weeks

  2. 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.

    Time frame: 24 weeks

  3. 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

    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)

  4. 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.

    Time frame: 002 Baseline, 003 Week 12, Week 24

Secondary outcomes

  1. 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.

    Time frame: 002 Baseline, 003 Week 8, 003 Week 16, 003 Week 24, 003 Week 26-29

  2. 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.

    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)

  3. 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.

    Time frame: 002 Baseline, 003 Week 12, 003 Week 24

  4. 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.

    Time frame: 002 Baseline, 003 Week 12, 003 Week 24

  5. 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.

    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)

  6. 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.

    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)

  7. 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.

    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)

  8. 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.

    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)

  9. 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.

    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)

  10. 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.

    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)

  11. 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.

    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)

07

Results

Posted Jul 23, 2019

Participant flow

Vamorolone 0.25 mg/kg/Day
Participant flow — Vamorolone 0.25 mg/kg/Day
MilestoneDose Level Group 1Dose Level Group 2Dose Level Group 3Dose Level Group 4
Started12000
Completed12000
Not completed0000
0.75 mg/kg/Day
Participant flow — 0.75 mg/kg/Day
MilestoneDose Level Group 1Dose Level Group 2Dose Level Group 3Dose Level Group 4
Started01200
Completed01200
Not completed0000
2.0 mg/kg/Day
Participant flow — 2.0 mg/kg/Day
MilestoneDose Level Group 1Dose Level Group 2Dose Level Group 3Dose Level Group 4
Started00120
Completed00120
Not completed0000
6.0 mg/kg/Day
Participant flow — 6.0 mg/kg/Day
MilestoneDose Level Group 1Dose Level Group 2Dose Level Group 3Dose Level Group 4
Started00012
Completed00012
Not completed0000

Outcome measures

PrimaryNumber 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.

Time frame:
24 weeks
Reported as:
Number · participants
Number of Participants With Adverse Events as Assessed by CTCAE Version 4.03
participantsDose Level Group 1Dose Level Group 2Dose Level Group 3Dose Level Group 4
Subjects with Any TEAE10101111
Subjects with Any Drug Related TEAE1245
Subjects with Any CTCAE Grade 3 or Higher TEAE0002
Discontinuation of Study Drug due to TEAE0000
Subjects with Any Serious TEAE0102
Death0000
PrimaryTotal 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.

Time frame:
24 weeks
Reported as:
Number · Events
Total Number of Adverse Events as Assessed by CTCAE Version 4.03
EventsDose Level Group 1Dose Level Group 2Dose Level Group 3Dose Level Group 4
Total Number of AEs48445473
Total Number of TEAEs48445472
PrimaryMuscle 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

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)
Reported as:
Mean · Rises/Second
Muscle Function Measured by Time to Stand Test (TTSTAND)- Velocity
Rises/SecondDose Level Group 1Dose Level Group 2Dose Level Group 3Dose Level Group 4
002 Baseline0.18 ± 0.0650.24 ± 0.0900.22 ± 0.0820.19 ± 0.056
003 Baseline0.15 ± 0.0450.22 ± 0.0770.24 ± 0.0780.22 ± 0.070
003 Week 120.18 ± 0.0720.23 ± 0.1020.24 ± 0.0890.22 ± 0.075
003 Week 12 Change from 002 Baseline-0.01 ± 0.0610.00 ± 0.0540.02 ± 0.0660.02 ± 0.034
003 Week 240.18 ± 0.0810.24 ± 0.1140.26 ± 0.1080.24 ± 0.086
003 Week 24 Change from 002 Baseline-0.01 ± 0.0660.00 ± 0.0620.05 ± 0.0610.04 ± 0.045
PrimaryBMI 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.

Time frame:
002 Baseline, 003 Week 12, Week 24
Reported as:
Mean · z score
BMI Z-score
z scoreDose Level Group 1Dose Level Group 2Dose Level Group 3Dose Level Group 4
002 Baseline1.165 ± 0.62190.703 ± 1.07381.200 ± 0.53250.695 ± 0.7189
003 Week 121.103 ± 0.64570.494 ± 1.06801.261 ± 0.39811.011 ± 0.7034
003 Week 12 Change from 002 Baseline-0.062 ± 0.2438-0.209 ± 0.40780.062 ± 0.38860.174 ± 0.5826
003 Week 241.004 ± 0.63810.493 ± 1.16961.242 ± 0.45961.330 ± 0.5857
003 Week 24 Change from 002 Baseline-0.161 ± 0.3234-0.210 ± 0.36290.043 ± 0.38490.493 ± 0.6363
SecondarySerum 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.

Time frame:
002 Baseline, 003 Week 8, 003 Week 16, 003 Week 24, 003 Week 26-29
Reported as:
Mean · % of HbA1c
Serum Pharmacodynamics Biomarkers Measured by Levels of HbA1c
% of HbA1cDose Level Group 1Dose Level Group 2Dose Level Group 3Dose Level Group 4
002 Baseline5.18 ± 0.2605.22 ± 0.2445.19 ± 0.1245.23 ± 0.231
003 Week 85.18 ± 0.2265.33 ± 0.2505.28 ± 0.2045.25 ± 0.216
003 Week 8 Change from 002 Baseline0.00 ± 0.1280.12 ± 0.1530.09 ± 0.1980.00 ± 0.100
003 Week 165.26 ± 0.2395.35 ± 0.2385.26 ± 0.1565.31 ± 0.270
003 Week 16 Change from 002 Baseline0.08 ± 0.2040.14 ± 0.2250.07 ± 0.1300.05 ± 0.113
003 Week 245.15 ± 0.3025.22 ± 0.2215.13 ± 0.1605.24 ± 0.254
003 Week 24 Change from 002 Baseline-0.10 ± 0.2200.00 ± 0.186-0.07 ± 0.130-0.02 ± 0.154
003 Week 26-295.07 ± 0.208———
003 Week 26-29 Change from 002 Baseline-0.07 ± 0.208———
SecondarySerum 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.

Time frame:
002 Baseline, 003 Week 8, 003 Week 16, 003 Week 24, 003 Week 26-29
Reported as:
Mean · % change
Serum Pharmacodynamics Biomarkers Measured by Levels of HbA1c
% changeDose Level Group 1Dose Level Group 2Dose Level Group 3Dose Level Group 4
003 Week 8 % Change from 002 Baseline0.06 ± 2.4702.28 ± 3.0081.79 ± 3.8640.02 ± 1.927
003 Week 16 % Change from 002 Baseline1.70 ± 3.9892.72 ± 4.4571.30 ± 2.5051.03 ± 2.14
003 Week 24 % Change from 002 Baseline-1.89 ± 4.8010.08 ± 3.629-1.27 ± 2.525-0.33 ± 2.895
003 Week 26-29 % Change from 002 Baseline-1.25 ± 3.942———
SecondarySerum 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.

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)
Reported as:
Mean · pg/mL
Serum Pharmacodynamics Biomarkers Measured by Levels of ACTH
pg/mLDose Level Group 1Dose Level Group 2Dose Level Group 3Dose Level Group 4
002 Baseline18.3 ± 2.9618.0 ± 6.8821.1 ± 6.1319.3 ± 8.67
003 Baseline15.9 ± 4.5218.6 ± 4.5618.2 ± 5.2918.4 ± 9.73
003 Week 813.0 ± 6.257.1 ± 5.847.8 ± 4.426.5 ± 5.23
003 Week 8 Change from 002 Baseline-5.3 ± 6.92-10.5 ± 9.32-13.3 ± 7.33-13.5 ± 7.84
003 Week 1612.2 ± 4.939.0 ± 5.869.0 ± 14.189.0 ± 4.49
003 Week 16 Change from 002 Baseline-6.2 ± 6.34-9.1 ± 9.89-12.0 ± 15.42-12.2 ± 8.16
003 Week 2419.8 ± 6.3214.0 ± 3.8815.7 ± 9.6311.3 ± 7.52
003 Week 24 Change from 002 Baseline0.6 ± 6.63-4.0 ± 5.48-5.4 ± 11.09-6.3 ± 7.78
003 Week 26-299.5 ± 7.26———
003 Week 26-29 Change from 002 Baseline-7.8 ± 4.19———
SecondarySerum 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.

Time frame:
002 Baseline, 003 Week 12, 003 Week 24
Reported as:
Mean · mg/dL
Serum Pharmacodynamics Biomarkers Measured by Levels of Fasting Glucose
mg/dLDose Level Group 1Dose Level Group 2Dose Level Group 3Dose Level Group 4
002 Baseline87.5 ± 9.4488.9 ± 18.7189.3 ± 7.9192.3 ± 8.19
003 Week 1281.5 ± 5.6181.7 ± 4.3584.3 ± 8.1386.5 ± 5.57
003 Week 12 Change from 002 Baseline-6.8 ± 8.29-7.6 ± 19.22-5.1 ± 9.01-5.2 ± 9.21
003 Week 2480.8 ± 6.5680.8 ± 4.0881.3 ± 7.9484.6 ± 6.53
003 Week 24 Change from 002 Baseline-6.3 ± 11.97-9.0 ± 20.87-8.1 ± 10.28-7.8 ± 9.44
SecondarySerum 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.

Time frame:
002 Baseline, 003 Week 12, 003 Week 24
Reported as:
Mean · uIU/mL
Serum Pharmacodynamics Biomarkers Measured by Levels of Fasting Insulin
uIU/mLDose Level Group 1Dose Level Group 2Dose Level Group 3Dose Level Group 4
002 Baseline5.54 ± 3.6513.09 ± 2.0333.40 ± 1.5483.96 ± 2.027
003 Week 124.17 ± 3.1672.97 ± 1.6693.89 ± 2.1896.97 ± 3.526
003 Week 12 Change from 002 Baseline-1.13 ± 3.822-0.14 ± 1.7560.49 ± 2.5922.97 ± 2.277
003 Week 244.23 ± 2.5603.12 ± 1.7884.82 ± 3.3937.21 ± 2.374
003 Week 24 Change from 002 Baseline-1.67 ± 4.4780.34 ± 2.8981.36 ± 3.2623.26 ± 2.862
SecondarySerum 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.

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)
Reported as:
Mean · ng/mL
Serum Pharmacodynamics Biomarkers Measured by Levels of Osteocalcin
ng/mLDose Level Group 1Dose Level Group 2Dose Level Group 3Dose Level Group 4
002 Baseline37.94 ± 11.62235.66 ± 6.80041.17 ± 5.61744.36 ± 5.979
003 Baseline39.20 ± 14.13641.84 ± 8.55247.91 ± 6.64842.81 ± 10.851
003 Week 836.21 ± 10.37441.78 ± 13.85644.45 ± 7.43941.55 ± 5.446
003 Week 8 Change from 002 Baseline-1.60 ± 8.8496.13 ± 10.4403.28 ± 8.325-2.01 ± 8.898
003 Week 1639.01 ± 9.62042.23 ± 9.39344.60 ± 9.53439.39 ± 6.972
003 Week 16 Change from 002 Baseline1.07 ± 9.9166.57 ± 6.7093.43 ± 10.718-4.17 ± 8.494
003 Week 2438.80 ± 6.29251.41 ± 11.26551.98 ± 9.37249.08 ± 7.771
003 Week 24 Change from 002 Baseline-1.34 ± 11.28915.75 ± 10.21110.81 ± 7.5425.29 ± 7.858
003 Week 26-2940.10 ± 18.729———
003 Week 26-29 Change from 002 Baseline-1.23 ± 17.943———
SecondarySerum 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.

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)
Reported as:
Mean · ng/mL
Serum Pharmacodynamics Biomarkers Measured by Levels of P1NP
ng/mLDose Level Group 1Dose Level Group 2Dose Level Group 3Dose Level Group 4
002 Baseline555.8 ± 184.72480.7 ± 118.20508.2 ± 94.36511.5 ± 106.50
003 Baseline573.9 ± 251.02489.3 ± 121.66492.0 ± 81.92566.3 ± 149.32
003 Week 8511.6 ± 190.94459.8 ± 101.93485.2 ± 105.12402.7 ± 70.46
003 Week 8 Change from 002 Baseline-20.3 ± 120.32-22.9 ± 128.79-23.0 ± 96.84-105.6 ± 121.07
003 Week 16481.9 ± 159.93431.8 ± 81.25455.7 ± 99.50488.5 ± 130.11
003 Week 16 Change from 002 Baseline-73.8 ± 109.31-42.4 ± 109.07-52.5 ± 104.05-19.8 ± 130.12
003 Week 24457.1 ± 129.21471.1 ± 121.10565.5 ± 158.89526.2 ± 130.18
003 Week 24 Change from 002 Baseline-30.8 ± 113.642.1 ± 165.1657.3 ± 150.368.7 ± 88.95
003 Week 26-29619.0 ± 379.07———
003 Week 26-29 Change from 002 Baseline-152.0 ± 331.46———
SecondarySerum 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.

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)
Reported as:
Mean · pg/mL
Serum Pharmacodynamics Biomarkers Measured by Levels of CTX
pg/mLDose Level Group 1Dose Level Group 2Dose Level Group 3Dose Level Group 4
002 Baseline871.0 ± 160.85935.8 ± 286.50936.8 ± 256.25889.3 ± 186.68
003 Baseline915.9 ± 263.13964.4 ± 319.26949.8 ± 303.76989.2 ± 216.29
003 Week 8897.1 ± 365.45933.3 ± 330.20928.3 ± 333.11825.5 ± 164.36
003 Week 8 Change from 002 Baseline26.1 ± 368.76-31.6 ± 236.34-8.5 ± 248.82-59.6 ± 259.08
003 Week 16885.4 ± 261.53912.8 ± 305.08939.8 ± 157.17953.7 ± 199.53
003 Week 16 Change from 002 Baseline-2.6 ± 304.50-46.3 ± 321.063.0 ± 244.71102.3 ± 230.44
003 Week 241109.3 ± 287.921235.6 ± 295.791248.7 ± 308.901237.0 ± 277.20
003 Week 24 Change from 002 Baseline212.3 ± 318.86295.6 ± 357.93346.5 ± 327.16321.4 ± 264.65
003 Week 26-291059.3 ± 536.26———
003 Week 26-29 Change from 002 Baseline569.5 ± 28.99———
SecondaryMuscle 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.

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)
Reported as:
Mean · tasks/ second
Muscle Strength, Mobility, and Functional Exercise Capacity as Measured by Time to Climb Test (TTCLIMB)- Velocity
tasks/ secondDose Level Group 1Dose Level Group 2Dose Level Group 3Dose Level Group 4
002 Baseline0.20 ± 0.0540.29 ± 0.1470.29 ± 0.1640.24 ± 0.086
003 Baseline0.20 ± 0.0650.29 ± 0.1680.31 ± 0.1440.25 ± 0.082
003 Week 120.21 ± 0.0640.34 ± 0.2380.31 ± 0.1570.26 ± 0.095
003 Week 12 Change from 002 Baseline0.01 ± 0.0440.05 ± 0.1150.02 ± 0.1070.02 ± 0.051
003 Week 240.20 ± 0.0710.30 ± 0.1660.34 ± 0.1480.29 ± 0.097
003 Week 24 Change from 002 Baseline0.00 ± 0.0760.01 ± 0.0660.04 ± 0.0900.05 ± 0.061
SecondaryMuscle 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.

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)
Reported as:
Mean · meters/ second
Muscle Strength, Mobility, and Functional Exercise Capacity as Measured by Time to Run/Walk 10 Meters Test (TTRW)- Velocity
meters/ secondDose Level Group 1Dose Level Group 2Dose Level Group 3Dose Level Group 4
002 Baseline1.60 ± 0.3121.77 ± 0.3671.84 ± 0.3471.64 ± 0.279
003 Baseline1.57 ± 0.3711.78 ± 0.4141.86 ± 0.4181.72 ± 0.295
003 Week 121.54 ± 0.3061.77 ± 0.5501.97 ± 0.5031.88 ± 0.341
003 Week 12 Change from 002 Baseline-0.06 ± 0.2610.00 ± 0.3070.13 ± 0.3160.26 ± 0.297
003 Week 241.55 ± 0.3841.84 ± 0.4861.90 ± 0.3211.89 ± 0.378
003 Week 24 Change from 002 Baseline-0.05 ± 0.3110.06 ± 0.2100.06 ± 0.2100.27 ± 0.254
SecondaryMuscle 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.

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)
Reported as:
Mean · scores on a scale
Muscle Strength, Mobility, and Functional Exercise Capacity as Measured by North Star Ambulatory Assessment (NSAA)
scores on a scaleDose Level Group 1Dose Level Group 2Dose Level Group 3Dose Level Group 4
002 Baseline19.0 ± 5.1320.5 ± 5.5820.0 ± 4.9519.7 ± 4.94
003 Baseline20.1 ± 7.3020.8 ± 5.6621.7 ± 3.8720.4 ± 4.01
003 Week 1219.3 ± 5.6021.2 ± 6.4521.0 ± 5.1320.4 ± 5.41
003 Week 12 Change from 002 Baseline0.3 ± 2.060.7 ± 2.711.0 ± 2.560.5 ± 2.38
003 Week 2419.8 ± 7.0921.6 ± 7.2322.3 ± 3.8022.3 ± 5.76
003 Week 24 Change from 002 Baseline0.8 ± 2.831.1 ± 2.942.3 ± 1.782.5 ± 2.62
SecondaryMuscle 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.

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)
Reported as:
Mean · Meters
Muscle Strength, Mobility, and Functional Exercise Capacity vs. Historical Controls as Measured by 6-minute Walk Test (6MWT) Meters
MetersDose Level Group 1Dose Level Group 2Dose Level Group 3Dose Level Group 4
002 Baseline316.2 ± 59.47331.5 ± 52.76353.9 ± 65.40336.8 ± 63.18
003 Baseline294.3 ± 60.62332.2 ± 56.83341.1 ± 49.42335.1 ± 80.13
003 Week 12312.9 ± 60.93358.7 ± 71.47393.7 ± 59.72369.9 ± 69.47
003 Week 12 Change from 002 Baseline6.0 ± 28.8120.8 ± 38.0939.8 ± 35.6127.6 ± 42.00
003 Week 24306.2 ± 68.08350.4 ± 64.23383.1 ± 63.38372.6 ± 69.12
003 Week 24 Change from 002 Baseline-11.6 ± 29.4518.9 ± 41.0829.2 ± 35.9143.9 ± 43.72

Adverse events

Collected over Adverse events, including Serious Adverse Events (SAEs), and concomitant medications will be assessed at each study visit and recorded throughout the 24 week treatment period.. Non-serious events are listed at a 5% frequency threshold.

Adverse event summary by group
GroupDeathsSeriousOther
Dose Level Group 10/12 (0%)0/12 (0%)10/12 (83.3%)
Dose Level Group 20/12 (0%)1/12 (8.3%)10/12 (83.3%)
Dose Level Group 30/12 (0%)0/12 (0%)11/12 (91.7%)
Dose Level Group 40/12 (0%)2/12 (16.7%)11/12 (91.7%)
Most frequent serious events
Most frequent serious events
EventDose Level Group 1Dose Level Group 2Dose Level Group 3Dose Level Group 4
PneumoniaInfections and infestations0/121/120/120/12
DehydrationMetabolism and nutrition disorders0/120/120/121/12
Testicular TorsionReproductive system and breast disorders0/120/120/121/12
HypoxiaRespiratory, thoracic and mediastinal disorders0/120/120/121/12
Most frequent other events
Showing 10 of 88
Most frequent other events
EventDose Level Group 1Dose Level Group 2Dose Level Group 3Dose Level Group 4
PyrexiaGeneral disorders2/127/125/123/12
Viral upper respiratory tract infectionInfections and infestations4/124/127/125/12
CoughRespiratory, thoracic and mediastinal disorders1/124/121/123/12
VomitingGastrointestinal disorders1/121/123/122/12
InfluenzaInfections and infestations1/120/120/123/12
Abdominal pain upperGastrointestinal disorders0/120/121/122/12
ConstipationGastrointestinal disorders1/120/121/122/12
DiarrhoeaGastrointestinal disorders2/120/122/121/12
ConjunctivitisInfections and infestations1/120/122/121/12
Eye infectionInfections and infestations2/120/120/120/12

Baseline characteristics

All subjects who receive at least one dose of vamorolone study medication in the extension study will be included in the Safety Population. The Safety Population is the primary analysis population for safety assessments.

Age, Customized
Age, Customized(years)Dose Level Group 1Dose Level Group 2Dose Level Group 3Dose Level Group 4Total
Age5.2 ± 1.034.8 ± 0.834.7 ± 0.894.8 ± 0.754.9 ± 0.87
Sex/Gender, Customized
Sex/Gender, Customized(Participants)Dose Level Group 1Dose Level Group 2Dose Level Group 3Dose Level Group 4Total
Male1212121248
Ethnicity (NIH/OMB)
Ethnicity (NIH/OMB)(Participants)Dose Level Group 1Dose Level Group 2Dose Level Group 3Dose Level Group 4Total
Hispanic or Latino01034
Not Hispanic or Latino121112944
Unknown or Not Reported00000
Race (NIH/OMB)
Race (NIH/OMB)(Participants)Dose Level Group 1Dose Level Group 2Dose Level Group 3Dose Level Group 4Total
American Indian or Alaska Native00000
Asian01001
Native Hawaiian or Other Pacific Islander00000
Black or African American10001
White1110121245
More than one race00000
Unknown or Not Reported01001
Region of Enrollment
Region of Enrollment(participants)Dose Level Group 1Dose Level Group 2Dose Level Group 3Dose Level Group 4Total
Canada30227
Sweden04004
United States932620
United Kingdom00426
Israel03205
Australia02226
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Study locations

12 sites
  • 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
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References and documents

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 42613652 ↗
  • 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. PubMed 38521535 ↗
  • 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. PubMed 32956407 ↗
  • Li 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 documents

  • Study protocol · Feb 22, 2017
  • Statistical analysis plan · Jul 27, 2018

Documents are hosted by the registry — open the source record to download them.

10

Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on Jul 23, 2019, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
11

Registry details

Key details

Study ID
NCT02760277
Lead sponsor
ReveraGen BioPharma, Inc.
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
First posted
May 3, 2016
Start date
Jul 28, 2016
Primary completion
Apr 26, 2018
Completion
Apr 26, 2018
Results posted
Jul 23, 2019
Last update
Jul 23, 2019

Study contacts

Paula R Clemens, MD
study chair · University of Pittsburgh

Oversight

Data monitoring committee
Yes
View the source record on ClinicalTrials.gov ↗

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