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| ID | Type | Description | Link |
|---|---|---|---|
| 1R44NS095423-01 | U.S. NIH Grant/Contract | View source | |
| 1U34AR068616-01 | U.S. NIH Grant/Contract | View source |
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| Name | Class |
|---|---|
| University of Pittsburgh | OTHER |
| National Institute of Neurological Disorders and Stroke (NINDS) | NIH |
| Cooperative International Neuromuscular Research Group | NETWORK |
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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.
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Dose Level Group 1 | Experimental | Participants enrolled in Dose Level Group 1 will receive 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. |
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| Dose Level Group 3 | Experimental | Participants enrolled in Dose Level Group 3 will receive vamorolone 2.0 mg/kg/day. |
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| Dose Level Group 4 | Experimental | Participants enrolled in Dose Level Group 4 will receive vamorolone 6.0 mg/kg/day. |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Vamorolone 0.25 mg/day/day | Drug | Oral administration of 0.25 mg/kg/day daily for 24 weeks. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants With Adverse Events as Assessed by CTCAE Version 4.03 | Treatment-emergent adverse events (TEAEs) are defined as any adverse event or worsening of an existing conditions after initiation of the investigational product and through the subject's last study visit (study completion or early termination). Serious adverse events were recorded for up to 30 days after the final administration of study drug; To evaluate the long-term safety and tolerability of vamorolone, administered orally at daily doses up to 6.0 mg/kg/day over a 24- week Treatment Period, in boys ages 4-7 years with DMD. | 24 weeks |
| Total Number of Adverse Events as Assessed by CTCAE Version 4.03 | Treatment-emergent adverse events (TEAEs) are defined as any adverse event or worsening of an existing conditions after initiation of the investigational product and through the subject's last study visit (study completion or early termination). Serious adverse events were recorded for up to 30 days after the final administration of study drug; To evaluate the long-term safety and tolerability of vamorolone, administered orally at daily doses up to 6.0 mg/kg/day over a 24- week Treatment Period, in boys ages 4-7 years with DMD. | 24 weeks |
| Muscle Function Measured by Time to Stand Test (TTSTAND)- Velocity | To compare the efficacy, as measured by the Time to Stand Test (TTSTAND), of vamorolone administered orally at daily doses up to 6.0 mg/kg over a 24-week Treatment Period vs. untreated DMD historical controls in boys ages 4-7 years with DMD | 002 Baseline, 003 Baseline, 003 Week 12, Week 24 (Note: 002 Baseline is from VBP15-002 4 week study (NCT02760264), previous to VBP15-003) |
| BMI Z-score | Summary of BMI Z-score of Safety Population. Please note 0 is the mean. A negative result indicates a response that is many standard deviations below the mean, and a positive result indicates a response that is many standard deviations above the mean. In this case, the closer the group mean BMI Z-score is to 0 is more favorable. |
| Measure | Description | Time Frame |
|---|---|---|
| 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. |
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Inclusion Criteria:
Exclusion Criteria:
Note: Participants may be re-evaluated if ineligible due to a transient condition which would prevent the subject from participating
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| Name | Affiliation | Role |
|---|---|---|
| Paula R Clemens, MD | University of Pittsburgh | Study Chair |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University of California Davis | Davis | California | 95616 | United States | ||
| University of Florida |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 38521535 | Derived | 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. | |
| 32956407 | Derived | 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. |
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| ID | Title | Description |
|---|---|---|
| FG000 | Dose Level Group 1 | Participants enrolled in Dose Level Group 1 will receive vamorolone 0.25 mg/kg/day. Vamorolone 0.25 mg/day/day: Oral administration of 0.25 mg/kg/day daily for 24 weeks. |
| FG001 | Dose Level Group 2 |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Vamorolone 0.25 mg/kg/Day |
|
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Feb 22, 2017 | Aug 14, 2018 |
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| National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS) |
| NIH |
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| Vamorolone 0.75 mg/day/day | Drug | Oral administration of 0.75 mg/kg/day daily for 24 weeks. |
|
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| Vamorolone 2.0 mg/day/day | Drug | Oral administration of 2.0 mg/kg/day daily for 24 weeks. |
|
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| Vamorolone 6.0 mg/day/day | Drug | Oral administration of 6.0 mg/kg/day daily for 24 weeks. |
|
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| 002 Baseline, 003 Week 12, Week 24 |
| 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. | 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. | 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. | 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. | 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. | 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. | 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. | 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. | 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. | 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. | 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) |
| 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 |
| 32434278 | Derived | 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. |
Participants enrolled in Dose Level Group 2 will receive vamorolone 0.75 mg/kg/day.
Vamorolone 0.75 mg/day/day: Oral administration of 0.75 mg/kg/day daily for 24 weeks.
| FG002 | Dose Level Group 3 | Participants enrolled in Dose Level Group 3 will receive vamorolone 2.0 mg/kg/day. Vamorolone 2.0 mg/day/day: Oral administration of 2.0 mg/kg/day daily for 24 weeks. |
| FG003 | Dose Level Group 4 | Participants enrolled in Dose Level Group 4 will receive vamorolone 6.0 mg/kg/day. Vamorolone 6.0 mg/day/day: Oral administration of 6.0 mg/kg/day daily for 24 weeks. |
| COMPLETED |
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| NOT COMPLETED |
|
| 0.75 mg/kg/Day |
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| 2.0 mg/kg/Day |
|
| 6.0 mg/kg/Day |
|
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.
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| ID | Title | Description |
|---|---|---|
| BG000 | Dose Level Group 1 | Participants enrolled in Dose Level Group 1 will receive vamorolone 0.25 mg/kg/day. Vamorolone 0.25 mg/day/day: Oral administration of 0.25 mg/kg/day daily for 24 weeks. |
| BG001 | Dose Level Group 2 | Participants enrolled in Dose Level Group 2 will receive vamorolone 0.75 mg/kg/day. Vamorolone 0.75 mg/day/day: Oral administration of 0.75 mg/kg/day daily for 24 weeks. |
| BG002 | Dose Level Group 3 | Participants enrolled in Dose Level Group 3 will receive vamorolone 2.0 mg/kg/day. Vamorolone 2.0 mg/day/day: Oral administration of 2.0 mg/kg/day daily for 24 weeks. |
| BG003 | Dose Level Group 4 | Participants enrolled in Dose Level Group 4 will receive vamorolone 6.0 mg/kg/day. Vamorolone 6.0 mg/day/day: Oral administration of 6.0 mg/kg/day daily for 24 weeks. |
| BG004 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Customized | Mean | Standard Deviation | years |
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| Sex/Gender, Customized | Count of Participants | Participants |
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| Ethnicity (NIH/OMB) | Count of Participants | Participants |
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| Race (NIH/OMB) | Count of Participants | Participants |
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| Region of Enrollment | Number | participants |
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| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||||||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | 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. | 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. | Posted | Number | participants | 24 weeks |
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| Primary | 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. | 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. | Posted | Number | Events | 24 weeks |
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| Primary | 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 | 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. | Posted | Mean | Standard Deviation | Rises/Second | 002 Baseline, 003 Baseline, 003 Week 12, Week 24 (Note: 002 Baseline is from VBP15-002 4 week study (NCT02760264), previous to VBP15-003) |
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| Primary | 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. | 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. | Posted | Mean | Standard Deviation | z score | 002 Baseline, 003 Week 12, Week 24 |
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| Secondary | 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. | 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. | Posted | Mean | Standard Deviation | % of HbA1c | 002 Baseline, 003 Week 8, 003 Week 16, 003 Week 24, 003 Week 26-29 |
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| Secondary | 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. | 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. | Posted | Mean | Standard Deviation | % change | 002 Baseline, 003 Week 8, 003 Week 16, 003 Week 24, 003 Week 26-29 |
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| Secondary | 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. | 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. | Posted | Mean | Standard Deviation | pg/mL | 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) |
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| Secondary | 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. | 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. | Posted | Mean | Standard Deviation | mg/dL | 002 Baseline, 003 Week 12, 003 Week 24 |
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| Secondary | 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. | 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. | Posted | Mean | Standard Deviation | uIU/mL | 002 Baseline, 003 Week 12, 003 Week 24 |
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| Secondary | 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. | 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. | Posted | Mean | Standard Deviation | ng/mL | 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) |
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| Secondary | 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. | 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. | Posted | Mean | Standard Deviation | ng/mL | 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) |
| ||||||||||||||||||||||||||||||||||||
| Secondary | 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. | 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. | Posted | Mean | Standard Deviation | pg/mL | 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) |
| ||||||||||||||||||||||||||||||||||||
| Secondary | 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. | 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. | Posted | Mean | Standard Deviation | tasks/ second | 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) |
| ||||||||||||||||||||||||||||||||||||
| Secondary | 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. | 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. | Posted | Mean | Standard Deviation | meters/ second | 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) |
| ||||||||||||||||||||||||||||||||||||
| Secondary | 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. | 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. | Posted | Mean | Standard Deviation | scores on a scale | 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) |
| ||||||||||||||||||||||||||||||||||||
| Secondary | 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. | 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. | Posted | Mean | Standard Deviation | Meters | 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) |
|
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.
Not provided
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Dose Level Group 1 | Participants enrolled in Dose Level Group 1 will receive vamorolone 0.25 mg/kg/day. Vamorolone 0.25 mg/day/day: Oral administration of 0.25 mg/kg/day daily for 24 weeks. | 0 | 12 | 0 | 12 | 10 | 12 |
| EG001 | Dose Level Group 2 | Participants enrolled in Dose Level Group 2 will receive vamorolone 0.75 mg/kg/day. Vamorolone 0.75 mg/day/day: Oral administration of 0.75 mg/kg/day daily for 24 weeks. | 0 | 12 | 1 | 12 | 10 | 12 |
| EG002 | Dose Level Group 3 | Participants enrolled in Dose Level Group 3 will receive vamorolone 2.0 mg/kg/day. Vamorolone 2.0 mg/day/day: Oral administration of 2.0 mg/kg/day daily for 24 weeks. | 0 | 12 | 0 | 12 | 11 | 12 |
| EG003 | Dose Level Group 4 | Participants enrolled in Dose Level Group 4 will receive vamorolone 6.0 mg/kg/day. Vamorolone 6.0 mg/day/day: Oral administration of 6.0 mg/kg/day daily for 24 weeks. | 0 | 12 | 2 | 12 | 11 | 12 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Pneumonia | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Dehydration | Metabolism and nutrition disorders | MedDRA | Systematic Assessment |
| |
| Testicular Torsion | Reproductive system and breast disorders | MedDRA | Systematic Assessment |
| |
| Hypoxia | Respiratory, thoracic and mediastinal disorders | MedDRA | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Left ventricular dysfunction | Cardiac disorders | MedDRA | Systematic Assessment |
| |
| Ear pain | Ear and labyrinth disorders | MedDRA | Systematic Assessment |
| |
| Cushingoid | Endocrine disorders | MedDRA | Systematic Assessment |
| |
| Excessive eye blinking | Eye disorders | MedDRA | Systematic Assessment |
| |
| Abdominal distension | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| Abdominal Pain | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| Abdominal pain upper | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| Faeces discoloured | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| Lip swelling | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| Gastrointestinal disorder | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| Gait disturbance | General disorders | MedDRA | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA | Systematic Assessment |
| |
| Thirst | General disorders | MedDRA | Systematic Assessment |
| |
| Seasonal allergy | Immune system disorders | MedDRA | Systematic Assessment |
| |
| Bronchitis | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Conjunctivitis | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Ear infection | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Enterobiasis | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Eye infection | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Gastroenteritis | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Gastroenteritis viral | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Gastrointestinal infection | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Hordeolum | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Influenza | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Otitis Media | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Pharyngitis | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Pharyngitis streptococcal | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Pneumonia | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Sinusitis | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Sinusitis bacterial | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Staphylococcal skin infection | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Tooth infection | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Viral infection | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Viral upper respiratory tract infection | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Arthropod Sting | Injury, poisoning and procedural complications | MedDRA | Systematic Assessment |
| |
| Foot fracture | Injury, poisoning and procedural complications | MedDRA | Systematic Assessment |
| |
| Human Bite | Injury, poisoning and procedural complications | MedDRA | Systematic Assessment |
| |
| Laceration | Injury, poisoning and procedural complications | MedDRA | Systematic Assessment |
| |
| Muscle strain | Injury, poisoning and procedural complications | MedDRA | Systematic Assessment |
| |
| Tendon injury | Injury, poisoning and procedural complications | MedDRA | Systematic Assessment |
| |
| Upper limb fracture | Injury, poisoning and procedural complications | MedDRA | Systematic Assessment |
| |
| Blood cortisol abnormal | Investigations | MedDRA | Systematic Assessment |
| |
| Urine output increased | Investigations | MedDRA | Systematic Assessment |
| |
| Weight increased | Investigations | MedDRA | Systematic Assessment |
| |
| Dehydration | Metabolism and nutrition disorders | MedDRA | Systematic Assessment |
| |
| Hyperlipidaemia | Metabolism and nutrition disorders | MedDRA | Systematic Assessment |
| |
| Hypoglycaemia | Metabolism and nutrition disorders | MedDRA | Systematic Assessment |
| |
| Vitamin D deficiency | Metabolism and nutrition disorders | MedDRA | Systematic Assessment |
| |
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA | Systematic Assessment |
| |
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA | Systematic Assessment |
| |
| Myalgia | Musculoskeletal and connective tissue disorders | MedDRA | Systematic Assessment |
| |
| Neck pain | Musculoskeletal and connective tissue disorders | MedDRA | Systematic Assessment |
| |
| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA | Systematic Assessment |
| |
| Pain in jaw | Musculoskeletal and connective tissue disorders | MedDRA | Systematic Assessment |
| |
| Tendon pain | Musculoskeletal and connective tissue disorders | MedDRA | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA | Systematic Assessment |
| |
| Presyncope | Nervous system disorders | MedDRA | Systematic Assessment |
| |
| Psychomotor hyperactivity | Nervous system disorders | MedDRA | Systematic Assessment |
| |
| Abnormal behavior | Psychiatric disorders | MedDRA | Systematic Assessment |
| |
| Anxiety | Psychiatric disorders | MedDRA | Systematic Assessment |
| |
| Emotional disorder | Psychiatric disorders | MedDRA | Systematic Assessment |
| |
| Insomnia | Psychiatric disorders | MedDRA | Systematic Assessment |
| |
| Personality change | Psychiatric disorders | MedDRA | Systematic Assessment |
| |
| Stereotypy | Psychiatric disorders | MedDRA | Systematic Assessment |
| |
| Tic | Psychiatric disorders | MedDRA | Systematic Assessment |
| |
| Testicular Torsion | Reproductive system and breast disorders | MedDRA | Systematic Assessment |
| |
| Asthma | Respiratory, thoracic and mediastinal disorders | MedDRA | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA | Systematic Assessment |
| |
| Epistaxis | Respiratory, thoracic and mediastinal disorders | MedDRA | Systematic Assessment |
| |
| Hypoxia | Respiratory, thoracic and mediastinal disorders | MedDRA | Systematic Assessment |
| |
| Nasal Congestion | Respiratory, thoracic and mediastinal disorders | MedDRA | Systematic Assessment |
| |
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA | Systematic Assessment |
| |
| Productive cough | Respiratory, thoracic and mediastinal disorders | MedDRA | Systematic Assessment |
| |
| Rhinorrhoea | Respiratory, thoracic and mediastinal disorders | MedDRA | Systematic Assessment |
| |
| Upper-airway cough syndrome | Respiratory, thoracic and mediastinal disorders | MedDRA | Systematic Assessment |
| |
| Dermatitis | Skin and subcutaneous tissue disorders | MedDRA | Systematic Assessment |
| |
| Dermatitis contact | Skin and subcutaneous tissue disorders | MedDRA | Systematic Assessment |
| |
| Eczema | Skin and subcutaneous tissue disorders | MedDRA | Systematic Assessment |
| |
| Hypertrichosis | Skin and subcutaneous tissue disorders | MedDRA | Systematic Assessment |
| |
| Rash | Skin and subcutaneous tissue disorders | MedDRA | Systematic Assessment |
| |
| Rash pruritic | Skin and subcutaneous tissue disorders | MedDRA | Systematic Assessment |
| |
| Swelling face | Skin and subcutaneous tissue disorders | MedDRA | Systematic Assessment |
| |
| Uriticaria | Skin and subcutaneous tissue disorders | MedDRA | Systematic Assessment |
|
Not provided
Not provided
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Dr. Eric P. Hoffman | ReveraGen Biopharma Inc. | 240-672-0295 | ericphoffman@gmail.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Jul 27, 2018 | Oct 3, 2018 | SAP_001.pdf |
| ID | Term |
|---|---|
| D020388 | Muscular Dystrophy, Duchenne |
| ID | Term |
|---|---|
| D009136 | Muscular Dystrophies |
| D020966 | Muscular Disorders, Atrophic |
| D009135 | Muscular Diseases |
| D009140 | Musculoskeletal Diseases |
| D009468 | Neuromuscular Diseases |
| D009422 | Nervous System Diseases |
| D040181 | Genetic Diseases, X-Linked |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
Not provided
Not provided
| ID | Term |
|---|---|
| C584811 | VBP15 compound |
Not provided
Not provided
Not provided
| Not Hispanic or Latino |
|
| Unknown or Not Reported |
|
| Asian |
|
| Native Hawaiian or Other Pacific Islander |
|
| Black or African American |
|
| White |
|
| More than one race |
|
| Unknown or Not Reported |
|
| Sweden |
|
| United States |
|
| United Kingdom |
|
| Israel |
|
| Australia |
|
| Subjects with Any Drug Related TEAE |
|
| Subjects with Any CTCAE Grade 3 or Higher TEAE |
|
| Discontinuation of Study Drug due to TEAE |
|
| Subjects with Any Serious TEAE |
|
| Death |
|
Participants enrolled in Dose Level Group 3 will receive vamorolone 2.0 mg/kg/day. Vamorolone 2.0 mg/day/day: Oral administration of 2.0 mg/kg/day daily for 24 weeks. |
| OG003 | Dose Level Group 4 | Participants enrolled in Dose Level Group 4 will receive vamorolone 6.0 mg/kg/day. Vamorolone 6.0 mg/day/day: Oral administration of 6.0 mg/kg/day daily for 24 weeks. |
|
|
| OG003 | Dose Level Group 4 | Participants enrolled in Dose Level Group 4 will receive vamorolone 6.0 mg/kg/day. Vamorolone 6.0 mg/day/day: Oral administration of 6.0 mg/kg/day daily for 24 weeks. |
|
|
| OG003 | Dose Level Group 4 | Participants enrolled in Dose Level Group 4 will receive vamorolone 6.0 mg/kg/day. Vamorolone 6.0 mg/day/day: Oral administration of 6.0 mg/kg/day daily for 24 weeks. |
|
|
| OG002 | Dose Level Group 3 | Participants enrolled in Dose Level Group 3 will receive vamorolone 2.0 mg/kg/day. Vamorolone 2.0 mg/day/day: Oral administration of 2.0 mg/kg/day daily for 24 weeks. |
| OG003 | Dose Level Group 4 | Participants enrolled in Dose Level Group 4 will receive vamorolone 6.0 mg/kg/day. Vamorolone 6.0 mg/day/day: Oral administration of 6.0 mg/kg/day daily for 24 weeks. |
|
|
| OG002 | Dose Level Group 3 | Participants enrolled in Dose Level Group 3 will receive vamorolone 2.0 mg/kg/day. Vamorolone 2.0 mg/day/day: Oral administration of 2.0 mg/kg/day daily for 24 weeks. |
| OG003 | Dose Level Group 4 | Participants enrolled in Dose Level Group 4 will receive vamorolone 6.0 mg/kg/day. Vamorolone 6.0 mg/day/day: Oral administration of 6.0 mg/kg/day daily for 24 weeks. |
|
|
| OG002 | Dose Level Group 3 | Participants enrolled in Dose Level Group 3 will receive vamorolone 2.0 mg/kg/day. Vamorolone 2.0 mg/day/day: Oral administration of 2.0 mg/kg/day daily for 24 weeks. |
| OG003 | Dose Level Group 4 | Participants enrolled in Dose Level Group 4 will receive vamorolone 6.0 mg/kg/day. Vamorolone 6.0 mg/day/day: Oral administration of 6.0 mg/kg/day daily for 24 weeks. |
|
|
| OG002 | Dose Level Group 3 | Participants enrolled in Dose Level Group 3 will receive vamorolone 2.0 mg/kg/day. Vamorolone 2.0 mg/day/day: Oral administration of 2.0 mg/kg/day daily for 24 weeks. |
| OG003 | Dose Level Group 4 | Participants enrolled in Dose Level Group 4 will receive vamorolone 6.0 mg/kg/day. Vamorolone 6.0 mg/day/day: Oral administration of 6.0 mg/kg/day daily for 24 weeks. |
|
|
| OG002 | Dose Level Group 3 | Participants enrolled in Dose Level Group 3 will receive vamorolone 2.0 mg/kg/day. Vamorolone 2.0 mg/day/day: Oral administration of 2.0 mg/kg/day daily for 24 weeks. |
| OG003 | Dose Level Group 4 | Participants enrolled in Dose Level Group 4 will receive vamorolone 6.0 mg/kg/day. Vamorolone 6.0 mg/day/day: Oral administration of 6.0 mg/kg/day daily for 24 weeks. |
|
|
| OG002 | Dose Level Group 3 | Participants enrolled in Dose Level Group 3 will receive vamorolone 2.0 mg/kg/day. Vamorolone 2.0 mg/day/day: Oral administration of 2.0 mg/kg/day daily for 24 weeks. |
| OG003 | Dose Level Group 4 | Participants enrolled in Dose Level Group 4 will receive vamorolone 6.0 mg/kg/day. Vamorolone 6.0 mg/day/day: Oral administration of 6.0 mg/kg/day daily for 24 weeks. |
|
|
| OG002 | Dose Level Group 3 | Participants enrolled in Dose Level Group 3 will receive vamorolone 2.0 mg/kg/day. Vamorolone 2.0 mg/day/day: Oral administration of 2.0 mg/kg/day daily for 24 weeks. |
| OG003 | Dose Level Group 4 | Participants enrolled in Dose Level Group 4 will receive vamorolone 6.0 mg/kg/day. Vamorolone 6.0 mg/day/day: Oral administration of 6.0 mg/kg/day daily for 24 weeks. |
|
|
| OG002 | Dose Level Group 3 | Participants enrolled in Dose Level Group 3 will receive vamorolone 2.0 mg/kg/day. Vamorolone 2.0 mg/day/day: Oral administration of 2.0 mg/kg/day daily for 24 weeks. |
| OG003 | Dose Level Group 4 | Participants enrolled in Dose Level Group 4 will receive vamorolone 6.0 mg/kg/day. Vamorolone 6.0 mg/day/day: Oral administration of 6.0 mg/kg/day daily for 24 weeks. |
|
|
Participants enrolled in Dose Level Group 3 will receive vamorolone 2.0 mg/kg/day. Vamorolone 2.0 mg/day/day: Oral administration of 2.0 mg/kg/day daily for 24 weeks. |
| OG003 | Dose Level Group 4 | Participants enrolled in Dose Level Group 4 will receive vamorolone 6.0 mg/kg/day. Vamorolone 6.0 mg/day/day: Oral administration of 6.0 mg/kg/day daily for 24 weeks. |
|
|
Participants enrolled in Dose Level Group 3 will receive vamorolone 2.0 mg/kg/day. Vamorolone 2.0 mg/day/day: Oral administration of 2.0 mg/kg/day daily for 24 weeks. |
| OG003 | Dose Level Group 4 | Participants enrolled in Dose Level Group 4 will receive vamorolone 6.0 mg/kg/day. Vamorolone 6.0 mg/day/day: Oral administration of 6.0 mg/kg/day daily for 24 weeks. |
|
|
| OG002 | Dose Level Group 3 | Participants enrolled in Dose Level Group 3 will receive vamorolone 2.0 mg/kg/day. Vamorolone 2.0 mg/day/day: Oral administration of 2.0 mg/kg/day daily for 24 weeks. |
| OG003 | Dose Level Group 4 | Participants enrolled in Dose Level Group 4 will receive vamorolone 6.0 mg/kg/day. Vamorolone 6.0 mg/day/day: Oral administration of 6.0 mg/kg/day daily for 24 weeks. |
|
|
Participants enrolled in Dose Level Group 3 will receive vamorolone 2.0 mg/kg/day. Vamorolone 2.0 mg/day/day: Oral administration of 2.0 mg/kg/day daily for 24 weeks. |
| OG003 | Dose Level Group 4 | Participants enrolled in Dose Level Group 4 will receive vamorolone 6.0 mg/kg/day. Vamorolone 6.0 mg/day/day: Oral administration of 6.0 mg/kg/day daily for 24 weeks. |
|
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