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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/show/NCT03439670




Registration number
NCT03439670
Ethics application status
Date submitted
9/01/2018
Date registered
20/02/2018
Date last updated
13/08/2019

Titles & IDs
Public title
A Study to Assess the Efficacy and Safety of Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD)
Scientific title
A Phase IIb Randomized, Double-blind, Parallel Group, Placebo- and Active-controlled Study With Double-Blind Extension to Assess the Efficacy and Safety of Vamorolone in Ambulant Boys With Duchenne Muscular Dystrophy (DMD)
Secondary ID [1] 0 0
VBP15-004
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Duchenne Muscular Dystrophy 0 0
Condition category
Condition code
Musculoskeletal 0 0 0 0
Other muscular and skeletal disorders
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Neurological 0 0 0 0
Other neurological disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Vamorolone
Treatment: Drugs - Prednisone
Other interventions - Placebo
Treatment: Drugs - Vamorolone
Treatment: Drugs - Prednisone
Other interventions - Placebo
Treatment: Drugs - Vamorolone
Treatment: Drugs - Vamorolone

Experimental: Treatment Group 1 - Patients enrolled in Treatment Group 1 (experimental group) will receive vamorolone 2.0 mg/kg/day for the duration of the study.

Experimental: Treatment Group 2 - Patients enrolled in Treatment Group 2 (experimental group) will receive vamorolone at 6.0 mg/kg/day for the duration of the study.

Active Comparator: Treatment Group 3 - Patients enrolled in Treatment Group 3 (active comparator group) will receive prednisone 0.75 mg/kg/day for 24 weeks followed by 20 weeks of treatment with 2.0 mg/kg/day vamorolone.

Active Comparator: Treatment Group 4 - Patients enrolled in Treatment Group 4 (active comparator group) will receive prednisone 0.75 mg/kg/day for 24 weeks followed by 20 weeks treatment with 6.0 mg/kg/day vamorolone.

Placebo Comparator: Treatment Group 5 - Patients enrolled in Treatment Group 5 (placebo comparator group) will receive placebo daily for 24 weeks followed by 20 weeks treatment with 2.0 mg/kg/day vamorolone.

Placebo Comparator: Treatment Group 6 - Patients enrolled in Treatment Group 6 (placebo comparator group) will receive placebo daily for 24 weeks followed by 20 weeks treatment with 6.0 mg/kg/day vamorolone.


Treatment: Drugs: Vamorolone
Oral administration of 2.0 mg/kg/day for the duration of the study.

Treatment: Drugs: Prednisone
Oral administration of 0.75 mg/kg/day for 24 weeks.

Other interventions: Placebo
Oral administration of placebo daily for 24 weeks.

Treatment: Drugs: Vamorolone
Oral administration of 6.0 mg/kg/day for the duration of the study.

Treatment: Drugs: Prednisone
Oral administration of 0.75 mg/kg/day for 24 weeks.

Other interventions: Placebo
Oral administration of placebo daily for 24 weeks.

Treatment: Drugs: Vamorolone
Oral administration of 2.0 mg/kg/day for 20 weeks.

Treatment: Drugs: Vamorolone
Oral administration of 6.0 mg/kg/day for 20 weeks.

Intervention code [1] 0 0
Treatment: Drugs
Intervention code [2] 0 0
Other interventions
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Muscle function measured by Time to Stand Test (TTSTAND) - (vamorolone at 2.0mg/kg/day and 6.0mg/kg/day vs. placebo)
Timepoint [1] 0 0
24 weeks
Primary outcome [2] 0 0
Body Size as measured by body mass index (BMI) z-score - (vamorolone at 2.0mg/kg/day and 6.0mg/kg/day vs. prednisone at 0.75 mg/kg/day)
Timepoint [2] 0 0
24 weeks
Secondary outcome [1] 0 0
Safety measure by Treatment emergent adverse events (TRAEs) and serious adverse events (SAEs) by organ system class (SOC). - Overall by treatment, by treatment and relationship, and by treatment and intensity.
Timepoint [1] 0 0
48 weeks
Secondary outcome [2] 0 0
Safety measure assessed by sitting blood pressure. - Change from baseline to each of the scheduled on-treatment and post-treatment assessment time points.
Timepoint [2] 0 0
Day 1, Week 2, Week 6, Week 12,Week 18, Week 24, Week 28, Week 30, Week 34, Week 40, Week 48.
Secondary outcome [3] 0 0
Safety measure assessed by heart rate. - Change from baseline to each of the scheduled on-treatment and post-treatment assessment time points.
Timepoint [3] 0 0
Day 1, Week 2, Week 6, Week 12,Week 18, Week 24, Week 28, Week 30, Week 34, Week 40, Week 48.
Secondary outcome [4] 0 0
Safety measured assessed by respiratory rate. - Change from baseline to each of the scheduled on-treatment and post-treatment assessment time points.
Timepoint [4] 0 0
Day 1, Week 2, Week 6, Week 12,Week 18, Week 24, Week 28, Week 30, Week 34, Week 40, Week 48.
Secondary outcome [5] 0 0
Safety measure assesed by body temperature. - Change from baseline to each of the scheduled on-treatment and post-treatment assessment time points.
Timepoint [5] 0 0
Day 1, Week 2, Week 6, Week 12,Week 18, Week 24, Week 28, Week 30, Week 34, Week 40, Week 48.
Secondary outcome [6] 0 0
Safety measure assessed by body weight. - Change from baseline to each of the scheduled on-treatment and post-treatment assessment time points.
Timepoint [6] 0 0
Week 2, Week 6, Week 12, Week 18, Week 24, Week 28, Week 30, Week 34, Week 40, Week 48.
Secondary outcome [7] 0 0
Safety measure assessed by height - Change from baseline to each of the scheduled on-treatment and post-treatment assessment time points.
Timepoint [7] 0 0
Week 12, Week 24, Week 34, Week 48.
Secondary outcome [8] 0 0
Cushingoid features measured by the presence of buffalo hump obesity, striations, adiposity, hypertension, diabetes, or osteoporosis - Change from baseline to each of the scheduled on-treatment and post-treatment assessment time points.
Timepoint [8] 0 0
Week 6, Week 12, Week 18, Week 24, Week 28, Week 34, Week 40, Week 48.
Secondary outcome [9] 0 0
Safety measure assessed by blood laboratory measures. - Change from baseline to each of the scheduled on-treatment and post-treatment assessment time points.
Timepoint [9] 0 0
Day 1, Week 2, Week 6, Week 12, Week 18, Week 24, Week 28, Week 30, Week 34, Week 40, Week 48.
Secondary outcome [10] 0 0
Safety measure assessed by urine laboratory measures. - Change from baseline to each of the scheduled on-treatment and post-treatment assessment time points.
Timepoint [10] 0 0
Day 1, Week 2, Week 6, Week 12, Week 18, Week 24, Week 28, Week 30, Week 34, Week 40, Week 48
Secondary outcome [11] 0 0
Cardiac function measured by 12 lead electrocardiogram (ECG) - Change from baseline to each of the scheduled on-treatment and post-treatment assessment time points.
Timepoint [11] 0 0
Week 12, Week 24, Week 40, Week 48.
Secondary outcome [12] 0 0
Cardiac function measured by 2-D echocardiogram. - Change from baseline to each of the scheduled on-treatment and post-treatment assessment time points.
Timepoint [12] 0 0
Week 24, Week 48.
Secondary outcome [13] 0 0
Safety measure based on dual-energy x-ray absorptiometry (DXA) scan. - Change from baseline to Week 24 and Week 48 in BMD Z-score.
Timepoint [13] 0 0
Week 24, Week 48.
Secondary outcome [14] 0 0
Spine Fracture measured by spine X-ray - Change from baseline to Week 24 assessment.
Timepoint [14] 0 0
24 weeks
Secondary outcome [15] 0 0
Cataracts measured by the presence of partial or complete opacity of the crystalline lens of one or both eyes. - Week 24 and Week 48 assessments compared to baseline
Timepoint [15] 0 0
Week 24, Week 48
Secondary outcome [16] 0 0
Glaucoma measured by measured by ocular pressure. - Week 24 and Week 48 assessments compared to baseline
Timepoint [16] 0 0
Week 24, Week 48
Secondary outcome [17] 0 0
Safety measured assessed by Synacthen (ACTH) test
Timepoint [17] 0 0
24, 48 weeks
Secondary outcome [18] 0 0
Efficacy measured by Time to Stand Test (TTSTAND) - (Change from baseline to each of the scheduled study assessment time points for each treatment group up to Week 48)
Timepoint [18] 0 0
48 weeks
Secondary outcome [19] 0 0
Efficacy measured by Time to Climb (TTCLIMB). - (vamorolone at 2.0mg/kg/day and vamorolone at 6.0 mg/kg/day vs. placebo)- 24 weeks ; (Change from baseline to each of the scheduled study assessment time points for each treatment group up to Week 48)
Timepoint [19] 0 0
24, 48 weeks
Secondary outcome [20] 0 0
Efficacy as measured by Time to Run/Walk Test (TTRW). - (vamorolone at 2.0mg/kg/day and vamorolone at 6.0 mg/kg/day vs. placebo)- 24 weeks ; (Change from baseline to each of the scheduled study assessment time points for each treatment group up to Week 48)
Timepoint [20] 0 0
24, 48 weeks
Secondary outcome [21] 0 0
Efficacy as measured by total distance traveled in meters, in completing the Six-minute Walk Test (6MWT) - (vamorolone at 2.0mg/kg/day and vamorolone at 6.0 mg/kg/day vs. placebo)- 24 weeks ; (Change from baseline to each of the scheduled study assessment time points for each treatment group up to Week 48)
Timepoint [21] 0 0
24, 48 weeks
Secondary outcome [22] 0 0
Efficacy as measured by the North Star Ambulatory Assessment (NSAA) - (vamorolone at 2.0mg/kg/day and vamorolone at 6.0 mg/kg/day vs. placebo)- 24 weeks ; (Change from baseline to each of the scheduled study assessment time points for each treatment group up to Week 48)
Timepoint [22] 0 0
24, 48 weeks
Secondary outcome [23] 0 0
Efficacy as measured by hand-held myometry (elbow flexors and knee extensors) - (vamorolone at 2.0mg/kg/day and vamorolone at 6.0 mg/kg/day vs. placebo)- 24 weeks ; (Change from baseline to each of the scheduled study assessment time points for each treatment group up to Week 48)
Timepoint [23] 0 0
24, 48 weeks
Secondary outcome [24] 0 0
Efficacy as measured by range of motion in the ankles (ROM) - (vamorolone at 2.0mg/kg/day and vamorolone at 6.0 mg/kg/day vs. placebo)- 24 weeks ; (Change from baseline to each of the scheduled study assessment time points for each treatment group up to Week 48)
Timepoint [24] 0 0
24, 48 weeks
Secondary outcome [25] 0 0
Safety as measured by serum pharmacodynamic biomarkers by morning cortisol.
Timepoint [25] 0 0
48 weeks
Secondary outcome [26] 0 0
Safety as measured by serum pharmacodynamic biomarkers by levels of fasting glucose.
Timepoint [26] 0 0
48 weeks
Secondary outcome [27] 0 0
Safety as measured by serum pharmacodynamic biomarkers by levels of fasting insulin.
Timepoint [27] 0 0
48 weeks
Secondary outcome [28] 0 0
Safety as measured by serum pharmacodynamic biomarkers by levels of osteocalcin.
Timepoint [28] 0 0
48 weeks
Secondary outcome [29] 0 0
Safety as measured by serum pharmacodynamic biomarkers by levels of CTX1.
Timepoint [29] 0 0
48 weeks
Secondary outcome [30] 0 0
Safety as measured by serum pharmacodynamic biomarkers by levels of P1NP.
Timepoint [30] 0 0
48 weeks
Secondary outcome [31] 0 0
Safety as measured by serum pharmacodynamic biomarkers by levels of differential lymphocyte percentage.
Timepoint [31] 0 0
48 weeks
Secondary outcome [32] 0 0
Extremity Fracture Questionnaire
Timepoint [32] 0 0
24, 48 weeks
Secondary outcome [33] 0 0
Physical examination findings at each of the pretreatment, on treatment, and post treatment assessment time points determined by change from baseline in physical examination findings, with assessment of clinical significance
Timepoint [33] 0 0
48 weeks

Eligibility
Key inclusion criteria
1. Subject's parent(s) or legal guardian(s) has (have) provided written informed consent
and Health Insurance Portability and Accountability Act (HIPAA) authorization, where
applicable, prior to any study-related procedures; participants will be asked to give
written or verbal assent according to local requirements

2. Subject has a centrally confirmed (by TRiNDS central genetic counselor[s]) diagnosis
of DMD as defined as:

- Dystrophin immunofluorescence and/or immunoblot showing complete dystrophin
deficiency, and clinical picture consistent with typical DMD, OR

- Identifiable mutation within the DMD gene (deletion/duplication of one or more
exons), where reading frame can be predicted as 'out-of-frame,' and clinical
picture consistent with typical DMD, OR

- Complete dystrophin gene sequencing showing an alteration (point mutation,
duplication, other) that is expected to preclude production of the dystrophin
protein (i.e., nonsense mutation, deletion/duplication leading to a downstream
stop codon), with a clinical picture consistent with typical DMD;

3. Subject is = 4 years and <7 years of age at time of enrollment in the study;

4. Subject weighs >13.0 kg and = 39.9 kg at the Screening Visit;

5. Subject is able to walk independently without assistive devices;

6. Subject is able to complete the Time to Stand Test (TTSTAND) without assistance in <10
seconds, as assessed at the Screening Visit;

7. Clinical laboratory test results are within the normal range at the Screening Visit,
or if abnormal, are not clinically significant, in the opinion of the Investigator.
[Notes: Serum gamma glutamyl transferase (GGT), creatinine, and total bilirubin all
must be = upper limit of the normal range at the Screening Visit. An abnormal vitamin
D level that is considered clinically significant will not exclude a subject from
randomization];

8. Subject has evidence of chicken pox immunity as determined by:

- Presence of IgG antibodies to varicella, as documented by a positive test result
from the local laboratory from blood collected during the Screening Period, OR

- Documentation, provided at the Screening Visit, that the subject has had 2 doses
of varicella vaccine, with or without serologic evidence of immunity; the second
of the 2 immunizations must have been given at least 14 days prior to
randomization.

9. Subject is able to swallow tablets, as confirmed by successful test swallowing of
placebo tablets during the Screening Period; and

10. Subject and parent(s)/guardian(s) are willing and able to comply with scheduled
visits, study drug administration plan, and study procedures.
Minimum age
4 Years
Maximum age
7 Years
Gender
Males
Can healthy volunteers participate?
No
Key exclusion criteria
1. Subject has current or history of major renal or hepatic impairment, diabetes mellitus
or immunosuppression;

2. Subject has current or history of chronic systemic fungal or viral infections;

3. Subject has had an acute illness within 4 weeks prior to the first dose of study
medication;

4. Subject has used mineralocorticoid receptor agents, such as spironolactone,
eplerenone, canrenone (canrenoate potassium), prorenone (prorenoate potassium),
mexrenone (mexrenoate potassium) within 4 weeks prior to the first dose of study
medication;

5. Subject has a history of primary hyperaldosteronism;

6. Subject has evidence of symptomatic cardiomyopathy [Note: Asymptomatic cardiac
abnormality on investigation would not be exclusionary];

7. Subject is currently being treated or has received previous treatment with oral
glucocorticoids or other immunosuppressive agents [Notes: Past transient use of oral
glucocorticoids or other oral immunosuppressive agents for no longer than 1 month
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, unless discontinued for
intolerance. Inhaled and/or topical glucocorticoids are permitted if last use is at
least 4 weeks prior to first dose of study medication or if administered at stable
dose beginning at least 4 weeks prior to first dose of study medication and
anticipated to be used at the stable dose regimen for the duration of the study];

8. Subject has an allergy or hypersensitivity to the study medication or to any of its
constituents;

9. Subject has used idebenone within 4 weeks prior to the first dose of study medication;

10. Subject has severe behavioral or cognitive problems that preclude participation in the
study, in the opinion of the Investigator;

11. Subject has previous or ongoing medical condition, medical history, physical findings
or laboratory abnormalities that could affect safety, make it unlikely that treatment
and follow-up will be correctly completed or impair the assessment of study results,
in the opinion of the Investigator;

12. Subject is taking (or has taken within 4 weeks prior to the first dose of study
medication) herbal remedies and supplements which can impact muscle strength and
function (e.g., Co-enzyme Q10, creatine, etc);

13. Subject is taking (or has taken within 3 months prior to the first dose of study
medication) any medication indicated for DMD, including Exondys51 and Translarna;

14. Subject has been administered a live attenuated vaccine within 14 days prior to the
first dose of study medication;

15. Subject is currently taking any other investigational drug or has taken any other
investigational drug within 3 months prior to the first dose of study medication;

16. Subject has a sibling who is currently enrolled in any vamorolone study or Expanded
Access Program, or who intends to enroll in any vamorolone study or Expanded Access
Program during the subject's participation in the VBP15-004 study; or

17. Subject has previously been enrolled in the study. Note: Any parameter/test may be
repeated at the Investigator's discretion during Screening to determine
reproducibility. In addition, subjects may be rescreened if ineligible due to a
transient condition which would prevent the subject from participating, such as an
upper respiratory tract infection or injury, or if ineligible due to negative
anti-varicella IgG antibody test result.

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 2
Type of endpoint(s)
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
Royal Children's Hospital - Melbourne
Recruitment hospital [2] 0 0
Sydney Children's Hospital - Westmead
Recruitment postcode(s) [1] 0 0
- Melbourne
Recruitment postcode(s) [2] 0 0
- Westmead
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
California
Country [2] 0 0
United States of America
State/province [2] 0 0
Colorado
Country [3] 0 0
United States of America
State/province [3] 0 0
Florida
Country [4] 0 0
United States of America
State/province [4] 0 0
Illinois
Country [5] 0 0
United States of America
State/province [5] 0 0
Minnesota
Country [6] 0 0
United States of America
State/province [6] 0 0
North Carolina
Country [7] 0 0
United States of America
State/province [7] 0 0
Texas
Country [8] 0 0
United States of America
State/province [8] 0 0
Virginia
Country [9] 0 0
United States of America
State/province [9] 0 0
Washington
Country [10] 0 0
Canada
State/province [10] 0 0
Alberta
Country [11] 0 0
Canada
State/province [11] 0 0
British Columbia
Country [12] 0 0
Canada
State/province [12] 0 0
Ontario
Country [13] 0 0
Canada
State/province [13] 0 0
Quebec
Country [14] 0 0
Israel
State/province [14] 0 0
Petah Tikvah
Country [15] 0 0
Netherlands
State/province [15] 0 0
Nijmegen
Country [16] 0 0
Sweden
State/province [16] 0 0
Gothenburg
Country [17] 0 0
United Kingdom
State/province [17] 0 0
Glasgow
Country [18] 0 0
United Kingdom
State/province [18] 0 0
Leeds
Country [19] 0 0
United Kingdom
State/province [19] 0 0
Liverpool
Country [20] 0 0
United Kingdom
State/province [20] 0 0
London
Country [21] 0 0
United Kingdom
State/province [21] 0 0
Newcastle upon Tyne

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
ReveraGen BioPharma, Inc.
Address
Country
Other collaborator category [1] 0 0
Other
Name [1] 0 0
European Union
Address [1] 0 0
Country [1] 0 0
Other collaborator category [2] 0 0
Other
Name [2] 0 0
Cooperative International Neuromuscular Research Group
Address [2] 0 0
Country [2] 0 0
Other collaborator category [3] 0 0
Other
Name [3] 0 0
Newcastle University
Address [3] 0 0
Country [3] 0 0
Other collaborator category [4] 0 0
Other
Name [4] 0 0
University of Pittsburgh
Address [4] 0 0
Country [4] 0 0

Ethics approval
Ethics application status

Summary
Brief summary
Brief Summary: This Phase IIb study is a randomized, double-blind, parallel group, placebo
and active-controlled study to evaluate the efficacy, safety, PD, and population PK of
vamorolone administered orally at daily doses of 2.0 mg/kg and 6.0 mg/kg versus prednisone
0.75 mg/kg/day and placebo over a Treatment Period of 24 weeks, and to evaluate persistence
of effect over a Treatment Period of 48 weeks in ambulant boys ages 4 to <7 years with DMD.
Trial website
https://clinicaltrials.gov/show/NCT03439670
Trial related presentations / publications
Mah JK, Korngut L, Dykeman J, Day L, Pringsheim T, Jette N. A systematic review and meta-analysis on the epidemiology of Duchenne and Becker muscular dystrophy. Neuromuscul Disord. 2014 Jun;24(6):482-91. doi: 10.1016/j.nmd.2014.03.008. Epub 2014 Mar 22. Review.
Hoffman EP, Brown RH Jr, Kunkel LM. Dystrophin: the protein product of the Duchenne muscular dystrophy locus. Cell. 1987 Dec 24;51(6):919-28.
Evans NP, Misyak SA, Robertson JL, Bassaganya-Riera J, Grange RW. Immune-mediated mechanisms potentially regulate the disease time-course of duchenne muscular dystrophy and provide targets for therapeutic intervention. PM R. 2009 Aug;1(8):755-68. doi: 10.1016/j.pmrj.2009.04.010. Review.
Kumar A, Boriek AM. Mechanical stress activates the nuclear factor-kappaB pathway in skeletal muscle fibers: a possible role in Duchenne muscular dystrophy. FASEB J. 2003 Mar;17(3):386-96.
Acharyya S, Villalta SA, Bakkar N, Bupha-Intr T, Janssen PM, Carathers M, Li ZW, Beg AA, Ghosh S, Sahenk Z, Weinstein M, Gardner KL, Rafael-Fortney JA, Karin M, Tidball JG, Baldwin AS, Guttridge DC. Interplay of IKK/NF-kappaB signaling in macrophages and myofibers promotes muscle degeneration in Duchenne muscular dystrophy. J Clin Invest. 2007 Apr;117(4):889-901. Epub 2007 Mar 22.
Dogra C, Changotra H, Wergedal JE, Kumar A. Regulation of phosphatidylinositol 3-kinase (PI3K)/Akt and nuclear factor-kappa B signaling pathways in dystrophin-deficient skeletal muscle in response to mechanical stretch. J Cell Physiol. 2006 Sep;208(3):575-85.
Pahl HL. Activators and target genes of Rel/NF-kappaB transcription factors. Oncogene. 1999 Nov 22;18(49):6853-66. Review.
Spencer MJ, Montecino-Rodriguez E, Dorshkind K, Tidball JG. Helper (CD4(+)) and cytotoxic (CD8(+)) T cells promote the pathology of dystrophin-deficient muscle. Clin Immunol. 2001 Feb;98(2):235-43.
Spencer MJ, Tidball JG. Do immune cells promote the pathology of dystrophin-deficient myopathies? Neuromuscul Disord. 2001 Sep;11(6-7):556-64. Review.
Malik V, Rodino-Klapac LR, Mendell JR. Emerging drugs for Duchenne muscular dystrophy. Expert Opin Emerg Drugs. 2012 Jun;17(2):261-77. doi: 10.1517/14728214.2012.691965. Review.
Reeves EKM, Hoffman EP, Nagaraju K, Damsker JM, McCall JM. VBP15: preclinical characterization of a novel anti-inflammatory delta 9,11 steroid. Bioorg Med Chem. 2013 Apr 15;21(8):2241-2249. doi: 10.1016/j.bmc.2013.02.009. Epub 2013 Feb 18. Erratum in: Bioorg Med Chem. 2015 Apr 1;23(7):1664.
Bushby K, Finkel R, Birnkrant DJ, Case LE, Clemens PR, Cripe L, Kaul A, Kinnett K, McDonald C, Pandya S, Poysky J, Shapiro F, Tomezsko J, Constantin C; DMD Care Considerations Working Group. Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and pharmacological and psychosocial management. Lancet Neurol. 2010 Jan;9(1):77-93. doi: 10.1016/S1474-4422(09)70271-6. Epub 2009 Nov 27. Review.
Pane M, Mazzone ES, Sivo S, Sormani MP, Messina S, D'Amico A, Carlesi A, Vita G, Fanelli L, Berardinelli A, Torrente Y, Lanzillotta V, Viggiano E, D Ambrosio P, Cavallaro F, Frosini S, Barp A, Bonfiglio S, Scalise R, De Sanctis R, Rolle E, Graziano A, Magri F, Palermo C, Rossi F, Donati MA, Sacchini M, Arnoldi MT, Baranello G, Mongini T, Pini A, Battini R, Pegoraro E, Previtali S, Bruno C, Politano L, Comi GP, Bertini E, Mercuri E. Long term natural history data in ambulant boys with Duchenne muscular dystrophy: 36-month changes. PLoS One. 2014 Oct 1;9(10):e108205. doi: 10.1371/journal.pone.0108205. eCollection 2014. Erratum in: PLoS One. 2015;10(3):e0121882. PLoS One. 2015;10(12):e0144079.
Manzur AY, Kuntzer T, Pike M, Swan A. Glucocorticoid corticosteroids for Duchenne muscular dystrophy. Cochrane Database Syst Rev. 2008 Jan 23;(1):CD003725. doi: 10.1002/14651858.CD003725.pub3. Review. Update in: Cochrane Database Syst Rev. 2016;(5):CD003725.
Heier CR, Damsker JM, Yu Q, Dillingham BC, Huynh T, Van der Meulen JH, Sali A, Miller BK, Phadke A, Scheffer L, Quinn J, Tatem K, Jordan S, Dadgar S, Rodriguez OC, Albanese C, Calhoun M, Gordish-Dressman H, Jaiswal JK, Connor EM, McCall JM, Hoffman EP, Reeves EK, Nagaraju K. VBP15, a novel anti-inflammatory and membrane-stabilizer, improves muscular dystrophy without side effects. EMBO Mol Med. 2013 Oct;5(10):1569-85. doi: 10.1002/emmm.201302621. Epub 2013 Sep 9.
Damsker JM, Dillingham BC, Rose MC, Balsley MA, Heier CR, Watson AM, Stemmy EJ, Jurjus RA, Huynh T, Tatem K, Uaesoontrachoon K, Berry DM, Benton AS, Freishtat RJ, Hoffman EP, McCall JM, Gordish-Dressman H, Constant SL, Reeves EK, Nagaraju K. VBP15, a glucocorticoid analogue, is effective at reducing allergic lung inflammation in mice. PLoS One. 2013 May 7;8(5):e63871. doi: 10.1371/journal.pone.0063871. Print 2013.
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Public notes

Contacts
Principal investigator
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Michela Guglieri, M.D.
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John Walton Muscular Dystrophy Research Centre
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Phone 0 0
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Contact person for public queries
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Andrea Smith
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+1 (412) 436-9139
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asmith@trinds.com
Contact person for scientific queries

Summary results
For IPD and results data, please see https://clinicaltrials.gov/show/NCT03439670