Trial Review

Technical difficulties have been reported by some users of the search function and is being investigated by technical staff. Thank you for your patience and apologies for any inconvenience caused.

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been endorsed by the ANZCTR. Before participating in a study, talk to your health care provider and refer to this information for consumers
Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/ct2/show/NCT03439670




Registration number
NCT03439670
Ethics application status
Date submitted
9/01/2018
Date registered
20/02/2018
Date last updated
9/03/2023

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
Efficacy Measured by Time to Stand Test (TTSTAND) Velocity in Rises/Second Change From Baseline
Timepoint [1] 0 0
24 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
Sex
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
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
29/06/2018
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
19/08/2021
Sample size
Target
Accrual to date
Final
121
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
Belgium
State/province [10] 0 0
Ghent
Country [11] 0 0
Belgium
State/province [11] 0 0
Leuven
Country [12] 0 0
Canada
State/province [12] 0 0
Alberta
Country [13] 0 0
Canada
State/province [13] 0 0
British Columbia
Country [14] 0 0
Canada
State/province [14] 0 0
Ontario
Country [15] 0 0
Canada
State/province [15] 0 0
Quebec
Country [16] 0 0
Czechia
State/province [16] 0 0
Brno
Country [17] 0 0
Czechia
State/province [17] 0 0
Prague
Country [18] 0 0
Greece
State/province [18] 0 0
Athens
Country [19] 0 0
Israel
State/province [19] 0 0
Petah Tikvah
Country [20] 0 0
Netherlands
State/province [20] 0 0
Leiden
Country [21] 0 0
Netherlands
State/province [21] 0 0
Nijmegen
Country [22] 0 0
Spain
State/province [22] 0 0
Barcelona
Country [23] 0 0
Spain
State/province [23] 0 0
Valencia
Country [24] 0 0
Sweden
State/province [24] 0 0
Gothenburg
Country [25] 0 0
United Kingdom
State/province [25] 0 0
Birmingham
Country [26] 0 0
United Kingdom
State/province [26] 0 0
Glasgow
Country [27] 0 0
United Kingdom
State/province [27] 0 0
Leeds
Country [28] 0 0
United Kingdom
State/province [28] 0 0
Liverpool
Country [29] 0 0
United Kingdom
State/province [29] 0 0
London
Country [30] 0 0
United Kingdom
State/province [30] 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/ct2/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.
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. doi: 10.1016/0092-8674(87)90579-4.
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.
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. doi: 10.1096/fj.02-0542com.
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. doi: 10.1172/JCI30556. 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. doi: 10.1002/jcp.20696.
Pahl HL. Activators and target genes of Rel/NF-kappaB transcription factors. Oncogene. 1999 Nov 22;18(49):6853-66. doi: 10.1038/sj.onc.1203239.
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. doi: 10.1006/clim.2000.4966.
Spencer MJ, Tidball JG. Do immune cells promote the pathology of dystrophin-deficient myopathies? Neuromuscul Disord. 2001 Sep;11(6-7):556-64. doi: 10.1016/s0960-8966(01)00198-5.
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.
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.
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.
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.
Dillingham BC, Knoblach SM, Many GM, Harmon BT, Mullen AM, Heier CR, Bello L, McCall JM, Hoffman EP, Connor EM, Nagaraju K, Reeves EKM, Damsker JM. VBP15, a novel anti-inflammatory, is effective at reducing the severity of murine experimental autoimmune encephalomyelitis. Cell Mol Neurobiol. 2015 Apr;35(3):377-387. doi: 10.1007/s10571-014-0133-y. Epub 2014 Nov 13.
Dadgar S, Wang Z, Johnston H, Kesari A, Nagaraju K, Chen YW, Hill DA, Partridge TA, Giri M, Freishtat RJ, Nazarian J, Xuan J, Wang Y, Hoffman EP. Asynchronous remodeling is a driver of failed regeneration in Duchenne muscular dystrophy. J Cell Biol. 2014 Oct 13;207(1):139-58. doi: 10.1083/jcb.201402079.
Raman SV, Hor KN, Mazur W, Halnon NJ, Kissel JT, He X, Tran T, Smart S, McCarthy B, Taylor MD, Jefferies JL, Rafael-Fortney JA, Lowe J, Roble SL, Cripe LH. Eplerenone for early cardiomyopathy in Duchenne muscular dystrophy: a randomised, double-blind, placebo-controlled trial. Lancet Neurol. 2015 Feb;14(2):153-61. doi: 10.1016/S1474-4422(14)70318-7. Epub 2014 Dec 30. Erratum In: Lancet Neurol. 2015 Feb;14(2):135.
Avioli LV. Glucocorticoid effects on statural growth. Br J Rheumatol. 1993 May;32 Suppl 2:27-30. doi: 10.1093/rheumatology/32.suppl_2.27.
Wolthers OD, Pedersen S. Short term linear growth in asthmatic children during treatment with prednisolone. BMJ. 1990 Jul 21;301(6744):145-8. doi: 10.1136/bmj.301.6744.145.
Bircan Z, Soran M, Yildirim I, Dogan M, Sahin A, Bilici A, Danaci M. The effect of alternate-day low dose prednisolone on bone age in children with steroid dependent nephrotic syndrome. Int Urol Nephrol. 1997;29(3):357-61. doi: 10.1007/BF02550936.
Manolagas SC, Weinstein RS. New developments in the pathogenesis and treatment of steroid-induced osteoporosis. J Bone Miner Res. 1999 Jul;14(7):1061-6. doi: 10.1359/jbmr.1999.14.7.1061. No abstract available.
Sali A, Guerron AD, Gordish-Dressman H, Spurney CF, Iantorno M, Hoffman EP, Nagaraju K. Glucocorticoid-treated mice are an inappropriate positive control for long-term preclinical studies in the mdx mouse. PLoS One. 2012;7(4):e34204. doi: 10.1371/journal.pone.0034204. Epub 2012 Apr 3.
Kauh E, Mixson L, Malice MP, Mesens S, Ramael S, Burke J, Reynders T, Van Dyck K, Beals C, Rosenberg E, Ruddy M. Prednisone affects inflammation, glucose tolerance, and bone turnover within hours of treatment in healthy individuals. Eur J Endocrinol. 2012 Mar;166(3):459-67. doi: 10.1530/EJE-11-0751. Epub 2011 Dec 17.
Wehling-Henricks M, Oltmann M, Rinaldi C, Myung KH, Tidball JG. Loss of positive allosteric interactions between neuronal nitric oxide synthase and phosphofructokinase contributes to defects in glycolysis and increased fatigability in muscular dystrophy. Hum Mol Genet. 2009 Sep 15;18(18):3439-51. doi: 10.1093/hmg/ddp288. Epub 2009 Jun 19.
Younes M, Neffati F, Touzi M, Hassen-Zrour S, Fendri Y, Bejia I, Ben Amor A, Bergaoui N, Najjar MF. Systemic effects of epidural and intra-articular glucocorticoid injections in diabetic and non-diabetic patients. Joint Bone Spine. 2007 Oct;74(5):472-6. doi: 10.1016/j.jbspin.2006.10.009. Epub 2007 Jul 6.
Moon HJ, Choi KH, Lee SI, Lee OJ, Shin JW, Kim TW. Changes in blood glucose and cortisol levels after epidural or shoulder intra-articular glucocorticoid injections in diabetic or nondiabetic patients. Am J Phys Med Rehabil. 2014 May;93(5):372-8. doi: 10.1097/PHM.0000000000000001.
Investigator's Brochure, Version 4, Vamorolone (17a,21-dihydroxy-16a-methyl-pregna-1,4,9(11)-triene-3,20-dione) 4% Oral Suspension, ReveraGen BioPharma, Inc., January 10, 2017.
Fleishaker DL, Mukherjee A, Whaley FS, Daniel S, Zeiher BG. Safety and pharmacodynamic dose response of short-term prednisone in healthy adult subjects: a dose ranging, randomized, placebo-controlled, crossover study. BMC Musculoskelet Disord. 2016 Jul 16;17:293. doi: 10.1186/s12891-016-1135-3.
Hathout Y, Conklin LS, Seol H, Gordish-Dressman H, Brown KJ, Morgenroth LP, Nagaraju K, Heier CR, Damsker JM, van den Anker JN, Henricson E, Clemens PR, Mah JK, McDonald C, Hoffman EP. Serum pharmacodynamic biomarkers for chronic corticosteroid treatment of children. Sci Rep. 2016 Aug 17;6:31727. doi: 10.1038/srep31727.
Agwu JC, Spoudeas H, Hindmarsh PC, Pringle PJ, Brook CG. Tests of adrenal insufficiency. Arch Dis Child. 1999 Apr;80(4):330-3. doi: 10.1136/adc.80.4.330.
van Staa TP, Leufkens HG, Abenhaim L, Zhang B, Cooper C. Oral corticosteroids and fracture risk: relationship to daily and cumulative doses. Rheumatology (Oxford). 2000 Dec;39(12):1383-9. doi: 10.1093/rheumatology/39.12.1383.
McDonald C, Camino E, Escandon R, Finkel RS, Fischer R, Flanigan K, Furlong P, Juhasz R, Martin AS, Villa C, Sweeney HL. Draft Guidance for Industry Duchenne Muscular Dystrophy, Becker Muscular Dystrophy, and Related Dystrophinopathies - Developing Potential Treatments for the Entire Spectrum of Disease. J Neuromuscul Dis. 2024;11(2):499-523. doi: 10.3233/JND-230219.
Brooke MH, Griggs RC, Mendell JR, Fenichel GM, Shumate JB, Pellegrino RJ. Clinical trial in Duchenne dystrophy. I. The design of the protocol. Muscle Nerve. 1981 May-Jun;4(3):186-97. doi: 10.1002/mus.880040304. No abstract available.
Mazzone ES, Messina S, Vasco G, Main M, Eagle M, D'Amico A, Doglio L, Politano L, Cavallaro F, Frosini S, Bello L, Magri F, Corlatti A, Zucchini E, Brancalion B, Rossi F, Ferretti M, Motta MG, Cecio MR, Berardinelli A, Alfieri P, Mongini T, Pini A, Astrea G, Battini R, Comi G, Pegoraro E, Morandi L, Pane M, Angelini C, Bruno C, Villanova M, Vita G, Donati MA, Bertini E, Mercuri E. Reliability of the North Star Ambulatory Assessment in a multicentric setting. Neuromuscul Disord. 2009 Jul;19(7):458-61. doi: 10.1016/j.nmd.2009.06.368. Epub 2009 Jun 23.
McDonald CM, Henricson EK, Han JJ, Abresch RT, Nicorici A, Elfring GL, Atkinson L, Reha A, Hirawat S, Miller LL. The 6-minute walk test as a new outcome measure in Duchenne muscular dystrophy. Muscle Nerve. 2010 Apr;41(4):500-10. doi: 10.1002/mus.21544.
Bello L, Kesari A, Gordish-Dressman H, Cnaan A, Morgenroth LP, Punetha J, Duong T, Henricson EK, Pegoraro E, McDonald CM, Hoffman EP; Cooperative International Neuromuscular Research Group Investigators. Genetic modifiers of ambulation in the Cooperative International Neuromuscular Research Group Duchenne Natural History Study. Ann Neurol. 2015 Apr;77(4):684-96. doi: 10.1002/ana.24370. Epub 2015 Mar 13.
Spurney C, Shimizu R, Morgenroth LP, Kolski H, Gordish-Dressman H, Clemens PR; CINRG Investigators. Cooperative International Neuromuscular Research Group Duchenne Natural History Study demonstrates insufficient diagnosis and treatment of cardiomyopathy in Duchenne muscular dystrophy. Muscle Nerve. 2014 Aug;50(2):250-6. doi: 10.1002/mus.24163. Epub 2014 May 14.
McDonald CM, Henricson EK, Abresch RT, Han JJ, Escolar DM, Florence JM, Duong T, Arrieta A, Clemens PR, Hoffman EP, Cnaan A; Cinrg Investigators. The cooperative international neuromuscular research group Duchenne natural history study--a longitudinal investigation in the era of glucocorticoid therapy: design of protocol and the methods used. Muscle Nerve. 2013 Jul;48(1):32-54. doi: 10.1002/mus.23807. Epub 2013 May 16.
Henricson EK, Abresch RT, Cnaan A, Hu F, Duong T, Arrieta A, Han J, Escolar DM, Florence JM, Clemens PR, Hoffman EP, McDonald CM; CINRG Investigators. The cooperative international neuromuscular research group Duchenne natural history study: glucocorticoid treatment preserves clinically meaningful functional milestones and reduces rate of disease progression as measured by manual muscle testing and other commonly used clinical trial outcome measures. Muscle Nerve. 2013 Jul;48(1):55-67. doi: 10.1002/mus.23808. Epub 2013 May 6.
Escolar DM, Hache LP, Clemens PR, Cnaan A, McDonald CM, Viswanathan V, Kornberg AJ, Bertorini TE, Nevo Y, Lotze T, Pestronk A, Ryan MM, Monasterio E, Day JW, Zimmerman A, Arrieta A, Henricson E, Mayhew J, Florence J, Hu F, Connolly AM. Randomized, blinded trial of weekend vs daily prednisone in Duchenne muscular dystrophy. Neurology. 2011 Aug 2;77(5):444-52. doi: 10.1212/WNL.0b013e318227b164. Epub 2011 Jul 13.
Public notes

Contacts
Principal investigator
Name 0 0
Michela Guglieri, M.D.
Address 0 0
John Walton Muscular Dystrophy Research Centre
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries