welcome to DuchenneXchange- a positively charged Duchenne muscular dystrophy community.
- join today!
A Study to Assess Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD)
study id #: NCT02760264
condition: Duchenne Muscular Dystrophy
The purpose of this study is to determine whether a new medication called vamorolone is safe and well-tolerated by boys with Duchenne muscular dystrophy (DMD) ages >= 4 and < 7 years old.
Vamorolone 0.25 mg/kg/day, Vamorolone 0.75 mg/kg/day, Vamorolone 2.0 mg/kg/day, Vamorolone 6.0 mg/kg/day
mechanism of action: Glucocorticoid to delay decline in muscle strength
last updated: January 07, 2019
start date: June 2016
estimated completion: May 1, 2018
phase of development: Phase 2
size / enrollment: 48
This study will evaluate the safety and tolerability of a new steroid-like medication called vamorolone in boys with DMD ages >=4 years and < 7 years. Enrolled participants will take the study medication for 14 days followed by a 14 day follow-up period. The potential effectiveness of vamorolone in treating DMD will also be explored.
- Number of participants with treatment-related adverse events as assessed by CTCAE Version 4.03. [ Time Frame: 4 weeks ]
- Peak plasma concentration (Cmax) of vamorolone [ Time Frame: Day 1 ]
- Peak plasma concentration (Cmax) of vamorolone [ Time Frame: Week 2 ]
- Serum pharmacodynamics biomarkers measured by levels of cortisol [ Time Frame: 4 weeks ]
- Serum pharmacodynamics biomarkers measured by levels of ACTH [ Time Frame: 4 weeks ]
- Serum pharmacodynamics biomarkers measured by levels of PINP [ Time Frame: 4 weeks ]
- Serum pharmacodynamics biomarkers measured by levels of osteocalcin [ Time Frame: 4 weeks ]
- Serum pharmacodynamics biomarkers measured by levels of CTX [ Time Frame: 4 weeks ]
- Serum pharmacodynamics biomarkers measured by levels of 17-hydroxyprogesterone [ Time Frame: 4 weeks ]
- Serum pharmacodynamics biomarkers measured by levels of testosterone [ Time Frame: 4 weeks ]
- Serum pharmacodynamics biomarkers measured by levels of corticosterone [ Time Frame: 4 weeks ]
- Serum pharmacodynamics biomarkers measured by levels of 11-deoxycortisol [ Time Frame: 4 weeks ]
- Serum pharmacodynamics biomarkers measured by levels of glucose [ Time Frame: 2 weeks ]
- Serum pharmacodynamics biomarkers measured by levels of insulin [ Time Frame: 2 weeks ]
- Serum metabolites of vamorolone [ Time Frame: Week 2 ]
• Subject's parent or legal guardian has provided written informed consent/Health Insurance Portability and Accountability Act (HIPAA) authorization prior to any study-related procedures;
• Subject has a confirmed (by Central Genetic Counselor) diagnosis of DMD as defined as:
-Dystrophin immunofluorescence and/or immunoblot showing complete dystrophin deficiency, and clinical picture consistent with typical DMD, OR
-Identifiable mutation within the DMD gene (deletion/duplication of one or more exons) where reading frame can be predicted as 'out-of-frame', and clinical picture consistent with typical DMD, OR
-Complete dystrophin gene sequencing showing an alteration (point mutation, duplication, other) that is expected to preclude production of the dystrophin protein (i.e. nonsense mutation, deletion/duplication leading to a downstream stop codon), with a typical clinical picture of DMD;
• Subject is >= 4 years and < 7 years of age at time of enrollment in the study;
• Subject is able to complete the Time to Stand Test (TTSTAND) without assistance, as assessed at the Screening and Baseline Visits;
• Clinical laboratory test results are within the normal range at the Screening Visit, or if abnormal, are not clinically significant, in the opinion of the Investigator. (Note: Serum gamma glutamyl transferase [GGT], creatinine, and total bilirubin all must be <= upper limit of the normal range at the Screening Visit);
• Subject has evidence of chicken pox immunity as determined by presence of IgG antibodies to varicella, as documented by a positive test result from the testing laboratory at the Screening Visit; and
• Subject and parent/guardian are willing and able to comply with scheduled visits, study drug administration plan, and study procedures.
• Subject has current or history of major renal or hepatic impairment, diabetes mellitus or immunosuppression;
• Subject has current or history of chronic systemic fungal or viral infections;
• Subject has had an acute illness within 4 weeks prior to the first dose of study medication;
• Subject has used mineralocorticoid receptor agents, such as spironolactone, eplerenone, canrenone (canrenoate potassium), prorenone (prorenoate potassium), mexrenone (mexrenoate potassium) within 4 weeks prior to the first dose of study medication;
• Subject has evidence of symptomatic cardiomyopathy. [Note: Asymptomatic cardiac abnormality on investigation would not be exclusionary];
• Subject is currently being treated or has received previous treatment with oral glucocorticoids or other immunosuppressive agents. [Notes: Past transient use of oral glucocorticoids or other oral immunosuppressive agents for no longer than 3 months cumulative, with last use at least 3 months prior to first dose of study medication, will be considered for eligibility on a case-by-case basis. Inhaled and/or topical corticosteroids prescribed for an indication other than DMD are permitted but must be administered at stable dose for at least 3 months prior to study drug administration];
• Subject has used idebenone within 4 weeks prior to the first dose of study medication;
• Subject has an allergy or hypersensitivity to the study medication or to any of its constituents;
• Subject has severe behavioral or cognitive problems that preclude participation in the study, in the opinion of the Investigator;
• Subject has previous or ongoing medical condition, medical history, physical findings or laboratory abnormalities that could affect safety, make it unlikely that treatment and follow-up will be correctly completed or impair the assessment of study results, in the opinion of the Investigator;
• Subject is taking any other investigational drug currently or has taken any other investigational drug within 3 months prior to the start of study treatment; or
• Subject has previously been enrolled in the study.
An Open-label Extension Study To Evaluate Safety Of PF-06252616 In Boys With Duchenne Muscular DystrophyThis study is an open-label extension to...
Gene Transfer Clinical Trial to Deliver rAAVrh74.MCK.GALGT2 for Duchenne Muscular DystrophyThe proposed clinical trial study of rAA...
Safety Study of Eteplirsen to Treat Advanced Stage Duchenne Muscular DystrophyThe primary objective of this study is t...
Extension Study of BMN 044 in Duchenne Muscular Dystrophy (DMD)The aim of this study is to provide cont...
Open Label, Extension Study of PRO044 in Duchenne Muscular Dystrophy (DMD)The purpose of this study is to see whet...
PoC Study to Assess Activity and Safety of SMT C1100 (Ezutromid) in Boys With DMDA Phase 2 Clinical Study to Assess the A...
Microdystrophin Gene Transfer Study in Adolescents and Children With DMDThis is a randomized, controlled, open-l...
A phase 2 trial of the safety and pharmacokinetics of ataluren in patients aged 2 to 5 years with nonsense mutation ...Nonsense mutation Duchenne muscular dyst...
ReveraGen Announces First Patient Enrollment in International Pivotal Trial of Vamorolone in Duchenne Muscular Dystr...ReveraGen BioPharma, Inc. today announce...
Wave Life Sciences Duchenne Muscular Dystrophy Clinical Trial Selected for FDA Complex Innovative Trial Designs Pilo...Wave Life Sciences Ltd., a biotechnology...
Solid Biosciences Reports Third Quarter 2018 Financial Results And Provides Business UpdateSolid Biosciences Inc. today reported fi...
Phase IIa trial in Duchenne muscular dystrophy shows vamorolone is a first-in-class dissociative steroidal anti-infl...We report a first-in-patient study of va...