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

This study is currently recruiting participants.
Verified August 2017 by ReveraGen BioPharma, Inc.
Sponsor:
ClinicalTrials.gov Identifier:
NCT02760264
First Posted: May 3, 2016
Last Update Posted: August 25, 2017
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 evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
Collaborators:
University of Pittsburgh
National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)
National Institute of Neurological Disorders and Stroke (NINDS)
Cooperative International Neuromuscular Research Group
Information provided by (Responsible Party):
ReveraGen BioPharma, Inc.
  Purpose
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.

Condition Intervention Phase
Duchenne Muscular Dystrophy Drug: Vamorolone 0.25 mg/kg/day Drug: Vamorolone 0.75 mg/kg/day Drug: Vamorolone 2.0 mg/kg/day Drug: Vamorolone 6.0 mg/kg/day Phase 2

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase IIa Open-Label, Multiple Ascending Dose Study to Assess the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Exploratory Efficacy of Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD)

Resource links provided by NLM:


Further study details as provided by ReveraGen BioPharma, Inc.:

Primary Outcome Measures:
  • Number of participants with treatment-related adverse events as assessed by CTCAE Version 4.03. [ Time Frame: 4 weeks ]

Secondary Outcome Measures:
  • 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 ]
    Metabolites in Safety Testing (MIST)


Other Outcome Measures:
  • Muscle strength measured by Quantitative Muscle Testing (QMT) [ Time Frame: 4 weeks ]
  • Muscle function measured by Time to Stand Test (TTSTAND) [ Time Frame: 4 weeks ]
  • Muscle function measured by Time to Climb Test (TTCLIMB) [ Time Frame: 4 weeks ]
  • Muscle function measured by Time to Run/Walk 10 Meters Test (TTRW) [ Time Frame: 4 weeks ]
  • Muscle function measured by North Star Ambulatory Assessment (NSAA) [ Time Frame: 4 weeks ]
  • Muscle function measured by Six-minute Walk Test (6MWT) [ Time Frame: 4 weeks ]
  • Evaluate Metabolites in Safety Testing (MIST) assessment [ Time Frame: 4 weeks ]

Estimated Enrollment: 48
Study Start Date: June 2016
Estimated Study Completion Date: August 2017
Estimated Primary Completion Date: August 2017 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Dose Level Group 1
Participants enrolled in Dose Level Group 1 will receive vamorolone 0.25 mg/kg/day.
Drug: Vamorolone 0.25 mg/kg/day
Oral administration of 0.25 mg/kg/day daily for 14 days.
Other Name: VBP15
Experimental: Dose Level Group 2
Participants enrolled in Dose Level Group 2 will receive vamorolone 0.75 mg/kg/day.
Drug: Vamorolone 0.75 mg/kg/day
Oral administration of 0.75 mg/kg/day daily for 14 days.
Other Name: VBP15
Experimental: Dose Level Group 3
Participants enrolled in Dose Level Group 3 will receive vamorolone 2.0 mg/kg/day.
Drug: Vamorolone 2.0 mg/kg/day
Oral administration of 2.0 mg/kg/day daily for 14 days.
Other Name: VBP15
Experimental: Dose Level Group 4
Participants enrolled in Dose Level Group 4 will receive vamorolone 6.0 mg/kg/day.
Drug: Vamorolone 6.0 mg/kg/day
Oral administration of 6 mg/kg/day daily for 14 days.
Other Name: VBP15

Detailed Description:
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.
  Eligibility

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Ages Eligible for Study:   4 Years to 6 Years   (Child)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. 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;
  2. Subject has a confirmed (by Central Genetic Counselor) diagnosis of DMD as defined as:

    1. Dystrophin immunofluorescence and/or immunoblot showing complete dystrophin deficiency, and clinical picture consistent with typical DMD, OR
    2. 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
    3. 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;
  3. Subject is ≥ 4 years and < 7 years of age at time of enrollment in the study;
  4. Subject is able to complete the Time to Stand Test (TTSTAND) without assistance, as assessed at the Screening and Baseline Visits;
  5. 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);
  6. 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
  7. Subject and parent/guardian are willing and able to comply with scheduled visits, study drug administration plan, and study procedures.

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 evidence of symptomatic cardiomyopathy. [Note: Asymptomatic cardiac abnormality on investigation would not be exclusionary];
  6. 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];
  7. Subject has used idebenone within 4 weeks prior to the first dose of study medication;
  8. Subject has an allergy or hypersensitivity to the study medication or to any of its constituents;
  9. Subject has severe behavioral or cognitive problems that preclude participation in the study, in the opinion of the Investigator;
  10. 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;
  11. 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
  12. Subject has previously been enrolled in the study.
  Contacts and Locations
Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02760264


Contacts
Contact: Andrea Smith, MS 412-436-9139 asmith@trinds.com
Contact: Eric P Hoffman, PhD eric.hoffman@reveragen.com

Locations
United States, California
University of California Davis Recruiting
Davis, California, United States, 95616
Contact: Colleen Anthonisen    916-734-4307    canthonisen@ucdavis.edu   
Principal Investigator: Craig McDonald, MD         
United States, Florida
University of Florida Recruiting
Gainesville, Florida, United States, 32611
Contact: Stephanie Salabarria    352-273-6582    ssalabarria@ufl.edu   
Principal Investigator: Barry Byrne, MD         
Nemours Children's Hospital Recruiting
Orlando, Florida, United States, 32827
Contact: Kristin McCrary    407-650-7175    kristin.mccrary@nemours.org   
Principal Investigator: Richard Finkel, MD         
United States, Illinois
Ann & Robert H. Lurie Children's Hospital Recruiting
Chicago, Illinois, United States, 60611
Contact: Duncan Schulte    312-227-3813    dschulte@luriechildrens.org   
Principal Investigator: Nancy Kuntz, MD         
United States, North Carolina
Duke University Recruiting
Durham, North Carolina, United States, 27710
Contact: Karen Cornett    919-684-1143    k.cornett@duke.edu   
Principal Investigator: Edward Smith, MD         
United States, Texas
University of Texas Southwestern Medical Center Recruiting
Dallas, Texas, United States, 75207
Contact: Maria Martinez    214-648-5606    MariaR.Martinez@UTSouthwestern.edu   
Principal Investigator: Diana Castro, MD         
Australia
Royal Children's Hospital Recruiting
Melbourne, Australia
Contact: Jemima Mitchell, RN. BNurs. GradDip Paed.    03 9936 6157    Jemima.Mitchell@rch.org.au   
Sydney Children's Hospital Recruiting
Westmead, Australia
Contact: Eshwini Tadiyal    (02) 02 9845 3048    eshwini.tadiyal@health.nsw.gov.au   
Canada, Alberta
Alberta Children's Hospital Recruiting
Calgary, Alberta, Canada, T3B 6A8
Contact: Tiffany Haig    403-955-3192    tiffany.haig@albertahealthservices.ca   
Principal Investigator: Jean Mah, MD, MSc         
Israel
Schneider Children's Medical Center Recruiting
Petah Tikvah, Israel, 49202
Contact: Ori Raz    +972-3-9253559    orira1@clalit.org.il   
Sweden
Queen Silvia Children's Hospital Recruiting
Gothenburg, Sweden, 41685
Contact: Mar Tulinius, MD    +46 (0)31 3434780    mar.tulinius@gu.se   
United Kingdom
Newcastle upon Tyne Hospitals NHS Foundation Trust Recruiting
Newcastle upon Tyne, United Kingdom, NE7 7DN
Contact: Becky Davis    +44 (0) 191 241 8649    becky.davis@newcastle.ac.uk   
Sponsors and Collaborators
ReveraGen BioPharma, Inc.
University of Pittsburgh
National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)
National Institute of Neurological Disorders and Stroke (NINDS)
Cooperative International Neuromuscular Research Group
Investigators
Study Chair: Paula R Clemens, MD University of Pittsburg
  More Information

Additional Information:
Responsible Party: ReveraGen BioPharma, Inc.
ClinicalTrials.gov Identifier: NCT02760264     History of Changes
Other Study ID Numbers: VBP15-002
1R44NS095423-01 ( U.S. NIH Grant/Contract )
1U34AR068616-01 ( U.S. NIH Grant/Contract )
First Submitted: April 28, 2016
First Posted: May 3, 2016
Last Update Posted: August 25, 2017
Last Verified: August 2017
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes

Keywords provided by ReveraGen BioPharma, Inc.:
Duchenne Muscular Dystrophy
vamorolone

Additional relevant MeSH terms:
Muscular Dystrophies
Muscular Dystrophy, Duchenne
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, Inborn
Genetic Diseases, X-Linked


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