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

This study is currently recruiting participants. (see Contacts and Locations)
Verified March 2017 by ReveraGen BioPharma, Inc.
Sponsor:
Collaborators:
University of Pittsburgh
National Institute of Neurological Disorders and Stroke (NINDS)
Cooperative International Neuromuscular Research Group
National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)
Information provided by (Responsible Party):
ReveraGen BioPharma, Inc.
ClinicalTrials.gov Identifier:
NCT02760277
First received: April 28, 2016
Last updated: March 20, 2017
Last verified: March 2017
  Purpose
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.

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

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: No masking
Primary Purpose: Treatment
Official Title: A Phase II Open-label, Multicenter Extension Study to Assess the Long-term Safety and 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: 24 weeks ]
  • Muscle function measured by Time to Stand Test (TTSTAND) [ Time Frame: 24 weeks ]
  • Body size as measured by body mass index (BMI) z-score [ Time Frame: 24 weeks ]

Secondary Outcome Measures:
  • Serum pharmacodynamics biomarkers measured by levels of cortisol [ Time Frame: 24 weeks ]
  • Serum pharmacodynamics biomarkers measured by levels of ACTH [ Time Frame: 24 weeks ]
  • Serum pharmacodynamics biomarkers measured by levels of PINP [ Time Frame: 24 weeks ]
  • Serum pharmacodynamics biomarkers measured by levels of osteocalcin [ Time Frame: 24 weeks ]
  • Serum pharmacodynamics biomarkers measured by levels of CTX [ Time Frame: 24 weeks ]
  • Serum pharmacodynamics biomarkers measured by levels of 17- hydroxyprogesterone [ Time Frame: 24 weeks ]
  • Serum pharmacodynamics biomarkers measured by levels of testosterone [ Time Frame: 24 weeks ]
  • Serum pharmacodynamics biomarkers measured by levels of corticosterone [ Time Frame: 24 weeks ]
  • Serum pharmacodynamics biomarkers measured by levels of 11-deoxycortisol [ Time Frame: 24 weeks ]
  • Serum pharmacodynamics biomarkers measured by levels of glucose [ Time Frame: 24 weeks ]
  • Serum pharmacodynamics biomarkers measured by levels of insulin [ Time Frame: 24 weeks ]
  • Muscle strength measured by Quantitative Muscle Testing (QMT) [ Time Frame: 24 weeks ]
  • Muscle function measured by Time to Climb Test (TTCLIMB) [ Time Frame: 24 weeks ]
  • Muscle function measured by Time to Run/Walk 10 Meters Test (TTRW) [ Time Frame: 24 weeks ]
  • Muscle function measured by North Star Ambulatory Assessment (NSAA) [ Time Frame: 24 weeks ]
  • Muscle function measured by Six-minute Walk Test (6MWT) [ Time Frame: 24 weeks ]

Estimated Enrollment: 48
Study Start Date: August 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/day/day
Oral administration of 0.25 mg/kg/day daily for 24 weeks.
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/day/day
Oral administration of 0.75 mg/kg/day daily for 24 weeks.
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/day/day
Oral administration of 2.0 mg/kg/day daily for 24 weeks.
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/day/day
Oral administration of 6.0 mg/kg/day daily for 24 weeks.
Other Name: VBP15

Detailed Description:
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.
  Eligibility

Ages Eligible for Study:   4 Years to 7 Years   (Child)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Participant's parent or legal guardian has provided written informed consent/HIPAA authorization prior to any extension study-specific procedures;
  2. Participant has previously completed study VBP15-002 up to and including the Week 4 Follow-up assessments within 8 weeks prior to enrollment; and
  3. Participant and parent/guardian are willing and able to comply with scheduled visits, study drug administration plan, and study procedures.

Exclusion Criteria:

  1. Participant had a serious or severe adverse event in study VBP15-002 that, in the opinion of the Investigator, was probably or definitely related to vamorolone use and precludes safe use of vamorolone for the subject in this study;
  2. Participant has current or history of major renal or hepatic impairment, diabetes mellitus or immunosuppression;
  3. Participant has current or history of chronic systemic fungal or viral infections;
  4. Participant 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. Participant has evidence of symptomatic cardiomyopathy. [Note: Asymptomatic cardiac abnormality on investigation would not be exclusionary];
  6. Participant 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. Participant has an allergy or hypersensitivity to the study medication or to any of its constituents;
  9. Participant has severe behavioral or cognitive problems that preclude participation in the study, in the opinion of the Investigator;
  10. Participant 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; or
  11. Participant is currently taking any investigational drug, or has taken any investigational drug other than vamorolone within 3 months prior to the start of study treatment.

Note: Participants may be re-evaluated if ineligible due to a transient condition which would prevent the subject from participating

  Contacts and Locations
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, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT02760277

Contacts
Contact: Andrea Smith 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: Katie Dickinson    352-273-7573    k.dickinson@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: Theresa Oswald    312-227-3019    TOswald@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 Not yet 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 Tikwah, Israel, 49202
Contact: Ori Raz    +972-3-9253559    orira1@clalit.org.il   
Sweden
Queen Silvia Children's Hospital Not yet 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 Not yet 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 Neurological Disorders and Stroke (NINDS)
Cooperative International Neuromuscular Research Group
National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)
Investigators
Study Chair: Paula R Clemens, MD University of Pittsburg
  More Information

Responsible Party: ReveraGen BioPharma, Inc.
ClinicalTrials.gov Identifier: NCT02760277     History of Changes
Other Study ID Numbers: VBP15-003
1R44NS095423-01 ( US NIH Grant/Contract Award Number )
1U34AR068616-01 ( US NIH Grant/Contract Award Number )
Study First Received: April 28, 2016
Last Updated: March 20, 2017

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

ClinicalTrials.gov processed this record on March 24, 2017