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

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional

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)

Brief Summary: 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.

Detailed Summary: 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.
Sponsor: ReveraGen BioPharma, Inc.

Current Primary Outcome:

  • 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 ]


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • 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 ]


Original Secondary Outcome:

  • Serum pharmacodynamics biomarkers measured by levels of cortisol, ACTH, PINP, osteocalcin, CTX, 17-hydroxyprogesterone, testosterone, corticosterone, 11-deoxycortisol, glucose and 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 ]


Information By: ReveraGen BioPharma, Inc.

Dates:
Date Received: April 28, 2016
Date Started: August 2016
Date Completion: August 2017
Last Updated: April 5, 2017
Last Verified: April 2017