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

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

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

Brief Summary: 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.

Detailed Summary: 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.
Sponsor: ReveraGen BioPharma, Inc.

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

Original Primary Outcome: Same as current

Current Secondary Outcome:

  • 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)


Original Secondary Outcome:

  • 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, ACTH, PINP, osteocalcin, CTX, 17-hydroxyprogesterone, testosterone, corticosterone, 11-deoxycortisol, glucose and insulin [ Time Frame: Week 2 ]
  • Serum metabolites of vamorolone [ Time Frame: Week 2 ]
    Metabolites in Safety Testing (MIST)


Information By: ReveraGen BioPharma, Inc.

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