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

Study Status: Enrolling by invitation
Recruit Status: Enrolling by invitation
Study Type: Interventional

Official Title: A 24-month Phase II Open-label, Multicenter Long-term Extension Study to Assess the Long‑Term Safety and Efficacy of Vamorolone in Boys With Duchenne Muscular Dystrophy

Brief Summary: This long-term extension study is an open-label, multiple‑dose study to evaluate the long‑term safety, tolerability, efficacy and PD of vamorolone administered once daily by liquid oral suspension over a Treatment Period of 24 months to young boys with DMD who participated in the VBP15‑002 Phase IIa and VBP15-003 Phase IIa extension core studies.

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 months.
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 months ]
  • 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 months ]


Original Primary Outcome: Same as current

Current Secondary Outcome:

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


Original Secondary Outcome: Same as current

Information By: ReveraGen BioPharma, Inc.

Dates:
Date Received: January 30, 2017
Date Started: January 2017
Date Completion: January 2021
Last Updated: April 27, 2017
Last Verified: April 2017