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Trial record 16 of 1004 for:    BMD

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

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. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03038399
Recruitment Status : Active, not recruiting
First Posted : January 31, 2017
Last Update Posted : September 30, 2019
Sponsor:
Collaborators:
University of Pittsburgh
Cooperative International Neuromuscular Research Group
Information provided by (Responsible Party):
ReveraGen BioPharma, Inc.

Tracking Information
First Submitted Date  ICMJE January 30, 2017
First Posted Date  ICMJE January 31, 2017
Last Update Posted Date September 30, 2019
Actual Study Start Date  ICMJE February 2, 2017
Estimated Primary Completion Date April 2020   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: January 30, 2017)
  • 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 Measures  ICMJE Same as current
Change History Complete list of historical versions of study NCT03038399 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: January 30, 2017)
  • 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 Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Long-term Extension Study to Assess Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD)
Official Title  ICMJE 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 (DMD)
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 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 months.
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Duchenne Muscular Dystrophy
Intervention  ICMJE
  • Drug: Vamorolone 0.25 mg/day/day
    Oral administration of 0.25 mg/kg/day daily for 24 months.
    Other Name: VBP15
  • Drug: Vamorolone 0.75 mg/day/day
    Oral administration of 0.75 mg/kg/day daily for 24 months.
    Other Name: VBP15
  • Drug: Vamorolone 2.0 mg/day/day
    Oral administration of 2.0 mg/kg/day daily for 24 months.
    Other Name: VBP15
  • Drug: Vamorolone 6.0 mg/day/day
    Oral administration of 6.0 mg/kg/day daily for 24 months.
    Other Name: VBP15
Study Arms  ICMJE
  • Experimental: Dose Level Group 1
    Participants enrolled in Dose Level Group 1 will receive vamorolone 0.25 mg/kg/day.
    Intervention: Drug: Vamorolone 0.25 mg/day/day
  • Experimental: Dose Level Group 2
    Participants enrolled in Dose Level Group 2 will receive vamorolone 0.75 mg/kg/day.
    Intervention: Drug: Vamorolone 0.75 mg/day/day
  • Experimental: Dose Level Group 3
    Participants enrolled in Dose Level Group 3 will receive vamorolone 2.0 mg/kg/day.
    Intervention: Drug: Vamorolone 2.0 mg/day/day
  • Experimental: Dose Level Group 4
    Participants enrolled in Dose Level Group 4 will receive vamorolone 6.0 mg/kg/day.
    Intervention: Drug: Vamorolone 6.0 mg/day/day
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Active, not recruiting
Actual Enrollment  ICMJE
 (submitted: September 9, 2019)
46
Original Estimated Enrollment  ICMJE
 (submitted: January 30, 2017)
48
Estimated Study Completion Date  ICMJE April 2020
Estimated Primary Completion Date April 2020   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  1. Subject's parent or legal guardian has provided written informed consent and HIPAA authorization (if applicable) prior to any VBP15-LTE long-term extension study-specific procedures;
  2. Subject has previously completed study VBP15-003 up to and including the Week 24 Final assessments, prior to enrolling in the VBP15-LTE study at the conclusion of the VBP15-003 Week 24 Visit [Note: if entering the dose-tapering period, subject is enrolling within 8 weeks after the VBP15‑003 final visit following dose‑tapering]; and
  3. Subject and parent/guardian are willing and able to comply with scheduled visits, study drug administration plan, and study procedures.

Exclusion Criteria:

  1. Subject had a serious or severe adverse event in study VBP15-003 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 long-term extension study;
  2. Subject has current or history of major renal or hepatic impairment, diabetes mellitus or immunosuppression;
  3. Subject has current or history of chronic systemic fungal or viral infections;
  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 glucocorticoids 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 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: Subjects may be re-evaluated if ineligible due to a transient condition which would prevent the subject from participating.

Sex/Gender  ICMJE
Sexes Eligible for Study: Male
Ages  ICMJE 4 Years to 7 Years   (Child)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Australia,   Canada,   Israel,   Sweden,   United Kingdom,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03038399
Other Study ID Numbers  ICMJE VBP15-LTE
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE Not Provided
Responsible Party ReveraGen BioPharma, Inc.
Study Sponsor  ICMJE ReveraGen BioPharma, Inc.
Collaborators  ICMJE
  • University of Pittsburgh
  • Cooperative International Neuromuscular Research Group
Investigators  ICMJE
Study Chair: Paula R Clemens, MD University of Pittsburgh
PRS Account ReveraGen BioPharma, Inc.
Verification Date September 2019

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP