ClinicalTrials.gov
ClinicalTrials.gov Menu
Trial record 5 of 372 for:    "Muscular Dystrophies"

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 : Enrolling by invitation
First Posted : January 31, 2017
Last Update Posted : August 24, 2017
Sponsor:
Collaborators:
University of Pittsburgh
Cooperative International Neuromuscular Research Group
Information provided by (Responsible Party):
ReveraGen BioPharma, Inc.

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.

Condition or disease Intervention/treatment 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

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 : Interventional  (Clinical Trial)
Estimated Enrollment : 48 participants
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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 (DMD)
Study Start Date : January 2017
Estimated Primary Completion Date : January 2021
Estimated Study Completion Date : January 2021


Arm Intervention/treatment
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 months.
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 months.
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 months.
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 months.
Other Name: VBP15




Primary Outcome Measures :
  1. Number of participants with treatment-related adverse events as assessed by CTCAE Version 4.03 [ Time Frame: 24 months ]
  2. Muscle function measured by Time to Stand Test (TTSTAND) [ Time Frame: 24 weeks ]
  3. Body size as measured by body mass index (BMI) z-score [ Time Frame: 24 months ]

Secondary Outcome Measures :
  1. Serum pharmacodynamics biomarkers measured by levels of cortisol [ Time Frame: 24 months ]
  2. Serum pharmacodynamics biomarkers measured by levels of ACTH [ Time Frame: 24 months ]
  3. Serum pharmacodynamics biomarkers measured by levels of PINP [ Time Frame: 24 months ]
  4. Serum pharmacodynamics biomarkers measured by levels of osteocalcin [ Time Frame: 24 months ]
  5. Serum pharmacodynamics biomarkers measured by levels of CTX [ Time Frame: 24 months ]
  6. Serum pharmacodynamics biomarkers measured by levels of 17- hydroxyprogesterone [ Time Frame: 24 months ]
  7. Serum pharmacodynamics biomarkers measured by levels of testosterone [ Time Frame: 24 months ]
  8. Serum pharmacodynamics biomarkers measured by levels of corticosterone [ Time Frame: 24 months ]
  9. Serum pharmacodynamics biomarkers measured by levels of 11-deoxycortisol [ Time Frame: 24 months ]
  10. Serum pharmacodynamics biomarkers measured by levels of glucose [ Time Frame: 24 months ]
  11. Serum pharmacodynamics biomarkers measured by levels of insulin [ Time Frame: 24 months ]
  12. Muscle strength measured by Quantitative Muscle Testing (QMT) [ Time Frame: 24 months ]
  13. Muscle function measured by Time to Climb Test (TTCLIMB) [ Time Frame: 24 months ]
  14. Muscle function measured by Time to Run/Walk 10 Meters Test (TTRW) [ Time Frame: 24 months ]
  15. Muscle function measured by North Star Ambulatory Assessment (NSAA) [ Time Frame: 24 months ]
  16. Muscle function measured by Six-minute Walk Test (6MWT) [ Time Frame: 24 months ]


Information from the National Library of Medicine

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, Learn About Clinical Studies.


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

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.


Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03038399


Locations
United States, California
University of California Davis
Davis, California, United States, 95616
United States, Florida
University of Florida
Gainesville, Florida, United States, 32611
Nemours Children's Hospital
Orlando, Florida, United States, 32827
United States, Illinois
Ann & Robert H. Lurie Children's Hospital
Chicago, Illinois, United States, 60611
United States, North Carolina
Duke University
Durham, North Carolina, United States, 27710
United States, Texas
University of Texas Southwestern Medical Center
Dallas, Texas, United States, 75207
Australia
Royal Children's Hospital
Melbourne, Australia
Sydney Children's Hospital
Westmead, Australia
Canada, Alberta
Alberta Children's Hospital
Calgary, Alberta, Canada, T3B 6A8
Israel
Schneider Children's Medical Center
Petah Tikwah, Israel, 49202
Sweden
Queen Silvia Children's Hospital
Gothenburg, Sweden, 41685
United Kingdom
Newcastle upon Tyne Hospitals NHS Foundation Trust
Newcastle upon Tyne, United Kingdom, NE7 7DN
Sponsors and Collaborators
ReveraGen BioPharma, Inc.
University of Pittsburgh
Cooperative International Neuromuscular Research Group
Investigators
Study Chair: Paula R Clemens, MD University of Pittsburgh

Responsible Party: ReveraGen BioPharma, Inc.
ClinicalTrials.gov Identifier: NCT03038399     History of Changes
Other Study ID Numbers: VBP15-LTE
First Posted: January 31, 2017    Key Record Dates
Last Update Posted: August 24, 2017
Last Verified: August 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