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 : Completed
First Posted : January 31, 2017
Results First Posted : May 20, 2021
Last Update Posted : May 20, 2021
- Study Details
- Tabular View
- Study Results
- How to Read a Study Record
|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|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||46 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||None (Open Label)|
|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)|
|Actual Study Start Date :||February 2, 2017|
|Actual Primary Completion Date :||April 30, 2020|
|Actual Study Completion Date :||April 30, 2020|
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
- Number of Participants With Treatment-related Adverse Events as Assessed by CTCAE Version 4.03 [ Time Frame: 24 months ]To evaluate the long-term safety and tolerability of vamorolone, administered orally at daily doses up to 6.0 mg/kg/day over a 24- month Treatment Period, in boys ages 4-7 years with DMD; Treatment-emergent adverse events (TEAEs) are defined as any adverse event or worsening of an existing conditions after initiation of the investigational product and through the subject's last study visit (study completion or early termination);
- Total Number of Adverse Events as Assessed by CTCAE Version 4.03 [ Time Frame: 24 Months ]To evaluate the long-term safety and tolerability of vamorolone, administered orally at daily doses up to 6.0 mg/kg/day over a 24-month Treatment Period, in boys ages 4-7 years with DMD. Treatment-emergent adverse events (TEAEs) are defined as any adverse event or worsening of an existing conditions after initiation of the investigational product and through the subject's last study visit (study completion or early termination).
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|
- 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;
- 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
- Subject and parent/guardian are willing and able to comply with scheduled visits, study drug administration plan, and study procedures.
- 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;
- Subject has current or history of major renal or hepatic impairment, diabetes mellitus or immunosuppression;
- Subject has current or history of chronic systemic fungal or viral infections;
- 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;
- Subject has evidence of symptomatic cardiomyopathy. [Note: Asymptomatic cardiac abnormality on investigation would not be exclusionary];
- 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];
- Subject has used idebenone within 4 weeks prior to the first dose of study medication;
- Subject has an allergy or hypersensitivity to the study medication or to any of its constituents;
- Subject has severe behavioral or cognitive problems that preclude participation in the study, in the opinion of the Investigator;
- 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
- 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.
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
|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|
|Durham, North Carolina, United States, 27710|
|United States, Texas|
|University of Texas Southwestern Medical Center|
|Dallas, Texas, United States, 75207|
|Royal Children's Hospital|
|Sydney Children's Hospital|
|Alberta Children's Hospital|
|Calgary, Alberta, Canada, T3B 6A8|
|Schneider Children's Medical Center|
|Petah Tikwah, Israel, 49202|
|Queen Silvia Children's Hospital|
|Gothenburg, Sweden, 41685|
|Newcastle upon Tyne Hospitals NHS Foundation Trust|
|Newcastle upon Tyne, United Kingdom, NE7 7DN|
|Study Chair:||Paula R Clemens, MD||University of Pittsburgh|
Documents provided by ReveraGen BioPharma, Inc.:
Publications of Results:
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
|Responsible Party:||ReveraGen BioPharma, Inc.|
|Other Study ID Numbers:||
|First Posted:||January 31, 2017 Key Record Dates|
|Results First Posted:||May 20, 2021|
|Last Update Posted:||May 20, 2021|
|Last Verified:||April 2021|
|Studies a U.S. FDA-regulated Drug Product:||Yes|
|Studies a U.S. FDA-regulated Device Product:||No|
Duchenne muscular dystrophy
Muscular Dystrophy, Duchenne
Muscular Disorders, Atrophic
Nervous System Diseases
Genetic Diseases, Inborn
Genetic Diseases, X-Linked