Becker Muscular Dystrophy
Conditions
Interventions
Product Name: vamorolone
Product Code: [vamorolone]
Pharmaceutical Form: Oral drops, powder for suspension
INN or Proposed INN: vamorolone
CAS Number: 13209-41-1
Current Sponsor code: N/A
Other descri
Sponsors
ReveraGen BioPharma Inc.
Eligibility
Sex/Gender
Male
Inclusion criteria
Inclusion criteria: 1.Subject has provided written informed consent and Health Insurance Portability and Accountability Act (HIPAA) authorization, where applicable, prior to any study-related procedures; 2. Subject is a male and has a confirmed diagnosis of Becker dystrophy as defined as: a. Identifiable mutation within the DMD gene (deletion/duplication of one or more exons), where reading frame can be predicted as 'in-frame', and clinical picture consistent with Becker dystrophy, OR b. Complete dystrophin gene sequencing showing an alteration (small mutation, duplication, other) that is expected to allow production of an internally deleted dystrophin protein, with a typical clinical picture of Becker dystrophy; 3. Subject is = 18 years of age and =65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: 1. Subject has current or history of major renal or hepatic impairment, diabetes mellitus or immunosuppression; 2. Subject has current or history of chronic systemic fungal or viral infections; 3. Subject has had an acute illness within 4 weeks prior to the first dose of study medication 4. Subject has used mineralocorticoid receptor agents, such as spironolactone, eplerenone, canrenone (canrenoate potassium), prorenone (prorenoate potassium), or mexrenone (mexrenoate potassium) within 4 weeks prior to administration of study medication; 5. Subject has evidence of symptomatic cardiomyopathy [Note: Asymptomatic cardiac abnormality on investigation would not be exclusionary unless cardiac ejection fraction is less than 40%]; 6. Subject has an allergy or hypersensitivity to the study medication or to any of its constituents; 7. Subject has severe behavioral or cognitive problems that preclude participation in the study, in the opinion of the Investigator; 8. 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; 9. Subject is taking (or has taken within 4 weeks prior to first dose of study medication) herbal remedies and supplements which can impact muscle strength and function (e.g., Co-enzyme Q10, creatine, etc); 10. Subject has been administered a live attenuated vaccine within 14 days prior to the first dose of study medication; 11. Subject is currently taking any other investigational drug or has taken any other investigational drug within 3 months prior to first dose of study medication; or 12. Subject has previously been enrolled in the VBP15-BMD-001 study or any other vamorolone study. Note: Any parameter/test may be repeated at the Investigator’s discretion during Screening to determine reproducibility. In addition, subjects may be rescreened if ineligible due to negative anti-varicella IgG antibody test result.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To evaluate the safety and tolerability of vamorolone 500mg (250mg if <50kg body weight) daily administered orally vs. placebo over a 24-week treatment period in subjects with Becker dystrophy.;Secondary Objective: - To evaluate the pharmacokinetics (PK) of vamorolone 500mg (250mg if <50kg body weight) daily administered orally in subjects with Becker dystrophy. - To evaluate the pharmacodynamic response using biomarkers bridged to clinical safety outcomes (adrenal suppression, bone turnover, and insulin resistance) and efficacy (CD23 and MDC) to vamorolone 500mg (250mg if <50kg body weight) daily administered orally vs. placebo in subjects with Becker dystrophy. ;Primary end point(s): Safety and tolerability of vamorolone : Clinical labs and AEs collection. ;Timepoint(s) of evaluation of this end point: Safety labs at Screening, day 1, Week4, week 12, and week 24. AEs will be collected from the date of informed consent and through the time of the subject’s last study visit. | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): 1) To evaluate the pharmacokinetics (PK) of vamorolone 2) To evaluate safety biomarkers (osteocalcin, hemoglobin A1c (HbA1c), insulin) 3) To evaluate efficacy biomarkers (CD23 and MDC) ;Timepoint(s) of evaluation of this end point: 1) Time point: day 1 2) Time point: Day 1 and Week 24 3) Time point: Day 1, Week 12 and Week 24 | — |
Countries
Italy, United States
Contacts
Public ContactJesse Damsker
ReveraGen BioPharma Inc.
Outcome results
None listed