IIa extension: VBP15-003
Research type
Research Study
Full title
Amended Clinical Protocol #1 for a Phase II Open-label, Multicenter Extension Study to Assess the Long-term Safety and Efficacy of Vamorolone in Boys with Duchenne Muscular Dystrophy (DMD).
IRAS ID
218683
Contact name
Michela Guglieri
Contact email
Sponsor organisation
ReveraGen BioPharma, Inc.
Eudract number
2016-004263-38
Clinicaltrials.gov Identifier
Duration of Study in the UK
0 years, 10 months, 5 days
Research summary
Duchenne muscular dystrophy (DMD) is a rapidly progressive form of muscular dystrophy that occurs primarily in males and manifests prior to the age of six years. Although significant advances have been made in understanding the etiology of DMD, a cure has not been found, and current treatment options are often medications used “off-label” to alleviate the symptoms of DMD. Despite scientific advances, only glucocorticoids, such as prednisone, have consistently demonstrated efficacy in clinical trials.
Many disease modifying technologies that are currently in development focus on subsets of dystrophin mutations and therefore do not address the unmet need in all persons with DMD. Moreover developmental drugs are likely to be used in conjunction with glucocorticoids. Therefore glucocorticoids will be needed.
However, glucocorticoids have extensive side effect profiles, often limiting long-term administration. The cumulative side effects of glucocorticoids, including excess weight, delayed puberty, fragile skin, loss of bone mineral density, bruising, and Cushingoid appearance continue to negatively impact on the quality of life of the individual, leading to significant variations in clinical practice. Glucocorticoids also contribute to further muscle damage with long-term administration.
Vamorolone has shown few if any of the side effects of traditional glucocorticoids in mouse models of DMD.
This Phase IIa 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 weeks to boys ages 4-7 years with DMD.
Vamorolone 4% oral suspension is administered once daily over a 14-day Treatment Period (total of 14 doses) at the following planned dose levels:
0.25 mg/kg/day
0.75 mg/kg/day
2.0 mg/kg/day
or 6.0 mg/kg/day.Subjects will begin dosing in this study on Study Day 1 at the same vamorolone dose level they received in the Phase IIa core study. Subjects will continue to receive vamorolone at the dose received in the Phase IIa core study for the duration of the 24-week Treatment Period, unless new safety data indicate the dose level should be de-escalated.
REC name
North East - York Research Ethics Committee
REC reference
16/NE/0387
Date of REC Opinion
28 Dec 2016
REC opinion
Further Information Favourable Opinion