IIa: VBP15-002
Research type
Research Study
Full title
VBP15-002 A Phase IIa Open-Label, Multiple Ascending Dose Study to Assess the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Exploratory Efficacy of Vamorolone in Boys with Duchenne Muscular Dystrophy (DMD)
IRAS ID
217871
Contact name
Michela Guglieri
Contact email
Sponsor organisation
ReveraGen BioPharma, Inc.
Eudract number
2016-004462-26
Clinicaltrials.gov Identifier
Duration of Study in the UK
0 years, 6 months, 16 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 study is an open-label, multiple ascending dose study to evaluate the safety, tolerability, PK, PD, and exploratory clinical efficacy of oral vamorolone over the course of a 14-day Treatment Period in ambulant 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.The study is comprised of a 26-day Pre-treatment Screening Period, a 1-day Pre-treatment Baseline Period, a 14-day Treatment Period, and a 14-day Post-treatment Follow-up Period. This study will help to select a dose for the phase 2b study and will explore whether vamorolone will show at least equal efficacy to glucocorticoids with a more favorable side effect profile, thereby improving the quality of life for DMD patients. This profile would enable use of vamorolone in DMD boys
Subjects who complete the 14-day Follow-up Period will be given the option of continuing vamorolone treatment in an extension study under a separate protocol (VBP15-003).
REC name
North East - York Research Ethics Committee
REC reference
16/NE/0386
Date of REC Opinion
28 Dec 2016
REC opinion
Further Information Favourable Opinion