VISION DMD
Research type
Research Study
Full title
A Phase IIb Randomized, Double-blind, Parallel Group, Placebo- and Active-controlled Study with Double-Blind Extension to Assess the Efficacy and Safety of Vamorolone in Ambulant Boys with Duchenne Muscular Dystrophy (DMD)
IRAS ID
244048
Contact name
Michela Guglieri
Contact email
Sponsor organisation
ReveraGen BioPharma, Inc.
Eudract number
2017-002704-27
Clinicaltrials.gov Identifier
Clinicaltrials.gov Identifier
118942, FDA IND Number
Duration of Study in the UK
3 years, 0 months, 0 days
Research summary
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.
Vamorolone, 1.33% and 4.0% wt/wt suspension for oral dosing will be administered at daily doses of 2.0 mg/kg and 6.0 mg/kg versus prednisone 0.75 mg/kg/day and placebo over a Treatment Period of 24 weeks, and to evaluate persistence of effect over a Treatment Period of 48 weeks in ambulant boys ages 4 to <7 years with DMD.
At the end subjects will be given the option of enrolling into a long-term extension study or to transition to standard of care treatment for DMD (which may include glucocorticoids).Summary of Research
: Vamorolone was effective in the treatment of steroid naïve ambulant boys between 4 to <7 years of age with DMD.
Clinically meaningful, statistically significant, and robust improvements in multiple, independent measurements of lower limb motor function were seen with vamorolone 2 and 6 mg/kg compared with placebo. The improvements in motor function with vamorolone were similar to those seen with prednisone at Week 24.
No clinically meaningful differences were seen in efficacy at Week 24 for vamorolone 2 mg/kg compared with 6 mg/kg.
The effects seen with vamorolone 6 mg/kg at Week 24 were maintained at Week 48 whereas slight declines were seen from Week 24 to Week 48 with vamorolone 2 mg/kg.
No loss of effectiveness was seen when subjects were switched from prednisone to vamorolone 6 mg/kg, whereas small declines were seen after the switch to vamorolone 2 mg/kg.
Greater improvements in lower limb function were seen for the prednisone and deflazacort groups from the FOR-DMD study compared with vamorolone 2 mg/kg at Week 48. No statistically significant differences were seen for TTSTAND velocity or 6MWT distance for vamorolone 6 mg/kg compared with the prednisone and deflazacort groups from the FOR-DMD study at 24 and 48 weeks of treatment.
REC name
North East - York Research Ethics Committee
REC reference
18/NE/0235
Date of REC Opinion
21 Sep 2018
REC opinion
Further Information Favourable Opinion