Researchers are seeking volunteers to participate in a clinical trial sponsored by ReveraGen BioPharma to test the safety and effectiveness of the investigational drug vamorolone in boys with Duchenne muscular dystrophy (DMD).
In the two-part, open-label study called “A Phase 2a and 2a Extension study to Assess Vamorolone in Boys with Duchenne Muscular Dystrophy,” all participants will receive treatment with vamorolone via an oral sweetened suspension (syrup). Study investigators will assess whether the drug has positive effects on muscle strength and timed function tests.
A steroid without the side effects
Vamorolone, a “dissociative steroid,” is an anti-inflammatory compound that researchers hope will convey the same benefits of traditional glucocorticoids, such as prednisone and deflazacort, without those drugs’ unwanted side effects — including stunted growth, insulin resistance and weight gain. In clinical trials, glucocorticoid steroids have been shown to benefit DMD patients, but many find the side effects to be burdensome or intolerable.
These first in-patient studies of vamorolone follow the successful completion of a phase 1 trial to test the drug’s safety and tolerability in healthy adult volunteers.
Two MDA Venture Philanthropy (MVP) grants totaling $1.9 million to ReveraGen supported preclinical development and validation of the drug, and another MVP grant for $1,015,200 supported the phase 1 clinical trial to evaluate safety and tolerability in healthy adult volunteers. MVP is the drug development arm that operates within MDA’s Translational Research Program.
Additionally, through its basic research program, MDA is supporting development of biomarkers for glucocorticoids including vamorolone with a grant to Yetrib Hathout for $300,000, and research by Eric Hoffman to understand the effects of glucocorticoids including vamorolone on muscle repair with a grant for $321,659.
Boys ages 4 to 6 years old with a confirmed genetic diagnosis of DMD and who meet additional criteria may be eligible to participate.
This study is recruiting at sites across the United States and Canada.
- The first part of the study will last approximately 28 days and will include six in-person visits and one telephone contact.
- The second (extension) part of the study will last six to seven months and include eight to nine in-person study visits.
Each clinic visit will include timed function tests and blood draws. Throughout the study, participants will continue to see their regular doctor for routine care.
Travel and hotel accommodations will be provided for those who must travel to a test site for clinic visits. For additional information on travel and accommodations, contact Suzanne Gaglianone at firstname.lastname@example.org.
To learn more or to inquire about participation, contact Andrea Smith at email@example.com. You can also find out more information on the study by visiting ClinicalTrials.gov and searching for “vamorolone” in the search box or on ReveraGen BioPharma’s website.