Clinical Trials

Vamorolone has been studied in a series of clinical trials in both adult healthy volunteers (Phase I), and in DMD boys (Phase 2).

The Phase 1 studies were carried out in 86 adult volunteers in 2015. The volunteers received either a single dose of vamorolone (0.1 – 20.0 mg/kg) or 14 doses (2 weeks daily dosing; 1.0 – 20.0 mg/kg/day). These studies have been published and showed that vamorolone was safe to the highest doses tested (20.0 mg/kg/day; or about 10-15 times higher than typical corticosteroid doses in DMD). While clinical benefit of vamorolone could not be studied in healthy volunteers, there was evidence of lessened side effects based on blood biomarker studies.

Clinical trials in DMD boys were initiated in 2016 with a Phase 2a study (VBP15-002) in 48 boys (ages 4 to <7 years old). This study was carried out by the Cooperative International Neuromuscular Research Group (CINRG) – a clinical trial network that has also carried out the largest natural history studies of DMD. The Phase 2a study was a 4 week study, where vamorolone was given at one of 4 doses for 2 weeks (0.25, 0.75, 2.0 and 6.0 mg/kg/day), followed by a 2 week washout (no drug). The primary findings were that vamorolone seemed safe (consistent with adult volunteer studies), and that the pharmacokinetics were similar to traditional corticosteroids (good bioavailability with short half-life). The pharmacokinetics of the adult volunteers and DMD boys were also analyzed together to calculate population pharmacokinetics (PopPK), and this has also been published.

As the DMD boys completed the 4-week VBP15-002 study, they were enrolled into the VBP15-003 study (6-month extension), where they were maintained at the same dose as in the 4-week study (0.25 – 6.0 mg/mg/day). The 6-month treatment showed that higher doses of vamorolone (2.0 and 6.0 mg/kg/day) resulted in significant improvements in muscle function in DMD boys, and these studies were published in two papers:

The large majority (46 of 48) of the boys enrolled in the VBP15-002/003 studies, their families and their physicians expressed a desire to continue treatment with vamorolone after they had completed these initial clinical trials. A 2-year long-term extension study was then implemented (VBP15-LTE), and this study permitted the physician, in discussion with patient families, to alter the dose of vamorolone. All the patients on the two lower doses (0.25 and 0.75 mg/kg/day) were escalated to the two higher doses (2.0 and 6.0 mg/kg/day) with most choosing the highest dose (6.0 mg/kg/day).

All patients in the long-term extension have completed 18-months of vamorolone treatment (mid-point of LTE). Patients treated for 18 and 30-months appear to show continued improvement, while lacking the corticosteroid side effect of stunting of growth seen with deflazacort and prednisone. These results have been published in two papers:

In 2018 a pivotal (Phase 3-type) clinical trial initiated enrollment (VBP15-004). This trial enrolled DMD boys ages 4 to <7 years. Boys were randomized into one of four dose groups for 6 months (2.0 or 6.0 mg/kg/day vamorolone, prednisone [25%], or placebo [25%]), then all patients were treated with 2.0 or 6.0 mg/kg/day vamorolone for the next 6 months (total 1 year study). The placebo controlled part of this clinical trial (24-week Period 1 has been published:

This publication concluded, “In this pivotal randomized clinical trial, vamorolone was shown to be effective and safe in the treatment of boys with DMD over a 24-week treatment period. Vamorolone may be a safer alternative than prednisone in this disease, in which long-term corticosteroid use is the standard of care”

In 2022 a Phase 2 pilot study was initiated in Becker Muscular Dystrophy (NCT05166109). This trial is recruiting BMD participants ages 18-64 years. Subjects are randomized to one of two treatment groups in a 1:2 ratio (placebo:vamorolone) and will be on study for 6 months.

The majority of patients, families and their physicians involved in clinical trials with vamorolone have expressed desire to continue treatment rather than transition to corticosteroid standard of care. An Expanded Access Protocol is available in the USA, Canada, and Israel for patients who have completed clinical studies, and similar mechanisms have been implemented in other countries to enable continued access to vamorolone after exit from clinical trials.