Date: 2012-10-02
Type of information: Results
phase: 3
Announcement: results
Company: Genzyme (USA - MA), a Sanofi company (France)
Product: eliglustat tartrate
Action
mechanism: specific inhibitor of glucosylceramide synthase.
Disease: Gaucher disease type 1
Therapeutic area: Rare diseases - Genetic diseases
Country: North America, South America, Europe, Asia and the Middle East
Trial
details: ENGAGE is a randomized, double-blind, placebo controlled study in treatment-naïve patients with Gaucher disease type 1 and evaluated the efficacy, safety and pharmacokinetics of twice-daily dosing of eliglustat tartrate in 40 patients untreated for at least six months. The study had a primary efficacy endpoint of improvement in spleen size in individual patients treated with eliglustat tartrate compared with treatment with placebo, after the nine month study period. Patients were stratified at baseline by their spleen volume. Thirty-nine out of 40 study participants completed at least nine months of treatment. One patient in the eliglustat treated group discontinued at six months for personal reasons. At the end of nine months, patients who were on placebo were transitioned to eliglustat tartrate. After the primary analysis period concluded, all 39 patients chose to remain on treatment. Eighteen medical centers in 12 countries in North America, South America, Europe, Asia and the Middle East are participating in this study.
Latest
news: Genzyme, a Sanofi company, has announced that ENGAGE, the first Phase 3 trial of its investigational oral therapy, eliglustat tartrate, in previously untreated patients with Gaucher disease type 1, met its primary endpoint. Patients treated with eliglustat tartrate had a statistically significant improvement in spleen size at nine months, compared with placebo.
Spleen volumes in eliglustat tartrate treated patients decreased from baseline by a mean of 28 percent versus a mean increase of two percent in placebo patients, for an absolute difference of 30 percent (p<0.0001). In addition, all secondary endpoints were met, including improvements in hemoglobin levels and platelet levels, as well as liver volumes compared with placebo-treated individuals.
The initial safety analysis from ENGAGE suggests that eliglustat tartrate was well tolerated. There were no serious adverse events reported in the primary analysis period and no clinically meaningful differences in the related adverse events reported between the two treatment groups.
Full results from the ENGAGE study are planned for presentation at the Lysosomal Disease Network WORLD meeting, February 12-15, 2013, in Orlando, Fla. Top-line data from Genzyme’s second Phase 3 trial, ENCORE, are expected in early 2013.
Genzyme previously reported that the 12-month Phase 2 trial had met its primary composite endpoint: a clinically meaningful response in at least two of three endpoints (improvements in spleen size, hemoglobin and platelet levels) in individual patients. All patients in the ongoing Phase 2 trial have been on treatment for at least five years.
Genzyme is currently completing the second Phase 3 study, ENCORE.
