NCT00891202

A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multi-Center Study Confirming the Efficacy and Safety of Genz-112638 in Patients With Gaucher Disease Type 1 (ENGAGE)

Completed Phase 3 Results posted Last updated 17 January 2017

What this trial tests

Phase 3 trial testing Eliglustat tartrate in Gaucher Disease, Type 1 in 40 participants. Completed in 1 January 2016.

Timeline

1 November 2009

Primary endpoint
1 July 2012

1 January 2016

Quick facts

Lead sponsor	Genzyme, a Sanofi Company
Phase	Phase 3
Status	Completed
Study type	INTERVENTIONAL
Allocation	randomized
Design	parallel
Masking	quadruple
Primary purpose	treatment
Enrollment	40
Start date	1 November 2009
Primary completion	1 July 2012
Estimated completion	1 January 2016
Sites	18 locations across United States, Bulgaria, Canada, Colombia, India, Israel, Lebanon, Mexico

Drugs / interventions tested

• Eliglustat tartrate — full drug profile →
• Placebo

Conditions studied

• Gaucher Disease, Type 1 — all drugs for Gaucher Disease, Type 1 →

Sponsor

Genzyme, a Sanofi Company — full company profile →

Who can join

16 and older, any sex, with Gaucher Disease, Type 1. Patients with the condition only — healthy volunteers not accepted.

What's being measured

Primary outcomes are the specific endpoints the trial is designed to prove or disprove.

• PAP: Percent Change From Baseline in Spleen Volume (in Multiples of Normal [MN]) at Week 39 of the Primary Analysis Period With Eliglustat Tartrate Treatment as Compared to Placebo
  Time frame: PAP Baseline (Day 1), Week 39
  Percent change in spleen volume = (\[spleen volume at Week 39 minus spleen volume at baseline\] divided by \[spleen volume at baseline\]) multiplied by 100, where all volumes are in MN.

Sponsor's own description

This Phase 3 study was designed to confirm the efficacy and safety of eliglustat tartrate (Genz-112638) in participants with Gaucher disease Type 1.

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

1. Effect of oral eliglustat on splenomegaly in patients with Gaucher disease type 1: the ENGAGE randomized clinical trial.
   Mistry PK, Lukina E, Ben Turkia H, Amato D, et al · · 2015 · cited 113× · PMID 25688781 · DOI 10.1001/jama.2015.459
2. Outcomes after 18 months of eliglustat therapy in treatment-naïve adults with Gaucher disease type 1: The phase 3 ENGAGE trial.
   Mistry PK, Lukina E, Ben Turkia H, Shankar SP, et al · · 2017 · cited 69× · PMID 28762527 · DOI 10.1002/ajh.24877
3. Eliglustat tartrate for the treatment of adults with type 1 Gaucher disease.
   Bennett LL, Turcotte K. · · 2015 · cited 34× · PMID 26345314 · DOI 10.2147/dddt.s77760
4. Clinical outcomes after 4.5 years of eliglustat therapy for Gaucher disease type 1: Phase 3 ENGAGE trial final results.
   Mistry PK, Lukina E, Ben Turkia H, Shankar SP, et al · · 2021 · cited 32× · PMID 34161616 · DOI 10.1002/ajh.26276
5. Long-term adverse event profile from four completed trials of oral eliglustat in adults with Gaucher disease type 1.
   Peterschmitt MJ, Freisens S, Underhill LH, Foster MC, et al · · 2019 · cited 32× · PMID 31174576 · DOI 10.1186/s13023-019-1085-6
6. Glucosylsphingosine (Lyso-Gb1) as a reliable biomarker in Gaucher disease: a narrative review.
   Giuffrida G, Markovic U, Condorelli A, Calafiore V, et al · · 2023 · cited 30× · PMID 36782327 · DOI 10.1186/s13023-023-02623-7
7. The design and clinical development of inhibitors of glycosphingolipid synthesis: will invention be the mother of necessity?
   Shayman JA. · · 2013 · cited 25× · PMID 23874009
8. Clinical response to eliglustat in treatment-naïve patients with Gaucher disease type 1: Post-hoc comparison to imiglucerase-treated patients enrolled in the International Collaborative Gaucher Group Gaucher Registry.
   Ibrahim J, Underhill LH, Taylor JS, Angell J, et al · · 2016 · cited 11× · PMID 27408819 · DOI 10.1016/j.ymgmr.2016.06.003