NCT00943111: ENCORE

A Phase 3, Randomized, Multi-Center, Multi-National, Open-Label, Active Comparator Study to Evaluate the Efficacy and Safety of Genz-112638 in Patients With Gaucher Disease Type 1 Who Have Reached Therapeutic Goals With Enzyme Replacement Therapy (ENCORE)

Completed Phase 3 Results posted Last updated 11 October 2016

What this trial tests

Phase 3 trial testing Eliglustat tartrate in Gaucher Disease, Type 1 in 160 participants. Completed in 1 June 2015.

Timeline

1 September 2009

Primary endpoint
1 November 2012

1 June 2015

Quick facts

Lead sponsor	Genzyme, a Sanofi Company
Phase	Phase 3
Status	Completed
Study type	INTERVENTIONAL
Allocation	randomized
Design	parallel
Masking	none
Primary purpose	treatment
Enrollment	160
Start date	1 September 2009
Primary completion	1 November 2012
Estimated completion	1 June 2015
Sites	34 locations across United States, Argentina, Australia, Brazil, Canada, Egypt, France, Germany

Drugs / interventions tested

• Eliglustat tartrate — full drug profile →
• Imiglucerase (IMIGLUCERASE) — full drug profile →

Conditions studied

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

Sponsor

Genzyme, a Sanofi Company — full company profile →

Who can join

18 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.

• Percentage of Participants Who Remained Stable for 52 Weeks During the Primary Analysis Period
  Time frame: Baseline up to Week 52
  For a participant to be classified as stable, the participant must have remained stable in hematological parameters (hemoglobin levels and platelet counts) and organ volumes (spleen, when applicable, and liver volumes in multiples of normal \[MN\]). Stable hematological parameters were defined as hemoglobin level did not decrease more than (\>) 1.5 gram per deciliter (g/dL) from baseline and plate
• Percentage of Participants Who Remained Stable Annually for 4 Years During the LTTP
  Time frame: Week 52 up to week 208
  For a participant to be classified as stable, the participant must have remained stable in hematological parameters (hemoglobin levels and platelet counts) and organ volumes (spleen, when applicable, and liver volumes in MN). Stable hematological parameters were defined as hemoglobin level did not decrease \>1.5 g/dL from baseline and platelet count did not decrease \>25% from baseline. Stable org

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 who had reached therapeutic goals with enzyme replacement therapy (ERT).

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. Eliglustat compared with imiglucerase in patients with Gaucher's disease type 1 stabilised on enzyme replacement therapy: a phase 3, randomised, open-label, non-inferiority trial.
   Cox TM, Drelichman G, Cravo R, Balwani M, et al · · 2015 · cited 109× · PMID 25819691 · DOI 10.1016/s0140-6736(14)61841-9
3. Eliglustat maintains long-term clinical stability in patients with Gaucher disease type 1 stabilized on enzyme therapy.
   Cox TM, Drelichman G, Cravo R, Balwani M, et al · · 2017 · cited 54× · PMID 28167660 · DOI 10.1182/blood-2016-12-758409
4. 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
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. The design and clinical development of inhibitors of glycosphingolipid synthesis: will invention be the mother of necessity?
   Shayman JA. · · 2013 · cited 25× · PMID 23874009
7. Pregnancy outcome in women with Gaucher disease type 1 who had unplanned pregnancies during eliglustat clinical trials.
   Lukina E, Balwani M, Belmatoug N, Watman N, et al · · 2021 · cited 10× · PMID 33473343 · DOI 10.1002/jmd2.12172
8. Stability is maintained in adults with Gaucher disease type 1 switched from velaglucerase alfa to eliglustat or imiglucerase: A sub-analysis of the eliglustat ENCORE trial.
   Pleat R, Cox TM, Burrow TA, Giraldo P, et al · · 2016 · cited 10× · PMID 27722092 · DOI 10.1016/j.ymgmr.2016.08.009