NCT01074944

A Phase 3, Randomized, Multi-Center, Multi-National, Double-Blind Study to Evaluate the Efficacy, Safety and Pharmacokinetics of Once Daily Versus Twice Daily Dosing of Genz-112638 in Patients With Gaucher Disease Type 1 Who Have Demonstrated Clinical Stability on a Twice Daily Dose of Genz-112638

Completed Phase 3 Results posted Last updated 12 December 2016

What this trial tests

Phase 3 trial testing Eliglustat tartrate in Gaucher Disease in 170 participants. Completed in 1 October 2015.

Timeline

1 June 2010

Primary endpoint
1 October 2015

1 October 2015

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	170
Start date	1 June 2010
Primary completion	1 October 2015
Estimated completion	1 October 2015
Sites	46 locations across United States, Australia, Austria, Brazil, Canada, China, Croatia, France

Drugs / interventions tested

• Eliglustat tartrate — full drug profile →

Conditions studied

• Gaucher Disease — all drugs for Gaucher Disease →

Sponsor

Genzyme, a Sanofi Company — full company profile →

Who can join

18 and older, any sex, with Gaucher Disease. 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: Percentage of Participants Who Remained Stable for 52 Weeks During the PAP
  Time frame: PAP Baseline up to the end of PAP (Week 52)
  Participants were considered as stable if they met all of the following criteria: 1) no more than 2 bone crisis during PAP (with no more than 1 bone crisis during either the first 6 months or the later 6 months of the period), and were free of other clinically symptomatic bone disease during the entire 52-week PAP; 2) hemoglobin level not decreased \>1.5 g/dL from Baseline for PAP; 3) platelet cou

Sponsor's own description

The primary objective of this study was to evaluate the efficacy and safety of once daily (QD) versus twice daily (BID) dosing of eliglustat tartrate (Genz-112638) in participants with Gaucher disease type 1 who had demonstrated clinical stability on BID dosing of eliglustat tartrate (Genz-112638). The secondary objective was to evaluate the pharmacokinetics (PK) of Genz-99067 when eliglustat tartrate (Genz-112638) was administered QD and BID in participants with Gaucher disease type 1 who had demonstrated clinical stability on BID dosing of eliglustat tartrate (Genz-112638).

Publications & conference data

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

1. 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
2. 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
3. The design and clinical development of inhibitors of glycosphingolipid synthesis: will invention be the mother of necessity?
   Shayman JA. · · 2013 · cited 25× · PMID 23874009
4. Once- versus twice-daily dosing of eliglustat in adults with Gaucher disease type 1: The Phase 3, randomized, double-blind EDGE trial.
   Charrow J, Fraga C, Gu X, Ida H, et al · · 2018 · cited 20× · PMID 29358012 · DOI 10.1016/j.ymgme.2017.12.001
5. 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
6. Rare disease patients in India are rarely involved in international orphan drug trials.
   Chakraborty M, Choudhury MC, Chakraborty I, Saberwal G. · · 2022 · cited 9× · PMID 36962798 · DOI 10.1371/journal.pgph.0000890
7. Oral Presentations
   · 2018