Clinical Trials

Multiple clinical trials evaluate the efficacy, safety, and pharmacokinetics of eliglustat across stages from Early Phase 1 through Phase 3, primarily focusing on type 1 non-neuronopathic Gaucher disease alongside exploratory investigations in healthy volunteers, neuroblastoma, colorectal cancer, solid tumors, and hematologic malignancies. Sponsoring organizations include industry partners such as Genzyme and Sanofi, as well as academic institutions like Peking Union Medical College Hospital and Chinese PLA General Hospital. Portfolio recruitment statuses encompass completed, actively recruiting, not yet recruiting, and unknown protocols.

NCT Number Recruitment Conditions Sponsor/Collaborators Start Date Phases
NCT07138547 RECRUITING
Lymphoma
Chinese PLA General Hospital
 2025-12-26 PHASE1; PHASE2
NCT06558773 RECRUITING
Colorectal Cancer
Chinese PLA General Hospital
 2026-03-01 PHASE2
NCT07090538 NOT_YET_RECRUITING
Neuroblastoma
Chinese PLA General Hospital
 2025-08-01 EARLY_PHASE1
NCT03485677 COMPLETED
Gaucher's Disease Type I; Gaucher's Disease Type III
Sanofi
 2018-04-11 PHASE3
NCT06523517 UNKNOWN
Gaucher Disease
Peking Union Medical College Hospital
 2024-08-01 PHASE2
NCT04944888 COMPLETED
Solid Tumor; Hematologic Malignancy
Chinese PLA General Hospital
 2021-07-01 PHASE1
NCT02536755 COMPLETED
Gaucher Disease
Genzyme, a Sanofi Company
 2015-10-27 PHASE3
NCT03519646 COMPLETED
Gaucher Disease, Type III
National Taiwan University Hospital
 2018-04-23
NCT06188325 COMPLETED
Gaucher's Disease
Sanofi
 2018-01-01 PHASE1
NCT02536937 COMPLETED
Gaucher Disease
Sanofi
 2015-09 PHASE1
NCT02536911 COMPLETED
Gaucher Disease
Genzyme, a Sanofi Company
 2015-09 PHASE1
NCT01074944 COMPLETED
Gaucher Disease
Genzyme, a Sanofi Company
 2010-06 PHASE3
NCT02422654 COMPLETED
Gaucher Disease
Genzyme, a Sanofi Company
 2015-04 PHASE1
NCT06193304 COMPLETED
Gaucher's Disease
Sanofi
 2014-08-25 PHASE1
NCT00943111 COMPLETED
Gaucher Disease, Type 1
Genzyme, a Sanofi Company
 2009-09 PHASE3
NCT00891202 COMPLETED
Gaucher Disease, Type 1
Genzyme, a Sanofi Company
 2009-11 PHASE3
NCT01659944 COMPLETED
Healthy
Genzyme, a Sanofi Company
 2012-05 PHASE1
NCT01357811 COMPLETED
Healthy Volunteer
Genzyme, a Sanofi Company
 2011-08 PHASE1
NCT01452542 COMPLETED
Healthy Volunteer
Genzyme, a Sanofi Company
 2011-10 PHASE1
NCT00891202 Completed
Gaucher Disease Type 1
Genzyme a Sanofi Company|Sanofi
 2009-11 Phase 3
NCT00358150 COMPLETED
Gaucher Disease, Type 1; Cerebroside Lipidosis Syndrome; Glucocerebrosidase Deficiency Disease; Glucosylceramide Beta-Glucosidase Deficiency Disease; Gaucher Disease, Non-Neuronopathic Form
Genzyme, a Sanofi Company
 2006-06 PHASE2

(data from https://clinicaltrials.gov, updated on 2026-01-02)

Check the Eliglustat product page for in-depth specifications, including solubility, stock solutions, MOA, and working concentrations.

Compliance for Clinical Use

Mechanism and Biochemical Profile

Eliglustat potently inhibits glucosylceramide synthase (IC50 = 20 nM in intact MDCK cells), blocking the initial committed step in glycosphingolipid biosynthesis to reduce cellular accumulation and lysosomal influx of glucosylceramide. This substrate reduction mechanism normalizes glycosphingolipid homeostasis, directly addressing the underlying pathology of Gaucher disease type 1 and enabling exploratory targeted strategies in solid tumors like neuroblastoma.

Appendix RUO and cGMP Quality Standards

Quality Dimension RUO (Research Use Only) cGMP (Current Good Manufacturing Practice)
Clinical Applicability Prohibited in human clinical trials or medical diagnostics. Mandatory for human clinical trials (Phase I–III) and therapies.
Regulatory Status Non-regulated grade; exempt from drug manufacturing laws. Legally enforced by health authorities (e.g., FDA, EMA, NMPA).
Facility Environment Unclassified analytical or research laboratories. Validated Cleanrooms (ISO Class 5–8) with continuous monitoring.
Quality Control Basic purity and activity testing. Rigorous safety release testing (Sterility, Endotoxin, Mycoplasma).
Process Validation Basic equipment calibration; no process validation required. Full qualification (IQ/OQ/PQ) and complete batch records.
Quality Assurance Vendor self-declared without required formal QMS. Mandatory QA/QC unit, Change Control, CAPA, and vendor audits.
Regulatory Impact High risk of IND rejection if used as a critical raw material. Required for IND/NDA filings, supported by Drug Master Files (DMF).

Footnotes

Regulatory Note: Governed by FDA (21 CFR Parts 210/211/312), EMA (EudraLex Vol 4), ICH Guidelines (E6/Q7/Q9/Q10), and compendial standards (USP <71>/<85>/<1043>).

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Note: Technical data last updated: Sep 1, 2026.