Eliglustat hemitartrate Clinical Trials

Size	Price	Qty
5mg	USD 159
25mg	USD 477

Clinical Trials

Eliglustat hemitartrate has been evaluated in a clinical trial to assess its safety and therapeutic efficacy in patients diagnosed with Gaucher Disease Type 1. Sponsored by Genzyme, a Sanofi company, this pivotal Phase 3 study has officially completed recruitment. Overall, these findings highlight the late-stage clinical evaluation of this substrate reduction therapy for lysosomal storage pathology.

NCT Number	Recruitment	Conditions	Sponsor/Collaborators	Start Date	Phases
NCT00891202	Completed	Gaucher Disease Type 1	Genzyme a Sanofi Company\|Sanofi	2009-11	Phase 3

(data from https://clinicaltrials.gov, updated on 2024-05-22)

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

Compliance for Clinical Use

Mechanism and Biochemical Profile

Eliglustat hemitartrate acts as a potent and specific inhibitor of glucosylceramide synthase with an IC50 of 24 nM, blocking the rate-limiting step in glycosphingolipid biosynthesis and preventing intracellular glucosylceramide accumulation. This reduction in substrate accumulation mitigates cellular storage pathology, providing therapeutic benefit in clinical conditions such as Gaucher Disease Type 1.

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>).