Lomitapide Mesylate Clinical Trials

Size	Price	Qty
10mg	USD 147
50mg	USD 377

Clinical Trials

Numerous clinical trials across early to late phases evaluate lomitapide mesylate for severe lipid disorders, such as homozygous familial hypercholesterolemia (HoFH) and dyslipidemia, while assessing pharmacokinetics, intra-subject variability, and drug interactions in healthy volunteers. Sponsored by entities including Aegerion Pharmaceuticals, Amryt Pharma, and Fondazione SISA, these studies encompass completed, active not recruiting, terminated, and withdrawn statuses. Together, these clinical trials characterize the compound's clinical efficacy, drug-interaction profile, and safety in severe hypercholesterolemia.

NCT Number	Recruitment	Conditions	Sponsor/Collaborators	Start Date	Phases
NCT04681170	COMPLETED	Homozygous Familial Hypercholesterolaemia (HoFH)	Amryt Pharma	2020-12-14	PHASE3
NCT02765841	WITHDRAWN	Homozygous Familial Hypercholesterolemia	Aegerion Pharmaceuticals, Inc.	2016-05	PHASE3
NCT02173158	COMPLETED	Familial Hypercholesterolemia - Homozygous	Aegerion Pharmaceuticals, Inc.	2014-04-02	PHASE3
NCT02080468	COMPLETED	Healthy	Aegerion Pharmaceuticals, Inc.	2014-02-19	PHASE1
NCT02080455	COMPLETED	Effect of Atorvastatin on the Pharmacokinetics of Lomitapide	Aegerion Pharmaceuticals, Inc.	2014-01-27	PHASE1
NCT02044419	COMPLETED	Healthy	Aegerion Pharmaceuticals, Inc.	2013-10-30	PHASE1
NCT01915771	COMPLETED	Intra-subject Variability of Pharmacokinetics	Aegerion Pharmaceuticals, Inc.	2013-07-29	PHASE1
NCT01760187	COMPLETED	Healthy Volunteer	Aegerion Pharmaceuticals, Inc.	2012-11-07	PHASE1
NCT00943306	COMPLETED	Familial Hypercholesterolemia	Aegerion Pharmaceuticals, Inc.	2009-10-29	PHASE3
NCT01556906	COMPLETED	Homozygous Familial Hypercholesterolemia	Aegerion Pharmaceuticals, Inc.	2003-06	PHASE2

(data from https://clinicaltrials.gov, updated on 2025-08-27)

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

Compliance for Clinical Use

Mechanism and Biochemical Profile

Lomitapide mesylate selectively binds to and inhibits microsomal triglyceride transfer protein (MTP), thereby preventing the assembly and secretion of apoB-containing lipoproteins, including very low-density lipoproteins (VLDL) and chylomicrons, in hepatocytes and enterocytes. This reduction in circulating atherogenic lipoproteins directly decreases plasma low-density lipoprotein cholesterol (LDL-C) levels, rendering the compound therapeutically effective in the management of severe dyslipidemias such as homozygous familial hypercholesterolemia (HoFH).

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>).