Clinical Trials

Numerous clinical trials across Phase I through III have evaluated lomitapide, focusing on severe lipid disorders such as homozygous familial hypercholesterolemia and dyslipidemia, as well as pharmacokinetic parameters and drug-drug interactions with atorvastatin in healthy volunteers. Sponsored by organizations including Aegerion Pharmaceuticals, Amryt Pharma, Fondazione SISA, and Richmond Pharmacology Limited, these studies encompass recruitment statuses ranging from completed to active not recruiting, terminated, or withdrawn.

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NCT02765841 WITHDRAWN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
NCT02080468 Completed
Healthy
Aegerion Pharmaceuticals Inc.
 2014-02-19 Phase 1
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
NCT01760187 Completed
Healthy Volunteer
Aegerion Pharmaceuticals Inc.|Richmond Pharmacology Limited
 2012-11-07 Phase 1
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 product page for in-depth specifications, including solubility, stock solutions, MOA, and working concentrations.

Compliance for Clinical Use

Mechanism and Biochemical Profile

Lomitapide directly binds to and inhibits microsomal triglyceride transfer protein (MTP), thereby blocking the lipid transfer process required for the assembly and secretion of apolipoprotein B-containing lipoproteins in hepatocytes and enterocytes. By preventing hepatic and intestinal lipoprotein release, the compound significantly lowers circulating low-density lipoprotein cholesterol levels, demonstrating clear therapeutic utility in clinical conditions such as homozygous familial hypercholesterolemia and severe dyslipidemia.

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.