Mipomersen Clinical Trials

Size	Price	Qty
1mg	USD 370
5mg	USD 970

Clinical Trials

Numerous Phase 1, Phase 2, and Phase 3 clinical trials have evaluated mipomersen for treating familial hypercholesterolemia, severe dyslipidemias, and coronary heart disease, alongside pharmacokinetic assessments in healthy volunteers. Sponsored by organizations such as Kastle Therapeutics, LLC and Ludwig-Maximilians - University of Munich, these completed studies investigated the efficacy and long-term tolerability of mipomersen as an adjunct therapy in high-risk patients. Overall, these trials demonstrate the clinical utility of mipomersen in managing severe lipid metabolism disorders.

NCT Number	Recruitment	Conditions	Sponsor/Collaborators	Start Date	Phases
NCT01475825	COMPLETED	Hypercholesterolemia; Heterozygous Familial	Kastle Therapeutics, LLC	2011-12	PHASE3
NCT01598948	COMPLETED	Atherosclerosis; LDL-hypercholesterolemia	Ludwig-Maximilians - University of Munich	2012-08	PHASE3
NCT00694109	COMPLETED	Lipid Metabolism, Inborn Errors; Hypercholesterolemia, Autosomal Dominant; Hyperlipidemias; Metabolic Diseases; Hyperlipoproteinemia Type II; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Infant, Newborn, Diseases; Metabolic Disorder; Congenital Abnormalities; Hypercholesterolemia; Hyperlipoproteinemias; Dyslipidemias; Lipid Metabolism Disorders	Kastle Therapeutics, LLC	2008-04	PHASE3
NCT01414881	COMPLETED	Healthy Volunteer	Kastle Therapeutics, LLC	2011-09	PHASE1
NCT01299298	COMPLETED	Healthy Volunteer	Kastle Therapeutics, LLC	2011-01	PHASE1
NCT00477594	COMPLETED	Lipid Metabolism, Inborn Errors; Hypercholesterolemia, Autosomal Dominant; Hyperlipidemias; Metabolic Diseases; Hyperlipoproteinemia Type II; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Infant, Newborn, Diseases; Metabolic Disorder; Congenital Abnormalities; Hypercholesterolemia; Hyperlipoproteinemias; Dyslipidemias; Lipid Metabolism Disorders	Kastle Therapeutics, LLC	2007-05	PHASE2
NCT00707746	COMPLETED	Metabolic Diseases; Hyperlipidemias; Metabolic Disorder; Hypercholesterolemia; Dyslipidemias; Lipid Metabolism Disorders	Kastle Therapeutics, LLC	2008-10	PHASE2
NCT00770146	COMPLETED	Hypercholesterolemia; Coronary Heart Disease	Kastle Therapeutics, LLC	2008-11	PHASE3
NCT00794664	COMPLETED	Hypercholesterolemia; Coronary Heart Disease	Kastle Therapeutics, LLC	2009-01	PHASE3
NCT01133366	COMPLETED	Healthy	Kastle Therapeutics, LLC	2010-05	PHASE1
NCT01090661	COMPLETED	Healthy	Kastle Therapeutics, LLC	2010-03	PHASE1
NCT00362180	COMPLETED	Lipid Metabolism, Inborn Errors; Hyperlipidemias; Metabolic Diseases; Hypolipoproteinemia; Hypolipoproteinemias; Hypobetalipoproteinemias; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Infant, Newborn, Diseases; Congenital Abnormalities; Metabolic Disorder; Hypercholesterolemia; Dyslipidemias; Lipid Metabolism Disorders	Kastle Therapeutics, LLC	2006-07	PHASE2
NCT01061814	COMPLETED	Healthy Volunteer	Kastle Therapeutics, LLC	2010-01	PHASE1
NCT00706849	COMPLETED	Heterozygous Familial Hypercholesterolemia; Coronary Artery Disease	Kastle Therapeutics, LLC	2008-07	PHASE3
NCT00607373	COMPLETED	Lipid Metabolism, Inborn Errors; Hypercholesterolemia, Autosomal Dominant; Hyperlipidemias; Metabolic Diseases; Hyperlipoproteinemia Type II; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Infant, Newborn, Diseases; Metabolic Disorder; Congenital Abnormalities; Hypercholesterolemia; Hyperlipoproteinemias; Dyslipidemias; Lipid Metabolism Disorders	Kastle Therapeutics, LLC	2007-07	PHASE3

(data from https://clinicaltrials.gov, updated on 2019-03-26)

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

Compliance for Clinical Use

Mechanism and Biochemical Profile

Mipomersen is an antisense oligonucleotide that sequence-specifically binds to apolipoprotein B-100 mRNA, inducing RNase H-mediated degradation to inhibit translation of the protein. This inhibition decreases hepatic synthesis and secretion of apolipoprotein B-containing lipoproteins, thereby lowering plasma low-density lipoprotein cholesterol to improve outcomes in conditions such as familial hypercholesterolemia and coronary heart disease.

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>).