Safety, Tolerability and Efficacy of Microsomal Triglyceride Protein (MTP) Inhibitor

Completed · Phase 2

Conditions studied: Homozygous Familial Hypercholesterolemia

In brief

The primary objective of this study is to evaluate the safety and tolerability of 4 doses of lomitapide (AEGR-733; BMS-201038) given as an initial low dose and then escalated through an additional 3 dose levels over a 16-week period. The secondary objectives of this study included the evaluation of the pharmacodynamics of lomitapide based on: * Percent change in low-density lipoprotein cholesterol (LDL-C), total cholesterol (TC), triglycerides, and very low density lipoprotein cholesterol (VLDL-C) concentrations at the end of each 4-week dosing period compared to the Baseline value of each parameter at the end of the previous dose phase(s). * Changes in other plasma lipoproteins: apolipoproteins (apo B, apo AI, apo AII, apo CIII, apo E) and lipoprotein a \[Lp(a)\].

Key facts

Study ID

NCT01556906

Run by

Aegerion Pharmaceuticals, Inc.

People needed

6

Starts

2003-06-01

Expected to finish

2004-02-01

Last updated by the study team

2013-04-10

Who can join

Age: 13 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Males and females ≥13 years of age
• Clinical diagnosis of HoFH AND one of the following (a, b, or c):
• Documented functional mutation in both LDL receptor alleles, OR
• Skin fibroblast LDL receptor activity <20% of normal, OR
• TC >500 mg/dL AND triglycerides < 300 mg/dL AND both parents with documented TC >250 mg/dL
• Body weight ≥40 kg
• Negative screening pregnancy test if female of child-bearing potential
• Subjects must be willing and able to comply with all study-related procedures
• Subjects must be willing and able to go off all lipid-lowering medications, dietary supplements (psyllium preparations) and LDL apheresis within 4 weeks prior to the Baseline visit until the end of the study.

You may not qualify if…

• Uncontrolled hypertension defined as: systolic blood pressure >180 mmHg, diastolic blood pressure >95 mmHg
• History of chronic renal insufficiency (serum creatinine >2.5 mg/dL)
• History of liver disease or abnormal LFTs at screening (>3x upper limit of normal [ULN])
• Any major surgical procedure occurring < 3 months prior to the screening visit
• Cardiac insufficiency defined by the New York Heart Association classification as functional Class III or Class IV
• History of a non-skin malignancy within the previous 5 years
• History of alcohol or drug abuse
• Participation in an investigational drug study within 6 weeks prior to the screening visit
• Serious or unstable medical or psychological conditions that, in the opinion of the Investigator, would compromise the patient's safety or successful participation in the study.

Where it is running

• University of Pennsylvania — Philadelphia, Pennsylvania, United States

Full record on ClinicalTrials.gov

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