Homozygous Familial Hypercholesterolemia MedDRA version: 9.1 Level: LLT Classification code 10057080
Conditions
Interventions
Product Name: Lomitapide
Product Code: AEGR-733
Pharmaceutical Form: Capsule, hard
Current Sponsor code: AEGR-733
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 20
Sponsors
AEGERION PHARMACEUTICALS
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: Patients completed 733-005 study Patients willing and able to provide consent and comply with the requirements of the study protocol. Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: Patients that met any of the stopping rules for study discontinuation at the final visit of study 733-005
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: The primary objective of the study is to evaluate the efficacy of lomitapide, as defined by percent change in LDL-C at the maximum tolerated dose at Week 48 compared to baseline, in combination with other lipid lowering therapy in patients with homozygous familial hypercholesterolemia (FH) who completed study UP1002 or 733-005.;Secondary Objective: The secondary objectives of the study are to evaluate other lipid parameters, percent change in hepatic fat, and long-term safety of lomitapide in combination with other lipid lowering agents in patients with homozygous FH. In particular we will assess: a. Percent change in TC, non-HDL-C, LDL-C, HDL-C, TG, VLDL-C, triglycerides, Lp(a), as well as apolipoproteins B and AI at all other time points. b. Long term safety of lomitapide in patients with homozygous FH as assessed by changes in laboratory parameters, reported adverse events, changes noted on the electrocardiogram, pulmonary function tests, physical examinations, and weight. c. Percent change in hepatic fat as measured by MRI/NMRS.;Primary end point(s): Efficacy will be assessed through the following lipid and lipoprotein parameters: LDL-C, non-HDL-C, TC, VLDL-C, HDL-C, triglycerides, apolipoproteins B and AI and Lp(a) Safety will be assessed through findings noted on the physical exam, PFTs and ECG, changes in laboratory parameters weight, and reported adverse events. | — |
Countries
Italy
Outcome results
None listed