A Phase III, Long Term, Open Label, Follow on Study of Microsomal Triglyceride Transfer Protein (MTP) Inhibitor ‘lomitapide’ (AEGR-733) in Patients with Homozygous Familial Hypercholesterolemia - ND

• Register: EUCTR
• Last refreshed on: 12 October 2015
• Main ID: EUCTR2010-023742-79-IT
• Date of registration: 21/02/2011
• Prospective Registration: Yes
• Primary sponsor: AEGERION PHARMACEUTICALS
• Public title: A Phase III, Long Term, Open Label, Follow on Study of Microsomal Triglyceride Transfer Protein (MTP) Inhibitor ‘lomitapide’ (AEGR-733) in Patients with Homozygous Familial Hypercholesterolemia - ND
• Scientific title: A Phase III, Long Term, Open Label, Follow on Study of Microsomal Triglyceride Transfer Protein (MTP) Inhibitor ‘lomitapide’ (AEGR-733) in Patients with Homozygous Familial Hypercholesterolemia - ND
• Date of first enrolment: 25/03/2011
• Target sample size: 23
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2010-023742-79
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: no Randomised: no Open: no Single blind: no Double blind: no Parallel group: no Cross over: no Other: no If controlled, specify comparator, Other Medicinial Product: Placebo: Other:

Countries of recruitment

Italy

Key inclusion & exclusion 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:
Patients that met any of the stopping rules for study discontinuation at the final visit of study 733-005


Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

Homozygous Familial Hypercholesterolemia
MedDRA version: 9.1 Level: LLT Classification code 10057080

Intervention(s)

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-

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: 5-

Primary Outcome(s)

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.

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 ID(s)

AEG 733-012