Safety, PK and PD of lomitapide in Japanese and Caucasasians

• Research type

  Research Study

• Full title

  A randomized, double-blind, placebo-controlled, single ascending and multiple ascending dose study of the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD) of lomitapide in Japanese and Caucasian volunteers with elevated LDL-C

• IRAS ID

  117050

• Contact name

  Ulrike Lorch

• Sponsor organisation

  Aegerion Pharmaceuticals, SAS.

• Eudract number

  2012-004220-37

• ISRCTN Number

  No number provided

• Clinicaltrials.gov Identifier

  No number provided

• Research summary

  We are conducting a clinical study with a new medicine known as lomitapide in up to 104 healthy male volunteers (52 Japanese and 52 Caucasians) at Richmond Pharmacology Ltd (RPL) clinical research unit. Lomitapide is being developed by Aegerion Pharmaceuticals, Inc (the sponsor company) as a potential treatment for adult patients with Homozygous Familial Hypercholesterolemia (HoFH). HoFH is a severe and rare disease in which patients have very high levels of LDL-C ("bad" cholesterol) that is inherited from both parents. Lomitapide has previously been tested in over 1000 non-Japanese healthy volunteers and patients in a number of clinical trials to assess its safety, tolerability and efficacy. The aim of the study is to compare the safety and tolerability of lomitapide when given to Japanese and Caucasian volunteers with elevated levels of bad cholesterol. We will also measure the levels of lomitapide in the blood (pharmacokinetics) as well as the effect of lomitapide on specific lipids in the blood (pharmacodynamics) following single and multiple oral doses to volunteers during one treatment period. This will help us to determine oral doses that can be given safely to adult Japanese patients for HoFH in future. Standard safety assessments will be conducted during the study including laboratory tests, vital signs, physical examinations, ECG, pharmacokinetics (measurement of drug levels in the blood), pharmacodynamics (measurement of specific lipids in the blood that may be affected by the drug) and observations of any side effects. An exploratory blood sample will also be taken for genetic (DNA) analysis. Volunteers will have to follow a low-fat diet (generally <20% of energy from fat) for the duration of the study to eliminate dietary factors which may otherwise affect the blood lipid levels and confuse the results. The study will take approximately 55 days for a volunteer to complete.

• REC name

  London - Brent Research Ethics Committee

• REC reference

  12/LO/1618

• Date of REC Opinion

  30 Oct 2012

• REC opinion

  Favourable Opinion