Efficacy and safety of a microsomal triglyceride transfer protein inhibitor in patients with homozygous familial hypercholesterolaemia: a single-arm, open-label, phase 3 study

Cuchel, Marina; Meagher, Emma A; du Toit Theron, Hendrik; Blom, Dirk J; Marais, A David; Hegele, Robert A; Averna, Maurizio R; Sirtori, Cesare R; Shah, Prediman K; Gaudet, Daniel; Stefanutti, Claudia; Vigna, Giovanni B; Du Plessis, Anna ME; Propert, Kathleen J; Sasiela, William J; Bloedon, LeAnne T; Rader, Daniel J

doi:10.1016/s0140-6736(12)61731-0

Efficacy and safety of a microsomal triglyceride transfer protein inhibitor in patients with homozygous familial hypercholesterolaemia: a single-arm, open-label, phase 3 study

Background Patients with homozygous familial hypercholesterolemia (HoFH) respond inadequately to existing drugs. We conducted a phase 3 study to assess the efficacy and safety of the microsomal triglyceride transfer protein inhibitor lomitapide in adults with HoFH. Methods Twenty-nine subjects en…