Hypercholesterolemia Clinical Trial
Official title:
A Randomized, Double-Blind, Placebo-Controlled Study to Assess the Safety and Efficacy of Mipomersen as Add-on Therapy in Homozygous Familial Hypercholesterolemia Subjects
The purpose of this study is to evaluate the safety and efficacy of mipomersen (ISIS 301012)
in subjects with homozygous familial hypercholesterolemia on lipid-lowering therapy.
This study consisted of a 26-week treatment period and a 24-week post-treatment follow-up
period. Following treatment and Week 28 evaluations, participants could elect to enroll in
an open-label extension study (301012-CS6; NCT00694109). Participants who were not eligible
or elected not to enroll in the open-label extension study or who discontinued during the
28-week treatment period were followed in this study for 24 weeks from administration of the
last dose of study drug.
Familial hypercholesterolemia (FH) is an autosomal dominant genetic disorder of lipoprotein
metabolism characterized by markedly elevated low density lipoprotein (LDL), premature onset
of atherosclerosis and development of xanthomata. Patients with homozygous familial
hypercholesterolemia (HoFH) have a severe disease that presents in childhood with total
cholesterol typically in the 650 to 1000 mg/dL range.
This was a randomized, double-blind, placebo-controlled study, which consisted of a 4-week
screening period, 26 weeks of treatment, and a 24-week post- treatment follow-up period
(with the exception of patients who enrolled in the open-label extension study, Study
301012-CS6; NCT00694109). Eligible patients were randomized in a 2:1 ratio to receive 200 mg
mipomersen or matching volume placebo subcutaneous (SC) injections weekly. Patients who
weighed <50 kg received a lower dose of 160 mg mipomersen or matching volume of placebo SC
injections weekly. Patients were to have been on a stable (>=12 weeks) regimen of allowed
lipid-lowering therapies at screening, and were required to remain on the same dose and
regimen throughout the study.
Patients returned to the study center for clinical evaluation every other week during the
first 4 weeks of treatment, once every 4 to 5 weeks for the remainder of the treatment
period, and monthly during the post-treatment evaluation (follow-up) period. The primary
endpoint assessment was at Week 28. Following treatment and Week 28 evaluations, eligible
patients who tolerated the study drug could elect to enroll in the open-label extension
study (Study 301012-CS6; NCT00694109). Patients who did not participate in the open-label
extension study were required to return to the study center for clinical evaluation at least
twice during the post-treatment follow-up period, including an end-of-study termination
visit at the end of this 24-week period.
;
Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Primary Purpose: Treatment
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