The primary objective of this Phase I exploratory study is to determine the effects of mipomersen on the hepatic production of apolipoprotein-B (apo B) in very low density lipoprotein (VLDL) compared to baseline levels. The study will consist of a Screening Period, a 1-week Run-in Period to establish a stable diet, an approximate 11-week Treatment Period with Placebo or Mipomersen, and a 25-week Post-Treatment Follow-up Period. The total duration of any given subject's participation will be approximately 40 weeks.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Masking
SINGLE
Enrollment
20
mipomersen 200mg subcutaneously (SC) once weekly
Placebo administered subcutaneously (SC) once weekly
Columbia-Presbyterian Medical Center, MS Care Center
New York, New York, United States
Percent change in the production rate (PR) of very low density lipoprotein (VLDL) apolipoprotein B (apo B)
Time frame: through approximately 11 weeks of treatment
Fractional clearance rate (FCR) of VLDL Triglyceride (TG), VLDL apo B, intermediate density lipoprotein (IDL) apo B, and low-density lipoprotein (LDL) apo B
Time frame: Through approximately 11 weeks of treatment
Production rate (PR) of VLDL-TG, IDL apo B, LDL apo B
Time frame: Through approximately 11 weeks of treatment
Conversion of VLDL apo B to low-density lipoprotein (LDL) apo B
Time frame: Through approximately 11 weeks of treatment
Direct removal of VLDL apo B from plasma
Time frame: Through approximately 11 weeks of treatment
Post-heparin hepatic lipase and lipoprotein lipase activities in serum
Time frame: Through approximately 11 weeks of treatment
Fasting plasma levels of fatty acids and beta-hydroxybutyrate
Time frame: Up to 40 weeks
Incidence of adverse events (AEs) and serious adverse events (SAEs)
Time frame: Up to 40 weeks
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