CompletedPhase 3

A Safety and Efficacy Study of AEGR-733 to Treat Homozygous Familial Hypercholesterolemia (FH)

United States, Canada, Italy29 participantsStarted 2007-12

Plain-language summary

The goal of this trial is to study the effects of AEGR-733 on LDL cholesterol, other lipids as well as measures of safety over the long-term.

Age range18 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

✓. Males and females at least 18 years of age
✓. Diagnosis of functional homozygous FH by at least one (a-c) of the following clinical criteria:
✓. Concurrent lipid lowering medication/apheresis must be stable for at least 6 weeks before the baseline visit and must remain stable for the first 26 weeks.
✓. Body weight at least 40 kg and less than 136 kg
✓. Negative screening pregnancy test if female of child-bearing potential (females of child-bearing potential and all males must be following a medically accepted form of contraception)
✓. Subjects must be willing to comply with all study-related procedures

✕. Uncontrolled hypertension
✕. History of chronic renal insufficiency
✕. History of biopsy proven cirrhosis or abnormal LFTs at screening (AST or ALT greater than 2 x upper limit of normal and/or Total Bilirubin greater than or equal to 1.5 mg/dl unless patient has unconjugated hyperbilirubinemia due to Gilbert's syndrome)
✕. Chronic hepatitis B or chronic hepatitis C
✕. Any major surgical procedure occurring less than 3 months prior to the screening visit
✕. Cardiac insufficiency defined by the NYHA classification as functional Class III or Class IV
✕. Previous organ transplantation
✕. History of a non-skin malignancy within the previous 3 years

Percent Change From Baseline in Low-density Lipoprotein Cholesterol (LDL-C)

Timeframe: Baseline and Week 26

NCT IDNCT00730236

SponsorAegerion Pharmaceuticals, Inc.

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2010-09

Results submitted2013-01-18

Who can participate

Age range18 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

✓. Males and females at least 18 years of age
✓. Diagnosis of functional homozygous FH by at least one (a-c) of the following clinical criteria:
✓. Concurrent lipid lowering medication/apheresis must be stable for at least 6 weeks before the baseline visit and must remain stable for the first 26 weeks.
✓. Body weight at least 40 kg and less than 136 kg
✓. Negative screening pregnancy test if female of child-bearing potential (females of child-bearing potential and all males must be following a medically accepted form of contraception)
✓. Subjects must be willing to comply with all study-related procedures

✕. Uncontrolled hypertension
✕. History of chronic renal insufficiency
✕. History of biopsy proven cirrhosis or abnormal LFTs at screening (AST or ALT greater than 2 x upper limit of normal and/or Total Bilirubin greater than or equal to 1.5 mg/dl unless patient has unconjugated hyperbilirubinemia due to Gilbert's syndrome)
✕. Chronic hepatitis B or chronic hepatitis C
✕. Any major surgical procedure occurring less than 3 months prior to the screening visit
✕. Cardiac insufficiency defined by the NYHA classification as functional Class III or Class IV
✕. Previous organ transplantation
✕. History of a non-skin malignancy within the previous 3 years