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A 52 Week Study To Assess The Use Of Bococizumab (PF-04950615; RN316) In Subjects With Heterozygous Familial Hypercholesterolemia

Phase 3
Completed
Conditions
Heterozygous Familial Hypercholesterolemia
Interventions
Registration Number
NCT01968980
Lead Sponsor
Pfizer
Brief Summary

This is a multicenter, randomized study in subjects with heterozygous familial hypercholesterolemia receiving highly effective statins to assess the safety, efficacy and tolerability of Bococizumab (PF-04950615; RN316) to lower LDL-C.

Detailed Description

Not available

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
370
Inclusion Criteria
  • Treated with a statin.
  • Fasting LDL-C > 70 mg/dL and triglyceride <=400 mg/dL.
  • High or very high risk of incurring a cardiovascular event.
  • Heterozygous familial hypercholesterolemia.
Exclusion Criteria
  • Pregnant or breastfeeding females.
  • Cardiovascular or cerebrovascular event of procedures during the past 30 days.
  • Congestive heart failure NYHA class IV.
  • Poorly controlled hypertension.

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
Bococizumab (PF-04950615;RN316)Bococizumab (PF-04950615;RN316)Bococizumab (PF-04950615;RN316)
PlaceboPlacebo-
Primary Outcome Measures
NameTimeMethod
Percent Change From Baseline in Low Density Lipoprotein Cholesterol (LDL-C) at Week 12Baseline, Week 12
Secondary Outcome Measures
NameTimeMethod
Percent Change From Baseline in Total Cholesterol (TC) at Week 12Baseline, Week 12
Percent Change From Baseline in Non High Density Lipoprotein Cholesterol (Non HDL-C) at Week 12Baseline, Week 12
Percentage of Participants Achieving Low Density Lipoprotein Cholesterol (LDL-C) Level Less Than or Equal to (<=) 70 Milligram Per Deciliter (1.81 Millimoles Per Liter) at Week 12, 24 and 52Week 12, 24, 52
Percent Change From Baseline in Very Low Density Lipoprotein Cholesterol (VLDL-C) at Week 12, 24 and 52Baseline, Week 12, 24, 52
Absolute Change From Baseline in Lipoprotein (a) at Week 12Baseline, Week 12
Percentage of Participants Achieving Low Density Lipoprotein Cholesterol (LDL-C) Level Less Than or Equal to (<=) 100 Milligram Per Deciliter (2.59 Millimoles Per Liter) at Week 12, 24 and 52Week 12, 24, 52
Percent Change From Baseline in Apolipoprotein B (ApoB) at Week 12Baseline, Week 12
Percent Change From Baseline in High Density Lipoprotein Cholesterol (HDL-C) at Week 24 and 52Baseline, Week 24, 52
Absolute Change From Baseline in High Density Lipoprotein Cholesterol (HDL-C) at Week 12Baseline, Week 12
Absolute Change From Baseline in Ratio of Total Cholesterol to High Density Lipoprotein Cholesterol (TC/HDL-C Ratio) at Week 12, 24 and 52Baseline, Week 12, 24, 52
Percentage of Participants With Positive Anti-Drug Antibodies (ADA) and Neutralizing Antibodies (nAb)Baseline up to the end of study (up to 58 weeks)

Percentage of participants with at least 1 positive ADA titer or 1 positive nAb titer were reported in this outcome measure. ADA titer greater than or equal to (\>=) 6.23 were considered as ADA positive and nAb titer level \>=1.58 were considered as nAb positive.

Percent Change From Baseline in Lipoprotein (a) at Week 12Baseline, Week 12
Percent Change From Baseline in High Density Lipoprotein Cholesterol (HDL-C) at Week 12Baseline, Week 12
Percent Change From Baseline in Low Density Lipoprotein Cholesterol (LDL-C) at Week 24 and 52Baseline, Week 24, 52
Percent Change From Baseline in Total Cholesterol (TC) at Week 24 and 52Baseline, Week 24, 52
Percent Change From Baseline in Non High Density Lipoprotein Cholesterol (Non HDL-C) at Week 24 and 52Baseline, Week 24, 52
Percent Change From Baseline in Apolipoprotein B (ApoB) at Week 24 and 52Baseline, Week 24, 52
Percent Change From Baseline in Lipoprotein (a) at Week 24 and 52Baseline, Week 24, 52
Percent Change From Baseline in Triglycerides (TG) at Week 12, 24 and 52Baseline, Week 12, 24, 52
Percent Change From Baseline in Apolipoprotein A-I (ApoA-I) at Week 12, 24 and 52Baseline, Week 12, 24, 52
Absolute Change From Baseline in Ratio of Apolipoprotein B to Apolipoprotein A-I (ApoB/ApoA-I Ratio) at Week 12, 24 and 52Baseline, Week 12, 24, 52
Plasma PF-04950615 Concentrations at Week 12, 24 and 52Week 12, 24, 52
Percent Change From Baseline in Apolipoprotein A-II (ApoA-II) at Week 12, 24 and 52Baseline, Week 12, 24, 52
Absolute Change From Baseline in Low Density Lipoprotein (LDL-C) at Week 12Baseline, Week 12
Absolute Change From Baseline in Total Cholesterol (TC) at Week 12Baseline, Week 12
Absolute Change From Baseline in Non- High Density Lipoprotein Cholesterol (Non HDL-C) at Week 12Baseline, Week 12
Absolute Change From Baseline in Apolipoprotein B (ApoB) at Week 12Baseline, Week 12
Number of Participants With Adverse Events Related to Type 1 or 3 Hypersensitivity Reactions and Injection Site ReactionsBaseline up to the end of study (up to 58 weeks)

Type 1 hypersensitivity or allergic reactions were possible in response to any injected protein and included shortness of breath, urticaria, anaphylaxis and angioedema. Type 3 hypersensitivity reactions were similar to Type 1 hypersensitivity reactions but were likely to be delayed from the time of injection and included symptoms such as rash, urticaria, polyarthritis, myalgia, polysynovitis, fever and if severe then included glomerulonephritis as well. Injection site reaction is a reaction at the site of the subcutaneous injection and characterized by the symptoms of erythema, swelling, tenderness and warmth. Participants with any of the above type 1 or type 3 hypersensitivity reactions and participants with any of the above injection site reactions were reported in this outcome measure.

Trial Locations

Locations (87)

IMD Medical Group

🇺🇸

Los Angeles, California, United States

Hartford Hospital, JB704

🇺🇸

Hartford, Connecticut, United States

Best Quality Research, Inc.

🇺🇸

Hialeah, Florida, United States

NewPhase Clinical Trials, Corp.

🇺🇸

Miami Beach, Florida, United States

Medical Research Center

🇺🇸

Miami, Florida, United States

Premier Research Associate, Inc

🇺🇸

Miami, Florida, United States

Columbus Clinical Services, LLC

🇺🇸

Miami, Florida, United States

Om Medical

🇺🇸

Las Vegas, Nevada, United States

The University of North Carolina at Chapel Hill Center for Heart & Vascular Care

🇺🇸

Chapel Hill, North Carolina, United States

The University of North Carolina Hospitals - Clinical and Translational Research Center Clinic

🇺🇸

Chapel Hill, North Carolina, United States

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IMD Medical Group
🇺🇸Los Angeles, California, United States

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