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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

Galenos Research

🇺🇸

Dallas, Texas, United States

UT Southwestern Medical Center

🇺🇸

Dallas, Texas, United States

Pioneer Research Solutions, Inc.

🇺🇸

Sugar Land, Texas, United States

Discovery Clinical Services Ltd.

🇨🇦

Victoria, British Columbia, Canada

Deventer Ziekenhuis

🇳🇱

Deventer, Netherlands

NewPhase Clinical Trials, Corp.

🇺🇸

Miami Beach, Florida, United States

Focus Clinical Research, LLC

🇺🇸

Draper, Utah, United States

Columbus Clinical Services, LLC

🇺🇸

Miami, Florida, United States

Ecogene-21

🇨🇦

Chicoutimi, Quebec, Canada

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

🇺🇸

Chapel Hill, North Carolina, United States

Clinique des maladies lipidiques de Quebec Inc

🇨🇦

Quebec, Canada

NZOZ Vitamed

🇵🇱

Bydgoszcz, Poland

UMC Utrecht

🇳🇱

Utrecht, Netherlands

Oslo Universitetssykehus HF, Ulleval

🇳🇴

Oslo, Norway

Hospital Clinico Universitario Miguel Servet; Medicina Interna

🇪🇸

Zaragoza, Spain

Hartford Hospital, JB704

🇺🇸

Hartford, Connecticut, United States

Premier Research Associate, Inc

🇺🇸

Miami, Florida, United States

Medical Research Center

🇺🇸

Miami, Florida, United States

Best Quality Research, Inc.

🇺🇸

Hialeah, Florida, United States

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

🇺🇸

Chapel Hill, North Carolina, United States

Oklahoma Heart Hospital Physicians

🇺🇸

Oklahoma City, Oklahoma, United States

Oklahoma Heart Hospital

🇺🇸

Oklahoma City, Oklahoma, United States

Om Medical

🇺🇸

Las Vegas, Nevada, United States

Metabolic and Atherosclerosis Research Center

🇺🇸

Cincinnati, Ohio, United States

Oklahoma Heart Hospital Research Foundation

🇺🇸

Oklahoma City, Oklahoma, United States

SHAT in Cardiology EAD

🇧🇬

Pleven, Bulgaria

OnSite Clinical Solutions, LLC

🇺🇸

Gaffney, South Carolina, United States

UMHAT "Sveti Georgi" EAD, Clinic of Cardiology

🇧🇬

Plovdiv, Bulgaria

Burke Internal Medicine & Research

🇺🇸

Burke, Virginia, United States

MHAT "Sveta Anna", Clinic of Internal Diseases

🇧🇬

Sofia, Bulgaria

St. Paul's Hospital, Healthy Heart

🇨🇦

Vancouver, British Columbia, Canada

Second MHAT - Sofia EAD

🇧🇬

Sofia, Bulgaria

Asper Clinical Research Institute

🇨🇦

Winnipeg, Manitoba, Canada

University of Ottawa Heart Institute

🇨🇦

Ottawa, Ontario, Canada

Health Sciences Centre

🇨🇦

St. John's, Newfoundland and Labrador, Canada

Montreal Heart Institute

🇨🇦

Montreal, Quebec, Canada

Institut de Recherches Cliniques de Montreal

🇨🇦

Montreal, Quebec, Canada

Pohjois-Karjala Projekti Saatio

🇫🇮

Joensuu, Finland

Ospedale "Santa Maria della Misericordia"

🇮🇹

Perugia, PG, Italy

Policlinico "Paolo Giaccone"

🇮🇹

Palermo, PA, Italy

St. Franciscus Gasthuis

🇳🇱

Rotterdam, Zuid-holland, Netherlands

Amphia Hospital

🇳🇱

Breda, Netherlands

Ospedale di Circolo e Fondazione Macchi

🇮🇹

Varese, VA, Italy

Azienda Ospedaliero Universitaria "Federico II" di Napoli

🇮🇹

Napoli, Italy

Rotterdam Research Institute

🇳🇱

Rotterdam, Netherlands

Academic Medical Center

🇳🇱

Amsterdam, Netherlands

Admiraal de Ruyter ziekenhuis

🇳🇱

Goes, Zeeland, Netherlands

Hagaziekenhuis

🇳🇱

Den Haag, Netherlands

Albert Schweitzer Hospital

🇳🇱

Sliedrecht, Netherlands

NZOZ Przychodnia Specjalistyczna Andrzej Wittek Henryk Rudzki S.C

🇵🇱

Ruda Slaska, Poland

IATROS International

🇿🇦

Bloemfontein, Free State, South Africa

Unitas hospital

🇿🇦

Centurion, Gauteng, South Africa

NZOZ Centrum Zdrowia i Profilaktyki Dabie Sp zo.o.

🇵🇱

Krakow, Poland

Midrand Medical Centre

🇿🇦

Halfway House, Gauteng, South Africa

Medipark Centre for Clinical Research

🇿🇦

Pretoria, Gauteng, South Africa

Jongaie Research

🇿🇦

Pretoria, Gauteng, South Africa

Synexus Watermeyer Clinical Research Centre

🇿🇦

Pretoria, Gauteng, South Africa

Roodepoort Medicross Clinical Research Centre

🇿🇦

Roodepoort, Gauteng, South Africa

Chelmsford Medical Centre 3

🇿🇦

Durban, Kwa-zulu Natal, South Africa

Synexus Helderberg Clinical Research Centre

🇿🇦

Somerset West, Western Cape, South Africa

Hospital Universitario de Bellvitge

🇪🇸

L'Hospitalet de Llobregat, Barcelona, Spain

Hospital Clinico Universitario, Santiago de Compostela

🇪🇸

Santiago de Compostela, Galicia, Spain

Hospital Universitario Sant Joan de Reus

🇪🇸

Reus, Tarragona, Spain

Hospital Clinic

🇪🇸

Barcelona, Spain

Hospital General Universitario Gregorio Maranon

🇪🇸

Madrid, Spain

Hospital Universitario Reina Sofia

🇪🇸

Cordoba, Spain

Hospital Clinico San Carlos; U. de Lipidos; Medicina Interna III

🇪🇸

Madrid, Spain

Fundacion Jimenez Diaz; Servicio de Medicina Interna

🇪🇸

Madrid, Spain

Pennine Acute Hospitals NHS Trust

🇬🇧

Oldham, Greater Manchester, United Kingdom

Hospital Universitario Virgen del Rocio

🇪🇸

Sevilla, Spain

Clinical Trials Pharmacy, 4th Floor Inpatient Pharmacy

🇬🇧

Manchester, Greater Manchester, United Kingdom

East and North Hertfordshire NHS Trust

🇬🇧

Stevenage, Hertfordshire, United Kingdom

Burton Hospitals NHS Foundation Trust

🇬🇧

Burton-on-Trent, Staffordshire, United Kingdom

Heart of England NHS Foundation Trust

🇬🇧

Birmingham, West Midlands, United Kingdom

Central Manchester University Hospitals NHS Foundation Trust

🇬🇧

Manchester, United Kingdom

Peterborough and Stamford Hospitals NHS Foundation Trust

🇬🇧

Peterborough, United Kingdom

Oulu University Hospital

🇫🇮

Oulu, Finland

Helsinki Central University Hospital

🇫🇮

Helsinki, Finland

Oslo Universitetssykehus HF

🇳🇴

Oslo, Norway

TREAD Research cc.

🇿🇦

Parow, Cape Town, Western Cape, South Africa

Pohjois-Karjala Projekti Saatio/Ita-Suomen

🇫🇮

Joensuu, Finland

Division of Medicine Turku University Hospital

🇫🇮

Turku, Finland

Laakarikeskus Aava Kerava/Aava Kerava Medical Center

🇫🇮

Kerava, Finland

Tiervlei Trial Centre, Karl Bremer Hospital

🇿🇦

Bellville, Cape Town, Western Cape, South Africa

Royal Free London NHS Foundation Trust

🇬🇧

London, United Kingdom

Thomas Jefferson University

🇺🇸

Philadelphia, Pennsylvania, United States

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