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Heterozygous Familial HypercholesterolemiaPhase 3SPIRE-FHNCT01968980

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

A Phase 3 study testing Bococizumab (PF-04950615;RN316) in people with Heterozygous Familial Hypercholesterolemia, run by Pfizer at 88 sites in 11 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
Search all trials

Trial 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. Read more about this study

Who is it for?
Ages 18 years and older
What is being tested?
Bococizumab (PF-04950615;RN316)
Type of treatment
Injection or infusion
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
370 participants
Where
88 sitesin 11 countries
Study dates
23 Oct 2013to 15 Apr 2016
Phase
Phase 3
Are there results?
Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • 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.

Who cannot join

  • Pregnant or breastfeeding females.
  • Cardiovascular or cerebrovascular event of procedures during the past 30 days.
  • Congestive heart failure NYHA class IV.
  • Poorly controlled hypertension.

Trial locations and contacts

88 sites in 11 countries.

88 sites in 11 countries

United States22 sites

  • Burke Internal Medicine & Research

    Burke, Virginia, 22015

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

    Chapel Hill, North Carolina, 27599-7075

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

    Chapel Hill, North Carolina, 27599-7600

  • Metabolic and Atherosclerosis Research Center

    Cincinnati, Ohio, 45227

  • Galenos Research

    Dallas, Texas, 75251

  • UT Southwestern Medical Center

    Dallas, Texas, 75390

  • Focus Clinical Research, LLC

    Draper, Utah, 84020

  • OnSite Clinical Solutions, LLC

    Gaffney, South Carolina, 29340

  • Hartford Hospital, JB704

    Hartford, Connecticut, 06102

  • Best Quality Research, Inc.

    Hialeah, Florida, 33016

  • Pioneer Research Solutions, Inc.

    Houston, Texas, 77099

  • Om Medical

    Las Vegas, Nevada, 89119

  • IMD Medical Group

    Los Angeles, California, 90020

  • Medical Research Center

    Miami, Florida, 33144

  • Premier Research Associate, Inc

    Miami, Florida, 33165

  • Columbus Clinical Services, LLC

    Miami, Florida, 33174

  • NewPhase Clinical Trials, Corp.

    Miami Beach, Florida, 33140

  • Oklahoma Heart Hospital Physicians

    Oklahoma City, Oklahoma, 73120

  • Oklahoma Heart Hospital Research Foundation

    Oklahoma City, Oklahoma, 73120

  • Oklahoma Heart Hospital

    Oklahoma City, Oklahoma, 73120

  • Thomas Jefferson University

    Philadelphia, Pennsylvania, 19107

  • Pioneer Research Solutions, Inc.

    Sugar Land, Texas, 77479

Other trials in United States

South Africa11 sites

  • Tiervlei Trial Centre, Karl Bremer Hospital

    Bellville, Cape Town, Western Cape, 7530

  • IATROS International

    Bloemfontein, Free State, 9301

  • Unitas hospital

    Centurion, Gauteng, 0157

  • Chelmsford Medical Centre 3

    Durban, KwaZulu-Natal, 4001

  • Midrand Medical Centre

    Halfway House, Gauteng, 1685

  • TREAD Research cc.

    Parow, Cape Town, Western Cape, 7500

  • Medipark Centre for Clinical Research

    Pretoria, Gauteng, 0158

  • Jongaie Research

    Pretoria, Gauteng, 0183

  • Synexus Watermeyer Clinical Research Centre

    Pretoria, Gauteng, 0184

  • Roodepoort Medicross Clinical Research Centre

    Roodepoort, Gauteng, 1724

  • Synexus Helderberg Clinical Research Centre

    Somerset West, Western Cape, 7130

Other trials in South Africa

Spain10 sites

  • Hospital Clinic

    Barcelona, 08036

  • Hospital Universitario Reina Sofia

    Córdoba, 14004

  • Hospital Universitario de Bellvitge

    L'Hospitalet de Llobregat, Barcelona, 08907

  • Hospital General Universitario Gregorio Maranon

    Madrid, 28007

  • Fundacion Jimenez Diaz; Servicio de Medicina Interna

    Madrid, 28040

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

    Madrid, 28040

  • Hospital Universitario Sant Joan de Reus

    Reus, Tarragona, 43204

  • Hospital Clinico Universitario, Santiago de Compostela

    Santiago de Compostela, Galicia, 15706

  • Hospital Universitario Virgen del Rocio

    Seville, 41013

  • Hospital Clinico Universitario Miguel Servet; Medicina Interna

    Zaragoza, 50009

Other trials in Spain

Canada9 sites

  • Ecogene-21

    Chicoutimi, Quebec, G7H 7K9

  • Montreal Heart Institute

    Montreal, Quebec, H1T 1C8

  • Institut de Recherches Cliniques de Montreal

    Montreal, Quebec, H2W 1R7

  • University of Ottawa Heart Institute

    Ottawa, Ontario, K1Y 4W7

  • Clinique des maladies lipidiques de Quebec Inc

    Québec, Quebec, G1V 4W2

  • Health Sciences Centre

    St. John's, Newfoundland and Labrador, A1B 3V6

  • St. Paul's Hospital, Healthy Heart

    Vancouver, British Columbia, V6Z 1Y6

  • Discovery Clinical Services Ltd.

    Victoria, British Columbia, V8T 5G4

  • Asper Clinical Research Institute

    Winnipeg, Manitoba, R2H 2A6

Other trials in Canada

Netherlands9 sites

  • Academic Medical Center

    Amsterdam, 1105 AZ

  • Amphia Hospital

    Breda, 4818 CK

  • Deventer Ziekenhuis

    Deventer, 7416 SE

  • Admiraal de Ruyter ziekenhuis

    Goes, Zeeland, 4462 RA

  • St. Franciscus Gasthuis

    Rotterdam, South Holland, 3045 PM

  • Rotterdam Research Institute

    Rotterdam, 3039 BD

  • Albert Schweitzer Hospital

    Sliedrecht, 3361 XV

  • Hagaziekenhuis

    The Hague, 2545 CH

  • UMC Utrecht

    Utrecht, 3584 CX

Other trials in Netherlands

United Kingdom8 sites

  • Heart of England NHS Foundation Trust

    Birmingham, West Midlands, B9 5SS

  • Burton Hospitals NHS Foundation Trust

    Burton-on-Trent, Staffordshire, DE13 0RB

  • Royal Free London NHS Foundation Trust

    London, NW3 2QG

  • Clinical Trials Pharmacy, 4th Floor Inpatient Pharmacy

    Manchester, Greater Manchester, M13 9WL

  • Central Manchester University Hospitals NHS Foundation Trust

    Manchester, M13 9WL

  • Pennine Acute Hospitals NHS Trust

    Oldham, Greater Manchester, OL1 2JH

  • Peterborough and Stamford Hospitals NHS Foundation Trust

    Peterborough, PE3 9GZ

  • East and North Hertfordshire NHS Trust

    Stevenage, Hertfordshire, SG1 4AB

Other trials in United Kingdom

Finland6 sites

  • Helsinki Central University Hospital

    Helsinki, 00014

  • Pohjois-Karjala Projekti Saatio/Ita-Suomen

    Joensuu, 80100

  • Pohjois-Karjala Projekti Saatio

    Joensuu, 80100

  • Laakarikeskus Aava Kerava/Aava Kerava Medical Center

    Kerava, 04200

  • Oulu University Hospital

    Oulu, 90220

  • Division of Medicine Turku University Hospital

    Turku, 20520

Other trials in Finland

Bulgaria4 sites

  • SHAT in Cardiology EAD

    Pleven, 5800

  • UMHAT "Sveti Georgi" EAD, Clinic of Cardiology

    Plovdiv, 4002

  • Second MHAT - Sofia EAD

    Sofia, 1202

  • MHAT "Sveta Anna", Clinic of Internal Diseases

    Sofia, 1709

Other trials in Bulgaria

Italy4 sites

  • Azienda Ospedaliero Universitaria "Federico II" di Napoli

    Naples, 80131

  • Policlinico "Paolo Giaccone"

    Palermo, PA, 90127

  • Ospedale "Santa Maria della Misericordia"

    Perugia, PG, 06156

  • Ospedale di Circolo e Fondazione Macchi

    Varese, VA, 21100

Other trials in Italy

Poland3 sites

  • NZOZ Vitamed

    Bydgoszcz, 85-079

  • NZOZ Centrum Zdrowia i Profilaktyki Dabie Sp zo.o.

    Krakow, 31-567

  • NZOZ Przychodnia Specjalistyczna Andrzej Wittek Henryk Rudzki S.C

    Ruda Śląska, 41-709

Other trials in Poland

Norway2 sites

  • Oslo Universitetssykehus HF

    Oslo, 0373

  • Oslo Universitetssykehus HF, Ulleval

    Oslo, 0450

Other trials in Norway

About this study

Official title: A 52 Week, Phase 3 Double-blind, Randomized, Placebo-controlled, Parallel-group Study To Assess The Efficacy, Safety And Tolerability Of Pf-04950615 In Subjects With Heterozygous Familial Hypercholesterolemia

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.

Being tested
Bococizumab (PF-04950615;RN316), Placebo
Keywords
High Risk of cardiovascular events, Heterozygous familial hypercholesterolemia

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Bococizumab (PF-04950615;RN316)Drug

    150 mg every 2 weeks, subcutaneous injection, 12 months

  • PlaceboOther

    subcutaneous injection every 2 weeks for 12 months

Study groups in NCT01968980
Study groupRoleWhat they receive
Bococizumab (PF-04950615;RN316)ExperimentalBococizumab (PF-04950615;RN316)
Receives: Drug: Bococizumab (PF-04950615;RN316)
PlaceboPlacebo comparatorNot described
Receives: Other: Placebo

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Percent Change From Baseline in Low Density Lipoprotein Cholesterol (LDL-C) at Week 12

    Measured: Baseline, Week 12

Secondary outcomes (28)

  1. Percent Change From Baseline in Total Cholesterol (TC) at Week 12

    Measured: Baseline, Week 12

  2. Percent Change From Baseline in Non High Density Lipoprotein Cholesterol (Non HDL-C) at Week 12

    Measured: Baseline, Week 12

  3. Percent Change From Baseline in Apolipoprotein B (ApoB) at Week 12

    Measured: Baseline, Week 12

  4. Percent Change From Baseline in Lipoprotein (a) at Week 12

    Measured: Baseline, Week 12

  5. Percent Change From Baseline in High Density Lipoprotein Cholesterol (HDL-C) at Week 12

    Measured: Baseline, Week 12

  6. Percent Change From Baseline in Low Density Lipoprotein Cholesterol (LDL-C) at Week 24 and 52

    Measured: Baseline, Week 24, 52

  7. Percent Change From Baseline in Total Cholesterol (TC) at Week 24 and 52

    Measured: Baseline, Week 24, 52

  8. Percent Change From Baseline in Non High Density Lipoprotein Cholesterol (Non HDL-C) at Week 24 and 52

    Measured: Baseline, Week 24, 52

  9. Percent Change From Baseline in Apolipoprotein B (ApoB) at Week 24 and 52

    Measured: Baseline, Week 24, 52

  10. Percent Change From Baseline in Lipoprotein (a) at Week 24 and 52

    Measured: Baseline, Week 24, 52

  11. Percent Change From Baseline in High Density Lipoprotein Cholesterol (HDL-C) at Week 24 and 52

    Measured: Baseline, Week 24, 52

  12. Percent Change From Baseline in Triglycerides (TG) at Week 12, 24 and 52

    Measured: Baseline, Week 12, 24, 52

  13. Percent Change From Baseline in Apolipoprotein A-I (ApoA-I) at Week 12, 24 and 52

    Measured: Baseline, Week 12, 24, 52

  14. Percent Change From Baseline in Apolipoprotein A-II (ApoA-II) at Week 12, 24 and 52

    Measured: Baseline, Week 12, 24, 52

  15. Percent Change From Baseline in Very Low Density Lipoprotein Cholesterol (VLDL-C) at Week 12, 24 and 52

    Measured: Baseline, Week 12, 24, 52

  16. Absolute Change From Baseline in Low Density Lipoprotein (LDL-C) at Week 12

    Measured: Baseline, Week 12

  17. Absolute Change From Baseline in Total Cholesterol (TC) at Week 12

    Measured: Baseline, Week 12

  18. Absolute Change From Baseline in Non- High Density Lipoprotein Cholesterol (Non HDL-C) at Week 12

    Measured: Baseline, Week 12

  19. Absolute Change From Baseline in Apolipoprotein B (ApoB) at Week 12

    Measured: Baseline, Week 12

  20. Absolute Change From Baseline in Lipoprotein (a) at Week 12

    Measured: Baseline, Week 12

  21. Absolute Change From Baseline in High Density Lipoprotein Cholesterol (HDL-C) at Week 12

    Measured: Baseline, Week 12

  22. Absolute Change From Baseline in Ratio of Total Cholesterol to High Density Lipoprotein Cholesterol (TC/HDL-C Ratio) at Week 12, 24 and 52

    Measured: Baseline, Week 12, 24, 52

  23. Absolute Change From Baseline in Ratio of Apolipoprotein B to Apolipoprotein A-I (ApoB/ApoA-I Ratio) at Week 12, 24 and 52

    Measured: Baseline, Week 12, 24, 52

  24. 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 52

    Measured: Week 12, 24, 52

  25. 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 52

    Measured: Week 12, 24, 52

  26. Plasma PF-04950615 Concentrations at Week 12, 24 and 52

    Measured: Week 12, 24, 52

  27. Number of Participants With Adverse Events Related to Type 1 or 3 Hypersensitivity Reactions and Injection Site Reactions

    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.

    Measured: Baseline up to the end of study (up to 58 weeks)

  28. Percentage of Participants With Positive Anti-Drug Antibodies (ADA) and Neutralizing Antibodies (nAb)

    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.

    Measured: Baseline up to the end of study (up to 58 weeks)

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted24 Oct 2013 (estimated)
  2. Study start23 Oct 2013
  3. Primary completion15 Apr 2016
  4. Study completion15 Apr 2016

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 3
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
Quadruple blind
Who is masked
Participant, Care provider, Investigator, Outcomes assessor
Enrolment
370 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT01968980
Sponsor's protocol ID
B1481021
Other IDs
2013-002644-87, SPIRE-HF
Acronym
SPIRE-FH
Status last verified
May 2017
First submitted
21 Oct 2013
Last updated
26 Jun 2017
Data monitoring committee
Yes

Publications

  • Wang EQ, Kaila N, Plowchalk D, Gibiansky L, Yunis C, Sweeney K. Population PK/PD modeling of low-density lipoprotein cholesterol response in hypercholesterolemic participants following administration of bococizumab, a potent anti-PCSK9 monoclonal antibody. CPT Pharmacometrics Syst Pharmacol. 2023 Dec;12(12):2013-2026. doi: 10.1002/psp4.13050. Epub 2023 Nov 22.

    PubMed 37994400

  • McCush F, Wang E, Yunis C, Schwartz P, Baltrukonis D. Anti-drug Antibody Magnitude and Clinical Relevance Using Signal to Noise (S/N): Bococizumab Case Study. AAPS J. 2023 Sep 2;25(5):85. doi: 10.1208/s12248-023-00846-x.

    PubMed 37658997

  • Chasman DI, Hyde CL, Giulianini F, Danning RD, Wang EQ, Hickling T, Ridker PM, Loomis AK. Genome-wide pharmacogenetics of anti-drug antibody response to bococizumab highlights key residues in HLA DRB1 and DQB1. Sci Rep. 2022 Mar 11;12(1):4266. doi: 10.1038/s41598-022-07997-5.

    PubMed 35277540

  • Ridker PM, Rose LM, Kastelein JJP, Santos RD, Wei C, Revkin J, Yunis C, Tardif JC, Shear CL; Studies of PCSK9 Inhibition and the Reduction of vascular Events (SPIRE) Investigators. Cardiovascular event reduction with PCSK9 inhibition among 1578 patients with familial hypercholesterolemia: Results from the SPIRE randomized trials of bococizumab. J Clin Lipidol. 2018 Jul-Aug;12(4):958-965. doi: 10.1016/j.jacl.2018.03.088. Epub 2018 Apr 3.

    PubMed 29685591

  • Ridker PM, Tardif JC, Amarenco P, Duggan W, Glynn RJ, Jukema JW, Kastelein JJP, Kim AM, Koenig W, Nissen S, Revkin J, Rose LM, Santos RD, Schwartz PF, Shear CL, Yunis C; SPIRE Investigators. Lipid-Reduction Variability and Antidrug-Antibody Formation with Bococizumab. N Engl J Med. 2017 Apr 20;376(16):1517-1526. doi: 10.1056/NEJMoa1614062. Epub 2017 Mar 17.

    PubMed 28304227

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing Bococizumab (PF-04950615;RN316) in people with Heterozygous Familial Hypercholesterolemia, run by Pfizer at 88 sites in 11 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 4 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 26 Jun 2017. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 88 sites in United States, South Africa, Spain, Canada, Netherlands and 6 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 19 May 2017). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Percent Change From Baseline in Low Density Lipoprotein Cholesterol (LDL-C) at Week 12 (time frame: Baseline, Week 12). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT01968980, which also lists 5 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 26 Jun 2017; AzTrials last refreshed its copy on 6 Nov 2026.

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