HypercholesterolemiaPhase 2NCT01266876

Study of the Safety and Efficacy of REGN727/SAR236553 in Patients With HeFH Hypercholesterolemia

A Phase 2 study testing Alirocumab in people with Hypercholesterolemia, run by Regeneron Pharmaceuticals at 22 sites in 2 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

The purpose of this study is to assess the efficacy and safety of REGN727/SAR236553 in participants diagnosed with heterozygous familial hypercholesterolemia (heFH) Read more about this study

Who is it for?: Ages 18 to 75 years
Conditions the study is for: Hypercholesterolemia
What is being tested?: Alirocumab
Type of treatment: Injection or infusion
Could I receive a placebo?: Yes, some participants may receive a placebo
How many people: 77 participants
Where: 22 sitesin 2 countries
Study dates: Jan 2011to Nov 2011
Phase: Phase 2
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 to 75 years
All sexes
No healthy volunteers
Adult, Older adult

Who may be able to join

Must meet the World Health Organization criteria for heFH
Participants must be on a stable statin dose, with or without ezetimibe, for at least 6 weeks before screening
Serum LDL-C levels ≥ 100 mg/dL at screening
Willing to follow the NCEP ATPIII TLC diet, or an equivalent diet plan, starting at screening and continuing until the last study visit
A negative urine/serum pregnancy test at each screening visit and start of the study, for women of childbearing potential

Who cannot join

Participants with homozygous FH (clinically or by previous genotyping)
Use of a medication (other than a statin or EZE) to alter serum lipids within 42 days (6 weeks) before screening including, but not limited to:
Fibrates
Niacin (>500 mg/day)
Omega-3 fatty acids (>1000 mg/day of DHA/EPA)
Bile acid resins

Show all 15Show fewer

Use of nutraceuticals or OTC medications that may alter lipid levels that are not stable for at least 6 weeks before screening and are not planned to remain constant throughout the study. Examples include:
Omega-3 fatty acids (≤1000 mg/day of DHA/EPA)
Niacin (≤500 mg/day)
Plant stanols, such as found in Benecol, flax seed oil, psyllium
Red yeast rice
Disorders known to influence lipid levels, such as nephrotic syndrome, significant liver disease, Cushing's disease, untreated hypothyroidism (patients on stable thyroid replacement for at least 12 weeks before the full screening visit, who are metabolically euthyroid by thyroid-stimulating hormone (TSH) testing are allowed)
Use of thyroid medications (except for replacement therapy which has been stable for at least 12 weeks before the full screening visit)
Fasting serum TG >350 mg/dL screening
LDL apheresis within 12 months before screening

Trial locations and contacts

22 sites in 2 countries.

Country

City, postcode or hospital

22 sites in 2 countries

United States17 sites

Study site
Auburn, Maine
Study site
Biddeford, Maine
Study site
Bridgeport, Connecticut
Study site
Chicago, Illinois
Study site
Cincinnati, Ohio
Study site
Concord, New Hampshire
Study site
Durham, North Carolina
Study site
Houston, Texas
Study site
Huntsville, Alabama
Study site
Jacksonville, Florida
Study site
Kansas City, Kansas
Study site
Miami, Florida
Study site
Mission Viejo, California
Study site
Nashville, Tennessee
Study site
Newport Beach, California
Study site
Port Orange, Florida
Study site
St Louis, Missouri

Other trials in United States

Canada5 sites

Study site
Chicoutimi, Quebec
Study site
London, Ontario
Study site
Montreal, Quebec
Study site
Saint Foy, Quebec
Study site
Winnipeg, Manitoba

Other trials in Canada

About this study

Official title: A Randomized, Double-Blind, Placebo-Controlled, 12-Week Study of the Safety and Efficacy of REGN727 in Patients With Heterozygous Familial Hypercholesterolemia

The purpose of this study is to assess the efficacy and safety of REGN727/SAR236553 in participants diagnosed with heterozygous familial hypercholesterolemia (heFH)

Conditions: Hypercholesterolemia
Being tested: Alirocumab, Placebo
Medical subject headings: Hypercholesterolemia

What participants receive

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

AlirocumabDrug
Also called REGN727/SAR236553
Alirocumab two SC injections in the abdomen only.
PlaceboDrug
Placebo two SC injections in the abdomen only.

Study groups in NCT01266876
Study group	Role	What they receive
Placebo	Placebo comparator	Placebo SC injection Q2W added to stable statin regimen with or without concomitant ezetimibe for 12 weeks. Receives: Drug: Placebo
Alirocumab 150 mg Q4W	Experimental	Alirocumab 150 mg SC injection Q4W added to stable statin regimen with or without concomitant ezetimibe for 12 weeks. Receives: Drug: Alirocumab
Alirocumab 200 mg Q4W	Experimental	Alirocumab 200 mg SC injection Q4W added to stable statin regimen with or without concomitant ezetimibe for 12 weeks. Receives: Drug: Alirocumab
Alirocumab 300 mg Q4W	Experimental	Alirocumab 300 mg SC injection Q4W added to stable statin regimen with or without concomitant ezetimibe for 12 weeks. Receives: Drug: Alirocumab
Alirocumab 150 mg Q2W	Experimental	Alirocumab 150 mg SC injection Q2W added to stable statin regimen with or without concomitant ezetimibe for 12 weeks. Receives: Drug: Alirocumab

What is being measured

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

Percent Change From Baseline in Calculated LDL-C at Week 12 - On-treatment Analysis
Calculated LDL-C values were obtained using the Friedewald formula. Baseline adjusted least squares (LS) means and standard errors were estimated using an analysis of covariance (ANCOVA) model including available post-baseline data on treatment from first investigational medicinal product (IMP) injection up to 21 days after last IMP injection (on-treatment analysis). Missing Week 12 data were imputed by last observation carried forward [LOCF] method.
Measured: From Baseline to Week 12 (LOCF)

Secondary outcomes (18)

Absolute Change From Baseline in Calculated LDL-C at Week 12 - On-treatment Analysis
Calculated LDL-C value was obtained from Friedewald formula. Adjusted LS means and standard errors were estimated using the same ANCOVA model as for primary endpoint.
Measured: From Baseline to Week 12 (LOCF)
Percentage of Participants Achieving Calculated LDL-C <100 mg/dL (2.59 mmol/L) at Week 12 - On-treatment Analysis
Calculated LDL-C value was obtained from Friedewald formula.
Measured: Week 12 (LOCF)
Percentage of Participants Achieving LDL-C < 70 mg/dL (1.81 mmol/L) at Week 12 - On-treatment Analysis
Calculated LDL-C value was obtained from Friedewald formula.
Measured: Week 12 (LOCF)
Percent Change From Baseline in Total Cholesterol at Week 12 - On-treatment Analysis
Adjusted LS means and standard errors were estimated using the same ANCOVA model as for primary endpoint.
Measured: From Baseline to Week 12 (LOCF)
Absolute Change From Baseline in Total Cholesterol at Week 12 - On-treatment Analysis
Adjusted LS means and standard errors were estimated using the same ANCOVA model as for primary endpoint.
Measured: From Baseline to Week 12 (LOCF)
Percent Change From Baseline in High Density Lipoprotein Cholesterol (HDL-C) at Week 12 - On-treatment Analysis
Adjusted LS means and standard errors were estimated using the same ANCOVA model as for primary endpoint..
Measured: From Baseline to Week 12 (LOCF)
Absolute Change From Baseline in HDL-C at Week 12 - On-treatment Analysis
Adjusted LS means and standard errors were estimated using the same ANCOVA model as for primary endpoint.
Measured: From Baseline to Week 12 (LOCF)
Percent Change From Baseline in Triglycerides at Week 12 - On-treatment Analysis
Since the assumptions of normal distribution and equality of variances were not verified for the lipid parameter, percent changes were expressed as median (interquartile range)
Measured: From Baseline to Week 12 (LOCF)
Absolute Change From Baseline in Triglycerides at Week at 12 - On-treatment Analysis
Since the assumptions of normal distribution and equality of variances were not verified for the lipid parameters, percent changes were expressed as median (interquartile range)
Measured: From Baseline to Week 12 (LOCF)
Percent Change From Baseline in Non-HDL-C at Week 12 - On-treatment Analysis
Adjusted LS means and standard errors were estimated using the same ANCOVA model as for primary endpoint.
Measured: From Baseline to Week 12
Absolute Change From Baseline in Non-HDL-C at Week 12 - On-treatment Analysis
Adjusted LS means and standard errors were estimated using the same ANCOVA model as for primary endpoint.
Measured: From Baseline to Week 12 (LOCF)
Percent Change From Baseline in Apo Lipoprotein B (Apo-B) at Week 12 - On-treatment Analysis
Adjusted LS means and standard errors were estimated using the same ANCOVA model as for primary endpoint.
Measured: From Baseline to Week 12 (LOCF)
Absolute Change From Baseline in Apo-B at Week 12 - On-treatment Analysis
Adjusted LS means and standard errors were estimated using the same ANCOVA model as for primary endpoint.
Measured: From Baseline to Week 12
Percent Change From Baseline in Apolipoprotein - A1 (Apo-A1) at Week 12 - On-treatment Analysis
Adjusted LS means and standard errors were estimated using the same ANCOVA model as for primary endpoint.
Measured: From Baseline to Week 12 (LOCF)
Absolute Change From Baseline in Apo-A1 at Week 12 - On-treatment Analysis
Adjusted LS means and standard errors were estimated using the same ANCOVA model as for primary endpoint.
Measured: From Baseline to Week 12 (LOCF)
Absolute Change in the Ratio ApoB/ApoA-1 From Baseline to Week 12 - On-treatment Analysis
Adjusted LS means and standard errors were estimated using the same ANCOVA model as for primary endpoint.
Measured: From Baseline to Week 12
Percent Change From Baseline in Lipoprotein(a) at Week 12 - On-treatment Analysis
Since the assumptions of normal distribution and equality of variances were not verified for the lipid parameters, percent changes were expressed as median (interquartile range)
Measured: From Baseline to Week 12 (LOCF)
Absolute Change From Baseline in Lipoprotein(a) at Week 12 - On-treatment Analysis
Since the assumptions of normal distribution and equality of variances were not verified for the lipid parameter, percent changes were expressed as median (interquartile range)
Measured: From Baseline to Week 12 (LOCF)

Timeline

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

Jan 2011Nov 2011

First posted24 Dec 2010 (estimated)
Study startJan 2011
Primary completionNov 2011
Study completionNov 2011

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Publications (4)Papers the sponsor has linked to this study.
Other trials for HypercholesterolemiaStudies of the same condition, open and under way.
More trials run by Regeneron PharmaceuticalsThe sponsor's other registered studies.
What the trial phases meanHow a treatment moves from first tests in people to approval.

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 2
Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: Triple blind
Who is masked: Participant, Investigator, Outcomes assessor
Enrolment: 77 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT01266876
Sponsor's protocol ID: R727-CL-1003
Status last verified: Aug 2015
First submitted: 23 Dec 2010
Last updated: 22 Sep 2015
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: Yes

Publications

Leiter LA, Tinahones FJ, Karalis DG, Bujas-Bobanovic M, Letierce A, Mandel J, Samuel R, Jones PH. Alirocumab safety in people with and without diabetes mellitus: pooled data from 14 ODYSSEY trials. Diabet Med. 2018 Dec;35(12):1742-1751. doi: 10.1111/dme.13817. Epub 2018 Oct 9.
PubMed 30183102
Defesche JC, Stefanutti C, Langslet G, Hopkins PN, Seiz W, Baccara-Dinet MT, Hamon SC, Banerjee P, Kastelein JJP. Efficacy of alirocumab in 1191 patients with a wide spectrum of mutations in genes causative for familial hypercholesterolemia. J Clin Lipidol. 2017 Nov-Dec;11(6):1338-1346.e7. doi: 10.1016/j.jacl.2017.08.016. Epub 2017 Sep 4.
PubMed 28964736
Toth PP, Hamon SC, Jones SR, Martin SS, Joshi PH, Kulkarni KR, Banerjee P, Hanotin C, Roth EM, McKenney JM. Effect of alirocumab on specific lipoprotein non-high-density lipoprotein cholesterol and subfractions as measured by the vertical auto profile method: analysis of 3 randomized trials versus placebo. Lipids Health Dis. 2016 Feb 13;15:28. doi: 10.1186/s12944-016-0197-4.
PubMed 26872608
Stein EA, Gipe D, Bergeron J, Gaudet D, Weiss R, Dufour R, Wu R, Pordy R. Effect of a monoclonal antibody to PCSK9, REGN727/SAR236553, to reduce low-density lipoprotein cholesterol in patients with heterozygous familial hypercholesterolaemia on stable statin dose with or without ezetimibe therapy: a phase 2 randomised controlled trial. Lancet. 2012 Jul 7;380(9836):29-36. doi: 10.1016/S0140-6736(12)60771-5. Epub 2012 May 26.
PubMed 22633824

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

Active, not recruitingObservational study
Study Evaluating LDL-C Change and Adherence to Inclisiran Lipid-lowering Therapy in ASCVD
Primary Hypercholesterolemia, Mixed Dyslipidemia
Active, not recruitingPhase 3
A Study of Inclisiran to Prevent Cardiovascular Events in High-risk Primary Prevention Patients.
Primary Prevention of Atherosclerotic Cardiovascular Disease
RecruitingObservational study
Causes and Natural History of Dyslipidemias
Hypercholesterolemia, Atherosclerosis
Active, not recruitingObservational study
Early Detection of Familial Hypercholesterolaemia in Pre-school Children ( CzeCH-IT)
Familial Hypercholesterolaemia
Not yet recruiting
Penn Family Screening for Familial Hypercholesterolemia (NHLBI R33) Trial 3
Familial Hypercholesterolemia

All Hypercholesterolemia trials

Study at a glance

From the registry record, last updated by the sponsor on 22 Sep 2015. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: A Randomized, Double-Blind, Placebo-Controlled, 12-Week Study of the Safety and Efficacy of REGN727 in Patients With Heterozygous Familial Hypercholesterolemia
ClinicalTrials.gov ID: NCT01266876
Acronym / sponsor ID: R727-CL-1003
Conditions: Hypercholesterolemia
Phase: Phase 2
Study type: Interventional
Recruitment status: Completed
Enrolment: 77 participants (actual)
Lead sponsor: Regeneron PharmaceuticalsIndustry (pharma, biotech, devices)
Collaborators: Sanofi
Study start: Jan 2011
Primary completion: Nov 2011 (actual)
Study completion: Nov 2011 (actual)
First posted: 24 Dec 2010 (estimated)
Results: Posted 22 Sep 2015

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

The purpose of this study is to assess the efficacy and safety of REGN727/SAR236553 in participants diagnosed with heterozygous familial hypercholesterolemia (heFH)

Study design

Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: Triple blind (participant, investigator, outcomes assessor)
Number of arms: 5

Arm	Type	Receives
Placebo	Placebo comparator	Drug: PlaceboPlacebo SC injection Q2W added to stable statin regimen with or without concomitant ezetimibe for 12 weeks.
Alirocumab 150 mg Q4W	Experimental	Drug: AlirocumabAlirocumab 150 mg SC injection Q4W added to stable statin regimen with or without concomitant ezetimibe for 12 weeks.
Alirocumab 200 mg Q4W	Experimental	Drug: AlirocumabAlirocumab 200 mg SC injection Q4W added to stable statin regimen with or without concomitant ezetimibe for 12 weeks.
Alirocumab 300 mg Q4W	Experimental	Drug: AlirocumabAlirocumab 300 mg SC injection Q4W added to stable statin regimen with or without concomitant ezetimibe for 12 weeks.
Alirocumab 150 mg Q2W	Experimental	Drug: AlirocumabAlirocumab 150 mg SC injection Q2W added to stable statin regimen with or without concomitant ezetimibe for 12 weeks.

Who may qualify

Ages: 18 to 75 years
Sex: All
Healthy volunteers: Not accepted
Age groups: Adult, Older adult

Key inclusion criteria (5 listed)

Must meet the World Health Organization criteria for heFH
Participants must be on a stable statin dose, with or without ezetimibe, for at least 6 weeks before screening
Serum LDL-C levels ≥ 100 mg/dL at screening
Willing to follow the NCEP ATPIII TLC diet, or an equivalent diet plan, starting at screening and continuing until the last study visit
A negative urine/serum pregnancy test at each screening visit and start of the study, for women of childbearing potential

Key exclusion criteria (15 listed)

Participants with homozygous FH (clinically or by previous genotyping)
Use of a medication (other than a statin or EZE) to alter serum lipids within 42 days (6 weeks) before screening including, but not limited to:
Use of nutraceuticals or OTC medications that may alter lipid levels that are not stable for at least 6 weeks before screening and are not planned to remain constant throughout the study. Examples include:
Disorders known to influence lipid levels, such as nephrotic syndrome, significant liver disease, Cushing's disease, untreated hypothyroidism (patients on stable thyroid replacement for at least 12 weeks before the full screening visit, who are metabolically euthyroid by thyroid-stimulating hormone (TSH) testing are allowed)
Use of thyroid medications (except for replacement therapy which has been stable for at least 12 weeks before the full screening visit)

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Intervention	Type	Arms
AlirocumabAlso: REGN727/SAR236553Alirocumab two SC injections in the abdomen only.	Drug	Alirocumab 150 mg Q2W, Alirocumab 150 mg Q4W, Alirocumab 200 mg Q4W, Alirocumab 300 mg Q4W
PlaceboPlacebo two SC injections in the abdomen only.	Drug	Placebo

Primary outcome

Percent Change From Baseline in Calculated LDL-C at Week 12 - On-treatment Analysis
Time frame: From Baseline to Week 12 (LOCF)
Calculated LDL-C values were obtained using the Friedewald formula. Baseline adjusted least squares (LS) means and standard errors were estimated using an analysis of covariance (ANCOVA) model including available post-baseline data on treatment from first investigational medicinal product (IMP) injection up to 21 days after last IMP injection (on-treatment analysis). Missing Week 12 data were imputed by last observation carried forward [LOCF] method.

18 secondary outcomes

Absolute Change From Baseline in Calculated LDL-C at Week 12 - On-treatment Analysis
Time frame: From Baseline to Week 12 (LOCF)
Percentage of Participants Achieving Calculated LDL-C <100 mg/dL (2.59 mmol/L) at Week 12 - On-treatment Analysis
Time frame: Week 12 (LOCF)
Percentage of Participants Achieving LDL-C < 70 mg/dL (1.81 mmol/L) at Week 12 - On-treatment Analysis
Time frame: Week 12 (LOCF)
Percent Change From Baseline in Total Cholesterol at Week 12 - On-treatment Analysis
Time frame: From Baseline to Week 12 (LOCF)
Absolute Change From Baseline in Total Cholesterol at Week 12 - On-treatment Analysis
Time frame: From Baseline to Week 12 (LOCF)
Percent Change From Baseline in High Density Lipoprotein Cholesterol (HDL-C) at Week 12 - On-treatment Analysis
Time frame: From Baseline to Week 12 (LOCF)
Absolute Change From Baseline in HDL-C at Week 12 - On-treatment Analysis
Time frame: From Baseline to Week 12 (LOCF)
Percent Change From Baseline in Triglycerides at Week 12 - On-treatment Analysis
Time frame: From Baseline to Week 12 (LOCF)
Absolute Change From Baseline in Triglycerides at Week at 12 - On-treatment Analysis
Time frame: From Baseline to Week 12 (LOCF)
Percent Change From Baseline in Non-HDL-C at Week 12 - On-treatment Analysis
Time frame: From Baseline to Week 12
Absolute Change From Baseline in Non-HDL-C at Week 12 - On-treatment Analysis
Time frame: From Baseline to Week 12 (LOCF)
Percent Change From Baseline in Apo Lipoprotein B (Apo-B) at Week 12 - On-treatment Analysis
Time frame: From Baseline to Week 12 (LOCF)
Absolute Change From Baseline in Apo-B at Week 12 - On-treatment Analysis
Time frame: From Baseline to Week 12
Percent Change From Baseline in Apolipoprotein - A1 (Apo-A1) at Week 12 - On-treatment Analysis
Time frame: From Baseline to Week 12 (LOCF)
Absolute Change From Baseline in Apo-A1 at Week 12 - On-treatment Analysis
Time frame: From Baseline to Week 12 (LOCF)
Absolute Change in the Ratio ApoB/ApoA-1 From Baseline to Week 12 - On-treatment Analysis
Time frame: From Baseline to Week 12
Percent Change From Baseline in Lipoprotein(a) at Week 12 - On-treatment Analysis
Time frame: From Baseline to Week 12 (LOCF)
Absolute Change From Baseline in Lipoprotein(a) at Week 12 - On-treatment Analysis
Time frame: From Baseline to Week 12 (LOCF)

Results and safety

Results posted to the registry on 22 Sep 2015, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Placebo	15	15
Alirocumab 150 mg Q4W	15	15
Alirocumab 200 mg Q4W	16	16
Alirocumab 300 mg Q4W	15	14
Alirocumab 150 mg Q2W	16	16

Primary outcome: Percent Change From Baseline in Calculated LDL-C at Week 12 - On-treatment Analysis

Time frame: From Baseline to Week 12 (LOCF). Least squares mean (Standard Error). Unit: percent change

Measure	Placebo n = 15	Alirocumab 150 mg Q4W n = 15	Alirocumab 200 mg Q4W n = 16	Alirocumab 300 mg Q4W n = 15	Alirocumab 150 mg Q2W n = 16
Value	-10.7 (5.0)	-28.9 (5.1)	-31.5 (4.9)	-42.5 (5.1)	-67.9 (4.9)

Placebo vs Alirocumab 150 mg Q2W: p = 0.0000, ANCOVA
Placebo vs Alirocumab 300 mg Q4W: p = 0.0000, ANCOVA
Placebo vs Alirocumab 200 mg Q4W: p = 0.0035, ANCOVA

Population analysed: Modified Intent-To-Treat (mITT) population included all randomized participants with one baseline and at least one post baseline on-treatment calculated LDL-C.

18 secondary outcomes are reported in the full results.

Adverse events

Time frame: From Baseline up to Week 12. Other adverse events reported at 5% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Placebo	1 of 15 (6.7%)	9 of 15 (60%)	Not reported
Alirocumab 150 mg Q4W	0 of 15 (0%)	12 of 15 (80%)	Not reported
Alirocumab 200 mg Q4W	0 of 16 (0%)	13 of 16 (81.3%)	Not reported
Alirocumab 300 mg Q4W	0 of 15 (0%)	13 of 15 (86.7%)	Not reported
Alirocumab 150 mg Q2W	0 of 16 (0%)	12 of 16 (75%)	Not reported

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 22 Sep 2015.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Derived from this study: PubMed 30183102Leiter LA, Tinahones FJ, Karalis DG, Bujas-Bobanovic M, Letierce A, Mandel J, Samuel R, Jones PH. Alirocumab safety in people with and without diabetes mellitus: pooled data from 14 ODYSSEY trials. Diabet Med. 2018 Dec;35(12):1742-1751. doi: 10.1111/dme.13817. Epub 2018 Oct 9.
Derived from this study: PubMed 28964736Defesche JC, Stefanutti C, Langslet G, Hopkins PN, Seiz W, Baccara-Dinet MT, Hamon SC, Banerjee P, Kastelein JJP. Efficacy of alirocumab in 1191 patients with a wide spectrum of mutations in genes causative for familial hypercholesterolemia. J Clin Lipidol. 2017 Nov-Dec;11(6):1338-1346.e7. doi: 10.1016/j.jacl.2017.08.016. Epub 2017 Sep 4.
Derived from this study: PubMed 26872608Toth PP, Hamon SC, Jones SR, Martin SS, Joshi PH, Kulkarni KR, Banerjee P, Hanotin C, Roth EM, McKenney JM. Effect of alirocumab on specific lipoprotein non-high-density lipoprotein cholesterol and subfractions as measured by the vertical auto profile method: analysis of 3 randomized trials versus placebo. Lipids Health Dis. 2016 Feb 13;15:28. doi: 10.1186/s12944-016-0197-4.
Derived from this study: PubMed 22633824Stein EA, Gipe D, Bergeron J, Gaudet D, Weiss R, Dufour R, Wu R, Pordy R. Effect of a monoclonal antibody to PCSK9, REGN727/SAR236553, to reduce low-density lipoprotein cholesterol in patients with heterozygous familial hypercholesterolaemia on stable statin dose with or without ezetimibe therapy: a phase 2 randomised controlled trial. Lancet. 2012 Jul 7;380(9836):29-36. doi: 10.1016/S0140-6736(12)60771-5. Epub 2012 May 26.

Related trials for Hypercholesterolemia

Active, not recruitingObservational study
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Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.

Clinical Trial Management study director

Regeneron Pharmaceuticals

22 sites in 2 countries: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Alirocumab in people with Hypercholesterolemia, run by Regeneron Pharmaceuticals at 22 sites in 2 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 5 inclusion and 15 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 22 Sep 2015. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 22 sites in United States, Canada. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

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 22 Sep 2015). 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 Calculated LDL-C at Week 12 - On-treatment Analysis (time frame: From Baseline to Week 12 (LOCF)). 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 NCT01266876, which also lists 4 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 22 Sep 2015.