NCT01604824 is a Phase 2 clinical trial of Alirocumab in Hypercholesterolemia, sponsored by Regeneron Pharmaceuticals.

Who is sponsoring NCT01604824?

NCT01604824 is sponsored by Regeneron Pharmaceuticals.

What drugs are being tested in NCT01604824?

Interventions in NCT01604824: Alirocumab, Placebo.

What condition does NCT01604824 study?

NCT01604824 studies Hypercholesterolemia.

Is NCT01604824 still recruiting?

NCT01604824 is currently completed. Last updated 17 June 2020.

Are results published for NCT01604824?

Yes — primary outcome results have been posted to ClinicalTrials.gov. See the outcomes section above for details.

Last reviewed 2020-06-17 · How we verify

NCT01604824

Name: Drug Landscape Pharmaceutical Database
Creator: Drug Landscape
Published: 2026-01-01
License: https://creativecommons.org/licenses/by/4.0/

A Study of Alirocumab in Participants With Autosomal Dominant Hypercholesterolemia (ADH) and Gain-of-Function Mutations (GOFm) of the Proprotein Convertase Subtilisin Kexin 9 (PCSK9) Gene or Loss-of-Function Mutations (LOFm) of the Apolipoprotein (Apo) B Gene

Completed Phase 2 Results posted Last updated 17 June 2020

What this trial tests

Phase 2 trial testing Alirocumab in Hypercholesterolemia in 23 participants. Completed in 28 July 2017.

Timeline

22 February 2012

Primary endpoint
2 June 2014

28 July 2017

Quick facts

Lead sponsor	Regeneron Pharmaceuticals
Phase	Phase 2
Status	Completed
Study type	INTERVENTIONAL
Allocation	randomized
Design	parallel
Masking	triple
Primary purpose	treatment
Enrollment	23
Start date	22 February 2012
Primary completion	2 June 2014
Estimated completion	28 July 2017
Sites	4 locations across France, United States

Drugs / interventions tested

Alirocumab (ALIROCUMAB) — full drug profile →
Placebo

Conditions studied

Hypercholesterolemia — all drugs for Hypercholesterolemia →

Sponsor

Regeneron Pharmaceuticals — full company profile →

Who can join

Adults 18 to 70, any sex, with Hypercholesterolemia. Patients with the condition only — healthy volunteers not accepted.

Results — posted to ClinicalTrials.gov

Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.

Percent Change in Measured Serum Low-Density Lipoprotein Cholesterol (LDL-C) From Baseline to Day 15 Primary · Baseline to Day 15

By day 15, participants in groups A and C had received 1 subcutaneous (SC) dose of 150 mg alirocumab and participants in group B and D had received 1 SC dose of placebo. \[Baseline adjusted least squares (LS) means and standard errors were obtained using analysis of covariance (ANCOVA) model specifying the treatment arm as the fixed effect and the baseline measured LDL-C value as a covariate.\]

Group	Value	95% CI
PCSK9 GOFm: Alirocumab From Day 1 (Cohort 1: Group A)	-62.48	± 8.217
PCSK9 GOFm: Alirocumab From Day 15 (Cohort 1: Group B)	-8.77	± 7.575
PCSK9 GOFm/ApoB LOFm: Alirocumab From Day1 (Cohort 2: Group C)	-48.21	± 7.660
PCSK9GOFm/ApoB LOFm: Alirocumab From Day 15(Cohort 2: Group D)	-4.93	± 7.660

Percent Change in Apolipoprotein (Apo) B100 From Baseline to Day 15 Secondary · Baseline to Day 15

Baseline adjusted LS means and standard errors were obtained using the same ANCOVA model as for primary endpoint specifying the treatment arm as the fixed effect and the parameter value as a covariate.

Group	Value	95% CI
PCSK9 GOFm: Alirocumab From Day 1 (Cohort 1: Group A)	-53.33	± 8.678
PCSK9 GOFm: Alirocumab From Day 15 (Cohort 1: Group B)	-3.78	± 8.008
PCSK9 GOFm/ApoB LOFm: Alirocumab From Day1 (Cohort 2: Group C)	-47.73	± 7.547
PCSK9GOFm/ApoB LOFm: Alirocumab From Day 15(Cohort 2: Group D)	-3.09	± 7.547

Percent Change in Non High-Density Lipoprotein Cholesterol (Non-HDL-C) From Baseline to Day 15 Secondary · Baseline to Day 15

Group	Value	95% CI
PCSK9 GOFm: Alirocumab From Day 1 (Cohort 1: Group A)	-56.87	± 8.217
PCSK9 GOFm: Alirocumab From Day 15 (Cohort 1: Group B)	-7.50	± 7.575
PCSK9 GOFm/ApoB LOFm: Alirocumab From Day1 (Cohort 2: Group C)	-44.40	± 7.357
PCSK9GOFm/ApoB LOFm: Alirocumab From Day 15(Cohort 2: Group D)	-4.04	± 7.357

Percent Change in Total Cholesterol (Total-C) From Baseline to Day 15 Secondary · Baseline to Day 15

Group	Value	95% CI
PCSK9 GOFm: Alirocumab From Day 1 (Cohort 1: Group A)	-36.94	± 5.203
PCSK9 GOFm: Alirocumab From Day 15 (Cohort 1: Group B)	-6.18	± 4.802
PCSK9 GOFm/ApoB LOFm: Alirocumab From Day1 (Cohort 2: Group C)	-29.40	± 4.422
PCSK9GOFm/ApoB LOFm: Alirocumab From Day 15(Cohort 2: Group D)	-7.18	± 4.422

Percent Change in Apolipoprotein (Apo) B100/ ApoA-1 Ratio From Baseline to Day 15 Secondary · Baseline to Day 15

Group	Value	95% CI
PCSK9 GOFm: Alirocumab From Day 1 (Cohort 1: Group A)	-55.26	± 7.188
PCSK9 GOFm: Alirocumab From Day 15 (Cohort 1: Group B)	-5.53	± 6.647
PCSK9 GOFm/ApoB LOFm: Alirocumab From Day1 (Cohort 2: Group C)	-48.34	± 8.090
PCSK9GOFm/ApoB LOFm: Alirocumab From Day 15(Cohort 2: Group D)	0.99	± 8.090

Adverse events — posted to ClinicalTrials.gov

Time frame: From the screening visit after the last alirocumab injection in the open-label period + 70 days through the end of study. Reporting threshold: 5%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.

PCSK9 GOFm: Alirocumab From Day 1 (Cohort 1: Group A)

Serious: 1/6 (17%)

Deaths: 0/6

PCSK9 GOFm: Alirocumab From Day 15 (Cohort 1: Group B)

Serious: 0/7 (0%)

Deaths: 0/7

PCSK9 GOFm/ApoB LOFm: Alirocumab From Day1 (Cohort 2: Group C)

Serious: 0/5 (0%)

Deaths: 0/5

PCSK9GOFm/ApoB LOFm: Alirocumab From Day 15(Cohort 2:Group D)

Serious: 0/5 (0%)

Deaths: 0/5

OLE Period: PCSK9 GOFm (Cohort 1) Group A & Group B

Serious: 3/11 (27%)

Deaths: 0/11

OLE Period: PCSK9 GOFm/ ApoB LOFm (Cohort 2) Group C & Group D

Serious: 0/10 (0%)

Deaths: 0/10

Serious adverse events (5 terms)

Reaction	System	PCSK9 GOFm: Alirocumab Fro…	PCSK9 GOFm: Alirocumab Fro…	PCSK9 GOFm/ApoB LOFm: Alir…	PCSK9GOFm/ApoB LOFm: Aliro…	OLE Period: PCSK9 GOFm (Co…	OLE Period: PCSK9 GOFm/ Ap…
Chest pain	General disorders	—	—	—	—	—	—
Angina unstable	Cardiac disorders	—	—	—	—	—	—
Myocardial ischaemia	Cardiac disorders	—	—	—	—	—	—
Salivary gland disorder	Gastrointestinal disorders	—	—	—	—	—	—
Obesity	Metabolism and nutrition disorders	—	—	—	—	—	—

Other adverse events (100 terms — click to expand)

Reaction	System	PCSK9 GOFm: Alirocumab Fro…	PCSK9 GOFm: Alirocumab Fro…	PCSK9 GOFm/ApoB LOFm: Alir…	PCSK9GOFm/ApoB LOFm: Aliro…	OLE Period: PCSK9 GOFm (Co…	OLE Period: PCSK9 GOFm/ Ap…
Viral upper respiratory tract infection	Infections and infestations	—	—	—	—	—	—
Angina pectoris	Cardiac disorders	—	—	—	—	—	—
Gastroenteritis	Infections and infestations	—	—	—	—	—	—
Rhinitis	Infections and infestations	—	—	—	—	—	—
Upper respiratory tract infection	Infections and infestations	—	—	—	—	—	—
Arthralgia	Musculoskeletal and connective tissue disorders	—	—	—	—	—	—
Headache	Nervous system disorders	—	—	—	—	—	—
Cough	Respiratory, thoracic and mediastinal disorders	—	—	—	—	—	—
Asthenia	General disorders	—	—	—	—	—	—
Urinary tract infection	Infections and infestations	—	—	—	—	—	—
Vertigo	Ear and labyrinth disorders	—	—	—	—	—	—
Dry eye	Eye disorders	—	—	—	—	—	—
Ocular hyperaemia	Eye disorders	—	—	—	—	—	—
Abdominal discomfort	Gastrointestinal disorders	—	—	—	—	—	—
Abdominal distension	Gastrointestinal disorders	—	—	—	—	—	—
Abdominal pain	Gastrointestinal disorders	—	—	—	—	—	—
Haemorrhoids	Gastrointestinal disorders	—	—	—	—	—	—
Constipation	Gastrointestinal disorders	—	—	—	—	—	—
Chest pain	General disorders	—	—	—	—	—	—
Herpes zoster	Infections and infestations	—	—	—	—	—	—
Influenza	Infections and infestations	—	—	—	—	—	—
Lower respiratory tract infection	Infections and infestations	—	—	—	—	—	—
Nasopharyngitis	Infections and infestations	—	—	—	—	—	—
Oral herpes	Infections and infestations	—	—	—	—	—	—
Pneumonia	Infections and infestations	—	—	—	—	—	—
Blood bilirubin increased	Investigations	—	—	—	—	—	—
Haemoglobin decreased	Investigations	—	—	—	—	—	—
Musculoskeletal pain	Musculoskeletal and connective tissue disorders	—	—	—	—	—	—
Back pain	Musculoskeletal and connective tissue disorders	—	—	—	—	—	—
Pain in extremity	Musculoskeletal and connective tissue disorders	—	—	—	—	—	—
Migraine	Nervous system disorders	—	—	—	—	—	—
Neuropathy peripheral	Nervous system disorders	—	—	—	—	—	—
Paraesthesia	Nervous system disorders	—	—	—	—	—	—
Sciatica	Nervous system disorders	—	—	—	—	—	—
Oropharyngeal pain	Respiratory, thoracic and mediastinal disorders	—	—	—	—	—	—
Eczema	Skin and subcutaneous tissue disorders	—	—	—	—	—	—
Acne	Skin and subcutaneous tissue disorders	—	—	—	—	—	—
Raynaud's phenomenon	Vascular disorders	—	—	—	—	—	—
Rash	Skin and subcutaneous tissue disorders	—	—	—	—	—	—
Ear pruritus	Ear and labyrinth disorders	—	—	—	—	—	—

Most-reported serious reactions: Chest pain, Angina unstable, Myocardial ischaemia, Salivary gland disorder, Obesity.

Data from ClinicalTrials.gov NCT01604824 adverse events section.

Sponsor's own description

The primary objective of the study is to assess the pharmacodynamic (PD) effect of alirocumab on serum low density lipoprotein cholesterol (LDL-C) during 14 weeks of subcutaneous (SC) administered alirocumab in patients with autosomal dominant hypercholesterolemia (ADH) and gain-of-function mutation (GOFm) in 1 or both alleles of the proprotein convertase subtilisin/kexin type 9 (PCSK9) gene or with loss-of-function mutation (LOFm) in 1 or more alleles of the apolipoprotein (ApoB) gene.

Publications & conference data

3 peer-reviewed publications reference this trial (live from Europe PMC):

Characterization of Autosomal Dominant Hypercholesterolemia Caused by PCSK9 Gain of Function Mutations and Its Specific Treatment With Alirocumab, a PCSK9 Monoclonal Antibody.
Hopkins PN, Defesche J, Fouchier SW, Bruckert E, et al · · 2015 · cited 86× · PMID 26374825 · DOI 10.1161/circgenetics.115.001129
Pharmacokinetic and pharmacodynamic assessment of alirocumab in patients with familial hypercholesterolemia associated with proprotein convertase subtilisin/kexin type 9 gain-of-function or apolipoprotein B loss-of-function mutations.
Hopkins PN, Krempf M, Bruckert E, Donahue S, et al · · 2019 · cited 9× · PMID 31767518 · DOI 10.1016/j.jacl.2019.10.007
Efficacy and Safety of Alirocumab in Patients With Autosomal Dominant Hypercholesterolemia Associated With Proprotein Convertase Subtilisin/Kexin Type 9 Gain-of-Function or Apolipoprotein B Loss-of-Function Mutations.
Krempf M, Hopkins PN, Bruckert E, Lee S, et al · · 2020 · cited 3× · PMID 31932084 · DOI 10.1016/j.amjcard.2019.12.028

Verify or expand the search:

Other trials of Alirocumab

Trials testing the same drug.

NCT07477704 — A Study to See How Safe and Effective Alirocumab is When Given Weekly to Adult Participants Who Have Hypercholesterolemi · Phase 2 · not yet recruiting
NCT06858332 — Lipoprotein(a) Levels in Patients With Atherosclerotic Cardiovascular Diseases in Russia · recruiting
NCT06385262 — TOP 2301: Neoadjuvant Chemo for NSCLC · Phase 2 · suspended
NCT06080256 — Extra Alirocumab in Addition to Statin Therapy in Asymptomatic Intracranial Atherosclerotic Stenosis (EAST-aICAS) · Phase 3 · unknown
NCT06083961 — The Effect of Early Administration of PCSK9 Inhibitor to Acute Ischemic Stroke Patients Associated With Atherosclerosis · Phase 4 · not yet recruiting

Other recruiting trials for Hypercholesterolemia

Currently open trials in the same condition.

NCT07406191 — WB-EMS Effects on Cardiometabolic Risk Factors · NA · recruiting
NCT07374861 — Multicenter Study on the Evaluation of Adherence, Persistence and Efficacy of Treatment With Bempedoic Acid in Italy · recruiting
NCT07295327 — Effect of Two Food Supplements on Lipid Profile in Patients With Mild Hypercholesterolemia · NA · recruiting
NCT06568601 — Pharmacogenomic Informed Statin Prescribing · NA · recruiting
NCT06423365 — A Tool to Help Patients With Muscle Symptoms After Taking a Statin Medication. · NA · active not recruiting

Other Regeneron Pharmaceuticals trials

Trials by the same sponsor.

NCT07428369 — A Study of Linvo-VR vs DVRd in Transplant-Eligible Adult Participants With Newly Diagnosed Multiple Myeloma (NDMM) · Phase 2, PHASE3 · not yet recruiting
NCT07526116 — A First in Human Study to Assess Safety, Tolerability and Pharmacokinetics of a Single Dose of REGN22044 in Healthy Adul · Phase 1 · not yet recruiting
NCT07527923 — First-in-Human Trial to Assess REGN20423 in Healthy Adult Participants and Adult Participants With Atopic Dermatitis · Phase 1 · not yet recruiting
NCT07527910 — A Phase 2a Study of ALN-PNP With and Without a GLP1R Agonist in Adult Patients With Homozygous PNPLA3-Related MASLD · Phase 2 · not yet recruiting
NCT07477704 — A Study to See How Safe and Effective Alirocumab is When Given Weekly to Adult Participants Who Have Hypercholesterolemi · Phase 2 · not yet recruiting

Verify against primary sources

ClinicalTrials.gov — authoritative US registry record
WHO ICTRP — international registry index
EU Clinical Trials Register
Sponsor press releases (Google)

Data sources for this page

Trial protocol + status: ClinicalTrials.gov NCT01604824 (US National Library of Medicine, public domain)
Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
Sponsor: as reported to ClinicalTrials.gov by Regeneron Pharmaceuticals
Last refreshed: 17 June 2020

Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT01604824.

Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing