HypercholesterolemiaPhase 2NCT01604824

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

A Phase 2 study testing Alirocumab in people with Hypercholesterolemia, run by Regeneron Pharmaceuticals at 4 sites in 2 countries.

Completed

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

Official record

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Trial summary

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… Read more about this study

Who is it for?: Ages 18 to 70 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: 23 participants
Where: 4 sitesin 2 countries
Study dates: 22 Feb 2012to 28 Jul 2017
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 70 years
All sexes
No healthy volunteers
Adult, Older adult

Who may be able to join

Inclusion criteria include, but are not limited to the following:
Between the ages of 18 and 70 years, inclusive
A history of molecularly confirmed PCSK9 GOFm for cohort 1 and a history of molecularly confirmed PCSK9 GOFm or ApoB LOFm
Plasma LDL-Cholesterol levels ≥70 mg/dL at the screening visit on a lipid-lowering therapy (LLT) regimen stable for at least 28 days

Who cannot join

Exclusion criteria include, but are not limited to the following:
Serum triglycerides >350 mg/dL at the screening visit
Known to be positive for human immunodeficiency virus (HIV), hepatitis B virus, or hepatitis C virus
Pregnant or breast-feeding women.
Sexually active man or woman of childbearing potential who is unwilling to practice adequate contraception during the study

Trial locations and contacts

4 sites in 2 countries.

Country

City, postcode or hospital

4 sites in 2 countries

France3 sites

Study site
Lille, Cedex
Study site
Nantes, Cedex
Study site
Paris

Other trials in France

United States1 site

Study site
Salt Lake City, Utah

Other trials in United States

About this study

Official title: A Phase 2 Pilot Study With a Randomized Double-Blind Treatment Phase to Evaluate the Pharmacodynamics and Safety of Alirocumab in Patients With Autosomal Dominant Hypercholesterolemia and Gain-of-Function Mutations in 1 or Both Alleles of the PCSK9 Gene or Loss-of-Function Mutations in 1 or More Alleles of the Loss-of-Function Mutations B Gene

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 Praluent®, REGN727, SAR236553
SC injection in the abdomen
PlaceboDrug
SC injection in the abdomen

Study groups in NCT01604824
Study group	Role	What they receive
GOFm PCSK9 (Cohort 1): Alirocumab From Day 1	Experimental	Participants with gain-of-function mutation (GOFm) in proprotein convertase subtilisin/kexin type 9 (PCSK9) gene (Cohort 1): Alirocumab 150 mg subcutaneous (SC) injection at Week 0 (Day 1), Week 2 (Day 15), Week 4, 6 and 10 (matching placebo at Week 8, 12 and 14) during the double-blind period (Group A). Afterwards, participants have the possibility to continue in an open-label extension period with 150 mg alirocumab SC twice per week (Q2W) for an additional 3 years. Receives: Drug: Alirocumab; Drug: Placebo
GOFm PCSK9 (Cohort 1): Alirocumab From Day 15	Experimental	Participants with gain-of-function mutation (GOFm) in proprotein convertase subtilisin/kexin type 9 (PCSK9) gene (Cohort 1): Alirocumab 150 mg subcutaneous (SC) injection at Week 2 (Day 15), Week 4, 6, 8 and 12 ([matching placebo at Week 0 (Day 1), 10 and 14]) during the double-blind period (Group B). Afterwards, participants have the possibility to continue in an open-label extension period with 150 mg alirocumab SC twice per week (Q2W) for an additional 3 years. Receives: Drug: Alirocumab; Drug: Placebo
GOFm PCSK9 or LOFm ApoB (Cohort 2): Alirocumab from Day 1	Experimental	Participants with gain-of-function mutation (GOFm) in the proprotein convertase subtilisin/kexin type 9 (PCSK9) gene or loss-of-function mutation (LOFm) in the apolipoprotein (Apo) B gene (Cohort 2): Alirocumab 150 mg subcutaneous (SC) injection at Week 0 (Day 1), Week 2 (Day 15), Week 4, 6 and 10 (matching placebo at Week 8, 12 and 14) during the double-blind period (Group C). Afterwards, participants have the possibility to continue in an open-label extension period with 150 mg alirocumab SC twice per week (Q2W) for an additional 3 years. Receives: Drug: Alirocumab; Drug: Placebo
GOFm PCSK9 or LOFm ApoB (Cohort 2): Alirocumab from Day 15	Experimental	Participants with gain-of-function mutation (GOFm) in proprotein convertase subtilisin/kexin type 9 (PCSK9) gene or loss-of-function mutation (LOFm) in apolipoprotein (Apo) B gene (Cohort 2): Alirocumab 150 mg subcutaneous (SC) injection at Week 2 (Day 15), Week 4, 6, 8 and 12 ([matching placebo at Week 0 (Day 1), 10 and 14]) during the double-blind period (Group D). Afterwards, participants have the possibility to continue in an open-label extension period with 150 mg alirocumab SC twice per week (Q2W) for an additional 3 years. Receives: Drug: Alirocumab; Drug: Placebo

What is being measured

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

Percent Change in Measured Serum Low-Density Lipoprotein Cholesterol (LDL-C) From 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.]
Measured: Baseline to Day 15

Secondary outcomes (4)

Percent Change in Apolipoprotein (Apo) B100 From 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.
Measured: Baseline to Day 15
Percent Change in Non High-Density Lipoprotein Cholesterol (Non-HDL-C) From Baseline to Day 15
Measured: Baseline to Day 15
Percent Change in Total Cholesterol (Total-C) From Baseline to Day 15
Measured: Baseline to Day 15
Percent Change in Apolipoprotein (Apo) B100/ ApoA-1 Ratio From Baseline to Day 15
Measured: Baseline to Day 15

Timeline

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

22 Feb 201228 Jul 2017

First posted24 May 2012 (estimated)
Study start22 Feb 2012
Primary completion2 Jun 2014
Study completion28 Jul 2017

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 (2)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: 23 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT01604824
Sponsor's protocol ID: R727-CL-1018
Status last verified: Jun 2020
First submitted: 22 May 2012
Last updated: 17 Jun 2020
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: Yes
FDA-regulated device: No

Publications

Hopkins PN, Krempf M, Bruckert E, Donahue S, Yang F, Zhang Y, DiCioccio AT. 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. J Clin Lipidol. 2019 Nov-Dec;13(6):970-978. doi: 10.1016/j.jacl.2019.10.007. Epub 2019 Oct 21.
PubMed 31767518
Hopkins PN, Defesche J, Fouchier SW, Bruckert E, Luc G, Cariou B, Sjouke B, Leren TP, Harada-Shiba M, Mabuchi H, Rabes JP, Carrie A, van Heyningen C, Carreau V, Farnier M, Teoh YP, Bourbon M, Kawashiri MA, Nohara A, Soran H, Marais AD, Tada H, Abifadel M, Boileau C, Chanu B, Katsuda S, Kishimoto I, Lambert G, Makino H, Miyamoto Y, Pichelin M, Yagi K, Yamagishi M, Zair Y, Mellis S, Yancopoulos GD, Stahl N, Mendoza J, Du Y, Hamon S, Krempf M, Swergold GD. Characterization of Autosomal Dominant Hypercholesterolemia Caused by PCSK9 Gain of Function Mutations and Its Specific Treatment With Alirocumab, a PCSK9 Monoclonal Antibody. Circ Cardiovasc Genet. 2015 Dec;8(6):823-31. doi: 10.1161/CIRCGENETICS.115.001129. Epub 2015 Sep 15.
PubMed 26374825

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 17 Jun 2020. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: A Phase 2 Pilot Study With a Randomized Double-Blind Treatment Phase to Evaluate the Pharmacodynamics and Safety of Alirocumab in Patients With Autosomal Dominant Hypercholesterolemia and Gain-of-Function Mutations in 1 or Both Alleles of the PCSK9 Gene or Loss-of-Function Mutations in 1 or More Alleles of the Loss-of-Function Mutations B Gene
ClinicalTrials.gov ID: NCT01604824
Acronym / sponsor ID: R727-CL-1018
Conditions: Hypercholesterolemia
Phase: Phase 2
Study type: Interventional
Recruitment status: Completed
Enrolment: 23 participants (actual)
Lead sponsor: Regeneron PharmaceuticalsIndustry (pharma, biotech, devices)
Collaborators: Sanofi
Study start: 22 Feb 2012 (actual)
Primary completion: 2 Jun 2014 (actual)
Study completion: 28 Jul 2017 (actual)
First posted: 24 May 2012 (estimated)
Results: Posted 17 Jun 2020

Clinical summary

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

Study design

Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: Triple blind (participant, investigator, outcomes assessor)
Number of arms: 4

Arm	Type	Receives
GOFm PCSK9 (Cohort 1): Alirocumab From Day 1	Experimental	Drug: Alirocumab, Drug: PlaceboParticipants with gain-of-function mutation (GOFm) in proprotein convertase subtilisin/kexin type 9 (PCSK9) gene (Cohort 1): Alirocumab 150 mg subcutaneous (SC) injection at Week 0 (Day 1), Week 2 (Day 15), Week 4, 6 and 10 (matching placebo at Week 8, 12 and 14) during the double-blind period (Group A). Afterwards, participants have the possibility to continue in an open-label extension period with 150 mg alirocumab SC twice per week (Q2W) for an additional 3 years.
GOFm PCSK9 (Cohort 1): Alirocumab From Day 15	Experimental	Drug: Alirocumab, Drug: PlaceboParticipants with gain-of-function mutation (GOFm) in proprotein convertase subtilisin/kexin type 9 (PCSK9) gene (Cohort 1): Alirocumab 150 mg subcutaneous (SC) injection at Week 2 (Day 15), Week 4, 6, 8 and 12 ([matching placebo at Week 0 (Day 1), 10 and 14]) during the double-blind period (Group B). Afterwards, participants have the possibility to continue in an open-label extension period with 150 mg alirocumab SC twice per week (Q2W) for an additional 3 years.
GOFm PCSK9 or LOFm ApoB (Cohort 2): Alirocumab from Day 1	Experimental	Drug: Alirocumab, Drug: PlaceboParticipants with gain-of-function mutation (GOFm) in the proprotein convertase subtilisin/kexin type 9 (PCSK9) gene or loss-of-function mutation (LOFm) in the apolipoprotein (Apo) B gene (Cohort 2): Alirocumab 150 mg subcutaneous (SC) injection at Week 0 (Day 1), Week 2 (Day 15), Week 4, 6 and 10 (matching placebo at Week 8, 12 and 14) during the double-blind period (Group C). Afterwards, participants have the possibility to continue in an open-label extension period with 150 mg alirocumab SC twice per week (Q2W) for an additional 3 years.
GOFm PCSK9 or LOFm ApoB (Cohort 2): Alirocumab from Day 15	Experimental	Drug: Alirocumab, Drug: PlaceboParticipants with gain-of-function mutation (GOFm) in proprotein convertase subtilisin/kexin type 9 (PCSK9) gene or loss-of-function mutation (LOFm) in apolipoprotein (Apo) B gene (Cohort 2): Alirocumab 150 mg subcutaneous (SC) injection at Week 2 (Day 15), Week 4, 6, 8 and 12 ([matching placebo at Week 0 (Day 1), 10 and 14]) during the double-blind period (Group D). Afterwards, participants have the possibility to continue in an open-label extension period with 150 mg alirocumab SC twice per week (Q2W) for an additional 3 years.

Who may qualify

Ages: 18 to 70 years
Sex: All
Healthy volunteers: Not accepted
Age groups: Adult, Older adult

Key inclusion criteria (4 listed)

Between the ages of 18 and 70 years, inclusive
A history of molecularly confirmed PCSK9 GOFm for cohort 1 and a history of molecularly confirmed PCSK9 GOFm or ApoB LOFm
Plasma LDL-Cholesterol levels ≥70 mg/dL at the screening visit on a lipid-lowering therapy (LLT) regimen stable for at least 28 days

Key exclusion criteria (5 listed)

Serum triglycerides >350 mg/dL at the screening visit
Known to be positive for human immunodeficiency virus (HIV), hepatitis B virus, or hepatitis C virus
Pregnant or breast-feeding women.
Sexually active man or woman of childbearing potential who is unwilling to practice adequate contraception during the study

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: Praluent®, REGN727, SAR236553SC injection in the abdomen	Drug	GOFm PCSK9 (Cohort 1): Alirocumab From Day 1, GOFm PCSK9 (Cohort 1): Alirocumab From Day 15, GOFm PCSK9 or LOFm ApoB (Cohort 2): Alirocumab from Day 1, GOFm PCSK9 or LOFm ApoB (Cohort 2): Alirocumab from Day 15
PlaceboSC injection in the abdomen	Drug	GOFm PCSK9 (Cohort 1): Alirocumab From Day 1, GOFm PCSK9 (Cohort 1): Alirocumab From Day 15, GOFm PCSK9 or LOFm ApoB (Cohort 2): Alirocumab from Day 1, GOFm PCSK9 or LOFm ApoB (Cohort 2): Alirocumab from Day 15

Primary outcome

Percent Change in Measured Serum Low-Density Lipoprotein Cholesterol (LDL-C) From Baseline to Day 15
Time frame: 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.]

4 secondary outcomes

Percent Change in Apolipoprotein (Apo) B100 From Baseline to Day 15
Time frame: Baseline to Day 15
Percent Change in Non High-Density Lipoprotein Cholesterol (Non-HDL-C) From Baseline to Day 15
Time frame: Baseline to Day 15
Percent Change in Total Cholesterol (Total-C) From Baseline to Day 15
Time frame: Baseline to Day 15
Percent Change in Apolipoprotein (Apo) B100/ ApoA-1 Ratio From Baseline to Day 15
Time frame: Baseline to Day 15

Results and safety

Results posted to the registry on 17 Jun 2020, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
PCSK9 GOFm: Alirocumab From Day 1 (Cohort 1: Group A)	6	6
PCSK9 GOFm: Alirocumab From Day 15 (Cohort 1: Group B)	7	7
PCSK9 GOFm/ApoB LOFm: Alirocumab From Day1 (Cohort 2: Group C)	5	5
PCSK9GOFm/ApoB LOFm: Alirocumab From Day 15(Cohort 2: Group D)	5	5
PCSK9 GOFm (Cohort 1: Group A and Group B)	0	0
PCSK9 GOFm/ApoB LOFm (Cohort 2: Group C and Group D)	0	0

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

Time frame: Baseline to Day 15. Least squares mean (Standard Error). Unit: percent change

Measure	PCSK9 GOFm: Alirocumab From Day 1 (Cohort 1: Group A) n = 6	PCSK9 GOFm: Alirocumab From Day 15 (Cohort 1: Group B) n = 7	PCSK9 GOFm/ApoB LOFm: Alirocumab From Day1 (Cohort 2: Group C) n = 5	PCSK9GOFm/ApoB LOFm: Alirocumab From Day 15(Cohort 2: Group D) n = 5
Value	-62.48 (8.217)	-8.77 (7.575)	-48.21 (7.660)	-4.93 (7.660)

PCSK9 GOFm: Alirocumab From Day 1 (Cohort 1: Group A) vs PCSK9 GOFm: Alirocumab From Day 15 (Cohort 1: Group B): LS Mean Difference: -53.72 (95% CI -79.31 to -28.12), p = 0.0009, ANCOVA
PCSK9 GOFm/ApoB LOFm: Alirocumab From Day1 (Cohort 2: Group C) vs PCSK9GOFm/ApoB LOFm: Alirocumab From Day 15(Cohort 2: Group D): LS Mean Difference: -43.28 (95% CI -69.21 to 17.35), p = 0.0056, ANCOVA

4 secondary outcomes are reported in the full results.

Adverse events

Time frame: From the screening visit after the last alirocumab injection in the open-label period + 70 days through the end of study. Other adverse events reported at 5% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
PCSK9 GOFm: Alirocumab From Day 1 (Cohort 1: Group A)	1 of 6 (16.7%)	6 of 6 (100%)	0 of 6
PCSK9 GOFm: Alirocumab From Day 15 (Cohort 1: Group B)	0 of 7 (0%)	6 of 7 (85.7%)	0 of 7
PCSK9 GOFm/ApoB LOFm: Alirocumab From Day1 (Cohort 2: Group C)	0 of 5 (0%)	5 of 5 (100%)	0 of 5
PCSK9GOFm/ApoB LOFm: Alirocumab From Day 15(Cohort 2:Group D)	0 of 5 (0%)	4 of 5 (80%)	0 of 5
OLE Period: PCSK9 GOFm (Cohort 1) Group A & Group B	3 of 11 (27.3%)	10 of 11 (90.9%)	0 of 11
OLE Period: PCSK9 GOFm/ ApoB LOFm (Cohort 2) Group C & Group D	0 of 10 (0%)	9 of 10 (90%)	0 of 10

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 17 Jun 2020.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Derived from this study: PubMed 31767518Hopkins PN, Krempf M, Bruckert E, Donahue S, Yang F, Zhang Y, DiCioccio AT. 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. J Clin Lipidol. 2019 Nov-Dec;13(6):970-978. doi: 10.1016/j.jacl.2019.10.007. Epub 2019 Oct 21.
Derived from this study: PubMed 26374825Hopkins PN, Defesche J, Fouchier SW, Bruckert E, Luc G, Cariou B, Sjouke B, Leren TP, Harada-Shiba M, Mabuchi H, Rabes JP, Carrie A, van Heyningen C, Carreau V, Farnier M, Teoh YP, Bourbon M, Kawashiri MA, Nohara A, Soran H, Marais AD, Tada H, Abifadel M, Boileau C, Chanu B, Katsuda S, Kishimoto I, Lambert G, Makino H, Miyamoto Y, Pichelin M, Yagi K, Yamagishi M, Zair Y, Mellis S, Yancopoulos GD, Stahl N, Mendoza J, Du Y, Hamon S, Krempf M, Swergold GD. Characterization of Autosomal Dominant Hypercholesterolemia Caused by PCSK9 Gain of Function Mutations and Its Specific Treatment With Alirocumab, a PCSK9 Monoclonal Antibody. Circ Cardiovasc Genet. 2015 Dec;8(6):823-31. doi: 10.1161/CIRCGENETICS.115.001129. Epub 2015 Sep 15.

Related trials for Hypercholesterolemia

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.
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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

4 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 4 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 4 inclusion and 5 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 17 Jun 2020. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 4 sites in France, United States. 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. 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 17 Jun 2020). 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 in Measured Serum Low-Density Lipoprotein Cholesterol (LDL-C) From Baseline to Day 15 (time frame: Baseline to Day 15). 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 NCT01604824, which also lists 2 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 17 Jun 2020.