A Study to Determine if REGN727(SAR236553) Will Have an Effect of Circulating Lipids in Patients With Gain-of-function (GOF) Mutations in Their PCSK9 Gene

This study is ongoing, but not recruiting participants.

Sponsor:

Collaborator:

Sanofi

Information provided by (Responsible Party):

Regeneron Pharmaceuticals

ClinicalTrials.gov Identifier:

NCT01604824

First received: May 22, 2012

Last updated: January 23, 2013

Last verified: January 2013

History of Changes

Tracking Information

First Received Date ^ICMJE

May 22, 2012

Last Updated Date

January 23, 2013

Start Date ^ICMJE

September 2012

Estimated Primary Completion Date

July 2013 (final data collection date for primary outcome measure)

Current Primary Outcome Measures ^ICMJE

Percent change in serum LDL-C [ Time Frame: Day 1(baseline) to Day 15 ] [ Designated as safety issue: No ]

Percent change in Low density lipoprotein cholesterol (LDL-C) from day 1 (baseline) to day 15 for group A compared to group B.

Original Primary Outcome Measures ^ICMJE

Same as current

Change History

Complete list of historical versions of study NCT01604824 on ClinicalTrials.gov Archive Site

Current Secondary Outcome Measures ^ICMJE

Number of TEAEs [ Time Frame: Day 1 to Day 155 ] [ Designated as safety issue: Yes ]
Incidence and severity of treatment-emergent adverse events (TEAEs) in patients treated with REGN727 reported between the first dose of REGN727 (day 1) and the end-of-study (EOS) visit (day 155)
Percent change in ApoB100 [ Time Frame: Day 1 to Day 15 ] [ Designated as safety issue: No ]
Percent change in Apolipoprotein (Apo) B100 from day 1 (baseline) to day 15 for group A compared to group B
Percent change in non-HDL-C [ Time Frame: Day 1 to Day 15 ] [ Designated as safety issue: No ]
Percent change in non-HDL-C (High Density Lipoprotein Cholesterol) from day 1 (baseline) to day 15 for group A compared to group B
Percent change in total cholesterol [ Time Frame: Day 1 to Day 15 ] [ Designated as safety issue: No ]
Percent change in total cholesterol from day 1 (baseline) to day 15 for group A compared to group B

Original Secondary Outcome Measures ^ICMJE

Same as current

Current Other Outcome Measures ^ICMJE

Not Provided

Original Other Outcome Measures ^ICMJE

Not Provided

Descriptive Information

Brief Title ^ICMJE

A Study to Determine if REGN727(SAR236553) Will Have an Effect of Circulating Lipids in Patients With Gain-of-function (GOF) Mutations in Their PCSK9 Gene

Official Title ^ICMJE

A Phase 2 Pilot Study With a Randomized Double-Blind Treatment Phase to Evaluate the Pharmacodynamics and Safety of REGN727 in Patients With Autosomal Dominant Hypercholesterolemia and Gain-of-Function Mutations in 1 or Both Alleles of the PCSK9 Gene

Brief Summary

The purpose of this study is to assess the pharmacodynamic (PD) effect of REGN727 on serum low-density lipoprotein cholesterol (LDL-C) during 14 weeks of subcutaneously (SC) administered REGN727 in patients with autosomal dominant hypercholesterolemia (ADH) and gain-of-function mutations (GOFm) in 1 or both alleles of the proprotein convertase subtilisin/kexin type 9 (PCSK9) gene.

Detailed Description

Not Provided

Study Type ^ICMJE

Interventional

Study Phase

Phase 2

Study Design ^ICMJE

Allocation: Randomized
Endpoint Classification: Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Investigator, Outcomes Assessor)
Primary Purpose: Treatment

Condition ^ICMJE

Hypercholesterolemia

Intervention ^ICMJE

Drug: REGN727 (SAR236553)
Patients in Group A will receive REGN727 (SAR236553) or placebo as per Regimen 1
Drug: REGN727 (SAR236553)
Patients in Group B will receive REGN727 (SAR236553) or placebo as per Regimen 2

Study Arm (s)

Experimental: Group A
Regimen 1

Intervention: Drug: REGN727 (SAR236553)
Experimental: Group B
Regimen 2

Intervention: Drug: REGN727 (SAR236553)

Publications *

Not Provided

* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.

Recruitment Information

Recruitment Status ^ICMJE

Active, not recruiting

Enrollment ^ICMJE

Estimated Completion Date

July 2013

Estimated Primary Completion Date

July 2013 (final data collection date for primary outcome measure)

Eligibility Criteria ^ICMJE

Inclusion Criteria:

Inclusion criteria include, but are not limited to the following:

Between the ages of 18 and 70 years, inclusive
Plasma LDL-Cholesterol levels greater than or equal to 70 mg/dL at the screening visit

Exclusion Criteria:

Exclusion criteria include, but are not limited to the following:

Serum Triglycerides greater than 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

Gender

Both

Ages

18 Years to 70 Years

Accepts Healthy Volunteers

Contacts ^ICMJE

Contact information is only displayed when the study is recruiting subjects

Location Countries ^ICMJE

United States, France

Administrative Information

NCT Number ^ICMJE

NCT01604824

Other Study ID Numbers ^ICMJE

R727-CL-1018

Has Data Monitoring Committee

Responsible Party

Regeneron Pharmaceuticals

Study Sponsor ^ICMJE

Regeneron Pharmaceuticals

Collaborators ^ICMJE

Sanofi

Investigators ^ICMJE

Study Director:

Clinical Trial Management

Regeneron Pharmaceuticals

Information Provided By

Regeneron Pharmaceuticals

Verification Date

January 2013

^ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP

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