A Study of ALN-PCSSC in Participants With Homozygous Familial Hypercholesterolemia (HoFH)

The Medicines Company9 enrolled

Overview

The purpose of this study is to assess the safety, tolerability, and efficacy of ALN-PCSSC in participants with homozygous familial hypercholesterolemia.

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Homozygous Familial Hypercholesterolemia

Interventions

ALN-PCSSCDRUG

ALN-PCSSC is a small interfering ribonucleic acid (siRNA) that inhibits PCSK9 synthesis and is given as SC injections.

Standard of CareDRUG

Included maximally-tolerated statin therapy and/or other low density lipoprotein-cholesterol (LDL-C)-lowering therapies.

Eligibility

Sex: ALLMin age: 12 Years

Medical Language ↔ Plain English

Inclusion Criteria: * Males and females, ≥12 years of age with a diagnosis of homozygous familial hypercholesterolemia by genetic confirmation or a clinical diagnosis based on a history of an untreated low-density lipoprotein cholesterol (LDL-C) concentration \>500 mg/deciliter (dL) \[13 millimoles/liter (mmol/L)\] together with either xanthoma before 10 years of age or evidence of heterozygous familial hypercholesterolemia in both parents. * Stable on a low-fat diet. * Stable on pre-existing, lipid-lowering therapies (such as statins, cholesterolabsorption inhibitors, bile-acid sequestrants, or combinations thereof) for at least 4 weeks with no planned medication or dose change for the duration of study participation. * Fasting central lab LDL-C concentration \>130 mg/dL (3.4 mmol/L) and triglyceride concentration \<400 mg/dL (4.5 mmol/L). * Body weight of 40 kilograms (kg) or greater at screening. Exclusion Criteria: * LDL or plasma apheresis within 8 weeks prior to the screening visit, and no plan to receive it during the study because of the attendant difficulty in maintaining stable concentrations of LDL-C while receiving apheresis. * Use of mipomersen or lomitapide therapy within 5 months of screening. * Previous treatment with monoclonal antibodies directed towards PCSK9 within 8 weeks of screening. The above information is not intended to contain all considerations relevant to a participant's potential participation in a clinical trial.

Locations (3)

Research Site 201001

Los Angeles, California, United States

Research Site 231001

Amsterdam, Netherlands

Research Site 227001

Parktown, Johannesburg, South Africa

Outcomes

Primary Outcomes

Percentage Change From Day 1 to Day 90 in LDL-C

Due to the small number of subjects, and the fact that the data are not normally distributed, the data are presented as descriptive statistics with no inferential and limited summary statistics presented.

Time frame: Day 1, Day 90

Percentage Change From Day 1 to Day 180 (or Final Visit) in LDL-C

Time frame: Day 1, Day 180 (or Final Visit)