A Study of Inclisiran in Participants With Homozygous Familial Hypercholesterolemia (HoFH)

Sponsor
Novartis Pharmaceuticals
Study ID
NCT03851705
Phase
PHASE3
Status
Completed

Conditions

  • Homozygous Familial Hypercholesterolemia

Eligibility Criteria

Sex
ALL
Age
18 Years - 80 Years
Healthy Volunteers
Accepted

Interventions

  • Inclisiran Sodium for injection — DRUG
    Inclisiran is a synthetic, chemically modified small interfering ribonucleic acid (siRNA) targeting proprotein convertase subtilisin kexin type 9 (PCSK9) messenger ribonucleic acid (mRNA) with a covalently attached triantennary N-acetylgalactosamine (GalNAc) ligand.
  • Placebo — DRUG
    Sterile normal saline (0.9% sodium chloride in water for injection)
  • Placebos — DRUG
    Sterile normal saline (0.9% sodium chloride in water for injection)

Study Details

This study was a Phase III,A two-part (double-blind placebo-controlled/open-label) multicenter study to evaluate safety, tolerability, and efficacy of inclisiran in subjects with homozygous familial hypercholesterolemia (HoFH).

Key Dates

Start date
Feb 6, 2019
Status verified
Jan 2023
Primary completion
Mar 2, 2020
Completion
Sep 9, 2021

Study Design

Enrollment
56 participants (actual)
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT

Arms

  • Experimental: Part 1 - Inclisiran
    Participants who received a dose of 300 milligram (mg) inclisiran sodium for injection administered by SC injection on Day 1 and Day 90.
  • Placebo Comparator: Part 1 - Placebo
    Participants who received a dose of placebos administered by SC injection on Day 1 and Day 90.
  • Experimental: Part 2 - Inclisiran
    Participants who received a dose of 300 mg inclisiran sodium for injection administered by SC injection on Day 270, Day 450 and Day 630. In addition, participants who were assigned to the placebo arm in Part 1 will receive a dose of 300 mg inclisiran sodium administered by SC injection on Day 180 after completion of Part 1.

Primary Outcome Measure

Percent Change in Low-Density Lipoprotein Cholesterol (LDL-C) From Baseline to Day 150 [ Time Frame: Baseline, Day 150 ]

Related Studies