Study to Evaluate Efficacy and Safety of Inclisiran in Adolescents With Heterozygous Familial Hypercholesterolemia

Part of paid clinical trials in Tucson, Arizona.

Sponsor
Novartis Pharmaceuticals
Study ID
NCT04652726
Phase
PHASE3
Status
Completed

Conditions

  • Familial Hypercholesterolemia - Heterozygous

Eligibility Criteria

Sex
ALL
Age
12 Years - 17 Years
Healthy Volunteers
Not accepted

Interventions

  • Inclisiran — DRUG
    Inclisiran sodium 300 mg (equivalent to 284 mg inclisiran) in 1.5 mL solution for subcutaneous injection
  • Placebo — DRUG
    Sterile normal saline (0.9% sodium chloride in water for subcutaneous injection)

Study Details

This was a pivotal phase III study designed to evaluate safety, tolerability, and efficacy of inclisiran in adolescents with heterozygous familial hypercholesterolemia (HeFH) and elevated low density lipoprotein cholesterol (LDL-C).

Key Dates

Start date
Jan 27, 2021
Status verified
Dec 2025
Primary completion
Nov 9, 2023
Completion
Nov 27, 2024

Study Design

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

Arms

  • Experimental: Inclisiran
    Year 1 - inclisiran sodium 300 mg subcutaneous injection (given at Days 1, 90, and 270) Day 360 only - placebo subcutaneous injection Year 2 - inclisiran sodium 300 mg subcutaneous injection (given at Days 450 and 630)
  • Placebo Comparator: Placebo
    Year 1 - placebo subcutaneous injection (given at Days 1, 90 and 270) Year 2 - inclisiran sodium 300 mg subcutaneous injection (given at Days 360, 450, and 630)

Primary Outcome Measure

Percentage Change in LDL-C From Baseline to Day 330 (Part 1/Year 1) [ Time Frame: Baseline and Day 330 ]

Locations (6)

FacilityCityStateZIPSite coordinators
Tucson Medical CenterTucsonArizona85712-
Icahn School of Medicine at Mount SinaiNew YorkNew York10029-
Wake Forest U of Health SciencesWinston-SalemNorth Carolina27157-
Cincinnati Children's Hospital Medical CenterCincinnatiOhio45229-3039-
Childrens Hospital Pittsburgh of UPMCPittsburghPennsylvania15224-
Primary Children's Medical CenterSalt Lake CityUtah84113-

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By research site
Tucson Medical Center· Tucson, AZIcahn School of Medicine at Mount Sinai· New York, NYWake Forest U of Health Sciences· Winston-Salem, NCCincinnati Children's Hospital Medical Center· Cincinnati, OHChildrens Hospital Pittsburgh of UPMC· Pittsburgh, PAPrimary Children's Medical Center· Salt Lake City, UT

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