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

Part of paid clinical trials in Boca Raton, Florida.

Sponsor
Novartis Pharmaceuticals
Study ID
NCT04659863
Phase
PHASE3
Status
Completed

Conditions

  • Familial Hypercholesterolemia - Homozygous

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 injection) for subcutaneous injection

Study Details

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

Key Dates

Start date
Feb 16, 2021
Status verified
Dec 2025
Primary completion
Oct 30, 2023
Completion
Nov 18, 2024

Study Design

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

Arms

  • Experimental: Part 1 - Inclisiran
    Inclisiran sodium 300 mg subcutaneous (sc) injection (given at Days 1, 90 and 270)
  • Placebo Comparator: Part 1 - Placebo
    Placebo sc injection (given at Day 1, 90 and 270)
  • Experimental: Part 2 - Inclisiran (Total)
    Inclisiran sodium 300 mg sc injection (given at Days 450 and 630). In addition, participants assigned to placebo in Part 1 received inclisiran sodium 300 mg sc injection on Day 360, while participants assigned to inclisiran in Part 1 received placebo sc injection on Day 360.

Primary Outcome Measure

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

Locations (1)

FacilityCityStateZIPSite coordinators
Excel Medical Clinical Trials LLCBoca RatonFlorida33434-

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Excel Medical Clinical Trials LLC· Boca Raton, FL

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