Study to Evaluate Safety, Tolerability and Efficacy of Inclisiran in Children With Homozygous Familial Hypercholesterolemia

Conditions

• Familial Hypercholesterolemia - Homozygous

Eligibility Criteria

Sex

ALL

Age

2 Years - 11 Years

Healthy Volunteers

Not accepted

Interventions

• Inclisiran — DRUG
  Inclisiran (inclisiran sodium 300 mg subcutaneous (s.c.) for participants with body weight ≥23 kg, inclisiran sodium 180 mg s.c. for participants with body weight \<23 kg to ≥16 kg, or inclisiran sodium 100 mg s.c. for participants with body weight \<16 kg. The dose level is based on the participant's body weight on Day 1 (for Part 1) and Day 360 (for Part 2), respectively.
• Placebo — DRUG
  Sterile normal saline (0.9% sodium chloride in water for subcutaneous injection)

Study Details

This is a pivotal phase III study designed to evaluate safety, tolerability, and efficacy of inclisiran in children (aged 2 to \<12 years) with homozygous familial hypercholesterolemia (HoFH) and elevated low density lipoprotein cholesterol (LDLC).

Key Dates

Start date

Feb 28, 2025

Status verified

Feb 2026

Primary completion

Mar 23, 2028

Completion

Apr 15, 2029

Study Design

Enrollment

9 participants (estimated)

Allocation

RANDOMIZED

Intervention model

PARALLEL

Primary purpose

TREATMENT

Arms

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

Primary Outcome Measure

Percentage change in LDL-C from baseline to Day 330 (Year 1) [ Time Frame: Baseline and Day 330 ]

Central Contacts

• Novartis Pharmaceuticals
  1-888-669-6682
  [email protected]
• Novartis Pharmaceuticals
  +41613241111

Locations (4)

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