Recruiting

Phase 3

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

Sponsor:

Novartis Pharmaceuticals

Code:

NCT06597006

Conditions

Familial Hypercholesterolemia - Homozygous

Eligibility Criteria

Sex: All

Age: 2 - 11

Healthy Volunteers: Not accepted

Interventions

Inclisiran

Placebo

Study Details

Brief summary:

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).

Conditions

Familial Hypercholesterolemia - Homozygous

Study ID

NCT06597006

Start date

Feb 28, 2025

Status verified date

Jul, 2026

Completion date

Apr 15, 2029

Anticipated

Primary completion date

Mar 21, 2028

Anticipated

Eligibility Criteria

Sex: All

Age: 2 - 11

Healthy Volunteers: Not accepted

Inclusion Criteria:

Male or female participants, 2 to <12 years of age at screening
HoFH diagnosed by genetic confirmation

\- Note: Participants with known null (negative) mutations in both LDLR alleles are not eligible (see also exclusion criteria)

Fasting LDL-C >130 mg/dL (3.4 mmol/L) at screening
On an optimal dose of statin (investigator's discretion), unless statin intolerant, with or without other lipid-lowering therapy (e.g. ezetimibe)
Participants on lipid-lowering therapies (such as e.g. statins, ezetimibe) must be on a stable dose for ≥30 days before screening with no planned medication or dose changes during study participation
Participants on a documented regimen of LDL-apheresis for ≥ 3 months before screening will be allowed to continue the apheresis during the study, if needed. The apheresis schedule/settings/duration must be stable prior to screening, are not allowed to change during the double-blind period of the trial and must permit that an apheresis coincides with each study visit.

Exclusion Criteria:

Documented evidence of a null (negative) mutation in both LDLR alleles
Previous treatment (within 90 days of screening) with monoclonal antibodies directed towards PCSK9
History of poor response to therapy with any monoclonal antibody directed towards PCSK9 (e.g. <15% reduction in LDL-C)
Treatment with mipomersen or lomitapide (within 5 months of screening)
Secondary hypercholesterolemia, e.g. hypothyroidism or nephrotic syndrome
Heterozygous familial hypercholesterolemia (HeFH)
Body weight (at the screening and/or randomization (Day 1) visit) <16 kg for participants 6 to <12 years (at screening) or <11 kg for participants 2 to <6 years (at screening)
Active liver disease defined as any known current infectious, neoplastic, or metabolic pathology of the liver or unexplained alanine aminotransferase (ALT), aspartate aminotransferase (AST) elevation >3x ULN, or total bilirubin elevation >2x ULN (except patients with Gilbert's syndrome)
Pregnant or nursing females
Recent and/or planned use of other investigational medicinal products or devices

Study Design

Enrollment

9 participants

Anticipated

Allocation

Randomized

Intervention Model

Parallel Assignment

Primary purpose

Treatment

Interventions and Outcome Measures

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)

Interventions

Inclisiran

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

Sterile normal saline (0.9% sodium chloride in water for subcutaneous injection)

Primary outcome measure

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

Central Contacts and Locations

Central contacts

Novartis Pharmaceuticals

1-888-669-6682 novartis.email@novartis.com

Novartis Pharmaceuticals

+41613241111

Locations

UC San Francisco Medical Center

Recruiting

San Francisco, California, United States, 94143-0348

Contacts

Luis Gay

+1 415 476 8338 luis.gay@ucsf.edu

Principal Investigator:

Martin Thelin

Childrens National Hospital

Recruiting

Washington D.C., District of Columbia, United States, 20010

Contacts

Desiree Tobechukwu Nwanze

+1 202 476 5000 dnwanze@childrensnational.org

Principal Investigator:

Sarah Clauss

Washington Univ School Of Medicine

Recruiting

St Louis, Missouri, United States, 63110

Contacts

Jodi Pagano

jpagano@wustl.edu

Principal Investigator:

Anne Goldberg

Primary Childrens Medical Center

Recruiting

Salt Lake City, Utah, United States, 84113

Contacts

Linda Lambert

Linda.lambert@hsc.utah.edu

Principal Investigator:

Adam Ware

More Information

Sponsor

Novartis Pharmaceuticals

Last update posted

Jul 24, 2026

Last verified

Jul, 2026

Keywords

Homozygous familial hypercholesterolemia (HoFH),
LDL-cholesterol (LDL-C), children, pediatric,
small interfering ribonucleic acid (siRNA),
inclisiran,
Familial Hypercholesterolemia,
Homozygous FH,
Hypercholesterolemia,
Lipoprotein(a),
Hyperlipidemia,
Dyslipidemia,
Cardiovascular Diseases,
Heart Failure,
Cholesterol,
Aortic Stenosis

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-09. This information was provided to ClinicalTrials.gov by Novartis Pharmaceuticals on 2026-07-24.