Evaluating inclisiran for children with a rare cholesterol condition

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This pivotal phase III study aims to assess the safety, tolerability, and efficacy of inclisiran in children aged 2 to less than 12 years who have been diagnosed with homozygous familial hypercholesterolemia (HoFH) and exhibit elevated levels of low-density lipoprotein cholesterol (LDL-C). The study consists of two parts: a one-year double-blind comparison of inclisiran versus placebo followed by a one-year open-label phase where all participants receive inclisiran. Participants must be on a stable background lipid-lowering therapy, and the study will monitor their response to the treatment over the course of two years.

Why it matters

Who can join: the full rules

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

Where it's running

• UC San Francisco Medical Center
  San Francisco, California, United States Recruiting
• Childrens National Hospital
  Washington D.C., District of Columbia, United States Recruiting
• Washington Univ School Of Medicine
  St Louis, Missouri, United States Recruiting
• Primary Childrens Medical Center
  Salt Lake City, Utah, United States Recruiting
• Novartis Investigative Site
  Vienna, Austria Recruiting
• Novartis Investigative Site
  Beijing, Beijing Municipality, China Recruiting

Study contacts

• Study coordinator: Novartis Pharmaceuticals
• Email: novartis.email@novartis.com
• Phone: 1-888-669-6682

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

• Detecting Familial Hypercholesterolemia in Children

  This study is trying to find out if we can identify children with a genetic condition that causes high cholesterol so they can get early treatment and avoid heart problems later in life.

  Recruiting at 2 sites Institut Investigacio Sanitaria Pere Virgili · Reus, Tarragona and 1 other locations · NCT04370899

• XEP527 in stable schizophrenia: safety and how it behaves in the body

  This small, early-stage study tests the safety and how XEP527 behaves in people with stable schizophrenia who are on a single antipsychotic.

  Recruiting Phase 1 Novartis · Bentonville, Arkansas · NCT07908680

• BTK inhibitor remibrutinib in hidradenitis suppurativa shows how it works in adults

  This phase 1 trial tests whether remibrutinib can safely affect HS in adults with moderate to severe disease.

  Recruiting at 2 sites Phase 1 Novartis · San Diego, California and 1 other locations · NCT07855276

• Dose-finding test of YMI024 in adults with stable coronary artery disease and high hsCRP

  This study tests several doses of YMI024 to see if it reduces inflammation and is safe for adults with stable CAD and elevated hsCRP, compared to placebo.

  Recruiting at 23 sites Phase 2 Novartis · Fairhope, Alabama and 22 other locations · NCT07830719

• Remibrutinib and omalizumab-based treatment approach for adults with chronic spontaneous urticaria not well controlled by antihistamines

  This trial tests a sequencing plan using remibrutinib and omalizumab to see if it helps adults with chronic spontaneous urticaria that isn’t well controlled by second-generation antihistamines.

  Recruiting at 10 sites Phase 3 Novartis · London, Ontario and 9 other locations · NCT07816328

• Single-dose combination therapy for uncomplicated malaria in young children

  This study tests whether a single oral dose of a combination of anti-malarial drugs can cure uncomplicated Plasmodium falciparum malaria in children aged 2 to under 12 years, compared with standard Coartem treatment.

  Recruiting at 5 sites Phase 2 Novartis · Banfora and 12 other locations · NCT07811908

How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.