RECRUITING

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

Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

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).

Official Title

Two Part (Double-blind Inclisiran Versus Placebo [Year 1] Followed by Open-label Inclisiran [Year 2]) Randomized Multicenter Study to Evaluate Safety, Tolerability, and Efficacy of Inclisiran in Children (2 to Less Than 12 Years) With Homozygous Familial Hypercholesterolemia and Elevated LDL-cholesterol

Quick Facts

Study Start:2025-02-28
Study Completion:2029-04-15
Study Type:Not specified
Phase:Not Applicable
Enrollment:Not specified
Status:RECRUITING

Study ID

NCT06597006

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:2 Years to 11 Years
Sexes Eligible for Study:ALL
Accepts Healthy Volunteers:No
Standard Ages:CHILD
Inclusion CriteriaExclusion Criteria
  1. * Male or female participants, 2 to \<12 years of age at screening
  2. * HoFH diagnosed by genetic confirmation
  3. * Fasting LDL-C \>130 mg/dL (3.4 mmol/L) at screening
  4. * On an optimal dose of statin (investigator's discretion), unless statin intolerant, with or without other lipid-lowering therapy (e.g. ezetimibe)
  5. * 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
  6. * 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.
  1. * Documented evidence of a null (negative) mutation in both LDLR alleles
  2. * Previous treatment (within 90 days of screening) with monoclonal antibodies directed towards PCSK9
  3. * History of poor response to therapy with any monoclonal antibody directed towards PCSK9 (e.g. \<15% reduction in LDL-C)
  4. * Treatment with mipomersen or lomitapide (within 5 months of screening)
  5. * Secondary hypercholesterolemia, e.g. hypothyroidism or nephrotic syndrome
  6. * Heterozygous familial hypercholesterolemia (HeFH)
  7. * 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)
  8. * 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)
  9. * Pregnant or nursing females
  10. * Recent and/or planned use of other investigational medicinal products or devices

Contacts and Locations

Study Contact

Novartis Pharmaceuticals
CONTACT
1-888-669-6682
novartis.email@novartis.com
Novartis Pharmaceuticals
CONTACT
+41613241111

Principal Investigator

Novartis Pharmaceuticals
STUDY_DIRECTOR
Novartis Pharmaceuticals

Study Locations (Sites)

UC San Francisco Medical Center
San Francisco, California, 94158
United States
Childrens National Medical Center
Washington, District of Columbia, 20010
United States
Washington Univ School Of Medicine
Saint Louis, Missouri, 63110
United States

Collaborators and Investigators

Sponsor: Novartis Pharmaceuticals

  • Novartis Pharmaceuticals, STUDY_DIRECTOR, Novartis Pharmaceuticals

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2025-02-28
Study Completion Date2029-04-15

Study Record Updates

Study Start Date2025-02-28
Study Completion Date2029-04-15

Terms related to this study

Keywords Provided by Researchers

  • 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

Additional Relevant MeSH Terms

  • Familial Hypercholesterolemia - Homozygous