RECRUITING

Study to Evaluate Efficacy and Safety of Inclisiran in Children With Heterozygous 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 6 to \<12 years) with heterozygous familial hypercholesterolemia (HeFH) 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 (6 to Less Than 12 Years) With Heterozygous Familial Hypercholesterolemia and Elevated LDL- Cholesterol

Quick Facts

Study Start:2024-12-09
Study Completion:2029-01-14
Study Type:Not specified
Phase:Not Applicable
Enrollment:Not specified
Status:RECRUITING

Study ID

NCT06597019

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:6 Years to 11 Years
Sexes Eligible for Study:ALL
Accepts Healthy Volunteers:No
Standard Ages:CHILD
Inclusion CriteriaExclusion Criteria
  1. * Male or female participants, 6 to \<12 years of age at screening
  2. * HeFH diagnosed either by genetic testing or on phenotypic criteria
  3. * Fasting LDL-C \>130 mg/dL (3.4 mmol/L) at screening
  4. * For participants 8 to \<12 years, on an optimal dose of statin (investigator's discretion) unless statin intolerant, with or without other lipid-lowering therapy (e.g. ezetimibe). For participants \<8 years, the use of background lipid-lowering treatment is based on investigator's discretion.
  5. * Participants on lipid-lowering therapies (such as statin and/or e.g. ezetimibe) must be on a stable dose for ≥30 days before screening with no planned medication or dose changes during study participation.
  1. * Previous treatment (within 90 days of screening) with monoclonal antibodies directed towards PCSK9
  2. * Secondary hypercholesterolemia, e.g. hypothyroidism or nephrotic syndrome
  3. * Homozygous familial hypercholesterolemia (HoFH)
  4. * Body weight \<16 kg at the screening and/or randomization (Day 1) visit
  5. * 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)
  6. * Pregnant or nursing females
  7. * 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)

Excel Medical Clinical Trials LLC
Boca Raton, Florida, 33434
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 Date2024-12-09
Study Completion Date2029-01-14

Study Record Updates

Study Start Date2024-12-09
Study Completion Date2029-01-14

Terms related to this study

Keywords Provided by Researchers

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

Additional Relevant MeSH Terms

  • Familial Hypercholesterolemia - Heterozygous