NCT06597006 Study to Evaluate Safety, Tolerability and Efficacy of Inclisiran in Children With Homozygous Familial Hypercholesterolemia
| NCT ID | NCT06597006 |
| Status | Recruiting |
| Phase | Phase 3 |
| Sponsor | Novartis Pharmaceuticals |
| Condition | Familial Hypercholesterolemia - Homozygous |
| Study Type | INTERVENTIONAL |
| Enrollment | 9 participants |
| Start Date | 2025-02-28 |
| Primary Completion | 2028-03-21 |
Eligibility & Interventions
Eligibility Fast-Check
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What to Expect as a Participant
You will actively receive the study intervention — which may be a drug, biologic, device, or procedure.
Phase 3 trials are large pivotal studies comparing the treatment to current standard of care or placebo. Your participation directly contributes to the evidence needed for regulatory approval.
This trial targets 9 participants in total. It began in 2025-02-28 with a primary completion date of 2028-03-21.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
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).
Eligibility Criteria
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
Contact & Investigator
Novartis Pharmaceuticals
STUDY DIRECTOR
Novartis Pharmaceuticals
Frequently Asked Questions
Who can join the NCT06597006 clinical trial?
This trial is open to participants of all sexes, aged 2 Years or older, up to 11 Years, studying Familial Hypercholesterolemia - Homozygous. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
What phase is the NCT06597006 trial and what does that mean for participants?
Phase 3 trials are large-scale studies comparing the new treatment to existing standards of care or a placebo. They provide the evidence needed for regulatory approval. This trial targets 9 participants.
Is NCT06597006 currently recruiting?
Yes, NCT06597006 is actively recruiting participants. Contact the research team at novartis.email@novartis.com for enrollment information.
Where is the NCT06597006 trial being conducted?
This trial is being conducted at San Francisco, United States, Washington D.C., United States, St Louis, United States, Salt Lake City, United States and 11 additional locations.
Who is sponsoring the NCT06597006 clinical trial?
NCT06597006 is sponsored by Novartis Pharmaceuticals. The principal investigator is Novartis Pharmaceuticals at Novartis Pharmaceuticals. The trial plans to enroll 9 participants.