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Tests treatment safety and results for Familial Hypercholesterolemia - Homozygous
Official title Study to Evaluate Safety, Tolerability and Efficacy of Inclisiran in Children With Homozygous Familial Hypercholesterolemia
ClinicalTrials.gov ID: NCT06597006
What this study is testing
What is Inclisiran?
Inclisiran is an investigational medicine, given as an injection under the skin, being studied as a potential treatment for familial hypercholesterolemia - homozygous.
Also referred to as KJX839.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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).
- Phase 3: a large, late-stage study
- You might receive a placebo (an inactive treatment) instead of the study drug. You may not know which one you got.
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 2 to 11
You may be able to join if
- 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
You likely can't join if
- 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...
- Participants on a documented regimen of LDL-apheresis for ≥ 3 months before screening will be allowed to continue the apheresis during the study, if...
- Documented evidence of a null (negative) mutation in both LDLR alleles
- Previous treatment (within 90 days of screening) with monoclonal antibodies directed towards PCSK9
See the full eligibility 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
- 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...
- 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...
- Pregnant or nursing females
- Recent and/or planned use of other investigational medicinal products or devices
The study team makes the final eligibility decision.
Where it's taking place
- San Francisco, California, United States
- Washington D.C., District of Columbia, United States
- St Louis, Missouri, United States
- Vienna, Austria
- Beijing, Beijing Municipality, China
- Frankfurt am Main, Hesse, Germany
- Ioannina, Greece
- Thessaloniki, Greece
- Kota Bharu, Kelantan, Malaysia
- Amsterdam, North Holland, Netherlands
- Bloemfontein, Free State, South Africa
- Taichung, Taiwan
- Taipei, Taiwan
- Adana, Saricam, Turkey (Türkiye)
- Ankara, Yenimahalle, Turkey (Türkiye)
- Izmir, Turkey (Türkiye)
- Southampton, United Kingdom
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 2 years to 11 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include San Francisco, California, United States; Washington D.C., District of Columbia, United States; St Louis, Missouri, United States; Vienna, Austria; Beijing, Beijing Municipality, China; Frankfurt am Main, Hesse, Germany and 11 more location(s). Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.