Familial Hypercholesterolemia - Homozygous study: Inclisiran
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?
This is an investigational injectable medicine being studied to help lower cholesterol in children with a specific genetic condition. It is designed to block the production of a protein in the liver that helps make LDL cholesterol.
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 study aims to see if the drug inclisiran is safe and helps lower high cholesterol levels in children.
- Who it's for
- The study is looking for children aged 2 to under 12 who have a severe inherited form of high cholesterol.
- What's involved
- Participants will receive injections of the study drug and have regular blood tests to check their cholesterol levels.
- Time commitment
- The study involves regular visits to the clinic for check-ups and blood draws over a set period.
- 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...
- 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)
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
- 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 is enrolling
- San Francisco, California, United States
- Washington D.C., District of Columbia, United States
- St Louis, Missouri, United States
- Salt Lake City, Utah, 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
- Johannesburg, Gauteng, 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, but 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?
This study is enrolling now at San Francisco, California, United States; Washington D.C., District of Columbia, United States; St Louis, Missouri, United States; Salt Lake City, Utah, United States; Vienna, Austria; Beijing, Beijing Municipality, China and 13 more locations. Enter your location above to see the nearest enrolling site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool that is independent of the study sponsor. Details come from ClinicalTrials.gov. The study team decides eligibility, and your own doctor gives medical advice.