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Tests treatment safety and results for Familial Hypercholesterolemia - Heterozygous
Official title Study to Evaluate Efficacy and Safety of Inclisiran in Children With Heterozygous Familial Hypercholesterolemia
ClinicalTrials.gov ID: NCT06597019
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 - heterozygous.
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 6 to \<12 years) with heterozygous familial hypercholesterolemia (HeFH) 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 6 to 11
You may be able to join if
- Male or female participants, 6 to \<12 years of age at screening
- HeFH diagnosed either by genetic testing or on phenotypic criteria
- Fasting LDL-C \>130 mg/dL (3.4 mmol/L) at screening
- For participants 8 to \<12 years, on an optimal dose of statin (investigator's discretion) unless statin intolerant, with or without other...
- 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...
You likely can't join if
- Previous treatment (within 90 days of screening) with monoclonal antibodies directed towards PCSK9
- Secondary hypercholesterolemia, e.g. hypothyroidism or nephrotic syndrome
- Homozygous familial hypercholesterolemia (HoFH)
- Body weight \<16 kg at the screening and/or randomization (Day 1) visit
- Active liver disease defined as any known current infectious, neoplastic, or metabolic pathology of the liver or unexplained alanine aminotransferase...
- Pregnant or nursing females
See the full eligibility criteria
- Male or female participants, 6 to \<12 years of age at screening
- HeFH diagnosed either by genetic testing or on phenotypic criteria
- Fasting LDL-C \>130 mg/dL (3.4 mmol/L) at screening
- 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...
- 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.
- Previous treatment (within 90 days of screening) with monoclonal antibodies directed towards PCSK9
- Secondary hypercholesterolemia, e.g. hypothyroidism or nephrotic syndrome
- Homozygous familial hypercholesterolemia (HoFH)
- Body weight \<16 kg at the screening and/or randomization (Day 1) visit
- 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
- Boca Raton, Florida, United States
- New York, New York, United States
- Salt Lake City, Utah, United States
- Richmond, Virginia, United States
- Morgantown, West Virginia, United States
- Buenos Aires, Argentina
- CABA, Argentina
- Salzburg, Austria
- Vienna, Austria
- Brussels, Belgium
- Leuven, Belgium
- Fortaleza, Ceará, Brazil
- Rio de Janeiro, Rio de Janeiro, Brazil
- Porto Alegre, Rio Grande do Sul, Brazil
- São Paulo, São Paulo, Brazil
- Beijing, Beijing Municipality, China
- Shanghai, China
- Prague, Czechia
+ 39 more site(s).
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, 6 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; Boca Raton, Florida, United States; New York, New York, United States; Salt Lake City, Utah, United States; Richmond, Virginia, United States and 53 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.