New shot could slash Sky-High cholesterol in teens with rare gene condition
NCT ID NCT04659863
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study tested a drug called inclisiran in teenagers aged 12 to 18 who have a rare inherited condition that causes extremely high cholesterol. The goal was to see if the drug safely lowers their LDL (bad) cholesterol. Participants received either inclisiran or a placebo for one year, followed by open-label inclisiran for a second year.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- Inclisiran
- What this could lead to
- If successful, inclisiran could offer a new, convenient treatment option to lower dangerously high cholesterol in teenagers with this rare genetic condition.
- What could go wrong
- This is a small, early-phase study with only 13 participants, so results may not apply to all patients. Long-term safety and effectiveness are still unknown.
This is an AI summary of the original study and may miss details. Read our disclaimer.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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American University of Beirut Medical Center
Beirut, 1107 2020, Lebanon
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Excel Medical Clinical Trials LLC
Boca Raton, Florida, 33434, United States
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Hotel Dieu de France Hospital
El Achrafiyé, 166830, Lebanon
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Metropolitan Hospital
Athens, 18547, Greece
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Novartis Investigative Site
Québec, Quebec, G1V 4W2, Canada
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Novartis Investigative Site
Bron, 69677, France
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Novartis Investigative Site
Kuala Lumpur, 59100, Malaysia
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Novartis Investigative Site
Amsterdam, 1105 AZ, Netherlands
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Novartis Investigative Site
Istanbul, TUR, 34098, Turkey (Türkiye)
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Novartis Investigative Site
Izmir, 35100, Turkey (Türkiye)
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University General Hospital of Ioannina
Ioannina, GR, 455 00, Greece
More trials for these conditions
Other studies related to the condition(s) this trial covers.