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.
Disclaimer
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This is a summary of
the original study
.
Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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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
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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