New drug TAK-625 shows promise for rare liver disease in kids
NCT ID NCT05543174
First seen Jun 24, 2026 · Last updated Jun 27, 2026 · Updated 2 times
Summary
This phase 3 study tested TAK-625 (maralixibat) in 7 Japanese children with Alagille syndrome, a rare genetic liver disorder. The goal was to see if the drug reduces bile acid levels and itching, and to find the best dose. Participants took the drug daily for up to 34 months, with regular clinic visits to monitor safety and effectiveness.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
TAK-625 (maralixibat chloride)
What this could lead to
If it works, this could lead to a treatment that reduces bile acid buildup and itching in children with Alagille syndrome, improving their daily life.
What could go wrong
This is a very small, completed study with only 7 participants, so results may not apply to everyone. The drug may cause side effects or not work as well in larger groups.
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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Juntendo University Hospital
Bunkyo-ku, Tokyo, Japan
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Kindai University Nara Hospital
Ikoma, Nara, Japan
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Kyoto University Hospital
Kyoto, Japan
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Miyagi Children's Hospital
Sendai, Miyagi, Japan
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Osaka University Hospital
Suita, Osaka, Japan
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Saitama Prefectural Children's Medical Center
Saitama, Japan
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University of Tsukuba Hospital
Tsukuba, Ibaraki, Japan
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Yokohamashi Tobu Hospital
Yokohama, Kanagawa, Japan