New drug shows promise for rare liver disease itch
NCT ID NCT05543187
First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 2 times
Summary
This study tested a drug called TAK-625 (maralixibat) in 5 Japanese children with progressive familial intrahepatic cholestasis (PFIC), a rare liver disease that causes severe itching. The main goal was to see if the drug reduces itching over 26 weeks. Researchers also checked safety and how the drug behaves in the body. The trial is complete, but results are not yet published.
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 point toward a treatment to ease severe itching in children with PFIC, a rare liver disease.
- What could go wrong
- This is a very small study (5 participants) in Japanese patients only, so results may not apply to others. The drug may not work or could have side effects.
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
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Locations
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Juntendo University Hospital
Bunkyo-ku, Tokyo, Japan
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Kyoto University Hospital
Kyoto, Japan
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Kyushu University Hospital
Fukuoka, 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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Tsuyama Chuo Hospital
Tsuyama, Okayama-ken, Japan
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University of Tsukuba Hospital
Tsukuba, Ibaraki, Japan
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Yokohamashi Tobu Hospital
Yokohama, Kanagawa, Japan
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