New hope for kids with rare liver disease: drug eases debilitating itch

NCT ID NCT05543187

Completed Disease control Sponsor: Takeda Source: ClinicalTrials.gov ↗

First seen Jan 30, 2026 · Last updated May 20, 2026 · Updated 9 times

Summary

This study tested a drug called TAK-625 in 5 Japanese children with Progressive Familial Intrahepatic Cholestasis (PFIC), a rare liver disease that causes severe, constant itching. The main goal was to see if the drug reduces itching and to check for side effects. Participants took the drug for up to 34 months, with frequent clinic visits to monitor their symptoms and drug levels.

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Contacts and locations

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Locations

Juntendo University Hospital

Bunkyo-ku, Tokyo, Japan
Kyoto University Hospital

Kyoto, Japan
Kyushu University Hospital

Fukuoka, Japan
Osaka University Hospital

Suita, Osaka, Japan
Saitama Prefectural Children's Medical Center

Saitama, Japan
Tsuyama Chuo Hospital

Tsuyama, Okayama-ken, Japan
University of Tsukuba Hospital

Tsukuba, Ibaraki, Japan
Yokohamashi Tobu Hospital

Yokohama, Kanagawa, Japan

Conditions

Explore the condition pages connected to this study.

PROGRESSIVE FAMILIAL INTRAHEPATIC CHOLESTASIS (PFIC)