NCT05543187

A Study of TAK-625 for the Treatment of Progressive Familial Intrahepatic Cholestasis (PFIC)

An Open-Label, Phase 3 Study to Evaluate the Efficacy and Safety of TAK-625 in the Treatment of Subjects With Progressive Familial Intrahepatic Cholestasis

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 5 (actual)
• Sponsor: Takeda · Industry
• Sex: All
• Age: 1 Month
• Healthy volunteers: Not accepted

Summary

The main aim of the study is to check if TAK-625 improves symptoms of Progressive Familial Intrahepatic Cholestasis (PFIC), side effect from the study treatment or TAK-625, and how much TAK-625 stays in their blood over time. This will help the study sponsor (Takeda) to work out the best dose to give people in the future. The participants will be treated with TAK-625 for up to the end of study (about 34 months). Participants will visit their study clinic 15 times from the start of study. After 15 times visits, participants will visit their study clinic every 12 weeks up to the end of study.

Conditions

• Progressive Familial Intrahepatic Cholestasis (PFIC)

Interventions

Type	Name	Description
DRUG	TAK-625	TAK-625 orally, twice daily (BID)

Timeline

• Start date: 2023-01-10
• Primary completion: 2023-09-04
• Completion: 2025-07-22
• First posted: 2022-09-16
• Last updated: 2026-01-28
• Results posted: 2024-09-24

Locations

8 sites across 1 country: Japan

Regulatory

• FDA-regulated drug study