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TAK-625

Phase 3

Alagille Syndrome (ALGS) | Small molecule | Rare Disease |Takeda Pharmaceutical Company Limited|Last Updated: Jan 29, 2026

Success Probability

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Trial Design

UNCONTROLLED
Total Trials1
Total Enrollment7

FDA Designations

No designations recorded

Clinical trial landscape

TAK-625 · 2 trials · 2 indications

Phase 3 2
NCT05543174A Study of TAK-625 for the Treatment of Alagille Syndrome (ALGS)Alagille Syndrome (ALGS)
COMPLETED7 Analytics
NCT05543187A Study of TAK-625 for the Treatment of Progressive Familial Intrahepatic Cholestasis (PFIC)Progressive Familial Intrahepatic Cholestasis (PFIC)
COMPLETED5 Analytics
PHASE3COMPLETED
A Study of TAK-625 for the Treatment of Alagille Syndrome (ALGS)
Alagille Syndrome (ALGS)Unlock trial analytics
PHASE3COMPLETED
A Study of TAK-625 for the Treatment of Progressive Familial Intrahepatic Cholestasis (PFIC)
Progressive Familial Intrahepatic Cholestasis (PFIC)Unlock trial analytics

Study Endpoints

Primary Endpoints

Change in Fasting Serum Bile Acid (sBA) Levels From Week 18 to Week 22
From Week 18 to Week 22

Change in fasting sBA levels from Week 18 to 22 was reported. Change was calculated as: observed value at Week 22- observed value at Week 18.

Change in the Average Morning Itch Reported Outcome (ItchRO) (Observer Instrument [Obs]) Severity Score Between Baseline and the Average of Week 15 Through Week 26
Baseline to Week 15 through Week 26

The ItchRO (Obs) scale measures severity of pruritus. The score on ItchRO (Obs) scale ranged from 0 to 4, where 0=None observed or reported, 1=Mild, 2=Moderate, 3=Severe, 4=Very severe. A higher score indicated more severe pruritus. Average baseline morning ItchRO (Obs) scores were calculated as sum of the morning scores divided by number of morning scores for the 4-week (28 days) time periods (that is \[i.e.\], Day -28 to Day -1). Average morning ItchRO (Obs) scores Week 15 through Week 26 were calculated as the sum of the morning scores divided by the number of morning scores from Week 15 to Week 26. Change was calculated as: Average value of Weeks 15 to 26 - Average value of Baseline (Day -28 to Day -1).

Secondary Endpoints

Change in Fasting sBA Levels From Baseline to Week 18
Baseline to Week 18
Change in Weekly Average Severity of Pruritus Measured by Itch Reported Outcome (ItchRO) (Observer Instrument [Obs]) From Baseline to Week 18
Baseline to Week 18
Change in Weekly Average Morning Severity of Pruritus Measured by Itch Reported Outcome (ItchRO) (Observer Instrument [Obs]) From Baseline to Week 18
Baseline to Week 18
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Study Design & Arms

AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
TAK-625EXPERIMENTALTAK-625 200 mcg per kilogram, orally, once daily for 1 week. After that, TAK-625 400 mcg per kilogram, orally, once daily after Week 1.
TAK-625, Primary cohortEXPERIMENTALTAK-625 orally, twice daily (BID) for 4 weeks as Dose Escalation Period. The dose in Dose Escalation Period will be increased weekly, 150 mcg/kilograms (kg), 300 mcg/kg, 450 mcg/kg, and 600 mcg/kg. After Dose Escalation Period, TAK-625 600 mcg/kg (or maximum tolerated dose \[MTD\]), orally, BID up to study completion.
TAK-625, Supplemental cohortEXPERIMENTALTAK-625 orally, twice daily (BID) for 4 weeks as Dose Escalation Period. The dose in Dose Escalation Period will be increased weekly, 150 mcg/kilograms (kg), 300 mcg/kg, 450 mcg/kg, and 600 mcg/kg. After Dose Escalation Period, TAK-625 600 mcg/kg (or maximum tolerated dose \[MTD\]), orally, BID up to study completion.

Interventions

NameTypeDescription
TAK-625DRUGTAK-625 200 mcg or 400 mcg per kilogram, orally, once daily
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Eligibility Criteria

Age Range1 Month to N/A
SexALL
Healthy VolunteersNo
Study Sites8

Inclusion Criteria: 1. The participant is Japanese male or female with a body weight \>=3.0 kilograms (kg) and who is \>=1 month of age at the time of informed consent. 2. The participant is diagnosed with ALGS. 3. The participant has one or more of the following evidences of cholestasis: 1. To...

Countries:Japan
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Frequently asked questions about TAK-625

What is TAK-625 used for?

TAK-625 is an investigational small molecule being developed for the treatment of Progressive Familial Intrahepatic Cholestasis (PFIC) and Alagille Syndrome (ALGS), both rare liver diseases. It is currently in Phase 3 clinical development and has not been approved by regulatory authorities.

Who makes TAK-625?

TAK-625 is being developed by Takeda Pharmaceutical Company Limited, a biopharmaceutical company traded on the New York Stock Exchange under the ticker symbol TAK. The drug is in Phase 3 clinical trials for rare cholestatic liver diseases.

What phase is TAK-625 in?

TAK-625 is in Phase 3 clinical development for Progressive Familial Intrahepatic Cholestasis (PFIC) and Alagille Syndrome (ALGS). It is an investigational drug, meaning it has not been approved by regulatory agencies and remains under study in clinical trials.

What clinical trials is TAK-625 in?

TAK-625 has completed two Phase 3 trials in Japan: NCT05543174 for Alagille Syndrome (ALGS) with 7 participants, and NCT05543187 for Progressive Familial Intrahepatic Cholestasis (PFIC) with 5 participants. Both trials enrolled patients aged 1 month and older.

What is the mechanism of action of TAK-625?

The specific molecular target of TAK-625 has not been disclosed in available clinical trial information. It is classified as a small molecule therapeutic being investigated for the treatment of cholestatic liver diseases, but its precise mechanism of action is not publicly detailed.