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Alagille Syndrome (ALGS)Phase 3NCT05543174

A Study of TAK-625 for the Treatment of Alagille Syndrome (ALGS)

A Phase 3 study testing TAK-625 in people with Alagille Syndrome (ALGS), run by Takeda at 8 sites in Japan.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

The main aim of the study is to check if TAK-625 improves symptoms of Alagille Syndrome (ALGS), 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. Read more about this study

Who is it for?
Ages 1 month and older
Conditions the study is for
Alagille Syndrome (ALGS)
What is being tested?
TAK-625
Type of treatment
Pill or oral medicine
Could I receive a placebo?
No, everyone receives an active treatment
How many people
7 participants
Where
8 sitesJapan
Study dates
16 Jan 2023to 25 Jul 2025
Phase
Phase 3
Are there results?
Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 1 month and older
  • All sexes
  • No healthy volunteers
  • Child, Adult, Older adult

Who may be able to join

  • 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.
  • The participant is diagnosed with ALGS.
  • The participant has one or more of the following evidences of cholestasis:
  • Total serum bile acid (sBA) >3^ upper limit of the normal range (ULN) for age.
  • Direct bilirubin (conjugated) >1 mg/dL.
  • Lipid soluble vitamin (LSV) deficiency otherwise unexplainable.
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  • Gamma-glutamyl transferase (GGT) >3^ ULN for age.
  • Intractable pruritus explainable only by liver disease.
  • The participant is expected to have a consistent caregiver(s) for the duration of the study.
  • The participant has an access to phone for scheduled calls from study site.
  • Both a caregiver and participant above the age of assent are capable of reading and understanding the questionnaires.
  • Caregivers (and age-appropriate participants) must be willing and able to use an eDiary device during the study.
  • Caregivers (and age-appropriate participants) must complete at least 10 eDiary reports (morning or evening) during each of 2 consecutive weeks of the screening period (maximum possible reports=14 per week), even if the participant is an adult (over 18 years old).
  • Average daily score >2 on the ItchRO questionnaire (maximum possible daily score of 4) for 2 consecutive weeks in the screening period, prior to dosing. A daily score is the higher of the scores for the morning and evening ItchRO. The average daily score is the sum of all daily scores divided by the number of days the ItchRO was completed. Since it is difficult to evaluate pruritus in infants, participants <12 months of age at screening whose pruritus is unavoidably difficult to be evaluated are not necessarily required to meet the above score.

Who cannot join

  • The participant has chronic diarrhea requiring ongoing intravenous (IV) fluid or nutritional intervention.
  • The participant has a previous history of surgical interruption of the enterohepatic circulation.
  • The participant has a previous liver transplant.
  • The participant decompensated cirrhosis (ALT >15^ ULN, international normalized ratio [INR] >1.5 [unresponsive to vitamin K therapy], albumin <3.0 g/dL, history or presence of clinically significant ascites, variceal hemorrhage, and/or encephalopathy).
  • The participant has a history or presence of other concomitant liver disease.
  • The participant has a history or presence of any other disease or condition known to interfere with the absorption, distribution, metabolism, or excretion of drugs, including bile salt metabolism in the intestine (eg, inflammatory bowel disease).
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  • The participant has a history or presence of gallstones or kidney stones.
  • The participant has a possible malignant liver mass in imaging, including screening ultrasound.
  • The participant has cancers, except for in situ carcinoma, or cancers treated at least 5 years prior to screening with no evidence of recurrence.
  • The participant has received bile acid/lipid binding resins or IBAT inhibitors within 28 days prior to screening and throughout the trial.
  • The participant who has received sodium phenylbutyrate for less than 6 months at the initiation of screening.

Trial locations and contacts

8 sites in 1 country.

8 sites in 1 country

Japan8 sites

  • Juntendo University Hospital

    Bunkyo-ku, Tokyo

  • Kindai University Nara Hospital

    Ikoma, Nara

  • Kyoto University Hospital

    Kyoto

  • Saitama Prefectural Children's Medical Center

    Saitama

  • Miyagi Children's Hospital

    Sendai, Miyagi

  • Osaka University Hospital

    Suita, Osaka

  • University of Tsukuba Hospital

    Tsukuba, Ibaraki

  • Yokohamashi Tobu Hospital

    Yokohama, Kanagawa

Other trials in Japan

About this study

Official title: An Open-Label, Phase 3 Study to Evaluate the Efficacy and Safety of TAK-625 in the Treatment of Subjects With Alagille Syndrome

The main aim of the study is to check if TAK-625 improves symptoms of Alagille Syndrome (ALGS), 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 9 times from the start of study. After 9 times visits, participants will visit their study clinic every 12 weeks up to the end of study.

Being tested
TAK-625
Medical subject headings
Alagille Syndrome

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • TAK-625Drug

    Also called Maralixibat chloride

    TAK-625 200 mcg or 400 mcg per kilogram, orally, once daily

Study groups in NCT05543174
Study groupRoleWhat they receive
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.
Receives: Drug: TAK-625

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Change in Fasting Serum Bile Acid (sBA) Levels 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.

    Measured: From Week 18 to Week 22

Secondary outcomes (13)

  1. Change in Fasting sBA Levels From Baseline to Week 18

    Change in fasting sBA levels from baseline to Week 18 was reported. Change was calculated as: observed value at Week 18 - observed value at baseline (before dosing).

    Measured: Baseline to Week 18

  2. Change in Weekly Average Severity of Pruritus Measured by Itch Reported Outcome (ItchRO) (Observer Instrument [Obs]) From Baseline to Week 18

    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. Weekly average severity of Pruritus is calculated based on daily maximum of morning and evening severity scores measured by ItchRO (Obs). Weekly average severity was calculated as the average of the daily maximum of morning and evening over the visit consisting of the 7 days on or before the scheduled Study Day (i.e., the sum of the scores divided by the number of non-missing scores). Change was calculated as: average value of Week 18 (Day 120 to Day 126) - average value of baseline (Day -7 to Day -1).

    Measured: Baseline to Week 18

  3. Change in Weekly Average Morning Severity of Pruritus Measured by Itch Reported Outcome (ItchRO) (Observer Instrument [Obs]) From Baseline to Week 18

    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. Weekly average morning severity was calculated as the average of the daily morning scores over the visit consisting of the 7 days on or before the scheduled Study Day (i.e., the sum of the scores divided by the number of non-missing scores). Change was calculated as: average value of Week 18 (Day 120 to Day 126) - average value of baseline (Day -7 to Day -1).

    Measured: Baseline to Week 18

  4. Change in Weekly Average Severity of Pruritus Measured by ItchRO (Obs) From Week 18 to Week 22

    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. Weekly average severity of Pruritus was calculated based on daily maximum of morning and evening severity scores measured by ItchRO (Obs). Weekly average severity were calculated as the average of the daily maximum of morning and evening over the visit consisting of the 7 days on or before the scheduled Study Day (i.e., the sum of the scores divided by the number of non-missing scores). Change was calculated as: average value of Week 22 (Day 148 to Day 154) - average value of Week 18 (Day 120 to Day 126).

    Measured: From Week 18 to Week 22

  5. Change in Weekly Average Morning Severity of Pruritus Measured by ItchRO (Obs) From Week 18 to Week 22

    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. Weekly average morning severity was calculated as the average of the daily morning scores over the visit consisting of the 7 days on or before the scheduled Study Day (i.e., the sum of the scores divided by the number of non-missing scores). Change was calculated as: average value of Week 22 (Day 148 to Day 154) - average value of Week 18 (Day 120 to Day 126).

    Measured: From Week 18 to Week 22

  6. Change in Weekly Average Severity of Pruritus Measured by ItchRO (Pt) From Baseline to Week 18

    The ItchRO (Pt) scale measures severity of pruritus. The score on ItchRO (Pt) scale ranged from 0 to 4, where 0= Not felt itchy, 1= Felt a little bit itchy, 2= Felt pretty itchy, 3= Felt very itchy, 4= Felt very, very itchy. A higher score indicated more severe pruritus. Weekly average severity of Pruritus was calculated based on daily maximum of morning and evening severity scores measured by ItchRO (Pt). Weekly average scores was calculated as the average of the daily maximum of morning and evening over the visit consisting of the 7 days on or before the scheduled Study Day (i.e., the sum of the scores divided by the number of non-missing scores). Change was calculated as: average value of Week 18 (Day 120 to Day 126) - average value of baseline (Day -7 to Day -1).

    Measured: Baseline to Week 18

  7. Change in Weekly Average Morning Severity of Pruritus Measured by ItchRO (Pt) From Baseline to Week 18

    The ItchRO (Pt) scale measures severity of pruritus. The score on ItchRO (Pt) scale ranged from 0 to 4, where 0= Not felt itchy, 1= Felt a little bit itchy, 2= Felt pretty itchy, 3= Felt very itchy, 4= Felt very, very itchy. A higher score indicated more severe pruritus. Weekly average morning severity was calculated as the average of the daily morning scores over the visit consisting of the 7 days on or before the scheduled Study Day (i.e., the sum of the scores divided by the number of non-missing scores). Change was calculated as: average value of Week 18 (Day 120 to Day 126) - average value of baseline (Day -7 to Day -1).

    Measured: Baseline to Week 18

  8. Change in Alanine Aminotransferase (ALT) and Alkaline Phosphatase (ALP) Levels From Baseline to Week 18

    Change in ALT and ALP levels from baseline to Week 18 were reported. Change was calculated as: observed value at Week 18 - observed value at baseline (before dosing).

    Measured: Baseline to Week 18

  9. Change in Bilirubin (Total and Direct) Levels From Baseline to Week 18

    Change in bilirubin (total and direct) from baseline to Week 18 was reported. Change was calculated as: observed value at Week 18 - observed value at baseline (before dosing).

    Measured: Baseline to Week 18

  10. Change in Weekly Average Severity of Pruritus Measured by ItchRO (Pt) From Week 18 to Week 22

    The ItchRO (Pt) scale measures severity of pruritus. The score on ItchRO (Pt) scale ranged from 0 to 4, where 0= Not felt itchy, 1= Felt a little bit itchy, 2= Felt pretty itchy, 3= Felt very itchy, 4= Felt very, very itchy. A higher score indicated more severe pruritus. Weekly average severity of Pruritus was calculated based on daily maximum of morning and evening severity scores measured by ItchRO (Pt). Weekly average scores was calculated as the average of the daily maximum of morning and evening over the visit consisting of the 7 days on or before the scheduled Study Day (i.e., the sum of the scores divided by the number of non-missing scores). Change was calculated as: average value of Week 22 (Day 148 to Day 154) - average value of Week 18 (Day 120 to Day 126).

    Measured: From Week 18 to 22

  11. Change in Weekly Average Morning Severity of Pruritus Measured by ItchRO (Pt) From Week 18 to Week 22

    The ItchRO (Pt) scale measures severity of pruritus. The score on ItchRO (Pt) scale ranged from 0 to 4, where 0= Not felt itchy, 1= Felt a little bit itchy, 2= Felt pretty itchy, 3= Felt very itchy, 4= Felt very, very itchy. A higher score indicated more severe pruritus. Weekly average morning severity was calculated as the average of the daily morning scores over the visit consisting of the 7 days on or before the scheduled Study Day (i.e., the sum of the scores divided by the number of non-missing scores). Change was calculated as: average value of Week 22 (Day 148 to Day 154) - average value of Week 18 (Day 120 to Day 126).

    Measured: From Week 18 to 22

  12. Change in ALT and ALP Levels From Week 18 to 22

    Change in ALT and ALP from Week 18 to 22 was reported. Change was calculated as: observed value at Week 22 - observed value at Week 18.

    Measured: From Week 18 to 22

  13. Change in Bilirubin (Total and Direct) Levels From Week 18 to 22

    Change in bilirubin (total and direct) from Week 18 to 22 was reported. Change was calculated as: observed value at Week 22 - observed value at Week 18.

    Measured: From Week 18 to 22

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted16 Sep 2022
  2. Study start16 Jan 2023
  3. Primary completion25 Oct 2023
  4. Study completion25 Jul 2025

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 3
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
7 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT05543174
Sponsor's protocol ID
TAK-625-3001
Other IDs
jRCT2051220098
Status last verified
Jan 2026
First submitted
14 Sep 2022
Last updated
29 Jan 2026
Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No: De-identified individual participant data from this particular study will not be shared as there is a reasonable likelihood that individual patients could be re-identified (due to the limited number of study participants/study sites).

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing TAK-625 in people with Alagille Syndrome (ALGS), run by Takeda at 8 sites in Japan.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 14 inclusion and 11 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 29 Jan 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 8 sites in Japan. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 15 Oct 2024). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Change in Fasting Serum Bile Acid (sBA) Levels From Week 18 to Week 22 (time frame: From Week 18 to Week 22). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT05543174.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 29 Jan 2026.

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