A Study of TAK-625 for the Treatment of Alagille Syndrome (ALGS) (NCT05543174) | Clinical Trial Compass
CompletedPhase 3
A Study of TAK-625 for the Treatment of Alagille Syndrome (ALGS)
Japan7 participantsStarted 2023-01-16
Plain-language 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.
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.
Who can participate
Age range
1 Month
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
. 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.
. Gamma-glutamyl transferase (GGT) \>3\^ ULN for age.
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Change in Fasting Serum Bile Acid (sBA) Levels From Week 18 to Week 22
. Intractable pruritus explainable only by liver disease.
Exclusion criteria
. 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).
. 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.