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 range1 Month
SexALL
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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.
✓. 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).
What they're measuring
1
Change in Fasting Serum Bile Acid (sBA) Levels From Week 18 to Week 22
. 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.