A phase 3 study is being launched to examine TAK-625 for the treatment of Alagille syndrome (ALGS).
The sponsor of this clinical trial is Takeda, who wrote, “The main aim of the study is to check if TAK-625 improves symptoms of ALGS, side effects from the study treatment or TAK-625, and how much TAK-625 stays in [patients’] blood over time.” This would allow researchers to determine the best doses to give patients in the future.
Five participants, each with a clear diagnosis of ALGS, will be recruited. They must be a Japanese male or female with a body weight greater or equal to 5 kg aged 1 year old or older at the time of informed consent.
Inclusion criteria include having high levels of total serum bile acids or gamma-glutamyl transferase, conjugated direct bilirubin greater than 1 mg/dL, lipid-soluble vitamin deficiency otherwise unexplainable, or intractable pruritus caused by liver disease.
Read more about ALGS etiology
The primary outcome measure of the trial is the change in fasting serum bile acid levels from week 18 to week 22. Other important secondary outcomes include the severity of pruritus, measured at various moments throughout the study. The researchers were also interested in understanding any changes to alanine aminotransferase, alkaline phosphatase, and bilirubin between baseline to week 18, as well as from week 18 to week 22.
The interventional, open-label, study is estimated to start on September 27, 2022, and end on July 31, 2025. By choosing outcome measures that reflect the severity of ALGS, researchers hope the study will uncover new ways to treat patients with ALGS.
A study of TAK-625 for the treatment of Alagille syndrome (ALGS). ClinicalTrials.gov. September 16, 2022. Accessed September 20, 2022.