Alagille Syndrome
Conditions
Interventions
Cohort 1:Subjects are subcutaneously injected with the 0.33mg/kg ART104 for a total of 5 times, the first three injections are administered q2w, and the rest two are administered q4w, spanning a tota
Cohort 2:Subjects are subcutaneously injected with the 1mg/kg ART104 for a total of 5 times, the first three injections are administered q2w, and the rest two are administered q4w, spanning a total t
Sponsors
Children’s Hospital of Fudan University
Eligibility
Sex/Gender
All
Age
2 Years to 15 Years
Inclusion criteria
Inclusion criteria: 1. The subjects and their legal guardian must voluntarily agree to participate in the study, sign the Written Informed Consent Form (ICF), and be willing to comply with all study procedures, including the completion of the Subject Diary Card (with assistance from the guardian for assessment and documentation). 2. Aged >= 2 years and 5 mg/dL, primarily composed of elevated conjugated bilirubin. 6. Subjects may continue previously initiated concomitant treatments for cholestasis and pruritus (e.g., ursodeoxycholic acid, cholestyramine, rifampicin), doses of medication, bile acid levels, total bilirubin levels, and clinical status must have been stable for at least 3 months prior to study entry, per the investigator's judgement.
Exclusion criteria
Exclusion criteria: 1. Patients who had previous liver transplantation. 2. Patients with history of prior surgical biliary diversion (e.g., external or internal biliary diversion) to improve hepatointestinal circulation. 3. Patients with vitamin K1–unresponsive coagulopathy (INR >= 1.5 or APTT >= 1.3×upper limit of normal). 4. Patients with decompensated cirrhosis (>= Child-Pugh class C, defined as along with history or current illness of clinically significant ascites or esophageal variceal bleeding, with or without hepatic encephalopathy). 5. Other conditions may increase biopsy risk, including thrombocytopenia (platelets count <60×10?/L), hepatic amyloidosis, or hepatic cystic lesions, as deemed by the investigator. 6. History or current of concomitant with other types of chronic liver diseases. 7. History or current cholelithiasis. 8. Heart failure or renal failure. 9. The proportion of reticulocytes in red blood cells is greater than 3%, or the platelet count is less than 60×109/L.. 10. Concurrent HIV infection. 11. A known history of cancer, except for carcinoma in situ or cancers with no evidence of recurrence within 5 years after treatment prior to signing informed consent. 12. Patients who have received investigational drugs, biological products, or medical devices within 28 days or 5 half-lives of the drug (whichever is longer) before signing the informed consent. 13. Patients known to be allergic to ONTs medications containing GalNAc, the study drug, and its components. 14. Psychiatric illness or factors affecting study compliance, as deemed by the investigator. 15. Recent or current illness likely to prevent study completion. 16. Any disease or abnormality that may affect safety, as deemed by the investigator.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Analysis of Subject Distribution and Baseline Characteristics;Safety Analysis; | — |
Secondary
| Measure | Time frame |
|---|---|
| Pharmacokinetic Analysis;Analysis of Dose Linear Relationship;Efficacy Analysis;Pharmacodynamic - Exposure Relationship Analysis;Pharmacodynamic Analysis;Pharmacodynamic - Dose Relationship Analysis; | — |
Countries
China
Contacts
Public ContactJianshe WANG
Children’s Hospital of Fudan University
Outcome results
None listed