Hepatolenticular Degeneration
Conditions
Interventions
Experimental Group:This study presets three dose groups: low, medium and high. The low-dose group is the accelerated titration group, while the medium and high-dose groups adopt the traditional "3+3"
Sponsors
The First Affiliated Hospital of Anhui Medical University
Eligibility
Sex/Gender
All
Age
18 Years to No maximum
Inclusion criteria
Inclusion criteria: 1.Age >=18 years old, gender not limited. 2.Meet the diagnostic criteria f Wilson's Disease in "Guidelines for Diagnosis and Treatment of Wilson's Disease (2022 Edition)", with a Leipzig score>=4, at least one year between diagnosis and screening; ceruloplasmin level = 25 µg/L and =100 µg/24 hours and <=900 µg/24 hours; (3) ALT < 2 times of upper limit of normal value (ULN); (4) The investigator believes that no other laboratory values or clinical symptoms would stop current standard therapy; 8.Subjects with good compliance, who can understand and cooperate to complete the requirements of protocol. 9.The subjects voluntarily participated in the trial and signed the informed consent form.
Exclusion criteria
Exclusion criteria: 1.Allergy or intolerance to the investigational drug; 2.Wilson's disease is accompanied by severe complications such as neurological and mental disorders; 3.History of liver transplantation; 4.Other liver-related diseases and clinical symptoms that can cause liver injury, such as acute and chronic hepatitis, alcoholic liver disease, autoimmune liver disease, drug-induced liver injury, liver cirrhosis, liver ascites, esophageal varices, hepatic encephalopathy, hepatorenal syndrome, liver failure, liver malignancy, etc.; Subjects with Model for end-stage liver disease score (MELD)>13; 5.Other diseases that can cause hemolysis or anemia, such as erythrocytosis, Mediterranean anemia, hemolytic anemia, various causes of infection, large area burns, etc.; 6.Other diseases that can cause dysfunction of the nervous system, such as Parkinson's disease, Parkinson syndrome, various causes of dystonia, chorea, primary tremor, epilepsy, mental abnormalities (such as history of schizophrenia or suicide attempts), etc.; 7.Screening period laboratory examination indicators: (1) Hemoglobin =2×ULN,ALP/TBil2.2; (4) Platelets < 70 ×10^9/L; (5) Neutrophils < 1.0 × 10^9 /L; 8.History of gastrointestinal bleeding within six months before screening; 9.Subjects with history of moderate to severe depression, suicidal thoughts or behaviors and serious psychiatric within 6 months prior to screening; 10.Subjects who have uncontrolled diseases of thr heart, liver, kidneys, endocrine system, digestive tract, metabolism, blood, or malignant tumors; 11.Active hepatitis B virus infection or active hepatitis C virus infection, or human immunodeficiency virus antibody positive; 12.Pregnant women or lactating women; 13.Subjects who have participated other clinical trial within 3 months prior to screening or plan to participate during the clinical trial; 14.Investigators evaluate other subjects who are not suitable to participate in this clinical trial.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Vital signs;Phusical examination;Laboratory tests;Abdominal ultrasound scan;12-lead electrocardiogram;Adverse event;Aerious adverse event; | — |
Secondary
| Measure | Time frame |
|---|---|
| Pharmacokinetics indicator;Immunogenicity indicator ADA;Cytokines;24-hour urine copper;Total copper in serum;Serum free copper;Ceruloplasmin concentration;Ceruloplasmin activity;Serum iron concentration;Serum ferritin concentration;Liver function;UWDRS score;Complement ;Magnetic resonance imaging of the brain; | — |
Countries
China
Contacts
Public ContactSun Beicheng;Zhou Huan
The First Affiliated Hospital of Anhui Medical University
Outcome results
None listed