Actively Recruiting
A Clinical Study on the Treatment of Wilson Disease With ATP7B mRNA/LNP (DSL101)
Led by DSciLab Co., Ltd. · Updated on 2026-01-20
18
Participants Needed
1
Research Sites
169 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
This study adopted an open, single-arm, non-randomized, dose-escalation research design, aiming to evaluate the safety, tolerability, preliminary efficacy, pharmacokinetic and immunogenicity characteristics of single and multiple intravenous infusions of DSL101 in patients with Wilson's disease.
CONDITIONS
Official Title
A Clinical Study on the Treatment of Wilson Disease With ATP7B mRNA/LNP (DSL101)
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Age 18 years or older, any gender
- Diagnosed with Wilson's Disease according to 2022 guidelines, Leipzig score of 4 or higher, with diagnosis at least one year before screening; ceruloplasmin level below 0.1 g/L
- Confirmed double-chromosome mutations in the ATP7B gene
- On a low copper diet for at least six months before screening and willing to continue during the study
- Fertile participants agree to use reliable contraception from screening until six months after last dose
- Stable Wilson's disease patients treated for at least six months without drug or dose changes and continuously using standard treatments such as D-penicillamine, sodium dihydroxypropane sulfonate, dimercaptosuccinic acid, trientine, or zinc preparations
- Condition fully controlled after treatment, meeting all: serum NCC level 25-150 µg/L; urinary copper 100-900 µg/24 hours; ALT less than twice upper limit of normal; no other lab values or symptoms requiring stopping standard therapy
- Good compliance and able to understand and follow protocol requirements
- Voluntarily consent to participate and sign informed consent form
You will not qualify if you...
- Allergy or intolerance to the investigational drug
- Wilson's disease with severe neurological or mental disorders
- History of liver transplantation
- Other liver diseases causing injury, including acute/chronic hepatitis, alcoholic liver disease, autoimmune liver disease, drug-induced liver injury, cirrhosis, ascites, esophageal varices, hepatic encephalopathy, hepatorenal syndrome, liver failure, malignancy; MELD score above 13
- Other diseases causing hemolysis or anemia, such as erythrocytosis, Mediterranean anemia, hemolytic anemia, infections, large burns
- Other nervous system dysfunction diseases including Parkinson's disease, dystonia, chorea, tremor, epilepsy, serious psychiatric history
- Screening lab abnormalities: hemoglobin below 90 g/L; creatinine clearance under 30 mL/min or GFR under 45 mL/min/1.73 m²; total bilirubin over twice upper limit; platelet count below 70 x10^9/L; neutrophils below 1.0 x10^9/L
- History of gastrointestinal bleeding within six months before screening
- History of moderate to severe depression, suicidal thoughts or serious psychiatric issues within six months prior to screening
- Uncontrolled heart, liver, kidney, endocrine, digestive, metabolic, blood diseases, or malignant tumors
- Active hepatitis B or C infection, or positive HIV antibody
- Pregnant or lactating women
- Participation in another clinical trial within three months prior to screening or planning to during this trial
- Investigator judged unsuitable for participation for other reasons
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 1 location
1
The First Affiliated Hospital of Anhui Medical University
Hefei, Anhui, China, 230000
Actively Recruiting
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
3
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