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New treatment option for Wilsons Disease
Official title A Clinical Study on the Treatment of Wilson Disease With ATP7B mRNA/LNP (DSL101)
ClinicalTrials.gov ID: NCT07240896
What this study is testing
What is Group 1: DSL101 Low dose?
Group 1: DSL101 Low dose is an investigational medicine, given as an infusion into a vein, being studied as a potential treatment for wilsons disease.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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.
- Phase 1: an early, usually small safety study
- Which group you join is decided by chance.
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 18 and older
You may be able to join if
- Age ≥18 years old, gender not limited.
- Meet the diagnostic criteria f Wilson's Disease in "Guidelines for Diagnosis and Treatment of Wilson's Disease (2022 Edition)", with a Leipzig score...
- Patinets with Wilson's disease confirmed by laboratory tests to have double-chromosome mutations in the ATP7B gene.
- Low copper diet for at least six months befoer screening and willing to continue low copper diet during study.
- Fertile people agreed to adopt reliable contrraceptive methods from the screening until 6 months after the last administration.
You likely can't join if
- Allergy or intolerance to the investigational drug.
- Wilson's disease is accompanied by severe complications such as neurological and mental disorders.
- History of liver transplantation.
- Other liver-related diseases and clinical symptoms that can cause liver injury, such as acute and chronic hepatitis, alcoholic liver disease...
- Other diseases that can cause hemolysis or anemia, such as erythrocytosis, Mediterranean anemia, hemolytic anemia, various causes of infection, large...
- Other diseases that can cause dysfunction of the nervous system, such as Parkinson's disease, Parkinson syndrome, various causes of dystonia, chorea...
See the full eligibility criteria
- Age ≥18 years old, gender not limited.
- 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...
- Patinets with Wilson's disease confirmed by laboratory tests to have double-chromosome mutations in the ATP7B gene.
- Low copper diet for at least six months befoer screening and willing to continue low copper diet during study.
- Fertile people agreed to adopt reliable contrraceptive methods from the screening until 6 months after the last administration.
- The people are atable patients with WD who have been trasted for at least six months without drug or dose changes for at least 6 momths at the time of screening , and have continuously used standard treatments [SOC...
- The subject's condition was fully controlled after treatment, and its definition must meet all of the following conditions:
- Serum NCC level ≥ 25 μg/L and ≤ 150 μg/L;
- Urinary copper ≥100 μg/24 hours and ≤900 μg/24 hours;;
- ALT \< 2 times of upper limit of normal value (ULN);
- The investigator believes that no other laboratory values or clinical symptoms would stop current standard therapy;
- people with good compliance, who can understand and cooperate to complete the requirements of protocol.
- The people voluntarily participated in the trial and signed the informed consent form.
- Allergy or intolerance to the investigational drug.
- Wilson's disease is accompanied by severe complications such as neurological and mental disorders.
- History of liver transplantation.
- 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...
- Other diseases that can cause hemolysis or anemia, such as erythrocytosis, Mediterranean anemia, hemolytic anemia, various causes of infection, large area burns, etc.
- 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...
- Screening period laboratory examination indicators:
- Hemoglobin \< 90 g/L;
- Creatinine clearance ≤30 mL/min, or glomerular filtration rate \<45 mL/min/1.73 m²;
- TBil≥2×ULN，ALP/TBil\ 2.2;
- Platelets \< 70 ×10\^9/L;
- Neutrophils \< 1.0 × 10\^9 /L.
- History of gastrointestinal bleeding within six months before screening.
- people with history of moderate to severe depression, suicidal thoughts or behaviors and serious psychiatric within 6 months prior to screening.
- people who have uncontrolled diseases of thr heart, liver, kidneys, endocrine system, digestive tract, metabolism, blood, or malignant tumors.
- Active hepatitis B virus infection or active hepatitis C virus infection, or human immunodeficiency virus antibody positive.
- Pregnant women or lactating women.
- people who have participated other clinical trial within 3 months prior to screening or plan to participate during the clinical trial.
- Investigators evaluate other people who are not suitable to participate in this clinical trial.
The study team makes the final eligibility decision.
Where it's taking place
- Hefei, Anhui, China
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 18 years and older. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Hefei, Anhui, China. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.