A Clinical Study on the Treatment of Wilson Disease With ATP7B mRNA/LNP (DSL101)

NCT07240896 | Recruiting Early Phase 1

• 1 other identifier: DSLIIT101
• Study Type: interventional
• Target: 18
• Locations: 1 country
• Sites: 1

Brief Summary

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

Wilsons Disease

Outcome Measures

Primary Outcomes (1)

• Incidence of adverse events and serious adverse events

  up to 56 weeks

Secondary Outcomes (14)

• Change in 24 hour urine copper

  up to week 20

• Change in sum total cpper [ serum copper bound by ceruloplasmin (NCC) ]

  up to week 20

• Change in serum free copper

  up to week 20

• Change from baseline in serum ceruloplasmin

  up to week20

• Change in serum iron concentration and serum ferritin concentration

  up to week 20

Other Outcomes (4)

• PK: Cmax

  up to week 6

• PK: Time to reach peak plasma concentration (Tmax)

  up to week 6

• PK: Area under the plasma concentration-time curve (AUC)

  up to week 6

Study Arms (3)

Group 1: DSL101

EXPERIMENTAL

Subjects will receive intravenous infusions of DSL101 once every four weeks.

Drug: Group 1: DSL101 Low dose

Group 2: DSL101

EXPERIMENTAL

Subjects will receive intravenous infusions of DSL101 once every four weeks.

Drug: Group 2: DSL101 Medium dose

Group 3: DSL101

EXPERIMENTAL

Subjects will receive intravenous infusions of DSL101 once every four weeks.

Drug: Group 3: DSL101 High dose

Interventions

Group 2: DSL101 Medium dose DRUG

Subjects will receive intravenous infusions of DSL101 once every four weeks.

Group 2: DSL101

Group 3: DSL101 High dose DRUG

Subjects will receive intravenous infusions of DSL101 once every four weeks.

Group 3: DSL101

Group 1: DSL101 Low dose DRUG

Subjects will receive intravenous infusions of DSL101 once every four weeks.

Group 1: DSL101

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify 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 ≥4, at least one year between diagnosis and screening; ceruloplasmin level \<0.1g/L.
• 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 subjects agreed to adopt reliable contrraceptive methods from the screening until 6 months after the last administration.
• The subjects 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 \[SOC, such as D-penicillamine, sodiu dihydroxypropane sulfonate, dimercaptosuccinic acid, trientine, and zinc preparations (zinc acetate, zinc gluconate, zinc sulfate)\] for at least 6 months screening, and allowed subjects to continue with their prior SOC treatment.
• 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;
• Subjects with good compliance, who can understand and cooperate to complete the requirements of protocol.
• The subjects voluntarily participated in the trial and signed the informed consent form.

You may not qualify 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, 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.
• 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 schizophrenia or suicide attempts), etc.
• 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\<4，AST/ALT\>2.2;
• Platelets \< 70 ×10\^9/L;
• Neutrophils \< 1.0 × 10\^9 /L.
• History of gastrointestinal bleeding within six months before screening.
• Subjects with history of moderate to severe depression, suicidal thoughts or behaviors and serious psychiatric within 6 months prior to screening.
• Subjects who have uncontrolled diseases of thr heart, liver, kidneys, endocrine system, digestive tract, metabolism, blood, or malignant tumors.

Sponsors & Collaborators

• DSciLab Co., Ltd.: lead

Study Sites (1)

The First Affiliated Hospital of Anhui Medical University

Hefei, Anhui, 230000, China

RECRUITING

MeSH Terms

Conditions

Hepatolenticular Degeneration

Condition Hierarchy (Ancestors)

Liver Diseases; Digestive System Diseases; Basal Ganglia Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Movement Disorders; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Metabolism, Inborn Errors; Metal Metabolism, Inborn Errors; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Design

• Study Type: interventional
• Phase: early phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 25, 2025
• First Posted: November 21, 2025
• Study Start: December 31, 2025
• Primary Completion (Estimated): April 1, 2028
• Study Completion (Estimated): April 1, 2029
• Last Updated: January 20, 2026

Record last verified: 2026-01

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)