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

Status: Recruiting
Location: See location...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Early Phase 1
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.

Eligibility
Participation Requirements
Sex: All
Minimum Age: 18
Healthy Volunteers: f
View:

• 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.

Locations
Other Locations
China
The First Affiliated Hospital of Anhui Medical University
RECRUITING
Hefei
Time Frame
Start Date: 2025-12-31
Estimated Completion Date: 2029-04
Participants
Target number of participants: 18
Treatments
Experimental: Group 1: DSL101
Subjects will receive intravenous infusions of DSL101 once every four weeks.
Experimental: Group 2: DSL101
Subjects will receive intravenous infusions of DSL101 once every four weeks.
Experimental: Group 3: DSL101
Subjects will receive intravenous infusions of DSL101 once every four weeks.
Sponsors
Leads: DSciLab Co., Ltd.

This content was sourced from clinicaltrials.gov