A Clinical Study on the Treatment of Wilson Disease With ATP7B mRNA/LNP (DSL101)
ConditionWilsons Disease
Trial statusRecruiting
Trial phaseEarly Phase 1
Trial typeInterventional
Biological sexAll
Age18+
SponsorDSciLab Co., Ltd.
About this trial
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 criteria
Qualifiers
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.
Disqualifiers
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.
Trial design
Treatments tested in this trial
- Group 1: DSL101 Low dose
- Group 2: DSL101 Medium dose
- Group 3: DSL101 High dose
Treatment groups
18 Participants
are divided into 3 treatment groupsSponsors and collaborators
Source ClinicalTrials.gov