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

This trial does not accept healthy volunteers

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

Design model

Sequential

Treatments tested in this trial

Group 1: DSL101 Low dose
Drug
Subjects will receive intravenous infusions of DSL101 once every four weeks.
Group 2: DSL101 Medium dose
Drug
Subjects will receive intravenous infusions of DSL101 once every four weeks.
Group 3: DSL101 High dose
Drug
Subjects will receive intravenous infusions of DSL101 once every four weeks.

Treatment groups

18 Participants

are divided into 3 treatment groups

Group A: Group 1: DSL101Experimental treatment 1 intervention

Group B: Group 2: DSL101Experimental treatment 1 intervention

Group C: Group 3: DSL101Experimental treatment 1 intervention

Trial outcomes

Primary outcomes

Incidence of adverse events and serious adverse events

Time frame

up to 56 weeks

Secondary outcomes

Change in 24 hour urine copper

Time frame

up to week 20

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

Time frame

up to week 20

Change in serum free copper

Time frame

up to week 20

Change from baseline in serum ceruloplasmin

Time frame

up to week20

Other outcomes

PK: Cmax

Time frame

up to week 6

PK: Time to reach peak plasma concentration (Tmax)

Time frame

up to week 6

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

Time frame

up to week 6

Immunogenicity: Positive rate of anti-drug antibody (ADA)

Time frame

up to week 20

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

DSciLab Co., Ltd.

Lead sponsor

Source ClinicalTrials.gov

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