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Wilsons DiseaseEarly Phase 1NCT07240896

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

A Early Phase 1 study testing Group 1: DSL101 Low dose, Group 2: DSL101 Medium dose and Group 3: DSL101 High dose in people with Wilsons Disease, run by DSciLab Co., Ltd. at 1 site in China.

Recruiting

Looking for participants now. Contact the study team or a site below to ask about taking part.

Sites and contacts
Official record
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Trial 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. Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Wilsons Disease
What is being tested?
Group 1: DSL101 Low dose, Group 2: DSL101 Medium dose and Group 3: DSL101 High dose
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
18 participants
Where
1 siteChina
Study dates
31 Dec 2025to Apr 2029
Phase
Early Phase 1
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • 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.
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  • 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.

Who cannot join

  • 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.
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  • 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.
  • Active hepatitis B virus infection or active hepatitis C virus infection, or human immunodeficiency virus antibody positive.
  • Pregnant women or lactating women.
  • Subjects who have participated other clinical trial within 3 months prior to screening or plan to participate during the clinical trial.
  • Investigators evaluate other subjects who are not suitable to participate in this clinical trial.

Trial locations and contacts

1 site in 1 country. Contact the study team or a site near you to ask about taking part.

China1 site, 1 recruiting

  • The First Affiliated Hospital of Anhui Medical University

    Hefei, Anhui, 230000

    Huan Zhou, 0551-62922017, zhouhuanbest@163.com

    Recruiting

Other trials in China

About this study

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.

Read the detailed description

In this study, low, medium and high dose groups were preset, the low dose group was accelerated titration group, and the medium and high dose groups used the traditional "3+3" method combined with Sentinel method for dose escalation.

Conditions
Wilsons Disease
Being tested
Group 1: DSL101 Low dose, Group 2: DSL101 Medium dose, Group 3: DSL101 High dose
Medical subject headings
Hepatolenticular Degeneration

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Group 1: DSL101 Low doseDrug

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

  • Group 2: DSL101 Medium doseDrug

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

  • Group 3: DSL101 High doseDrug

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

Study groups in NCT07240896
Study groupRoleWhat they receive
Group 1: DSL101ExperimentalSubjects will receive intravenous infusions of DSL101 once every four weeks.
Receives: Drug: Group 1: DSL101 Low dose
Group 2: DSL101ExperimentalSubjects will receive intravenous infusions of DSL101 once every four weeks.
Receives: Drug: Group 2: DSL101 Medium dose
Group 3: DSL101ExperimentalSubjects will receive intravenous infusions of DSL101 once every four weeks.
Receives: Drug: Group 3: DSL101 High dose

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Incidence of adverse events and serious adverse events

    Measured: up to 56 weeks

Secondary outcomes (14)

  1. Change in 24 hour urine copper

    Measured: up to week 20

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

    Measured: up to week 20

  3. Change in serum free copper

    Measured: up to week 20

  4. Change from baseline in serum ceruloplasmin

    Measured: up to week20

  5. Change in serum iron concentration and serum ferritin concentration

    Measured: up to week 20

  6. Clinical laboratory test: Biochemistry - alanine aminotransferase (ALT)

    The physician will judge whether an abnormality is clinically significant

    Measured: up to week 20

  7. Change in the total score of the Unified Wilson Disease Rating Scale (UWDRS )

    The UWDRS scale consists of three parts: neurological, liver function and mental symptom. The higher scores mean a worse outcome.

    Measured: up to week 20

  8. Number of subjects and percentage decrease in standard of care (SOC) medication euse within 20 weeks of administration

    Measured: up to week 20

  9. PK: Average steady-state concentration (Cav,ss)

    Measured: up to week 6

  10. Change from baseline in seurm ceruloplasmin activity

    Measured: up to week20

  11. Clinical laboratory test: Biochemistry - aspartate aminotransferase (AST)

    Measured: up to week 20

  12. Clinical laboratory test: Biochemistry - alkaline phosphatase (ALP)

    Measured: up to week 20

  13. Clinical laboratory test: Biochemistry - gamma-glutamyltransferase (γ-GGT)

    Measured: up to week 20

  14. Clinical laboratory test: Biochemistry - total bilirubin (TBIL)

    Measured: up to week 20

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted21 Nov 2025
  2. Study start31 Dec 2025
  3. Primary completionApr 2028 (estimated)
  4. Study completionApr 2029 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Early Phase 1
Primary purpose
Treatment
Allocation
Non-randomised
Intervention model
Sequential
Masking
None (open label)
Enrolment
18 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT07240896
Sponsor's protocol ID
DSLIIT101
Status last verified
Jan 2026
First submitted
25 Sep 2025
Last updated
20 Jan 2026
Results posted
No
Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
No: The study is in early stages and will consider releasing related information when sufficient data is available in subjects.

Questions about this trial

What is this clinical trial studying?

A Early Phase 1 study testing Group 1: DSL101 Low dose, Group 2: DSL101 Medium dose and Group 3: DSL101 High dose in people with Wilsons Disease, run by DSciLab Co., Ltd. at 1 site in China.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 13 inclusion and 19 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: looking for participants now, as of the sponsor's last update on 20 Jan 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Early Phase 1 (sometimes called Phase 0) studies give a very small amount of a treatment to a few people, mainly to see how the body handles it.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

Each site lists its own contact: choose the nearest one under Trial locations. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Incidence of adverse events and serious adverse events (time frame: up to 56 weeks). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT07240896.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 20 Jan 2026.

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