Wilson DiseaseNCT06663878
An Exploratory Study to Evaluate the Tolerability and Safety of MWAV201 in Subjects With Wilson Disease
A study testing MWAV201 in people with Wilson Disease, run by Xinhua Hospital, Shanghai Jiao Tong University School of Medicine at 1 site in China.
Trial summary
The primary objective of this study is to evaluate the tolerability and safety of MWAV201 in patients with Wilson disease. Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Wilson Disease
- What is being tested?
- MWAV201
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 9 participants
- Where
- 1 siteChina
- Study dates
- 28 Oct 2024to May 2031
- Phase
- Interventional
- 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 to 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Male or female aged 18 and 65 years inclusive;
- Confirmed diagnosis of Wilson disease;
- Low copper diet and standardized medication for ≥ 1 year;
- Stable Wilson disease for ≥ 1 year;
- Able to understand and willing to follow study procedures.
Who cannot join
- Significant hepatic inflammation as evidenced by liver function test.
- Liver biopsy or liver stiffness measurement show progressive liver fibrosis.
- Laboratory tests or clinical symptoms indicate decreased liver reserve function.
- Other chronic liver disease (such as hepatitis B).
- Any signs of decompensated liver function (such as ascites).
- History of liver transplant or plan to receive liver transplant.
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- Other diseases with clinical significance, such as cardiovascular and cerebrovascular diseases, kidney diseases, respiratory system diseases, neurological diseases, mental illnesses, active infections, etc.
- Body Mass Index ≥ 30 kg/m2.
- Other conditions that, in the Investigator's opinion, may not be suitable for the subject to be enrolled.
Trial locations and contacts
1 site in 1 country.
China1 site
Xinhua Hospital, Shanghai Jiao Tong University School of Medicine
Shanghai, Shanghai Municipality, 200092
About this study
Official title: An Open-Label, Dose Escalation Study to Evaluate the Tolerability, Safety, and Preliminary Efficacy of MWAV201 in Subjects With Wilson Disease
The primary objective of this study is to evaluate the tolerability and safety of MWAV201 in patients with Wilson disease.
Read the detailed description
This is an open-label, dose escalation study to evaluate the tolerability, safety, and preliminary efficacy of MWAV201 in patients with Wilson disease. Participants will receive a single, peripheral intravenous (IV) infusion of MWAV201. The dose escalation plan and the number of dose levels may be adjusted during study period.
- Conditions
- Wilson Disease
- Being tested
- MWAV201
- Medical subject headings
- Hepatolenticular Degeneration
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
MWAV201Genetic
Nonreplicating, recombinant gene transfer vector
| Study group | Role | What they receive |
|---|---|---|
| MWAV201 | Experimental | All subjects will receive a single intravenous (IV) infusion of MWAV201. Receives: Genetic: MWAV201 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety and tolerability profile [including adverse events (AEs) and serious adverse events (SAEs)]
AEs will be summarized based on incidence and the date of onset for the event.
Measured: At 1-Year post treatment
Secondary outcomes (3)
A Laboratory Outcome Measure: Total serum copper
Total serum copper will be summarized descriptively for all patients by dose cohort and planned visit, for absolute values, changes from baseline and percent change from baseline.
Measured: At 1-Year post treatment
A Laboratory Outcome Measure: 24-hour urinary copper
24-hour urinary copper will be summarized descriptively for all patients by dose cohort and planned visit, for absolute values, changes from baseline and percent change from baseline.
Measured: At 1-Year post treatment
A Laboratory Outcome Measure: Aspartate aminotransferase to platelet ratio index
Aspartate aminotransferase to platelet ratio index will be summarized descriptively for all patients by dose cohort and planned visit, for absolute values, changes from baseline and percent change from baseline.
Measured: At 1-Year post treatment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Oct 2024
- Study start28 Oct 2024
- Primary completionMar 2031 (estimated)
- Study completionMay 2031 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Wilson DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- Participants will receive a single, peripheral intravenous (IV) infusion of MWAV201.
- Masking
- None (open label)
- Enrolment
- 9 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Xinhua Hospital, Shanghai Jiao Tong University School of Medicine
- Sponsor type
- University, hospital or other
- Collaborators
- Mabwell (Shanghai) Bioscience Co., Ltd.
- Responsible party
- Sponsor
- Investigators
- Jian-Gao Fan, doctor, Principal investigator, Xinhua Hospital, Shanghai Jiao Tong University School of Medicine
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06663878
- Sponsor's protocol ID
- MWAV201-CP101
- Other IDs
- 2021YFC2700802
- Status last verified
- Jul 2026
- First submitted
- 10 Oct 2024
- Last updated
- 12 Aug 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No: This is a study initiated by investigator.
Study at a glance
From the registry record, last updated by the sponsor on 12 Aug 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-Label, Dose Escalation Study to Evaluate the Tolerability, Safety, and Preliminary Efficacy of MWAV201 in Subjects With Wilson Disease
- ClinicalTrials.gov ID
- NCT06663878
- Acronym / sponsor ID
- MWAV201-CP101
- Conditions
- Wilson Disease
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 9 participants (estimated)
- Lead sponsor
- Xinhua Hospital, Shanghai Jiao Tong University School of MedicineUniversity, hospital or other
- Collaborators
- Mabwell (Shanghai) Bioscience Co., Ltd.
- Study start
- 28 Oct 2024 (actual)
- Primary completion
- Mar 2031 (estimated)
- Study completion
- May 2031 (estimated)
- First posted
- 29 Oct 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The primary objective of this study is to evaluate the tolerability and safety of MWAV201 in patients with Wilson disease.
Detailed description
This is an open-label, dose escalation study to evaluate the tolerability, safety, and preliminary efficacy of MWAV201 in patients with Wilson disease. Participants will receive a single, peripheral intravenous (IV) infusion of MWAV201. The dose escalation plan and the number of dose levels may be adjusted during study period.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- Participants will receive a single, peripheral intravenous (IV) infusion of MWAV201.
| Arm | Type | Receives |
|---|---|---|
| MWAV201 | Experimental | Genetic: MWAV201All subjects will receive a single intravenous (IV) infusion of MWAV201. |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- Male or female aged 18 and 65 years inclusive;
- Confirmed diagnosis of Wilson disease;
- Low copper diet and standardized medication for ≥ 1 year;
- Stable Wilson disease for ≥ 1 year;
- Able to understand and willing to follow study procedures.
Key exclusion criteria (9 listed)
- Significant hepatic inflammation as evidenced by liver function test.
- Liver biopsy or liver stiffness measurement show progressive liver fibrosis.
- Laboratory tests or clinical symptoms indicate decreased liver reserve function.
- Other chronic liver disease (such as hepatitis B).
- Any signs of decompensated liver function (such as ascites).
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| MWAV201Nonreplicating, recombinant gene transfer vector | Genetic | MWAV201 |
Primary outcome
Safety and tolerability profile [including adverse events (AEs) and serious adverse events (SAEs)]
Time frame: At 1-Year post treatment
AEs will be summarized based on incidence and the date of onset for the event.
3 secondary outcomes
A Laboratory Outcome Measure: Total serum copper
Time frame: At 1-Year post treatment
A Laboratory Outcome Measure: 24-hour urinary copper
Time frame: At 1-Year post treatment
A Laboratory Outcome Measure: Aspartate aminotransferase to platelet ratio index
Time frame: At 1-Year post treatment
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 12 Aug 2026.
- Individual participant dataSharing planned: No. This is a study initiated by investigator.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Jian-Gao Fan, doctor principal investigator
Xinhua Hospital, Shanghai Jiao Tong University School of Medicine
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing MWAV201 in people with Wilson Disease, run by Xinhua Hospital, Shanghai Jiao Tong University School of Medicine 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 5 inclusion and 9 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: under way, not looking for participants, as of the sponsor's last update on 12 Aug 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.
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?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Safety and tolerability profile [including adverse events (AEs) and serious adverse events (SAEs)] (time frame: At 1-Year post treatment). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 12 Aug 2026.