Clinical trial NCT06663878: An Exploratory Study to Evaluate the Tolerability and Safety of MWAV201 in Subjects With Wilson Disease
Official title: An Open-Label, Dose Escalation Study to Evaluate the Tolerability, Safety, and Preliminary Efficacy of MWAV201 in Subjects With Wilson Disease
Study type: INTERVENTIONAL
Overall status: NOT_YET_RECRUITING
Phase: NA
Conditions: Wilson Disease
Lead sponsor: Xinhua Hospital, Shanghai Jiao Tong University School of Medicine
Sponsor class: OTHER
Collaborators: name: Mabwell (Shanghai) Bioscience Co., Ltd.
First posted: 2024-10-29
Enrollment: 9
Sex: ALL
Minimum age (years): 18
Maximum age (years): 65
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Intervention model description: Participants will receive a single, peripheral intravenous (IV) infusion of MWAV201.
Primary purpose: TREATMENT

Brief summary: 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.

Eligibility criteria: Inclusion Criteria:

* 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.

Exclusion Criteria:

* 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.
* 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.

Primary outcomes:
measure: Safety and tolerability profile [including adverse events (AEs) and serious adverse events (SAEs)]
timeFrame: At 1-Year post treatment
Secondary outcomes:
measure: A Laboratory Outcome Measure: Total serum copper
timeFrame: At 1-Year post treatment
measure: A Laboratory Outcome Measure: 24-hour urinary copper
timeFrame: At 1-Year post treatment
measure: A Laboratory Outcome Measure: Aspartate aminotransferase to platelet ratio index
timeFrame: At 1-Year post treatment
Other outcomes:
Locations:
city: Shanghai
country: China
facility: Xinhua Hospital, Shanghai Jiao Tong University School of Medicine
state: Shanghai
Study officials:
affiliation: Xinhua Hospital, Shanghai Jiao Tong University School of Medicine
name: Jian-Gao Fan, doctor
role: PRINCIPAL_INVESTIGATOR
References: