An Exploratory Study to Evaluate the Tolerability and Safety of MWAV201 in Subjects With Wilson Disease

NCT06663878 | Not Yet Recruiting

• 2 other identifiers: MWAV201-CP1012021YFC2700802
• Study Type: interventional
• Target: 9
• Locations: 1 country
• Sites: 1

Brief Summary

The primary objective of this study is to evaluate the tolerability and safety of MWAV201 in patients with Wilson disease.

Conditions

Wilson Disease

Outcome Measures

Primary Outcomes (1)

• 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.

  At 1-Year post treatment

Secondary Outcomes (3)

• A Laboratory Outcome Measure: Total serum copper

  At 1-Year post treatment

• A Laboratory Outcome Measure: 24-hour urinary copper

  At 1-Year post treatment

• A Laboratory Outcome Measure: Aspartate aminotransferase to platelet ratio index

  At 1-Year post treatment

Study Arms (1)

MWAV201

EXPERIMENTAL

All subjects will receive a single intravenous (IV) infusion of MWAV201.

Genetic: MWAV201

Interventions

MWAV201 GENETIC

Nonreplicating, recombinant gene transfer vector

MWAV201

Eligibility Criteria

• Age: 18 Years - 65 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Xinhua Hospital, Shanghai Jiao Tong University School of Medicine: lead
• Mabwell (Shanghai) Bioscience Co., Ltd.: collaborator

Study Sites (1)

Xinhua Hospital, Shanghai Jiao Tong University School of Medicine

Shanghai, Shanghai Municipality, 200092, China

Location

MeSH Terms

Conditions

Hepatolenticular Degeneration

Condition Hierarchy (Ancestors)

Liver Diseases; Digestive System Diseases; Basal Ganglia Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Movement Disorders; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Metabolism, Inborn Errors; Metal Metabolism, Inborn Errors; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Officials

• Jian-Gao Fan, doctor

  Xinhua Hospital, Shanghai Jiao Tong University School of Medicine

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Jian-Gao Fan, doctor

CONTACT

86-21-25077344; fanjiangao@xinhuamed.com.cn

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Model Details: Participants will receive a single, peripheral intravenous (IV) infusion of MWAV201.

Study Record Dates

• First Submitted: October 10, 2024
• First Posted: October 29, 2024
• Study Start: October 1, 2024
• Primary Completion: March 1, 2026
• Study Completion (Estimated): May 1, 2031
• Last Updated: October 29, 2024

Record last verified: 2024-10

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)