A Clinical Study to Evaluate the Safety and Efficacy of LY-M003 Injection in Patients With Wilson Disease
Recruiting
Early Phase 1
Interventional Study
Wilson Disease
No Placebo Group
Every participant receives an active treatment — no one gets a placebo.
At a Glance
- Age
- 10 – 60
- Sex
- Any
- Trial phase
- Early Phase 1
- Study type
- Interventional
- Purpose
- Treatment
- Participants needed
- 18 (estimated)
- Sponsor
- Chaohui Yu · Other
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About This Trial
Wilson's disease (WD), also known as Wilson's disease, is a rare autosomal recessive metabolic disorder caused by a mutation of the copper transport ATPase β (ATP7B) gene located on the long arm of chromosome 13 (13q14.3). This leads to accumulation of copper ions in multiple organs such as liver, brain and kidney, resulting in organ involvement. In this study, LY-M003 Injection is a gene therapy …
Trial Locations
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Eligibility Criteria
Inclusion Criteria:
1. The subject must be able to fully understood the purpose, nature, method, and possible adverse effects of the study, must be able to voluntarily participate in the study and voluntarily able to provide the written informed consent form (ICF).
2. Patients diagnosed with Wilson…
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