A Study of the Safety and Preliminary Efficacy of LY-M001 Injection in the Treatment of Adult Patients With Gaucher Disease Type I

Recruiting N/A Interventional Study
Gaucher Disease Type I
No Placebo Group Every participant receives an active treatment — no one gets a placebo.
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At a Glance
Age
18 – 60
Sex
Any
Study type
Interventional
Purpose
Treatment
Participants needed
3 (estimated)
Sponsor
He Huang · Other
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About This Trial

This is a prospective single-center, open, single-arm, single-dose intravenous infusion study to evaluate the safety and initial efficacy, pharmacodynamic characteristics, immunogenicity, biodistribution, and viral shedding of LY-M001 injection.This study mainly includes the main study stage and the long-term follow-up study stage.

Trial Locations
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Eligibility Criteria
Inclusion Criteria: 1. Age ≥ 18 years and ≤ 60 years, male or female. 2. The subjects should fully understand the purpose, nature and method of this study as well as possible adverse reactions, and sign the informed consent form (ICF) voluntarily. 3. Patients with GD1 who have confirmed double muta…
Contacts

He Huang, PhD

0571-87236703

hehuangyu@126.com

CONTACT