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.
This study included the screening period (weeks -8 to days -2), the baseline period (days -1), the treatment and safety observation period (days 0 to 28), and the short-term follow-up period (weeks 5 \[from day 29\] to week 38). Participants eligible for the screening period will be admitted to the study center for a single LY-M001 treatment and a short-term follow-up period after the end of the treatment and safety observation period. Participants who complete all follow-up during the main study phase or who withdraw early from the study are required to complete all assessments required for the End of Study (EOS) visit.The study included up to three adult Gaucher disease type I subjects at preset dose group levels.With 5.0 × 1012 vg/kg as the initial effective dose (first dose group), 1 to 2 subjects are expected to be included. The first dose group was enrolled by sentinel method, and the first subject in this group was observed for at least 28 days after receiving LY-M001 (dose-limited toxicity \[DLT\] observation period) to enroll the next subject. Participants enrolled in the long-term follow-up study were those who completed the main study or withdrew early, and the duration of the long-term follow-up study and the main study lasted for a total of 5 years.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
3
With 5.0 × 10\^12 vg/kg as the initial effective dose (first dose group), 1 to 2 subjects are expected to be included. The first dose group was enrolled by sentinel method, and the first subject in this group was observed for at least 28 days after receiving LY-M001 (dose-limited toxicity \[DLT\] observation period) to enroll the next subject. After the first participant completes the safety evaluation of dosing for at least 28 days, the investigator (PI) and the partner discuss the safety and efficacy data and decide to maintain the first dose or increase/reduce it to another dose group
The First Affiliated Hospital of Zhejiang University School of Medicine
Hanzhou, Zhejiang, China
RECRUITINGIncidence of treatment- related adverse events
Number and severity of adverse events and serious adverse events and relationship to LY-M001
Time frame: within 38 weeks after LY-M001 infusion
Incidence of events adjudicated as dose limiting toxicity (DLT)
Number and severity of dose limiting toxicity
Time frame: within 28 days after LY-M001 infusion
Glucocerebrosidase (GCase) protein in plasma and GCase enzyme activity
Change from baseline in plasma GCase protein and GCase enzyme activity level
Time frame: Within 38 Weeks after LY-M001 infusion
Glucosylsphingosine (Lyso-GL1)in plasma
Change from baseline in plasma Lyso-GL1 level
Time frame: Within 38 Weeks after LY-M001 infusion
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