A Prospective, Single-center, Open-arm, Single-arm Clinical Study to Evaluate the Safety and Efficacy of a Single Intravenous Infusion of LY-M001 Injection in Adolescents With Type I Gaucher Disease
The purpose of this study was to evaluate the safety, tolerability, efficacy, immunogenicity, PD and PK characteristics of LY-M001 injection in children with GD1 aged 12 years ≤ age \< 18 years. This study mainly includes the main study stage and the long-term follow-up study stage.
• The subject and/or parent, caregiver, or legal representative must be willing and able to provide written informed consent/consent for the study in accordance with applicable regulations and guidelines and comply with all study access and procedures, including the use of any data collection devices that can be used to directly record participant data;
• Gender is not limited, 12 years old ≤ 18 years old;
• Patients with double allele mutation of glucocerebrosidase gene (GBA1) and decreased glucocerebrosidase activity were confirmed by laboratory tests and met the clinical manifestations of type I Gaucher disease;
• Subjects were newly treated or treated patients with type I Gaucher disease; For patients treated with enzyme replacement therapy (ERT) or substrate clearance therapy (SRT) before screening, 5 drug half-lives are required before administration;
• The subject is willing to participate in all study follow-up and comply with all study procedures and evaluations;
• The subject must be willing to refrain from donating blood, organs, tissues, or cells at any time after receiving treatment;
• Pregnant Women (WOCBP) subjects tested negative for pregnancy. Note: WOCBP is defined as a female subject who has not achieved postmenopausal status (continuous amenorrhea for at least 12 months with no clear cause other than menopause) after menstruating, and who has no surgical (i.e., bilateral ovariectomy, fallopian tube removal, and/or hysterectomy) or other investigator identified cause of permanent infertility (e.g., mallear tube agenesis).