Clinical Study of LY-M001 Injection in the Treatment of Adolescents With Type I Gaucher Disease
NCT Number: NCT06528080
Status: RECRUITING
Study Type: INTERVENTIONAL
Phases: EARLY_PHASE1
Conditions: Gaucher Disease
Interventions: GENETIC: LY-M001
Sponsor: Shanghai Jiao Tong University School of Medicine
Sex: ALL
Age: CHILD, ADULT
Enrollment: 9
Study Design: Allocation: NA|Intervention Model: SINGLE_GROUP|Masking: NONE|Primary Purpose: TREATMENT
Start Date: 2024-08-01
Completion Date: 2029-08
Locations: Shanghai Children's Medical Center, School of Medicine, Shanghai Jiao Tong University, Shanghai, Shanghai, China

Summary:
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.

Primary Outcome Measures: Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability], The incidence of adverse events (AE) and serious adverse events (SAE), Within 52 weeks of infusion
Secondary Outcome Measures: pharmacodynamics on blood glucocerebrosidase, Blood glucocerebrosidase (GCase) protein level and activity level, at 52 weeks after infusion and Week 53 to 5 years after infusion of LY-M001|pharmacodynamics on Blood glucose sphingosine, Blood glucose sphingosine (Lyso-GL1) levels, at 52 weeks after infusion and Week 53 to 5 years after infusion of LY-M001|Effectiveness of symptom improvement on Liver volume, Liver volume, at 52 weeks after infusion and Week 53 to 5 years after infusion of LY-M001|Effectiveness of symptom improvement on spleen volume, spleen volume, at 52 weeks after infusion and Week 53 to 5 years after infusion of LY-M001|Effectiveness of symptom improvement on Hemoglobin levels, Hemoglobin levels, at 52 weeks after infusion and Week 53 to 5 years after infusion of LY-M001|Effectiveness of symptom improvement on platelet counts, platelet counts, at 52 weeks after infusion and Week 53 to 5 years after infusion of LY-M001|Effectiveness of symptom improvement on Bone mineral density (BMD), Bone mineral density (BMD), at 52 weeks after infusion and Week 53 to 5 years after infusion of LY-M001|Effectiveness of symptom improvement on bone marrow load (BMB) after administration, bone marrow load (BMB) after administration, at 52 weeks after infusion and Week 53 to 5 years after infusion of LY-M001|Effectiveness of symptom improvement on GD patient Quality of Life scale score, GD patient Quality of Life scale score, at 52 weeks after infusion and Week 53 to 5 years after infusion of LY-M001|Effectiveness of symptom improvement on GD Fatigue Scale-14 score, GD Fatigue Scale-14 score, at 52 weeks after infusion and Week 53 to 5 years after infusion of LY-M001|Immunogenicity of production of AAV8 anti-drug antibodies (ADA) in the blood, AAV8 anti-drug antibody (ADA) production in the blood, at 52 weeks after infusion and Week 53 to 5 years after infusion of LY-M001|immunogenicity of production of AAV8 neutralizing antibody (NAb) in the blood, AAV8 neutralizing antibody (NAb) production in the blood, at 52 weeks after infusion and Week 53 to 5 years after infusion of LY-M001|immunogenicity of production of anti-drug antibody (ADA) in the blood, anti-drug antibody (ADA) production of GCase in blood, at 52 weeks after infusion and Week 53 to 5 years after infusion of LY-M001|immunogenicity in the cells, Enzyme-linked immunospot assay (ELISPOT) was used to detect the immunogenicity in the cells, at 52 weeks after infusion and Week 53 to 5 years after infusion of LY-M001