Gaucher disease
Conditions
Interventions
Treatment group:LY-M001 investigational drug is administered by a single intravenous injection
Sponsors
Shanghai Children's Medical Center
Eligibility
Sex/Gender
All
Age
12 Years to 18 Years
Inclusion criteria
Inclusion criteria: 1.Subjects and/or parents, caregivers, or legal representatives 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 visits and procedures, including the use of any data collection devices that can be used to directly record participant data; 2.Gender is not limited, 12 years = age < 18 years; 3.Subjects with double allele mutation of the glucocerebrosidase gene (GBA1) and decreased glucocerebrosidase activity were confirmed by laboratory tests and met the clinical manifestations of type I Gaucher disease; 4.Subjects were newly treated or treated patients with type I Gaucher disease; Patients treated with enzyme replacement therapy (ERT) or substrate clearance therapy (SRT) prior to screening need to eluate five drug half-lives before dosing; 5.Subjects are willing to participate in all study follow-up and comply with all study procedures and evaluations; 6.Subjects must be willing to refrain from donating blood, organs, tissues, or cells at any time after receiving treatment. 7.Pregnant women (WOCBP) subjects tested negative for pregnancy
Exclusion criteria
Exclusion criteria: 1.Positive for AAV8 neutralizing antibody; 2.Subjects with type II or III Gaucher disease (GD2 or GD3), or suspected to have type III Gaucher disease as assessed by the investigator (e.g., subjects with Gaucher disease-related central nervous system manifestations or abnormal electroencephalography [EEG] examination); 3.Active and progressive bone disease that is expected to require surgical treatment within the next 6 months; 4.As determined by the investigator, the subjects had idiopathic thrombocytopenic purpura (ITP), thrombotic thrombocytopenic purpura (TTP), thrombocytopenia, anemia, hepatomegalia, splenomegalia, and/or osteoporosis unrelated to GD; 5.Treatment with an investigational drug from another clinical study within 28 days prior to screening or 5 half-lives, whichever is older; 6.Subject blood blood indicators have any of the following:? The hemoglobin value was 103 copies /mL); Or take hepatitis B drugs (such as interferon, lamivudine, adefovir and entecavir); Or hepatitis C virus (HCV) antibody positive; Or human immunodeficiency virus (HIV) antibody positive or treponema syphilis antibody positive; 9.Subjects have received or plan to receive bone marrow transplantation, hematopoietic stem cell transplantation, and/or major organ transplant, including but not limited to liver transplantation, kidney transplantation, etc; 10. 3 months before screening, subjects received erythropoietin, whole blood transfusion, or red blood cell transfusion; Or received platelet transfusion 1 month before screening; 11. Allergic to any component of LY-M001 injection; 12. Prior treatment with any type of gene therapy or cell therapy.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Incidence of adverse events (AE) and serious adverse events (SAE) within 52 weeks after infusion of LY-M001; | — |
Secondary
| Measure | Time frame |
|---|---|
| Blood glucocerebrosidase (GCase) protein levels and activity levels;Blood glucose sphingosine (Lyso-GL1) levels;Liver volume and spleen volume;Hemoglobin levels and platelet counts; | — |
Countries
China
Contacts
Public ContactXiumin Wang
Shanghai Children's Medical Center
Outcome results
None listed