A Clinical Study on the Safety and Effectiveness of CD7 CAR-T Cell Sequential Allogeneic Hematopoietic Stem Cell Transplantation for Non-malignant Blood and Immune System Diseases
1. Non-malignant blood and immune system diseases include: hereditary bone marrow failure, congenital immune deficiency, hemoglobinopathy and other non-malignant blood and immune system diseases,
Confirmed hereditary bone marrow failure syndrome. Including: Fanconi anemia, congenital pure red cell aplastic anemia, congenital dyskeratosis, Scheux-Day syndrome, congenital neutropenia, various bone marrow failure related congenital thrombocytopenia and other unclassified congenital bone marrow exhaustion diseases;
It meets the criteria of clinical manifestation, immune function and gene diagnosis of immune deficiency disease;
Diagnosed with hemoglobinopathy and dependent on blood transfusions; serum ferritin levels are < 3000 μg/L, with cardiac and hepatic iron content indicating moderate or lower iron overload; documentation of iron chelation therapy (including prescriptions or invoices) for at least three months prior to screening is available; no hydroxyurea, ruxolitinib, decitabine, or cytarabine has been administered in the three months preceding enrollment. The spleen size must not extend beyond the umbilical horizontal line or the midline of the abdomen. Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is indicated, and suitable donors for related allo-HSCT are available.
1. People with a history of epilepsy or other central nervous system disorders;
2. Epstein-Barr virus (EBV) DNA positive;
3. People with a history of prolonged QT interval or serious heart disease;
4. People with active hepatitis B or C virus;
Single group
CD7 CAR-T cells injection
Biological/VaccineEach subject receive CD7 CAR T-cells by intravenous infusion
Allo-HSCT
Procedure/Surgeryallogeneic hematopoietic stem cell transplantation
Incidence of treatment-emergent adverse events (TEAEs)
Incidence of treatment-emergent adverse events
Transplant related mortality rate
The proportion of patients who died after transplantation to the total number of transplant patients during the same period
Allogeneic hematopoietic stem cell transplant implantation rate
The proportion of the number of patients who achieved hematopoietic reconstitution to the total number of allogeneic hematopoietic stem cell transplantation patients in the same period.
Time to neutrophil and platelet engraftment
The time for neutrophils and platelets to reach the implantation criteria after stem cell reinfusion
Disease-feesurvival，DFS
The proportion of disease-free patients who survived to the total number of patients who transplanted allogeneic hematopoietic stem cells during the same period.
Overall survival, OS
After transplantation until death from any cause.
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Zhejiang University
Lead sponsor
First Affiliated Hospital of Zhejiang University
Sponsor institution
Yake Biotechnology Ltd.
Collaborator
