A Clinical Study on the Safety and Effectiveness of CD7 CAR-T Cell Sequential Allo-HSCT and Kidney Transplantation in the treatment of Schimke immuno-osseous dysplasia
This is a single-arm, open-label, dose-escalation clinical trial to evaluate the safety and efficacy of CD7 CAR-T Cell Sequential Allo-HSCT and Kidney Transplantation in patients with Schimke immuno-osseous dysplasia. It is planned to enroll 20 participants in this trial.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
20
Intravenous infusion, single dose
allogeneic hematopoietic stem cell transplantation
Kidney Transplantation
The first affiliated hospital of medical college of zhejiang university
Hangzhou, Zhejiang, China
RECRUITINGIncidence of treatment-emergent adverse events (TEAEs)
Incidence of treatment-emergent adverse events
Time frame: Up to 2 years after Treatment
Transplant related mortality rate
The proportion of patients who died after transplantation to the total number of transplant patients during the same period
Time frame: Up to 100 days after Treatment
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 frame: Up to 100 days after Treatment
Kidney transplantation implantation rate
The ratio of successfully implanted kidneys to the total implanted kidneys
Time frame: Up to 100 days after Treatment
Time to neutrophil and platelet engraftment
The time for neutrophils and platelets to reach the implantation criteria after stem cell reinfusion
Time frame: Up to 30 days after Treatment
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.
Time frame: Up to 2 years after Treatment
Overall survival, OS
After transplantation until death from any cause.
Time frame: Up to 2 years after Treatment
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