cd7 car-t bridging to allohsct for severe aplastic anemia

Conditions: Aplastic Anemia

Summary: this is a single-arm, open-label, single-center, phase i study. the primary objective is to evaluate the safety of cd7 car-t bridging to allo-hsct therapy for patients with severe aplastic anemia

this is a prospective, open-label, single-center clinical trial. this study will evaluate the safety and efficacy of cd7 car-t bridging to allo-hsct in the treatment of severe aplastic anemia.the primary endpoints are dose limiting toxicity (dlt) and the incidence of treatment emergent adverse event (teae).

Eligibility criteria:
inclusion criteria: * chinese expert consensus on the diagnosis and treatment of aplastic anemia（2017）, diagnosis of severe aplastic anemia ，1. the degree of bone marrow cell proliferation \< 25%, or 25%-50% but residual hematopoietic cells \< 30%；2. with pancytopenia (at least two of the following peripheral blood parameters) : (1) absolute neutrophil \<0.5×10\^9/l; (2) platelet count\< 20×10\^9/l; (3) the absolute value of reticulocytes \<20×109/l; * suitable donors (relatives) with allogeneic hsct indications and at least haploid allogeneic transplantation; * patients who are not suitable or unwilling to undergo traditional allogeneic hematopoietic stem cell transplantation; * creatinine clearance \> 60ml/min; without liver invasion, serum total bilirubin ≤ 1.5 times the upper limit of normal, and serum alanine aminotransferase (alt) and aspartate aminotransferase (ast) were both ≤ 3 times the upper limit of the normal range. if there is liver invasion, serum erythrambirubin ≤ 3 times the upper limit of normal, and serum alt and ast are both ≤ 5 times the upper limit of the normal range; * echocardiogram shows left ventricular ejection fraction (lvef) ≥ 50%; * no active infection in the lungs, blood oxygen saturation in indoor air is ≥ 92%; * estimated survival time ≥ 3 months; * ecog performance status 0 to 1; * females and males of childbearing potential must agree to use adequate contraception prior to study entry, during study participation, and for 6 months after infusion (the safety of this therapy for unborn children is not known and has unknown risks); * those who voluntarily participated in this trial and provided informed consent; exclusion criteria: * allergy to pre-treatment measures； * those with acute graft versus host disease (gvhd) or moderate to severe chronic gvhd within 4 weeks before screening; those who have received systemic drug therapy for gvhd within 4 weeks before the reinfusion； * history of epilepsy or other central nervous system disorders； * electrocardiogram shows prolonged qt interval, severe heart diseases such as severe arrhythmia in the past; * less than 100 days after allogeneic hematopoietic stem cell transplantation； * patients with hiv infection，active infection of hepatitis b virus or hepatitis c virus，uncured active infection； * the proiferation rate is less than 5 times response to cd3/cd28 co-stimulation signal; * received anti-cancer chemotherapy or other drug treatment within 2 weeks prior to screening; * any condition that, in the opinion of the investigator, may increase the risk to the subject or interfere with the results of the study.

Keywords: allo-hsct cd7 car-t