cd7 car-t cell sequential allo-hsct for non-malignant blood and immune system diseases

Conditions: Bone Marrow Failure Syndrome

Summary: 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

this is a single-arm, open-label clinical trial to evaluate the safety and efficacy of cd7 car-t cell sequential allogeneic hematopoietic stem cell transplantation for non-malignant blood and immune system diseases. it is planned to enroll 12-20 participants in this trial.

Eligibility criteria:
inclusion criteria: * 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, 1. 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; 2. it meets the criteria of clinical manifestation, immune function and gene diagnosis of immune deficiency disease; 3. 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. * 2. serum total bilirubin ≤1.5 times the upper limit of normal value, serum alanine aminotransferase (alt) and aspartate aminotransferase (ast) ≤ 3 times the upper limit of normal value range; * 3. echocardiography showed left ventricular ejection fraction (lvef) \>50%; * 4. pulse oxygen saturation ≥92% (non-oxygen state); * 5. the estimated survival is more than 3 months; * 6. ecog score 0-1; * 7. abdominal b-ultrasonography and other examinations were performed to evaluate spleen size. splenectomy should be evaluated before transplantation for patients with giant spleen; * 8. women and men who are fertile must consent to the use of appropriate contraception before entering the study, during study participation, and for 6 months after transfusion (the safety of this therapy for the unborn child is not known, with unknown risks); * 9. subjects who are willing to participate in the study are able to understand and have the ability to sign informed consent. exclusion criteria: * 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; * 5. tuberculosis, aids and other major infectious diseases; * 6. sepsis, pulmonary infection, intestinal infection and other major organ infection and poor control, and/or hypersensitive c-reactive protein, procalcitonin significantly elevated; * 7. people who have previously received other clinical studies and gene therapy; * 8. any situation that the investigator believes may increase the risk to the subject or interfere with the test results.

Keywords: car-t allo-hsct immuno deficiency disorders