Overview
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
Description
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.
Eligibility
Inclusion Criteria:
- 1. Diagnosed as SIOD and was in stage 5 of chronic kidney disease
- 2. Having allogeneic HSCT indications, at least suitable donors (relatives) for haploidentical allogeneic transplantation and kidneys from stem cell transplantation donors;
- 3. 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.
- 4. Echocardiogram shows left ventricular ejection fraction (LVEF) ≥ 50%;
- 5. There is no active pulmonary infection, and the oxygen saturation during air inhalation is more than 92%；
- 6. Estimated survival time ≥ 3 months;
- 7. ECOG performance status 0 to 1;
- 8. Pregnant/lactating women, or male or female patients who have fertility and are willing to take effective contraceptive measures at least 6 months after the last cell infusion during the study period;
- 9. Those who voluntarily participated in this trial and provided informed consent;
Exclusion Criteria:
- 1. Allergic to pretreatment measures
- 2. received any containing ATG/ALG such IST、alemtuzumab、high-dose cyclophosphamide (≥ 45mg/kg/day) , received CsA treatment within 6 months, or used thrombopoietin receptor (tpo-r) agonists in the past;
- 3. Patients with the history of epilepsy or other CNS disease;
- 4. Patients with prolonged QT interval time or severe heart disease;
- 5. Previous recipients of allogeneic hematopoietic stem cell transplantation or organ transplantation
- 6. People infected with HIV, active hepatitis B or hepatitis C virus, and patients with active infection who are not cured;
- 7. The proiferation rate is less than 5 times response to CD3/CD28 co-stimulation signal;
- 8. Patients with malignant tumor;
- 9. People with other genetic diseases；
- 10. After receiving CD7 car-t treatment, patients who were unable to accept subsequent kidney transplantation due to severe infection or poor amplification of car-t in vivo.
- 11. Any situation that researchers believe may increase the risk to the subjects or interfere with the trial results.