Clinical Study on the Safety and Efficacy of CD7 CAR-T Cell Sequential Allo-HSCT and Kidney Transplantation in the Treatment of SIOD

Zhejiang University

Status and phase

Enrolling

Early Phase 1

Conditions

Schimke Immuno-osseous Dysplasia

Treatments

Biological: CD7 CAR-T cells injection

Procedure: Kidney Transplantation

Procedure: Allo-HSCT

Study type

Interventional

Funder types

Other

Industry

Identifiers

NCT06769191

TXB2024022

Details and patient eligibility

About

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

Full 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.

Enrollment

20 estimated patients

Sex

All

Volunteers

No Healthy Volunteers

Inclusion criteria

1. Diagnosed as SIOD and was in stage 5 of chronic kidney disease
1. Having allogeneic HSCT indications, at least suitable donors (relatives) for haploidentical allogeneic transplantation and kidneys from stem cell transplantation donors;
1. 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.
1. Echocardiogram shows left ventricular ejection fraction (LVEF) ≥ 50%;
1. There is no active pulmonary infection, and the oxygen saturation during air inhalation is more than 92%；
1. Estimated survival time ≥ 3 months;
1. ECOG performance status 0 to 1;
1. 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;
1. Those who voluntarily participated in this trial and provided informed consent;

Exclusion criteria

1. Allergic to pretreatment measures
1. 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;
1. Patients with the history of epilepsy or other CNS disease;
1. Patients with prolonged QT interval time or severe heart disease;
1. Previous recipients of allogeneic hematopoietic stem cell transplantation or organ transplantation
1. People infected with HIV, active hepatitis B or hepatitis C virus, and patients with active infection who are not cured;
1. The proiferation rate is less than 5 times response to CD3/CD28 co-stimulation signal;
1. Patients with malignant tumor;
1. People with other genetic diseases；
1. 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.
1. Any situation that researchers believe may increase the risk to the subjects or interfere with the trial results.