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

Launched by ZHEJIANG UNIVERSITY · Jan 8, 2025

Keywords

ClinConnect Summary

This clinical trial is studying a new treatment approach for Schimke Immuno-osseous Dysplasia (SIOD), a rare genetic condition that can lead to serious health issues, including kidney problems. The trial is looking at a combination of advanced therapies: CD7 CAR-T cell therapy followed by a stem cell transplant and kidney transplant. The goal is to see if this approach is safe and effective for patients in the later stages of chronic kidney disease.

To participate in this study, patients must be diagnosed with SIOD and have stage 5 chronic kidney disease. They should have suitable donors for the stem cell and kidney transplants and meet certain health criteria, such as having stable liver function and good heart health. Participants can expect thorough monitoring during the treatment process, and they will need to provide informed consent to join. This trial is currently recruiting participants of all ages and genders, and it aims to improve treatment options for those affected by this challenging condition.

Gender

ALL

Eligibility criteria

• 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.