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

Early Phase 1

Recruiting

• Conditions: Schimke Immuno-osseous Dysplasia
• Interventions: Biological: CD7 CAR-T cells injection; Procedure: Allo-HSCT; Procedure: Kidney Transplantation

• Registration Number: NCT06769191
• Lead Sponsor: Zhejiang University

Brief Summary

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

Detailed 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.

Study Timeline

• Status Verified: 2025-01
• Study First Submitted: 2025-01-02
• Study First Submitted QC: 2025-01-08
• Study First Posted: 2025-01-10
• Last Update Submitted: 2025-01-16
• Last Update Posted: 2025-01-20
• Study Start: 2025-01-30
• Primary Completion: 2028-01-30
• Study Completion: 2028-01-30

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 20

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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Treatment Group	Allo-HSCT	Schimke Immuno-osseous Dysplasia
Treatment Group	Kidney Transplantation	Schimke Immuno-osseous Dysplasia
Treatment Group	CD7 CAR-T cells injection	Schimke Immuno-osseous Dysplasia

Primary Outcome Measures

Name	Time	Method
Incidence of treatment-emergent adverse events (TEAEs)	Up to 2 years after Treatment	Incidence of treatment-emergent adverse events
Transplant related mortality rate	Up to 100 days after Treatment	The proportion of patients who died after transplantation to the total number of transplant patients during the same period

Secondary Outcome Measures

Name	Time	Method
Overall survival, OS	Up to 2 years after Treatment	After transplantation until death from any cause.
Allogeneic hematopoietic stem cell transplant implantation rate	Up to 100 days after Treatment	The proportion of the number of patients who achieved hematopoietic reconstitution to the total number of allogeneic hematopoietic stem cell transplantation patients in the same period.
Kidney transplantation implantation rate	Up to 100 days after Treatment	The ratio of successfully implanted kidneys to the total implanted kidneys
Time to neutrophil and platelet engraftment	Up to 30 days after Treatment	The time for neutrophils and platelets to reach the implantation criteria after stem cell reinfusion
Disease-feesurvival，DFS	Up to 2 years after Treatment	The proportion of disease-free patients who survived to the total number of patients who transplanted allogeneic hematopoietic stem cells during the same period.

Trial Locations

Locations (1)

The first affiliated hospital of medical college of zhejiang university; 🇨🇳 Hangzhou, Zhejiang, China