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

NCT06769191 | Recruiting Early Phase 1

• 1 other identifier: TXB2024022
• Study Type: interventional
• Target: 20
• Locations: 1 country
• Sites: 1

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

Conditions

Schimke Immuno-osseous Dysplasia

Keywords

CAR-T; Allo-HSCT; Kidney Transplantation

Outcome Measures

Primary Outcomes (2)

• Incidence of treatment-emergent adverse events (TEAEs)

  Incidence of treatment-emergent adverse events

  Up to 2 years after Treatment

• Transplant related mortality rate

  The proportion of patients who died after transplantation to the total number of transplant patients during the same period

  Up to 100 days after Treatment

Secondary Outcomes (5)

• Allogeneic hematopoietic stem cell transplant implantation rate

  Up to 100 days after Treatment

• Kidney transplantation implantation rate

  Up to 100 days after Treatment

• Time to neutrophil and platelet engraftment

  Up to 30 days after Treatment

• Disease-feesurvival，DFS

  Up to 2 years after Treatment

• Overall survival, OS

  Up to 2 years after Treatment

Study Arms (1)

Treatment Group

EXPERIMENTAL

Schimke Immuno-osseous Dysplasia

Biological: CD7 CAR-T cells injection; Procedure: Allo-HSCT; Procedure: Kidney Transplantation

Interventions

CD7 CAR-T cells injection BIOLOGICAL

Intravenous infusion, single dose

Treatment Group

Allo-HSCT PROCEDURE

allogeneic hematopoietic stem cell transplantation

Treatment Group

Kidney Transplantation PROCEDURE

Kidney Transplantation

Treatment Group

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

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

You may not qualify if:

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

Sponsors & Collaborators

• Zhejiang University: lead
• Yake Biotechnology Ltd.: collaborator

Study Sites (1)

The first affiliated hospital of medical college of zhejiang university

Hangzhou, Zhejiang, 310003, China

RECRUITING

MeSH Terms

Conditions

Schimke immunoosseous dysplasia

Interventions

Kidney Transplantation

Intervention Hierarchy (Ancestors)

Renal Replacement Therapy; Therapeutics; Organ Transplantation; Transplantation; Surgical Procedures, Operative; Urologic Surgical Procedures; Urogenital Surgical Procedures

Study Officials

• He Huang, MD

  Zhejiang University

  PRINCIPAL INVESTIGATOR

Central Study Contacts

He Huang, MD

CONTACT

057187233772; hehuangyu@126.com

Yongxian Hu, MD

CONTACT

057187233772; huyongxian2000@aliyun.com

Study Design

• Study Type: interventional
• Phase: early phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Principal Investigator

Study Record Dates

• First Submitted: January 2, 2025
• First Posted: January 10, 2025
• Study Start: January 30, 2025
• Primary Completion (Estimated): January 30, 2028
• Study Completion (Estimated): January 30, 2028
• Last Updated: January 20, 2025

Record last verified: 2025-01

Locations

CN (1)