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Recruiting NCT06769191

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 I Interventional Schimke Immuno-osseous Dysplasia

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: CD7 CAR-T cells injection, Allo-HSCT, Kidney Transplantation.
Who it may be relevant to
Registry conditions: Schimke Immuno-osseous Dysplasia. Basic parameters: No limits · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
China
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Unsure about the terms? Read our patient guide →

Official title

Clinical Study on the Safety and Efficacy of CD7 CAR-T Cell Sequential Allogeneic Hematopoietic Stem Cell Transplantation and Kidney Transplantation in the Treatment of Schimke Immuno-osseous Dysplasia

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

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.

Interventions

  • Biological CD7 CAR-T cells injection
    Intravenous infusion, single dose
  • Procedure Allo-HSCT
    allogeneic hematopoietic stem cell transplantation
  • Procedure Kidney Transplantation
    Kidney Transplantation

Primary outcome measures

  • Incidence of treatment-emergent adverse events (TEAEs) [Time frame: Up to 2 years after Treatment]
  • Transplant related mortality rate [Time frame: Up to 100 days after Treatment]
Secondary outcome measures (5)
  • Allogeneic hematopoietic stem cell transplant implantation rate [Time frame: Up to 100 days after Treatment]
  • Kidney transplantation implantation rate [Time frame: Up to 100 days after Treatment]
  • Time to neutrophil and platelet engraftment [Time frame: Up to 30 days after Treatment]
  • Disease-feesurvival，DFS [Time frame: Up to 2 years after Treatment]
  • Overall survival, OS [Time frame: Up to 2 years after Treatment]

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.

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation
N/A
Model
Single group
Masking
Open label
Primary purpose
Treatment

Study locations

China · 1 center
  • The first affiliated hospital of medical college of zhejiang university — Hangzhou

Identifiers

NCT: NCT06769191 · TXB2024022

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗