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

Официальное название

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

Обзор

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

Подробное описание

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.

Вмешательства

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

Первичные конечные точки

• Incidence of treatment-emergent adverse events (TEAEs) [Срок оценки: Up to 2 years after Treatment]
• Transplant related mortality rate [Срок оценки: Up to 100 days after Treatment]

Вторичные конечные точки (5)

• Allogeneic hematopoietic stem cell transplant implantation rate [Срок оценки: Up to 100 days after Treatment]
• Kidney transplantation implantation rate [Срок оценки: Up to 100 days after Treatment]
• Time to neutrophil and platelet engraftment [Срок оценки: Up to 30 days after Treatment]
• Disease-feesurvival，DFS [Срок оценки: Up to 2 years after Treatment]
• Overall survival, OS [Срок оценки: Up to 2 years after Treatment]

Критерии участия

Критерии включения

• 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;

Критерии исключения

• 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.

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Распределение

Не применимо

Модель

Одна группа

Маскирование

Открытое

Основная цель

Лечение

Центры проведения

Китай · 1 центр

• The first affiliated hospital of medical college of zhejiang university — Ханчжоу

Идентификаторы

NCT: NCT06769191 · TXB2024022

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

Открыть это исследование на ClinicalTrials.gov ↗