CD7 CAR-T Bridging to alloHSCT for Severe Aplastic Anemia

Официальное название

Clinical Study on the Safety and Efficacy of Donor Derived CD7 CAR-T Cell Bridging Allogeneic Hematopoietic Stem Cell Transplantation for the for Patients With Severe Aplastic Anemia

Обзор

This is a single-arm, open-label, single-center, phase I study. The primary objective is to evaluate the safety of CD7 CAR-T Bridging to allo-HSCT therapy for patients with severe aplastic anemia

Подробное описание

This is a prospective, open-label, single-center clinical trial. This study will evaluate the safety and efficacy of CD7 CAR-T Bridging to allo-HSCT in the treatment of severe aplastic anemia.The primary endpoints are dose limiting toxicity (DLT) and the incidence of treatment emergent adverse event (TEAE).

Вмешательства

• Препарат CD7 CAR-T cells injection
  CD7 CAR T cells treat patients with severe aplastic anemia
• Другое Allogeneic hematopoietic stem cell transplantation
  In this study, Allogeneic hematopoietic stem cell transplantation is used as a bridge therapy to CD7 CAR T cells infusion to treat patients with severe aplastic anemia

Первичные конечные точки

• Dose-limiting toxicity (DLT) [Срок оценки: Up to 28 days after Treatment]
• Incidence of treatment-emergent adverse events (TEAEs) [Срок оценки: Up to 2 years after Treatment]

Вторичные конечные точки (4)

• Allogeneic hematopoietic stem cell transplant 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

• Chinese expert consensus on the diagnosis and treatment of aplastic anemia（2017）, Diagnosis of severe aplastic anemia ，1. The degree of bone marrow cell proliferation < 25%, or 25%-50% but residual hematopoietic cells < 30%；2. With pancytopenia (at least two of the following peripheral blood parameters) : (1) absolute neutrophil <0.5×10\^9/L; (2) Platelet count< 20×10\^9/L; (3) The absolute value of reticulocytes <20×109/L;
• Suitable donors (relatives) with allogeneic HSCT indications and at least haploid allogeneic transplantation;
• Patients who are not suitable or unwilling to undergo traditional allogeneic hematopoietic stem cell transplantation;
• creatinine clearance > 60ml/min; without liver invasion, 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. If there is liver invasion, serum erythrambirubin ≤ 3 times the upper limit of normal, and serum ALT and AST are both ≤ 5 times the upper limit of the normal range;
• Echocardiogram shows left ventricular ejection fraction (LVEF) ≥ 50%;
• No active infection in the lungs, blood oxygen saturation in indoor air is ≥ 92%;
• Estimated survival time ≥ 3 months;
• ECOG performance status 0 to 1;
• Females and males of childbearing potential must agree to use adequate contraception prior to study entry, during study participation, and for 6 months after infusion (the safety of this therapy for unborn children is not known and has unknown risks);
• Those who voluntarily participated in this trial and provided informed consent;

Критерии исключения

• Allergy to pre-treatment measures；
• Those with acute graft versus host disease (GvHD) or moderate to severe chronic GvHD within 4 weeks before screening; Those who have received systemic drug therapy for GvHD within 4 weeks before the reinfusion；
• History of epilepsy or other central nervous system disorders；
• Electrocardiogram shows prolonged QT interval, severe heart diseases such as severe arrhythmia in the past;
• Less than 100 days after allogeneic hematopoietic stem cell transplantation；
• Patients with HIV infection，Active infection of hepatitis B virus or hepatitis C virus，Uncured active infection；
• The proiferation rate is less than 5 times response to CD3/CD28 co-stimulation signal;
• Received anti-cancer chemotherapy or other drug treatment within 2 weeks prior to screening;
• Any condition that, in the opinion of the investigator, may increase the risk to the subject or interfere with the results of the study.

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 Zhejiang University School of Medicine — Hanzhou

Идентификаторы

NCT: NCT06633328 · TXB2024009

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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