CD7 CAR-T therapy for severe aplastic anemia before stem cell transplant

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

This study is testing a new CAR-T cell therapy for people with severe aplastic anemia to see if it can help them before they receive a stem cell transplant.

Quick facts

• Phase: EARLY_PHASE1
• Study type: Interventional
• Enrollment: 30 (estimated)
• Sex: All
• Sponsor: Zhejiang University (other)
• Drugs / interventions: chemotherapy, CAR-T
• Locations: 1 site (Hanzhou, Zhejiang)
• Trial ID: NCT06633328 on ClinicalTrials.gov

What this trial studies

This clinical trial is a single-arm, open-label, phase I study aimed at evaluating the safety and efficacy of CD7 CAR-T cell therapy as a bridging treatment to allogeneic hematopoietic stem cell transplantation (alloHSCT) for patients with severe aplastic anemia. The study will assess dose limiting toxicity (DLT) and the incidence of treatment emergent adverse events (TEAE) in participants. It is conducted at a single center, focusing on a specific patient population with severe aplastic anemia who are not suitable for traditional transplantation methods.

Who should consider this trial

Good fit: Ideal candidates for this study are patients diagnosed with severe aplastic anemia who have limited bone marrow cell proliferation and are not suitable for traditional allogeneic hematopoietic stem cell transplantation.

Not a fit: Patients with mild aplastic anemia or those who are suitable and willing to undergo traditional allogeneic hematopoietic stem cell transplantation may not benefit from this study.

Why it matters

Potential benefit: If successful, this approach could provide a new treatment option for patients with severe aplastic anemia, potentially improving their outcomes and quality of life.

How similar studies have performed: While this approach is novel, similar CAR-T therapies have shown promise in other hematological conditions, suggesting potential for success in this context.

Eligibility criteria

Inclusion Criteria:

* 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;

Exclusion Criteria:

* 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.

Where this trial is running

Hanzhou, Zhejiang

• The First Affiliated Hospital of Zhejiang University School of Medicine — Hanzhou, Zhejiang, China (RECRUITING)

Study contacts

• Principal investigator: He Huang, MD — Zhejiang University
• Study coordinator: He Z Huang, MD

Conditions: Aplastic Anemia, allo-HSCT, CD7 CAR-T