CD7 CAR-T Bridging to alloHSCT for Severe Aplastic Anemia

Trial statusRecruiting
Trial phaseEarly Phase 1
Trial typeInterventional
Biological sexAll
AgeNot listed
SponsorZhejiang University

About this trial

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

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

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;

Disqualifiers

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;

Trial design

Design model

Single group

Treatments tested in this trial

  • CD7 CAR-T cells injection

    Drug

    CD7 CAR T cells treat patients with severe aplastic anemia

  • Allogeneic hematopoietic stem cell transplantation

    Other intervention

    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

Treatment groups

30 Participants
are divided into 1 treatment group
Group A: Treatment GroupExperimental treatment 2 interventions

Trial outcomes

Primary outcomes

1

Dose-limiting toxicity (DLT)

Adverse events assessed according to NCI-CTCAE v5.0 criteria

Time frame
Up to 28 days after Treatment
2

Incidence of treatment-emergent adverse events (TEAEs)

Incidence of treatment-emergent adverse events \[Safety and Tolerability\]

Time frame
Up to 2 years after Treatment

Secondary outcomes

1

Allogeneic hematopoietic stem cell transplant implantation rate

The proportion of the number of patients who achieved hematopoietic reconstitution to the total number of allogeneic hematopoietic stem cell transplantation patients in the same period.

Time frame
Up to 100 days after Treatment
2

Time to neutrophil and platelet engraftment

The time for neutrophils and platelets to reach the implantation criteria after stem cell reinfusion

Time frame
Up to 30 days after Treatment
3

Disease-feesurvival,DFS

The proportion of disease-free patients who survived to the total number of patients who transplanted allogeneic hematopoietic stem cells during the same period.

Time frame
Up to 2 years after Treatment
4

Overall survival, OS

After transplantation until death from any cause.

Time frame
Up to 2 years after Treatment

Other outcomes

Locations

1

Map coordinates are unavailable for these locations. Locations are shown below instead.

The First Affiliated Hospital of Zhejiang University School of MedicineRecruiting310003, Hanzhou, ZhejiangChinaChina

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Zhejiang University

Lead sponsor

Yake Biotechnology Ltd.

Collaborator