CD7 CAR-T Bridging to alloHSCT for Severe Aplastic Anemia

NCT06633328 | Recruiting Early Phase 1

• 1 other identifier: TXB2024009
• Study Type: interventional
• Target: 30
• Locations: 1 country
• Sites: 1

Brief Summary

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

Conditions

Aplastic Anemia

Keywords

allo-HSCT; CD7 CAR-T

Outcome Measures

Primary Outcomes (2)

• Dose-limiting toxicity (DLT)

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

  Up to 28 days after Treatment

• Incidence of treatment-emergent adverse events (TEAEs)

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

  Up to 2 years after Treatment

Secondary Outcomes (4)

• Allogeneic hematopoietic stem cell transplant implantation rate

  Up to 100 days after Treatment

• Time to neutrophil and platelet engraftment

  Up to 30 days after Treatment

• Disease-feesurvival，DFS

  Up to 2 years after Treatment

• Overall survival, OS

  Up to 2 years after Treatment

Study Arms (1)

Treatment Group

EXPERIMENTAL

Aplastic Anemia

Drug: CD7 CAR-T cells injection; Other: Allogeneic hematopoietic stem cell transplantation

Interventions

CD7 CAR-T cells injection DRUG

CD7 CAR T cells treat patients with severe aplastic anemia

Treatment Group

Allogeneic hematopoietic stem cell transplantation OTHER

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 Group

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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;

You may not qualify if:

• 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.

Sponsors & Collaborators

• Zhejiang University: lead
• Yake Biotechnology Ltd.: collaborator

Study Sites (1)

The First Affiliated Hospital of Zhejiang University School of Medicine

Hanzhou, Zhejiang, 310003, China

RECRUITING

MeSH Terms

Conditions

Anemia, Aplastic

Condition Hierarchy (Ancestors)

Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Bone Marrow Failure Disorders; Bone Marrow Diseases

Study Officials

• He Huang, MD

  Zhejiang University

  PRINCIPAL INVESTIGATOR

Central Study Contacts

He Z Huang, MD

CONTACT

13605714822; hehuangyu@126.com

Yongxian Hu, MD

CONTACT

86-15957162012; huyongxian2000@aliyun.com

Study Design

• Study Type: interventional
• Phase: early phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Clinical Professor

Study Record Dates

• First Submitted: October 8, 2024
• First Posted: October 9, 2024
• Study Start: October 20, 2024
• Primary Completion (Estimated): October 20, 2027
• Study Completion (Estimated): October 20, 2027
• Last Updated: October 9, 2024

Record last verified: 2024-08

Locations

CN (1)