Anti-CLL1 CAR T-cell Therapy in CLL1 Positive Relapsed/Refractory Acute Myeloid Leukemia (AML)

Sponsor

The First Affiliated Hospital of Soochow University (Other)

Overall Status

Recruiting

CT.gov ID

NCT04884984

Collaborator

(none)

Enrollment

Location

Arm

Anticipated Duration (Months)

0.2

Patients Per Site Per Month

Study Details

Study Description

Brief Summary

This is a single center, open-label phase 1/2 study to evaluate the safety and efficacy of anti-CLL1 chimeric antigen receptor engineered T cell immunotherapy (CART) in the treatment of CLL1 positive relapsed or refractory acute myeloid leukemia.

Condition or Disease	Intervention/Treatment	Phase
Acute Myeloid Leukemia	Biological: anti-CLL1 CART	Phase 1/Phase 2

Detailed Description

The patients will receive infusion of anti-CLL1 CAR T-cells to confirm the safety and efficacy of anti-CLL1 CAR T-cells in relapsed or refractory acute myeloid leukemia.

Study Design

Study Type:

Interventional

Anticipated Enrollment :

20 participants

Allocation:

N/A

Intervention Model:

Single Group Assignment

Masking:

None (Open Label)

Primary Purpose:

Treatment

Official Title:

Pilot Study of the Safety and Efficacy of Anti-CLL1 Chimeric Antigen Receptor Engineered T-Cells in the Treatment of CLL1 Positive Relapsed or Refractory Acute Myeloid Leukemia (AML)

Actual Study Start Date :

Jul 1, 2017

Anticipated Primary Completion Date :

Dec 31, 2022

Anticipated Study Completion Date :

Dec 31, 2024

Arms and Interventions

Arm	Intervention/Treatment
Experimental: CLL1 positive relapsed or refractory acute myeloid leukemia	Biological: anti-CLL1 CART Split intravenous infusion of anti-CLL1 CAR T cells [dose escalating infusion of (5-20)x10^6 anti-CLL1 CAR T cells/kg].

Arm

Intervention/Treatment

Experimental: CLL1 positive relapsed or refractory acute myeloid leukemia

Biological: anti-CLL1 CART

Split intravenous infusion of anti-CLL1 CAR T cells [dose escalating infusion of (5-20)x10^6 anti-CLL1 CAR T cells/kg].

Outcome Measures

Primary Outcome Measures

Number of Adverse Events [12 months]
Adverse events are evaluated with CTCAE V5.0

Secondary Outcome Measures

Overall response rate (ORR) [2 years]
ORR includes CR, CRi, MLFS and PR. Complete remission (CR)#Bone marrow blasts <5%; absence of circulating blasts and blasts with Auer rods; absence of extramedullary disease; absolute neutrophil count >1.0x 10^9/L; platelet count >100x10^9/L. CR with incomplete hematologic recovery (CRi)#All CR criteria except for residual neutropenia (<1.0x10^9/L) or thrombocytopenia (<100x10^9/L). Morphologic leukemia-free state (MLFS): Bone marrow blasts <5%; absence of blasts with Auer rods; absence of extramedullary disease; no hematologic recovery required. Partial remission (PR): All hematologic criteria of CR; decrease of bone marrow blast percentage to 5% to 25%; and decrease of pretreatment bone marrow blast percentage by at least 50%.
overall survival (OS) [2 years]
The date of enrollment to the date of death from any cause
Event-free survival (EFS) [2 years]
Time from enrollment to the date of primary refractory disease, or relapse from CR or CRi, or death from any cause
Cumulative incidence of relapse(CIR) [2 years]
Time from the date of achievement of a remission to the date of relapse

Eligibility Criteria

Criteria

Ages Eligible for Study:

6 Years to 65 Years

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

Inclusion Criteria:

CLL1 positive relapsed/refractory acute myeloid leukemia
Age 6-65 years
Left ventricular ejection fractions ≥ 0.5 by echocardiography
Creatinine < 1.6 mg/dL
Aspartate aminotransferase/aspartate aminotransferase < 3x upper limit of normal
Total bilirubin <2.0 mg/dL
Karnofsky performance status ≥ 60
Expected survival time ≥ 3 months (according to investigator's judgement)

Exclusion Criteria:

Patients are pregnant or lactating
Uncontrolled active infection
Class III/IV cardiovascular disability according to the New York Heart Association Classification
Active hepatitis B or hepatitis C infection
Patients with HIV infection
Patients with history of seizure
Active central nervous system leukemia