Anti-FLT3 CAR T-cell Therapy in FLT3 Positive Relapsed/Refractory Acute Myeloid Leukemia

Pilot Study of the Safety and Efficacy of Anti-FLT3 Chimeric Antigen Receptor Engineered T-Cells in the Treatment of Relapsed or Refractory Acute Myeloid Leukemia (AML)

This is a prospective,open-label, phase1/2 study to evaluate the safety and efficacy of anti-FLT3 chimeric antigen receptor engineered T cell immunotherapy (CART) in the treatment of FLT3 positive relapsed or refractory acute myeloid leukemia.

Study Overview

• Status: Recruiting
• Conditions: Acute Myeloid Leukemia
• Intervention / Treatment: Biological: anti-FLT3 CAR-T

Detailed Description

The patients will receive infusion of anti-FLT3 CAR T-cells targeting FLT3 to evaluate the safety and efficacy of anti-FLT3 CAR T-Cells in relapsed or refractory acute myeloid leukemia.

• Study Type: Interventional
• Enrollment (Anticipated): 5
• Phase: Phase 2; Phase 1

Contacts and Locations

• Study Contact: Name: Lin Yang, Ph.D; Phone Number: (0086)18896802149; Email: huimin.meng@persongen.com.cn

Study Locations

• China
  • Jiangsu
    • Suzhou, Jiangsu, China, 215006
      • Recruiting
      • the First Affiliated Hospital of Soochow University
      • Contact: Xiaowen Tang, Ph.D; Phone Number: 86-512677801856; Email: xwtang1020@163.com

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 16 years to 65 years (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• FLT3 positive relapsed/refractory acute myeloid leukemia
• Age 16-65 years.
• Left ventricular ejection fractions ≥ 0.5 by echocardiography
• Creatinine < 1.5x upper limit of normal.
• Aspartate aminotransferase/aspartate aminotransferase ≤ 2.5x upper limit of normal
• Total bilirubin ≤ 1.5x upper limit of normal
• Karnofsky performance status ≥ 60
• Expected survival time ≥ 3 months (according to investigator's judgement)

Exclusion Criteria:

• Patients are pregnant or lactating
• Uncontrolled active infection
• Grade 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 a history of seizure
• Patients with other contraindications considered unsuitable for participation in this study (according to investigator's judgement)

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: CAR-T infusion; FLT3 positive relapsed or refractory acute myeloid leukemia	Biological: anti-FLT3 CAR-T; Single intravenous infusion of anti-FLT3 CAR-T cells

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of Adverse Events	Adverse events are evaluated with CTCAE V5.0	12 months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Overall response rate (ORR)	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 (<1.0 x 109/L) or thrombocytopenia (<100 x 109/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%.	2 years
Event-free survival (EFS)	time from enrollment to the date of primary refractory disease, or relapse from CR or CRi, or death from any cause	2 years
Cumulative incidence of relapse(CIR)	time from the date of achievement of a remission until the date of relapse	2 years

Collaborators and Investigators

• Sponsor: The First Affiliated Hospital of Soochow University
• Collaborators: The First Affiliated Hospital with Nanjing Medical University; The Affiliated Nanjing Drum Tower Hospital of Nanjing University Medical School; The Second People's Hospital of Huai'an; PersonGen BioTherapeutics (Suzhou) Co., Ltd.

Study record dates

Study Major Dates

• Study Start (Anticipated): December 31, 2021
• Primary Completion (Anticipated): June 30, 2023
• Study Completion (Anticipated): June 30, 2025

Study Registration Dates

• First Submitted: August 10, 2021
• First Submitted That Met QC Criteria: August 23, 2021
• First Posted (Actual): August 26, 2021

Study Record Updates

• Last Update Posted (Actual): December 30, 2021
• Last Update Submitted That Met QC Criteria: December 29, 2021
• Last Verified: July 1, 2021

More Information

Terms related to this study

• Keywords: FLT3,CAR-T,AML
• Additional Relevant MeSH Terms: Neoplasms by Histologic Type; Neoplasms; Leukemia; Leukemia, Myeloid; Leukemia, Myeloid, Acute
• Other Study ID Numbers: 2021136

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No