CD38-targeted Chimeric Antigen Receptor T Cell (CART) in Relapesd or Refractory Acute Myeloid Leukemia

Pilot Study of the Efficacy and Safety of CD38 Targeted Chimeric Antigen Receptor Engineered T-Cells in the Treatment of CD38 Positive Relapsed or Refractory Acute Myeloid Leukemia (AML)

This is a single center, open-label phase 1/2 study to evaluate the safety and efficacy of targeted CD38 chimeric antigen receptor engineered T cell immunotherapy (CART) in the treatment of CD38 positive relapsed or refractory acute myeloid leukemia.

Study Overview

• Status: Recruiting
• Conditions: Acute Myeloid Leukemia
• Intervention / Treatment: Biological: CART-38

Detailed Description

The patients will receive infusion of CAR T-cells targeting CD38 to confirm the safety and efficacy of CD38 CAR T-Cells in relapsed or refractory acute myeloid leukemia.

• Study Type: Interventional
• Enrollment (Anticipated): 20
• Phase: Phase 2; Phase 1

Contacts and Locations

• Study Contact: Name: Lei Yu, Ph.D; Phone Number: (0086)13818629089; Email: ylyh188@163.com

Study Locations

• China
  • Jiangsu
    • Suzhou, Jiangsu, China
      • Recruiting
      • The First Affiliated Hospital of Soochow University
      • Contact: Xiaowen Tang, Ph.D; Phone Number: (0086) 51267781856; Email: xwtang1020@163.com

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 6 years to 65 years (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• CD38 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

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: CD38 positive relapsed or refractory acute myeloid leukemia	Biological: CART-38; Split intravenous infusion of CART-38 cells [dose escalating infusion of (5-20)x10^6 CART-38 cells/kg].

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
Cumulative incidence of relapse(CIR)	time from the date of achievement of a remission until the date of relapse.	2 years
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 (<1.0x10^9/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%.	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

Collaborators and Investigators

• Sponsor: The First Affiliated Hospital of Soochow University

Publications and helpful links

General Publications

• Cui Q, Qian C, Xu N, Kang L, Dai H, Cui W, Song B, Yin J, Li Z, Zhu X, Qu C, Liu T, Shen W, Zhu M, Yu L, Wu D, Tang X. CD38-directed CAR-T cell therapy: a novel immunotherapy strategy for relapsed acute myeloid leukemia after allogeneic hematopoietic stem cell transplantation. J Hematol Oncol. 2021 May 25;14(1):82. doi: 10.1186/s13045-021-01092-4.

Study record dates

Study Major Dates

• Study Start (Actual): July 1, 2017
• Primary Completion (Anticipated): December 31, 2021
• Study Completion (Anticipated): December 31, 2023

Study Registration Dates

• First Submitted: April 15, 2020
• First Submitted That Met QC Criteria: April 15, 2020
• First Posted (Actual): April 17, 2020

Study Record Updates

• Last Update Posted (Actual): April 24, 2020
• Last Update Submitted That Met QC Criteria: April 21, 2020
• Last Verified: April 1, 2020

More Information

Terms related to this study

• Keywords: CD38, CART, AML
• Additional Relevant MeSH Terms: Neoplasms by Histologic Type; Neoplasms; Leukemia; Leukemia, Myeloid; Leukemia, Myeloid, Acute
• Other Study ID Numbers: UnicarTherapy2020001

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

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