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Universal CAR-T Cells Targeting AML

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ClinicalTrials.gov Identifier: NCT05995041
Recruitment Status : Recruiting
First Posted : August 16, 2023
Last Update Posted : October 12, 2023
Sponsor:
Information provided by (Responsible Party):
Shenzhen Geno-Immune Medical Institute

Tracking Information
First Submitted Date  ICMJE August 8, 2023
First Posted Date  ICMJE August 16, 2023
Last Update Posted Date October 12, 2023
Estimated Study Start Date  ICMJE October 31, 2023
Estimated Primary Completion Date September 30, 2026   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: August 8, 2023)		 Safety of infusion [ Time Frame: 6 months ]
Treatment-related adverse events are assessed by NCI CTCAE V4.0 criteria.
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: August 8, 2023)		 Clinical response [ Time Frame: 1 year ]
Objective responses (complete response (CR) + partial response (PR)) are assessed by the Response Evaluation Criteria in Solid Tumors (RECIST) v1.1 criteria.
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Universal CAR-T Cells Targeting AML
Official Title  ICMJE Universal CAR T Cells for the Treatment of Acute Myeloid Leukemia
Brief Summary The purpose of this clinical trial is to assess the feasibility, safety and efficacy of universal CAR T-cell products targeting CLL-1, CD33, CD38 and CD123 in patients with relapsed and refractory AML. The study also aims to learn more about the function of the universal CAR T cells and their persistency in AML patients.
Detailed Description

Acute myeloid leukemia (AML) is a malignant disease characterized by the rapid growth of myeloblasts that grow in the bone marrow and interferes with the generation of normal blood cells.Over the past years, several groups have demonstrated that CLL1, CD33, CD38 and CD123 are potential AML targets. CLL-1 (C-type lectin-like molecule-1) is a transmembrane glycoprotein, which is overexpressed in leukemic stem cells but absent in normal hematopoietic stem cells, suggesting that CLL-1 can be a promising target for targeted AML therapy. Although CAR-T cells have shown impressive anti-leukemic effect in B cell disease, CAR-T treatment for AML has proven to be more difficult. One of the reasons is because AML patients often has highly suppressed bone marrow function, and it is often difficult to obtain good quality of T cells for CAR-T preparation. In addition, AML progression can be acute and rapid, which can outpace the CAR-T expansion, and the time-consuming CAR-T manufacture process makes it more difficult to treat AML with autologous source of T cells By using universal type of CAR-T cells, the product can be supplied off-the-shelf without being customized from individual patients. In addition, the immediate availability means that patients with short disease remission time under severe bone marrow suppression may get a chance to be treated with CAR-T cells to achieve disease remission. In addition, those patients who suffer from long-term immunosuppression due to tumor microenvironment or myelosuppressive chemotherapy would have the option of treatment with the universal CAR-T cells.

The purpose of this study is to assess the feasibility, safety and efficacy of several AML-specific universal CAR-T products. Another goal is to learn more about the function of the universal CAR T cells and their persistency in the patients.
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Acute Myeloid Leukemia
Intervention  ICMJE Biological: CLL-1, CD33, CD38 and/or CD123-specific universal CAR- T cells
Infusion of CLL-1, CD33, CD38 and/or CD123-specific universal CAR- T cells
Study Arms  ICMJE Experimental: Multiple universal CAR T cells to treat AML
Intervention: Biological: CLL-1, CD33, CD38 and/or CD123-specific universal CAR- T cells
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: August 8, 2023)		 30
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE December 31, 2026
Estimated Primary Completion Date September 30, 2026   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  1. Age older than 6 months.
  2. Confirmed expression of CLL-1, CD123, CD38 and/or CD33 in AML blasts by immuno-histochemical staining or flow cytometry.
  3. Karnofsky performance status (KPS) score is higher than 80 and life expectancy > 3 months.
  4. Adequate bone marrow, liver and renal function as assessed by the following laboratory requirements: cardiac ejection fraction ≥ 50%, oxygen saturation ≥ 90%, creatinine ≤ 2.5 × upper limit of normal, aspartate aminotransferase (AST) and alanine aminotransferase (ALT) ≤ 3 × upper limit of normal, total bilirubin ≤ 2.0mg/dL.
  5. Hgb≥80g/L.
  6. No cell separation contraindications.
  7. Abilities to understand and the willingness to provide written informed consent.

Exclusion Criteria:

  1. Sever illness or medical condition, which would not permit the patient to be managed according to the protocol, including active uncontrolled infection.
  2. Active bacterial, fungal or viral infection not controlled by adequate treatment.
  3. Known HIV or active hepatitis C virus (HCV) infection.
  4. Pregnant or nursing women may not participate.
  5. Use of glucocorticoid for systemic therapy within one week prior to entering the trial.
  6. Previous treatment with any gene therapy products.
  7. The bone marrow AML burden (MRD) is above 50%.
  8. Patients, in the opinion of investigators, may not be able to comply with the study.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 6 Months to 75 Years   (Child, Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Lung-Ji Chang, Ph.D 86-0755-8672 5195 c@szgimi.org
Contact: Ying Deng 86-0755-8672 5195 ying.deng@szgimi.org
Listed Location Countries  ICMJE China
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT05995041
Other Study ID Numbers  ICMJE GIMI-IRB-23004
Has Data Monitoring Committee No
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Current Responsible Party Shenzhen Geno-Immune Medical Institute
Original Responsible Party Same as current
Current Study Sponsor  ICMJE Shenzhen Geno-Immune Medical Institute
Original Study Sponsor  ICMJE Same as current
Collaborators  ICMJE Not Provided
Investigators  ICMJE Not Provided
PRS Account Shenzhen Geno-Immune Medical Institute
Verification Date October 2023

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP