RECRUITING

CD123 Redirected T Cells for AML in Pediatric Subjects

Conditions

Acute Myeloid Leukemia, in Relapse Acute Myeloid Leukemia, Pediatric Acute Myeloid Leukemia, Refractory refractory relapsed Acute Myeloid leukemia AML

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

Phase 1 open-label study to evaluate the safety of intravenously administered, lentivirally transduced T cells expressing anti-CD123 chimeric antigen receptors expressing tandem TCRζ and 4-1BB (TCRζ /4-1BB) costimulatory domains in pediatric subjects with relapsed/refractory Acute Myeloid Leukemia (AML).

Official Title

Phase 1 Study of Lentivirally Transduced T Cells Engineered to Contain Anti-CD123 Linked to TCRζ and 4-1BB Signaling Domains in Pediatric Subjects With Refractory or Relapsed Acute Myeloid Leukemia

Quick Facts

Study Start:2021-02-15

Study Completion:2036-01

Study Type:Not specified

Phase:Not Applicable

Enrollment:Not specified

Status:RECRUITING

Study ID

NCT04678336

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:1 Year to 29 Years

Sexes Eligible for Study:ALL

Accepts Healthy Volunteers:No

Standard Ages:CHILD, ADULT

Inclusion Criteria	Exclusion Criteria
1. Male and female patients ≥ 1 and ≤ 29 years of age at time of consent. 2. AML in second or greater relapse, post-transplant relapse, or chemotherapy-refractory disease. Specifically: 1. Second or greater relapse defined as flow cytometric confirmation of myeloid leukemia of at least 0.1% after second documented complete remission; OR 2. Any detectable disease post-allogeneic transplant with flow cytometric confirmation (MRD) of myeloid leukemia of at least 0.1% (as confirmed by Hematologics); OR 3. Refractory disease, defined as persistent bone marrow involvement with \>5% blasts after two courses of induction chemotherapy for patients at initial presentation or \>5% bone marrow blasts after one course of re-induction chemotherapy for patients who have relapsed after previously achieving a CR. 3. Subjects must have a suitable stem cell donor identified with projected ability to proceed to transplant within 6-8 weeks of CART123 infusion. 4. Adequate organ function defined as: 1. A serum creatinine based on age/gender 2. Adequate liver function 5. Adequate performance status defined as Lansky or Karnofsky score ≥ 50 6. Signed informed consent must be obtained. 7. No contraindications for leukapheresis (unless apheresis product previously acquired). 8. Subjects of reproductive potential must agree to use acceptable birth control methods.	1. Pregnant or lactating (nursing) women. 2. Patients with relapsed AML with t(15:17). 3. Patients \< 6 months from alloHSCT. 4. HIV infection. 5. Active hepatitis B or hepatitis C infection. 6. Active acute or chronic graft-versus-host disease (GVHD) requiring systemic therapy 7. Patients requiring chronic treatment with systemic steroids or immunosuppressant medications. Low-dose physiologic replacement therapy with corticosteroids, topical steroids and inhaled steroids are acceptable. For additional details regarding use of steroids and immunosuppressant medications (including washout requirements prior to CAR T cell administration). 8. Any uncontrolled active medical disorder that would preclude participation as outlined. 9. Uncontrolled active infection 10. Subjects with CNS3 disease that is progressive on therapy or with CNS parenchymal lesions that may increase the risk of CNS toxicity. Subjects with adequately treated CNS leukemia are eligible. 11. Known history of allergy or hypersensitivity to study product excipients (human serum albumin, DMSO, and Dextran 40). 12. Patients with any prior history of myeloproliferative neoplasm. 13. Patients with somatic JAK2 V617F mutation by PCR or next generation sequencing.

Inclusion Criteria

Exclusion Criteria

1. Male and female patients ≥ 1 and ≤ 29 years of age at time of consent.
2. AML in second or greater relapse, post-transplant relapse, or chemotherapy-refractory disease. Specifically:
1. Second or greater relapse defined as flow cytometric confirmation of myeloid leukemia of at least 0.1% after second documented complete remission; OR
2. Any detectable disease post-allogeneic transplant with flow cytometric confirmation (MRD) of myeloid leukemia of at least 0.1% (as confirmed by Hematologics); OR
3. Refractory disease, defined as persistent bone marrow involvement with \>5% blasts after two courses of induction chemotherapy for patients at initial presentation or \>5% bone marrow blasts after one course of re-induction chemotherapy for patients who have relapsed after previously achieving a CR.
3. Subjects must have a suitable stem cell donor identified with projected ability to proceed to transplant within 6-8 weeks of CART123 infusion.
4. Adequate organ function defined as:
1. A serum creatinine based on age/gender
2. Adequate liver function
5. Adequate performance status defined as Lansky or Karnofsky score ≥ 50
6. Signed informed consent must be obtained.
7. No contraindications for leukapheresis (unless apheresis product previously acquired).
8. Subjects of reproductive potential must agree to use acceptable birth control methods.

1. Pregnant or lactating (nursing) women.
2. Patients with relapsed AML with t(15:17).
3. Patients \< 6 months from alloHSCT.
4. HIV infection.
5. Active hepatitis B or hepatitis C infection.
6. Active acute or chronic graft-versus-host disease (GVHD) requiring systemic therapy
7. Patients requiring chronic treatment with systemic steroids or immunosuppressant medications. Low-dose physiologic replacement therapy with corticosteroids, topical steroids and inhaled steroids are acceptable. For additional details regarding use of steroids and immunosuppressant medications (including washout requirements prior to CAR T cell administration).
8. Any uncontrolled active medical disorder that would preclude participation as outlined.
9. Uncontrolled active infection
10. Subjects with CNS3 disease that is progressive on therapy or with CNS parenchymal lesions that may increase the risk of CNS toxicity. Subjects with adequately treated CNS leukemia are eligible.
11. Known history of allergy or hypersensitivity to study product excipients (human serum albumin, DMSO, and Dextran 40).
12. Patients with any prior history of myeloproliferative neoplasm.
13. Patients with somatic JAK2 V617F mutation by PCR or next generation sequencing.

Contacts and Locations

Study Contact

Michelle Ashford

CONTACT

267-426-0762

oncointake@email.chop.edu

Claire White

CONTACT

whiteC3@email.chop.edu

Study Locations (Sites)

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104

United States

Collaborators and Investigators

Sponsor: University of Pennsylvania

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2021-02-15

Study Completion Date2036-01

Study Record Updates

Study Start Date2021-02-15

Study Completion Date2036-01

Terms related to this study

Keywords Provided by Researchers

refractory
relapsed
Acute
Myeloid
leukemia
AML

Additional Relevant MeSH Terms

Acute Myeloid Leukemia, in Relapse
Acute Myeloid Leukemia, Pediatric
Acute Myeloid Leukemia, Refractory