Actively Recruiting

Phase 2
Age: 0Years - 29Years
All Genders
NCT04276870

Orphan Indications for CD19 Redirected Autologous T Cells

Led by Stephan Grupp MD PhD · Updated on 2026-03-06

133

Participants Needed

1

Research Sites

886 weeks

Total Duration

On this page

Sponsors

S

Stephan Grupp MD PhD

Lead Sponsor

U

University of Pennsylvania

Collaborating Sponsor

AI-Summary

What this Trial Is About

This is an open-label, four-cohort, phase 2 study to determine the efficacy of CART19 in pediatric and young adult patientswith hypodiploid (Cohort A) or t(17;19) B-ALL (Cohort B), infants with very high risk KMT2A B-ALL (Cohort C), and in patients with central nervous system (CNS) relapse who did not receive cranial radiation (XRT) or bone marrow transplantation (BMT) (Cohort D).

CONDITIONS

Official Title

Orphan Indications for CD19 Redirected Autologous T Cells

Who Can Participate

Age: 0Years - 29Years
All Genders

Eligibility Criteria

Eligible

You may qualify if you...

  • Signed informed consent form must be obtained prior to any study procedure.
  • Male and female patients with documented CD19-positive B-cell acute lymphoblastic leukemia (B-ALL).
  • Cohort A: Patients with confirmed hypodiploid karyotype (fewer than 45 chromosomes).
  • Cohort B: Patients with cytogenetic confirmation of chromosomal translocation t(17;19).
  • Cohort C: Infants with newly diagnosed KMT2A rearranged B-ALL classified as very high risk by age and other clinical criteria.
  • Cohort D: Patients in first or greater central nervous system relapse prior to cranial radiation or hematopoietic stem cell transplantation.
  • Documentation of CD19 tumor expression in bone marrow, peripheral blood, cerebrospinal fluid, or tumor tissue.
  • Age from birth up to 29 years.
  • Adequate organ function including specific limits for serum creatinine based on age and gender.
  • Adequate liver function with limits on ALT and total bilirubin, or abnormalities attributed to leukemia infiltration.
  • Pulmonary reserve defined by specific dyspnea and hypoxia grades and lung diffusion capacity if appropriate.
  • Heart function with left ventricular shortening fraction ≥28% or ejection fraction ≥45%.
  • Performance status with Lansky or Karnofsky score ≥50.
  • Agreement to use acceptable birth control methods if of reproductive potential.
Not Eligible

You will not qualify if you...

  • For central nervous system relapse patients, prior cranial radiation or bone marrow transplantation for the current relapse.
  • Active hepatitis B or hepatitis C infection.
  • HIV infection.
  • Active acute or chronic graft-versus-host disease requiring systemic therapy.
  • Concurrent use of systemic steroids at time of cell infusion or collection, or conditions likely requiring steroid therapy during these times.
  • Progressive CNS3 disease or CNS parenchymal lesions increasing risk of CNS toxicity.
  • Pregnant or nursing (lactating) women.
  • Uncontrolled active infection.

AI-Screening

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Trial Site Locations

Total: 1 location

1

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, United States, 19104

Actively Recruiting

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Research Team

C

CART Nurse Navigator

CONTACT

M

Melissa S Varghese, M.S.

CONTACT

How is the study designed?

Study Type

INTERVENTIONAL

Masking

NONE

Allocation

RANDOMIZED

Model

PARALLEL

Primary Purpose

TREATMENT

Number of Arms

4

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