CAR T cell therapy for children with acute lymphoblastic leukemia

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This phase I study aims to evaluate the safety and determine the recommended phase 2 dose of autologous CD19-CD22 bispecific CAR T cells in pediatric patients with recurrent or refractory acute lymphoblastic leukemia. Participants aged 21 years or younger will receive a single course of lymphodepleting chemotherapy followed by the infusion of CAR T cells. The study will also assess the anti-leukemic activity of the treatment. The dose of CAR T cells will be adjusted based on a predefined escalation scheme considering the number of CAR+ T cells and the participant's weight.

Why it matters

Who can join: the full rules

Collection and Manufacturing Eligibility

Inclusion Criteria:

* Age \<21 years old
* Relapsed/refractory CD19- and/or CD22-positive acute leukemia defined as:

  \*CD19 and/or CD22-positivity confirmed within 2 months and after receipt of any CD19 or CD22-directed therapy
  * Second or greater relapse
  * Any relapse after allogeneic HCT
  * Refractory disease (primary or in relapse) despite therapy designed to induce remission
* Estimated life expectancy of \> 12 weeks
* Karnofsky or Lansky (age-dependent) performance score ≥50 (Appendix A)
* For females of childbearing age:

  * Not lactating with intent to breastfeed
  * Not pregnant with negative serum or urine pregnancy test within 7 days prior to enrollment

Exclusion Criteria:

* Known primary immunodeficiency
* Known HIV positivity
* Known contraindication to receiving protocol defined lymphodepleting
* chemotherapy regimen
* History of hypersensitivity reaction to murine protein-containing products

Treatment Eligibility

Inclusion Criteria:

* Age \< 21 years old
* Detectable disease in the bone marrow
* Estimated life expectancy of \> 8 weeks
* Karnofsky or Lansky (age-dependent) performance score \> 50 (Appendix A)
* Adequate cardiac function defined as left ventricular ejection fraction \>40%, or shortening fraction \> 25%
* EKG without evidence of clinically significant arrhythmia
* Adequate renal function defined as creatinine clearance or radioisotope GFR \>50 mL/min/1.73m2 (GFR \>40 mL/min/1.73m2 if \<2 years of age)
* Adequate pulmonary function defined as forced vital capacity (FVC) \>50% of predicted value; or pulse oximetry \>92% on room air
* Total bilirubin \< 3 times the upper limit of normal for age, except in subjects with Gilbert's syndrome
* Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) \< 5 times the upper limit of normal for age
* Has recovered from all NCI CTAE grade III-IV, non-hematologic acute toxicities from prior therapy
* Prior to planned CAR T cell infusion, patients with a history of prior allogeneicHCT must be at least 3 months from HCT, have no evidence of acute GVHD, and have not received a donor lymphocyte infusion (DLI) within the 28 daysprior to planned infusion
* For females of childbearing age:

  * Not lactating with intent to breastfeed
  * Not pregnant with negative serum or urine pregnancy test within 7 days prior to enrollment
  * If sexually active, agreement to use birth control until 3 months after T cell infusion. Male partners should use a condom.

Exclusion Criteria:

* Known primary immunodeficiency
* Known HIV positivity
* Known contraindication to receiving protocol defined lymphodepleting
* chemotherapy regimen
* History of hypersensitivity reactions to murine protein-containing products
* Severe, uncontrolled bacterial, viral or fungal infection
* Active CNS-3 disease
* Evidence of active, uncontrolled neurologic disease

Where it's running

• St. Jude Children's Research Hospital
  Memphis, Tennessee, United States Recruiting

Study contacts

• Principal investigator: Rebecca Epperly, MD — St. Jude Children's Research Hospital
• Study coordinator: Rebecca Epperly, MD
• Email: referralinfo@stjude.org
• Phone: 888-226-4343

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.