A Pediatric and Young Adult Trial of Genetically Modified T Cells Directed Against CD22 for Relapsed/Refractory Leukemia or Lymphoma

Pediatric and Young Adult Leukemia Adoptive Therapy (PLAT)-07: A Phase 1/2 Study of CD22-Specific CAR T Cells for CD22+ Leukemia or Lymphoma

Patients with relapsed or refractory leukemia or lymphoma are often refractory to further chemotherapy. In this study, the investigators will attempt to use T cells obtained directly from the patient, which can be genetically engineered to express a chimeric antigen receptor (CAR). The CAR used in this study can recognize CD22, a protein expressed on the surface of leukemia and lymphoma cells. The phase 1 part of this study will determine the safety and appropriate dose level of these CAR T cells, and the phase 2 part of the study will determine how effective this CAR T cell therapy is. Both patients who have never had prior CAR T cell therapy and those who have had prior CAR T cell therapy may be eligible to participate in this study.

Study Overview

• Status: Recruiting
• Conditions: Lymphoma; Leukemia
• Intervention / Treatment: Biological: SCRI-CAR22v2

• Study Type: Interventional
• Enrollment (Estimated): 42
• Phase: Phase 2; Phase 1

Contacts and Locations

• Study Contact: Name: Corinne Summers, MD; Phone Number: 206-987-2106; Email: CBDCIntake@seattlechildrens.org

Study Locations

• United States
  • California
    • Los Angeles, California, United States, 90027
      • Recruiting
      • Children's Hospital Los Angeles
      • Contact: Lee Chen
      • Principal Investigator: Emily Hsieh, MD
  • District of Columbia
    • Washington, District of Columbia, United States, 20010
      • Withdrawn
      • Children's National Hospital
  • Indiana
    • Indianapolis, Indiana, United States, 46202
      • Recruiting
      • Riley Hospital for Children
      • Contact: Jodi Skiles, MD
      • Principal Investigator: Jodi Skiles, MD
  • Texas
    • Houston, Texas, United States, 77030
      • Recruiting
      • Texas Children's Hospital
      • Contact: Rayne Rouce, MD
      • Principal Investigator: Rayne Rouce, MD
  • Washington
    • Seattle, Washington, United States, 98105
      • Recruiting
      • Seattle Children's Hospital
      • Contact: Corinne Summers, MD; Phone Number: 206-987-2106; Email: CBDCIntake@seattlechildrens.org
      • Principal Investigator: Corinne Summers, MD

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: No older than 30 years (Child, Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• Male and female subjects aged ≤ 30 years. First 2 enrolled subjects: age ≥ 18 and ≤ 30 years
• Evidence of refractory or recurrent CD22+ leukemia or lymphoma
• Able to tolerate apheresis, or subject with sufficient existing apheresis product or T cells for manufacturing investigational product.
• Life expectancy ≥ 8 weeks
• Lansky or Karnofsky, as applicable, score ≥ 50
• Recovered from acute toxic effects of all prior chemotherapy, immunotherapy, and radiotherapy, if the subject does not have a previously obtained apheresis product that is acceptable and available for manufacturing of CAR T cells
• ≥ 7 days post last chemotherapy and biologic therapy, with the exception of intrathecal chemotherapy and maintenance chemotherapy
• ≥ 7 days post last corticosteroid therapy
• ≥ 3 days post Tyrosine Kinase Inhibitor (TKI) use
• ≥ 1 day post hydroxyurea
• 30 days post most recent CAR T cell infusion
• Adequate organ function
• Adequate laboratory values, including absolute lymphocyte count ≥ 100 cells/uL
• Subjects of childbearing or child-fathering potential must agree to use highly effective contraception from consent through 12 months following infusion of investigational product on trial
• Subject and/or legally authorized representative has signed the informed consent form for this study

Exclusion Criteria:

• Presence of active malignancy other than disease under study
• History of symptomatic CNS pathology or ongoing symptomatic CNS pathology
• CNS involvement of leukemia or lymphoma that is symptomatic and in the opinion of the investigator, cannot be controlled during the interval between enrollment and CAR T cell infusion
• Subjects with uniform expression of CD19 on their malignant cells who are eligible but have not attempted CD19 directed CAR T cell therapy
• For subjects having had a previous stem cell transplant: presence of active GVHD, or receiving immunosuppressive therapy for treatment or prevention of GVHD within 4 weeks prior to enrollment
• Presence of active severe infection,
• Presence of primary immunodeficiency syndrome
• Subject has received prior virotherapy
• Pregnant or breastfeeding
• Subject and/or legally authorized representative unwilling to provide consent/assent for participation in the 15-year follow-up period, required if CAR T cell therapy is administered
• Presence of any condition that, in the opinion of the investigator, would prohibit the subject from undergoing treatment under this protocol

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: SCRI-CAR22v2; Patients will receive SCRI-CAR22v2 in either Phase I or Phase II	Biological: SCRI-CAR22v2; Single infusion of SCRI-CAR22v2

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
he adverse events associated with CAR T cell product infusions will be assessed	The type, frequency, severity, and duration of adverse events will be summarized	28 days post-infusion
The ability to successfully manufacture SCRI-CAR22v2	We will measure the number of successfully manufactured SCRI-CAR22v2 products	28 days
The leukemia response to SCRI-CAR22v2 in subjects with relapsed or refractory CD22+ leukemia will be assessed	The efficacy of the T cell infusion will be estimated based on the number of participants who have an MRD negative bone marrow aspirate following the T cell infusion	28 days post-infusion

Collaborators and Investigators

• Sponsor: Seattle Children's Hospital
• Investigators: Study Chair: Corinne Summers, MD, Seattle Children's Hospital

Study record dates

Study Major Dates

• Study Start (Actual): September 25, 2020
• Primary Completion (Estimated): June 1, 2025
• Study Completion (Estimated): February 1, 2040

Study Registration Dates

• First Submitted: September 2, 2020
• First Submitted That Met QC Criteria: September 25, 2020
• First Posted (Actual): September 30, 2020

Study Record Updates

• Last Update Posted (Actual): April 9, 2024
• Last Update Submitted That Met QC Criteria: April 6, 2024
• Last Verified: April 1, 2024

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Immune System Diseases; Neoplasms by Histologic Type; Neoplasms; Lymphoproliferative Disorders; Lymphatic Diseases; Immunoproliferative Disorders; Hematologic Diseases; Lymphoma; Leukemia
• Other Study ID Numbers: PLAT-07

Drug and device information, study documents

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