NCT02650414

Brief Summary

This is a pilot study to determine the feasibility and safety of a single dose of autologous T cells expressing CD22 chimeric antigen receptors expressing tandem TCR-ζ and 4-1BB signaling domains (CART22/CART22-65s cells) in pediatric and young adult subjects with relapsed or refractory B cell acute lymphoblastic leukemia.

Trial Health

75
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
41

participants targeted

Target at P50-P75 for phase_1

Timeline
140mo left

Started Jan 2016

Longer than P75 for phase_1

Geographic Reach
1 country

1 active site

Status
active not recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress47%
Jan 2016Dec 2037

First Submitted

Initial submission to the registry

December 22, 2015

Completed
17 days until next milestone

First Posted

Study publicly available on registry

January 8, 2016

Completed
5 days until next milestone

Study Start

First participant enrolled

January 13, 2016

Completed
21.9 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

December 1, 2037

Expected
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

December 1, 2037

Last Updated

December 12, 2025

Status Verified

December 1, 2025

Enrollment Period

21.9 years

First QC Date

December 22, 2015

Last Update Submit

December 5, 2025

Conditions

B Cell LeukemiasB Cell Lymphomas

Keywords

Biological: CART 22

Outcome Measures

Primary Outcomes (1)

  • Frequency and severity of adverse events, including, but not limited to, cytokine release syndrome (CRS)

    grade 3 and higher toxicity rate (toxicity possibly attributed to CART22)

    From date of dosing ( day 1 ) up 15 years

Secondary Outcomes (14)

  • Percentage of manufacturing products that do not meet release criteria.

    3 months

  • Overall Complete Remission Rate (ORR) at Day 28.

    4 months

  • Evaluate overall response rate (CR/CRi by or at Month 6) at Month 6.

    9 months

  • Evaluate disease status at Month 6.

    9 months

  • Overall survival (OS)

    15 years

  • +9 more secondary outcomes

Study Arms (1)

Pediatric patients with relapsed or refractory B-cell acute lymphoblastic leukemia

EXPERIMENTAL
Biological: Cohort 1Biological: Cohorts 2Biological: Cohort 3

Interventions

Cohorts 2BIOLOGICAL

Subjects \<50kg will receive 0.2-1 x 10\^7 CART22-65s cells/kg as a split dose over three days as follows: Day 1, 10% fraction: 0.2-1x10\^6 CART22-65s cells/kg Day 2, 30% fraction: 0.6-3x10\^6 CART22-65s cells/kg Day 3, 60% fraction: 1.2-6x10\^6 CART22-65s cells/kg Subjects ≥50kg will receive 1-5x10\^8 CART22-65s cells as a split dose over three days as follows: Day 1, 10% fraction: 1-5x10\^7 CART22-65s cells/kg Day 2, 30% fraction: 0.3-1.5x10\^8 CART22-65s cells/kg Day 3, 60% fraction: 0.6-3x10\^8 CART22-65s cells/kg

Pediatric patients with relapsed or refractory B-cell acute lymphoblastic leukemia
Cohort 3BIOLOGICAL

Subjects \<50kg will receive 0.2-1 x 10\^7 CART22-65s cells as a split dose over two days as follows: Day 1, 25% fraction: 0.5-2.5x10\^6 CAR T cells/kg Day 2, 75% fraction: 1.5-7.5x10\^6 CAR T cells/kg Subjects ≥50kg will receive 1-5x10\^8 CART22-65s cells as a split dose over two days as follows: Day 1, 25% fraction: 0.25-1.25x10\^8 CART22-65s cells Day 2, 75% fraction: 0.75-3.75x10\^8 CART22-65s cells

Pediatric patients with relapsed or refractory B-cell acute lymphoblastic leukemia
Cohort 1BIOLOGICAL

Subjects \<50kg will receive 0.2-1 x 10\^7 CART22 cells/kg as a split dose over three days as follows: Day 1, 10% fraction: 0.2-1x10\^6 CART22 cells/kg Day 2, 30% fraction: 0.6-3x10\^6 CART22 cells/kg Day 3, 60% fraction: 1.2-6x10\^6 CART22 cells/kg Subjects ≥50kg will receive 1-5x10\^8 CART22 cells as a split dose over three days as follows: Day 1, 10% fraction: 1-5x10\^7 CART22 cells/kg Day 2, 30% fraction: 0.3-1.5x10\^8 CART22 cells/kg Day 3, 60% fraction: 0.6-3x10\^8 CART22 cells/kg

Pediatric patients with relapsed or refractory B-cell acute lymphoblastic leukemia

Eligibility Criteria

Age1 Year - 29 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64)

You may not qualify if:

  • Active hepatitis B or active hepatitis C.
  • HIV Infection.
  • Active acute or chronic graft-versus-host disease (GVHD) requiring systemic therapy.
  • CNS3 disease that is progressive on therapy, or with CNS parenchymal lesions that might increase the risk of CNS toxicity.
  • Pregnant or nursing (lactating) women.
  • Receipt of a prior investigational study agent within 4 weeks prior to screening visit. \*Note - patients who have received anti-CD19 CART cells (e.g., CART19/CTL019) on an investigational study where cell infusion occurred greater than 4 weeks before the screening visit are NOT excluded.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

Location

Related Publications (2)

  • Myers RM, DiNofia AM, Li Y, Diorio C, Liu H, Wertheim G, Fraietta JA, Gonzalez V, Plesa G, Siegel DL, Iannone E, Shinehouse L, Brogdon JL, Taylor C, Jadlowsky JK, Hexner EO, Engels B, Baniewicz D, Callahan C, Ruella M, Aplenc R, Barz Leahy A, McClory SE, Rheingold SR, Wray L, June CH, Maude SL, Frey NV, Grupp SA. CD22-targeted chimeric antigen receptor-modified T cells for children and adults with relapse of B-cell acute lymphoblastic leukemia after CD19-directed immunotherapy. J Immunother Cancer. 2025 Apr 17;13(4):e011549. doi: 10.1136/jitc-2025-011549.

    PMID: 40246579DERIVED

  • Singh N, Frey NV, Engels B, Barrett DM, Shestova O, Ravikumar P, Cummins KD, Lee YG, Pajarillo R, Chun I, Shyu A, Highfill SL, Price A, Zhao L, Peng L, Granda B, Ramones M, Lu XM, Christian DA, Perazzelli J, Lacey SF, Roy NH, Burkhardt JK, Colomb F, Damra M, Abdel-Mohsen M, Liu T, Liu D, Standley DM, Young RM, Brogdon JL, Grupp SA, June CH, Maude SL, Gill S, Ruella M. Antigen-independent activation enhances the efficacy of 4-1BB-costimulated CD22 CAR T cells. Nat Med. 2021 May;27(5):842-850. doi: 10.1038/s41591-021-01326-5. Epub 2021 Apr 22.

    PMID: 33888899DERIVED

MeSH Terms

Conditions

Leukemia, B-CellLymphoma, B-Cell

Condition Hierarchy (Ancestors)

Leukemia, LymphoidLeukemiaNeoplasms by Histologic TypeNeoplasmsHematologic DiseasesHemic and Lymphatic DiseasesLymphoproliferative DisordersLymphatic DiseasesImmunoproliferative DisordersImmune System DiseasesLymphoma, Non-HodgkinLymphoma

Study Officials

  • Stephan Grupp, MD, PhD

    Children's Hospital of Philadelphia

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

December 22, 2015

First Posted

January 8, 2016

Study Start

January 13, 2016

Primary Completion (Estimated)

December 1, 2037

Study Completion (Estimated)

December 1, 2037

Last Updated

December 12, 2025

Record last verified: 2025-12

Locations

US(1)