B Cell LeukemiasPhase 1NCT02650414
CD22 Redirected Autologous T Cells for ALL
A Phase 1 study testing Cohort 1, Cohorts 2 and Cohort 3 in people with B Cell Leukemias and B Cell Lymphomas, run by University of Pennsylvania at 1 site in United States.
Trial 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… Read more about this study
- Who is it for?
- Ages 1 year to 29 years
- Conditions the study is for
- B Cell Leukemias, B Cell Lymphomas
- What is being tested?
- Cohort 1, Cohorts 2 and Cohort 3
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 41 participants
- Where
- 1 siteUnited States
- Study dates
- 13 Jan 2016to Dec 2037
- Phase
- Phase 1
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 1 year to 29 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
None listed.
Who cannot join
- Active hepatitis B or active hepatitis C.
- HIV Infection.
- Active acute or chronic graft-versus-host disease (GVHD) requiring systemic therapy.
- Concurrent use of systemic steroids or immunosuppressant medications. Recent or current use of inhaled steroids or physiologic replacement with hydrocortisone is not exclusionary.
- 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.
Trial locations and contacts
1 site in 1 country.
United States1 site
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
About this study
Official title: Pilot Study of Autologous Anti-CD22 Chimeric Antigen Receptor Redirected T Cells in Pediatric Patients With Chemotherapy Resistant Or Refractory Acute Lymphoblastic Leukemia
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.
- Conditions
- B Cell Leukemias, B Cell Lymphomas
- Being tested
- Cohort 1, Cohorts 2, Cohort 3, Cohort 3A
- Keywords
- Biological: CART 22
- Medical subject headings
- Leukemia, B-Cell, Lymphoma, B-Cell
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
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
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
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
Cohort 3ABiological
Subjects <50kg will receive 0.2-2 x 10^6 CART22-65s cells/kg as a split dose over two days as follows:
Day 1, 25% fraction: 25% fraction: 0.05-0.5x10^6 CAR T cells/kg Day 2, 75% fraction: 75% fraction: 0.15-1.5x10^6 CAR T cells/kg
Subjects ≥50kg will receive 0.1-1x10^8 CART22-65s cells as a split dose over two days as follows:
Day 1, 25% fraction: 0.25-2.5x10^7 CART22-65s cells Day 2, 75% fraction: 0.75-7.5x10^7 CART22-65s cells
| Study group | Role | What they receive |
|---|---|---|
| Pediatric patients with relapsed or refractory B-cell acute lymphoblastic leukemia | Experimental | Not described Receives: Biological: Cohort 1; Biological: Cohorts 2; Biological: Cohort 3; Biological: Cohort 3A |
What is being measured
The primary outcome is the main question the study is designed to answer.
The frequency and severity of adverse events.
Grade 3 and higher toxicity rate (toxicity possibly attributed to CART22 or CART22-65s) that is unmanageable, unexpected and unrelated to chemotherapy.
Measured: From date of dosing ( day 1 ) up 15 years
Secondary outcomes (14)
Percentage of manufacturing products that do not meet release criteria.
Product must pass for vector transduction efficiency, T cell product purity, viability, sterility or due to tumor contamination.
Measured: 3 months
Overall Complete Remission Rate (ORR) at Day 28.
Includes CR and CR with incomplete blood count recovery (CRi)
Measured: 4 months
Evaluate overall response rate (CR/CRi by or at Month 6) at Month 6.
Measured: 9 months
Evaluate disease status at Month 6.
Measured: 9 months
Overall survival (OS)
Measured: From date of dosing ( day 1 ) up 15 years
Duration of remission (DOR)
Measured: 15 Years
Number of subjects with relapse free survival (RFS)
Measured: 15 years
Number of subjects with event free survival (EFS).
Measured: 15 years
Describe cause of death (COD) when appropriate
Measured: 15 years
Describe response in terms of minimal residual disease (MRD).
Percentage of patients who achieve a CR associated with minimal residual disease (MRD) negative bone marrow as determined by high sensitivity flow cytometry.
Measured: 1 year
Incidence of any acute GVHD
Measured: 15 year
Incidence of any grade II-IV aGVHD
Measured: 15 year
Incidence any chronic GVHD.
Measured: 15 year
Incidence any extensive, limited cGVHD.
Measured: 15 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Jan 2016 (estimated)
- Study start13 Jan 2016
- Primary completionDec 2037 (estimated)
- Study completionDec 2037 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for B Cell LeukemiasStudies of the same condition, open and under way.
- More trials run by University of PennsylvaniaThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 41 participants (estimated)
Sponsor and investigators
- Lead sponsor
- University of Pennsylvania
- Sponsor type
- University, hospital or other
- Collaborators
- Children's Hospital of Philadelphia
- Responsible party
- Sponsor
- Investigators
- Stephan Grupp, MD, PhD, Principal investigator, Children's Hospital of Philadelphia
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02650414
- Sponsor's protocol ID
- 15CT055; 15-012219; 15-0152
- Status last verified
- Jul 2026
- First submitted
- 22 Dec 2015
- Last updated
- 7 Jul 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Publications
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.
PubMed 40246579Singh 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.
PubMed 33888899
Study at a glance
From the registry record, last updated by the sponsor on 7 Jul 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Pilot Study of Autologous Anti-CD22 Chimeric Antigen Receptor Redirected T Cells in Pediatric Patients With Chemotherapy Resistant Or Refractory Acute Lymphoblastic Leukemia
- ClinicalTrials.gov ID
- NCT02650414
- Acronym / sponsor ID
- 15CT055; 15-012219; 15-0152
- Conditions
- B Cell Leukemias, B Cell Lymphomas
- Keywords
- Biological: CART 22
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 41 participants (estimated)
- Lead sponsor
- University of PennsylvaniaUniversity, hospital or other
- Collaborators
- Children's Hospital of Philadelphia
- Study start
- 13 Jan 2016 (actual)
- Primary completion
- Dec 2037 (estimated)
- Study completion
- Dec 2037 (estimated)
- First posted
- 8 Jan 2016 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Pediatric patients with relapsed or refractory B-cell acute lymphoblastic leukemia | Experimental | Biological: Cohort 1, Biological: Cohorts 2, Biological: Cohort 3, Biological: Cohort 3A |
Who may qualify
- Ages
- 1 year to 29 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (0 listed)
Key exclusion criteria (7 listed)
- Active hepatitis B or active hepatitis C.
- HIV Infection.
- Active acute or chronic graft-versus-host disease (GVHD) requiring systemic therapy.
- Concurrent use of systemic steroids or immunosuppressant medications. Recent or current use of inhaled steroids or physiologic replacement with hydrocortisone is not exclusionary.
- CNS3 disease that is progressive on therapy, or with CNS parenchymal lesions that might increase the risk of CNS toxicity.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Cohort 1Subjects <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 | Biological | Pediatric patients with relapsed or refractory B-cell acute lymphoblastic leukemia |
| Cohorts 2Subjects <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 | Biological | Pediatric patients with relapsed or refractory B-cell acute lymphoblastic leukemia |
| Cohort 3Subjects <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 | Biological | Pediatric patients with relapsed or refractory B-cell acute lymphoblastic leukemia |
| Cohort 3ASubjects <50kg will receive 0.2-2 x 10^6 CART22-65s cells/kg as a split dose over two days as follows: Day 1, 25% fraction: 25% fraction: 0.05-0.5x10^6 CAR T cells/kg Day 2, 75% fraction: 75% fraction: 0.15-1.5x10^6 CAR T cells/kg Subjects ≥50kg will receive 0.1-1x10^8 CART22-65s cells as a split dose over two days as follows: Day 1, 25% fraction: 0.25-2.5x10^7 CART22-65s cells Day 2, 75% fraction: 0.75-7.5x10^7 CART22-65s cells | Biological | Pediatric patients with relapsed or refractory B-cell acute lymphoblastic leukemia |
Primary outcome
The frequency and severity of adverse events.
Time frame: From date of dosing ( day 1 ) up 15 years
Grade 3 and higher toxicity rate (toxicity possibly attributed to CART22 or CART22-65s) that is unmanageable, unexpected and unrelated to chemotherapy.
14 secondary outcomes
Percentage of manufacturing products that do not meet release criteria.
Time frame: 3 months
Overall Complete Remission Rate (ORR) at Day 28.
Time frame: 4 months
Evaluate overall response rate (CR/CRi by or at Month 6) at Month 6.
Time frame: 9 months
Evaluate disease status at Month 6.
Time frame: 9 months
Overall survival (OS)
Time frame: From date of dosing ( day 1 ) up 15 years
Duration of remission (DOR)
Time frame: 15 Years
Number of subjects with relapse free survival (RFS)
Time frame: 15 years
Number of subjects with event free survival (EFS).
Time frame: 15 years
Describe cause of death (COD) when appropriate
Time frame: 15 years
Describe response in terms of minimal residual disease (MRD).
Time frame: 1 year
Incidence of any acute GVHD
Time frame: 15 year
Incidence of any grade II-IV aGVHD
Time frame: 15 year
Incidence any chronic GVHD.
Time frame: 15 year
Incidence any extensive, limited cGVHD.
Time frame: 15 year
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 7 Jul 2026.
- Derived from this study: PubMed 40246579Myers 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.
- Derived from this study: PubMed 33888899Singh 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.
Related trials for B Cell Leukemias
Not yet recruitingPhase 1/2
A Study of Sonrotoclax (BGB-11417) in Children With Relapsed or Refractory Acute Myeloid Leukemia and B-cell Acute Lymphoblastic Leukemia
Relapsed Acute Myeloid Leukemia, Refractory Acute Myeloid Leukemia
RecruitingPhase 1
Anti-CRLF2-R/TSLPR Chimeric Antigen Receptor T Cells (TSLPR-CART) in Participants With Recurrent or Refractory CRLF2-R/TSLPR-Overexpressing B-Cell Acute Lymphoblastic Leukemia (B-ALL)
B-All, Acute Lymphoblastic Leukemia
RecruitingPhase 2
CD22 CAR T-cells to Extend Remission Following Commercial CD19 CAR T-cells in Children, Adolescents, and Adults With Relapsed/Refractory B-cell Acute Lymphoblastic Leukemia
Acute Lymphoblastic Leukemia, B-All
RecruitingPhase 3
International Study for Treatment of Childhood Relapsed Precursor B-Cell ALL 2020 (IntReALL BCP 2020)
B-Cell Acute Lymphoblastic Leukaemia
RecruitingObservational study
Natural History Study of Monoclonal B Cell Lymphocytosis (MBL), Chronic Lymphocytic Leukemia/Small Lymphocytic Lymphoma (CLL/SLL), Lymphoplasmacytic Lymphoma (LPL)/Waldenstrom Macroglobulinemia (WM), and Splenic Marginal Zone Lymphoma (SMZL)
Waldenstrom Macroglobulinemia, Lymphoplasmacytic Lymphoma
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Stephan Grupp, MD, PhD principal investigator
Children's Hospital of Philadelphia
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Cohort 1, Cohorts 2 and Cohort 3 in people with B Cell Leukemias and B Cell Lymphomas, run by University of Pennsylvania at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 0 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 7 Jul 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
The frequency and severity of adverse events. (time frame: From date of dosing ( day 1 ) up 15 years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02650414, which also lists 2 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 7 Jul 2026.