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Acute Lymphoblastic LeukemiaPhase 1/2NCT04787263

CD19-CAR_Lenti T Cells in Pediatric Patients Affected by Relapsed/Refractory CD19+ ALL and DLBCL or PML

A Phase 1/2 study testing CD19-CAR_Lenti T cell in people with Acute Lymphoblastic Leukemia and Diffuse Large B Cell Lymphoma, run by Bambino Gesù Hospital and Research Institute at 1 site in Italy.

Active, not recruiting

Under way, not looking for participants. Everyone needed has enrolled. The study is still running.

Official record
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Trial summary

This study aims at evaluating the feasibility and safety of the administration of autologous T cells that have been modified through the introduction of a chimeric antigen receptor targeting the B-cell surface antigen CD19, following administration of lymphodepleting chemotherapy regimen, in children and adults with… Read more about this study

Who is it for?
Ages 1 year to 25 years
What is being tested?
CD19-CAR_Lenti T cell
Could I receive a placebo?
No, everyone receives an active treatment
How many people
32 participants
Where
1 siteItaly
Study dates
4 Mar 2021to 3 Mar 2038
Phase
Phase 1/2
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 25 years
  • All sexes
  • No healthy volunteers
  • Child, Adult

Who may be able to join

  • Diagnosis of CD19 expressing B-ALL or DLBCL or PML and one of the following:
  • Patients in 1st relapse, with High-Risk (HR) features including: MLL- rearrangements, E2A/TCF3-PBX1, TCF3-HLF [t(17;19)], hypodiploidy (i.e., <44 chromosomes), TP53 alterations, early (i.e., <30 months from diagnosis)/very early (i.e., <18 months from diagnosis) isolated or combined bone marrow relapse
  • MRD > 0.1% after either reinduction therapy or any course of consolidation for relapsed ALL
  • Patients with DLBCL or PML in 1st or subsequent relapse, after at least one standard frontline chemotherapy
  • Age: 1 year - 25 years for Bcp-ALL and 1-35 years for B-NHL.
  • Voluntary informed consent is given. For subjects < 18 year-old their legal guardian must give informed consent. Pediatric subjects will be included in age-appropriate discussion and verbal assent will be obtained for those greater than or equal to 12 years of age, when appropriate.
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  • Clinical performance status: Patients > 16 years of age: Karnofsky greater than or equal to 60%; Patients < 16 years of age: Lansky scale greater than or equal to 60%.
  • Patients of child-bearing or child-fathering potential must be willing to practice birth control from the time of enrollment on this study and for four months after receiving the preparative regimen.
  • Females of child-bearing potential must have a negative pregnancy test because of the potentially dangerous effects on the fetus.

Who cannot join

  • Pregnant or lactating women
  • Severe, uncontrolled active infections
  • HIV, or active HCV and/or HBV infection (detection of viral RNA/DNA in blood)
  • Life-expectancy < 6 weeks
  • Hepatic function: Inadequate liver function defined as total bilirubin > 4x upper limit of normal (ULN) or transaminase (ALT and AST) > 6 x ULN
  • Renal function: serum creatinine > 3x ULN for age.
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  • Blood oxygen saturation < 90%.
  • Cardiac function: Left ventricular ejection fraction lower than 45% by ECHO.
  • Congestive heart failure, cardiac arrhythmia, psychiatric illness, or social situations that would limit compliance with study requirements or in the opinion of the PI would pose an unacceptable risk to the subject.
  • BM blasts > 50% pre-infusion.
  • Hyperleukocytosis (greater than or equal to 20,000 blasts/microliter) or rapidly progressive disease that in the evaluation of the investigator would compromise ability to complete study therapy
  • Presence of active, grade 2-4 acute or moderate-severe chronic GvHD
  • Recurrent or refractory ALL with testicular involvement
  • Concurrent or recent prior therapies, before infusion:
  • Systemic steroids (at a dose > 2 mg/kg prednisone) in the 2 weeks before infusion. Recent or current use of inhaled/topical/non-absorbable steroids is not exclusionary.
  • Systemic chemotherapy in the week preceding infusion.
  • Anti-thymocyte globulin (ATG) in the 4 weeks preceding infusion.
  • Immunosuppressive agents in the 1 week preceding infusion.
  • Radiation therapy must have been completed at least 3 weeks prior to enrollment.
  • Other anti-neoplastic investigational agents currently administered or within 30 days prior to infusion (i.e. start of protocol therapy);
  • Exceptions:
  • There is no time restriction with respect to prior intrathecal chemotherapy, provided that there is complete recovery from any acute toxic effects of such; ii. Patients who relapse while receiving standard ALL maintenance chemotherapy will not be required to have a waiting period before entry onto this study provided that they meet all other eligibility criteria; iii. Subjects receiving steroid therapy at physiologic replacement doses only are allowed provided that there has been no increase in dose for at least 2 weeks prior to starting apheresis;
  • Patient-derived CD19-CAR_Lenti production failure: vitality of the fresh product <80%, CD3+ cells <80%, CD3+ CAR+ cells <10%, non-sterility in IPC at day 5, endotoxin contamination (> 5 EU/ml) in IPC at day 5, mycoplasma contamination in IPC at day 5, failure of the visual inspection.

Trial locations and contacts

1 site in 1 country.

Italy1 site

  • IRCCS Ospedale Pediatrico Bambino Gesù

    Roma, 00165

Other trials in Italy

About this study

Official title: Phase I/II Study of Anti-CD19 Chimeric Antigen Receptor-Expressing T Cells in Pediatric Patients Affected by Relapsed/Refractory CD19+ Acute Lymphoblastic Leukemia and Diffuse Large B Cell Lymphoma (DLBCL) or Primary Mediastinal B Cell Lymphoma (PML)

This study aims at evaluating the feasibility and safety of the administration of autologous T cells that have been modified through the introduction of a chimeric antigen receptor targeting the B-cell surface antigen CD19, following administration of lymphodepleting chemotherapy regimen, in children and adults with relapsed/refractory B-cell acute lymphoblastic leukemia (B- ALL) or aggressive B-cell Non-Hodgkin lymphoma (B-NHL). The phase II extension is aimed at testing the efficacy of the treatment at the optimal dose defined in the phase I. In addition, the investigators hypothesize that it is feasible to successfully manufacture CAR T cells to meet the established release criteria at a maximum target dose of 3.0 x 10^6 cells/kilogram recipient total body weight in this patient population using the Miltenyi CliniMACS Prodigy® closed transduction system.

Being tested
CD19-CAR_Lenti T cell
Medical subject headings
Precursor Cell Lymphoblastic Leukemia-Lymphoma, Lymphoma, Large B-Cell, Diffuse

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • CD19-CAR_Lenti T cellBiological

    Fresh peripheral blood mononuclear cells are manufactured to obtain CD19-CAR_Lenti T cell, second generation CAR T cells incorporating the 4-1BB costimulatory domain. The fresh product is infused following lymphodepletion chemotherapy at a dose of 1.0-3.0x10^6 CAR+ cells/kg.

Study groups in NCT04787263
Study groupRoleWhat they receive
CD19-CAR_LentiExperimentalFollowing lymphodepletion with chemotherapy (fludarabine + cyclophosphamide), patients will be treated with 1.0 to 3.0 x 10^6/kg CD19-Chimeric Antigen Receptor (CAR)_Lenti positive cells as a single dose. The product will be infused fresh, at the end of manufacturing.
Receives: Biological: CD19-CAR_Lenti T cell

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Phase I - Identification of dose limiting toxicities (DLTs) and recommended dose (RD)

    Toxicity will be assessed according to the NCI Common Terminology Criteria for Adverse Events (CTCAE) scale, version 5.0

    Measured: 4 weeks after CAR T cell infusion

  2. Phase I - Identification of recommended dose (RD)

    The recommended dose of CD19-CAR_Lenti will be defined as the maximum tolerated dose (MTD) or the highest dose studied, if an MTD is not reached.

    Measured: 4 weeks after CAR T cell infusion of the last patient in the last dose level

  3. Phase II - Efficacy

    Bone marrow morphological and minimal residual disease complete remission rate at day 28 after infusion for BCP-ALL; Overall Response Rate (CR, CRi, PR and SD) at day 28, day 90 and day 180 after CAR T cells infusion

    Measured: Up to 6 months after CAR T cell infusion

Secondary outcomes (5)

  1. Relapse Rate (RR)

    Measured: Up to 2 years

  2. Overall survival (OS)

    Measured: Up to 2 years

  3. Disease-Free Survival (DFS)

    Measured: Up to 2 years

  4. In vivo persistence/expansion of infused CAR T cell

    Detection of infused CAR T cell in the peripheral and bone marrow blood

    Measured: Up to 2 years

  5. Cytokine profiling

    Define serum cytokine profile (Th1/Th2) after T cell infusion and correlation with cytokine release syndrome (CRS)

    Measured: Up to 10 days after CAR T cell infusion

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted8 Mar 2021
  2. Study start4 Mar 2021
  3. Primary completionMar 2027 (estimated)
  4. Study completion3 Mar 2038 (estimated)

Resources

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 1/2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
32 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04787263
Sponsor's protocol ID
CD19-CAR_Lenti
Status last verified
Nov 2025
First submitted
2 Mar 2021
Last updated
2 Dec 2025
Results posted
No
FDA-regulated drug
No
FDA-regulated device
No

Questions about this trial

What is this clinical trial studying?

A Phase 1/2 study testing CD19-CAR_Lenti T cell in people with Acute Lymphoblastic Leukemia and Diffuse Large B Cell Lymphoma, run by Bambino Gesù Hospital and Research Institute at 1 site in Italy.

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 9 inclusion and 23 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 2 Dec 2025. 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 Italy. 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 combined 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.

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

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?

Phase I - Identification of dose limiting toxicities (DLTs) and recommended dose (RD) (time frame: 4 weeks after CAR T cell infusion). The study lists 3 primary outcomes in all. 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 NCT04787263.

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 2 Dec 2025.

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