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B-cell Acute Lymphoblastic LeukemiaPhase 2NCT05460533

A Second Infusion (Early Reinfusion) of Tisagenlecleucel in Children and Young Adults With B-Cell Acute Lymphoblastic Leukemia(B-ALL)

A Phase 2 study testing Tisagenlecleucel in people with B-cell Acute Lymphoblastic Leukemia, run by Memorial Sloan Kettering Cancer Center at 6 sites in United States.

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

The researchers are doing this study to see if early reinfusion of tisagenlecleucel can keep participants in B-CEll ApLasia at 6 months after their first infusion. The researchers will also look at the safety of early reinfusion and how effective it is at treating B-ALL. Read more about this study

Who is it for?
Ages 1 day to 25 years
Conditions the study is for
B-cell Acute Lymphoblastic Leukemia
What is being tested?
Tisagenlecleucel
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
30 participants
Where
6 sitesUnited States
Study dates
12 Jul 2022to Jul 2027
Phase
Phase 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 day to 25 years
  • All sexes
  • No healthy volunteers
  • Child, Adult

Who may be able to join

  • Patients with R/R B-ALL who have received commercial tisagenlecleucel and have (an) additional dose(s) available for early reinfusion
  • History of CD19 expressing (in peripheral blood or bone marrow by flow cytometry) BALL prior to tisagenlecleucel infusion
  • Peripheral blood B-cell aplasia (BCA) within 14 days prior to reinfusion (See section 13.8: B-cell aplasia will be defined as peripheral blood (PB) absolute B lymphocyte count ≤ 50/μL. If BCA evaluation is repeated at any timepoint prior to reinfusion, it must be negative to proceed with reinfusion
  • Minimal residual disease negative complete remission (CR/CRi) in bone marrow within 14 days prior to reinfusion, including resolution of extramedullary disease
  • Patients with tisagenlecleucel that is deemed out of specification (OOS) will be permitted on this protocol if the reason for OOS is deemed to not impact the toxicity and efficacy profile of CAR T cell therapy
  • °Reasons for product being OOS include cell viability < 80%, total nucleated cell count <2 × 10^9 in leukapheresis product, failed interferon-γ release assay, leukapheresis product collected >9 months prior, and determination of residual beads >50 beads per 3 × 10^6 cells
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  • Patients age: < 26 years at time of first tisagenlecleucel order placement
  • Recovered from severe toxicities following initial dose of tisagenlecleucel
  • CRS
  • Neurotoxicity/ICANS
  • Adequate organ function at time of treatment is required and is defined:
  • Hepatic: Serum bilirubin ≤ 2 mg/dL, unless benign congenital hyperbilirubinemia
  • Hepatic: AST and ALT < 5x the upper limit of normal for age, unless thought to be disease-related
  • Renal: Serum creatinine <1.5x normal for age. If serum creatinine is outside the normal range, then CrCl > 60 mL/min/1.73m2 (calculated or estimated) or GFR (mL/min/1.72m2 ) >55% of predicted normal for age
  • Age Mean GFR +/-SD (mL/min/1.73 m2)
  • 1 week 40.6 + / - 14.8
  • 2 - 8 weeks 65.8 + / - 24.8
  • > 8 weeks 95.7 +/- 21.7
  • 2 - 12 years 133 +/- 27
  • 13 - 21 years (males) 140 +/- 30
  • 13 - 21 years (females) 126.0 + / - 22.0 Abbreviations: GFR, glomerular filtration rate; SD, standard deviation. Greater than 2 years old: Normal GFR is 100 mL/ min. Infants: GFR must be corrected for body surface area.
  • Cardiac: LVEF ≥ 50% by multi-gated acquisition scan (MUGA), resting echocardiogram, or cardiac magnetic resonance imaging (MRI) within 6 weeks of screening
  • Pulmonary: Oxygen saturation as recorded by pulse oximetry of ≥ 90% on room air
  • Adequate performance status:
  • Age ≥ 16 years: ECOG ≤ 1 or Karnofsky > 60% at treatment
  • Age < 16 years: Lansky > 60% at treatment
  • Patient must be enrolled on institutional CIBMTR reporting protocol for immune effector cells (IEC)/CAR T cells
  • Each patient must be willing to participate as a research subject, and patient or legal guardian must sign an informed consent form, along with patient assent if between 7 to 17 years of age. Legally authorized representatives of adult patients with impaired decision-making capacity will also be able to sign consent.

Who cannot join

  • Greater than 60 days from first tisagenlecleucel infusion
  • Ongoing severe toxicities from initial CAR T cell infusion
  • Received non-standard lymphodepleting chemotherapy (LDC) prior to initial tisagenlecleucel dose
  • Standard LDC is defined as:
  • Fludarabine 30mg/m^2/dose x 4 doses
  • Cyclophosphamide 500mg/m^2/dose x 2 doses
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  • Loss of BCA at any timepoint prior to reinfusion
  • Clinically significant active infection confirmed by clinical evidence, imaging, or positive laboratory tests (e.g., blood cultures, PCR for DNA/RNA, etc.)
  • Patient/parent/guardian unable to give informed consent or unable to comply with the treatment protocol
  • Pregnant or lactating women; women of child-bearing potential, defined as all women physiologically capable of becoming pregnant, unless they are using highly effective methods of contraception while taking study treatment and for at least 12 months after the tisagenlecleucel infusion and until CAR T-cells are no longer present by flow cytometry on two consecutive tests.
  • Sexually active males, unless they are willing to use a condom during intercourse while taking study treatment and for at least 12 months after the tisagenlecleucel infusion and until CAR T-cells are no longer present by flow cytometry on two consecutive tests.
  • Other uncontrolled, symptomatic, intercurrent illness including but not limited to infection, 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
  • Any other issue which, in the opinion of the treating physician, would make the patient ineligible for the study

Trial locations and contacts

6 sites in 1 country.

6 sites in 1 country

United States6 sites

  • Children's Hospital Colorado (Data Collection Only)

    Aurora, Colorado, 80045

  • Johns Hopkins University (Data Collection Only)

    Baltimore, Maryland, 21287

  • Cincinnati Children's Hospital Medical Center (Data Collection Only)

    Cincinnati, Ohio, 45229

  • Children's Hospital of Los Angeles (Data Collection Only)

    Los Angeles, California, 90027

  • Memorial Sloan Kettering Cancer Center

    New York, New York, 10065

  • Stanford University (Data Collection Only)

    Stanford, California, 94305

Other trials in United States

About this study

Official title: Phase II Open Label Multicenter Study of Early REinFusion of Tisagenlecleucel to Promote DUrable B-CEll ApLasia in Pediatric and Young Adult Patients With Relapsed/Refractory CD19-Positive B-Cell Acute Lymphoblastic Leukemia (REFUEL)

The researchers are doing this study to see if early reinfusion of tisagenlecleucel can keep participants in B-CEll ApLasia at 6 months after their first infusion. The researchers will also look at the safety of early reinfusion and how effective it is at treating B-ALL.

Read the detailed description

Prior to initial tisagenlecleucel cell infusion, lymphodepleting chemotherapy (LDC) is required with standard dosing cyclophosphamide and fludarabine as per standard-of-care (fludarabine 30mg/m^2 /dose x 4 doses and cyclophosphamide 500mg/m^2 /dose x 2 doses). Dose adjustments based off ideal body weight (IBW) and/or per institutional guidelines are allowed. LDC should be completed 2 to 14 days prior to the first tisagenlecleucel infusion. LDC may be repeated in cases where tisagenlecleucel has been delayed by more than 4 weeks. No additional LDC will be given prior to the early reinfusion.

Being tested
Tisagenlecleucel
Keywords
Tisagenlecleucel, Relapsed/Refractory CD19-Positive B-Cell, REinFusion, REFUEL, 22-220
Medical subject headings
Burkitt Lymphoma, Recurrence

What participants receive

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

  • TisagenlecleucelBiological

    Tisagenlecleucel will be infused based on institutional guidelines. Reinfusion of tisagenlecleucel will occur 30-60 days following the first dose.

Study groups in NCT05460533
Study groupRoleWhat they receive
TisagenlecleucelExperimentalPatients will receive reinfusion of tisagenlecleucel on day +30-60 after their initial infusion if meeting eligibility criteria. Tisagenlecleucel is an autologous cellular immunotherapy product that is comprised of CD3+T cells that have undergone ex vivo T cell activation, gene modification, expansion, and formulation in infusible cryomedia.
Receives: Biological: Tisagenlecleucel

What is being measured

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

  1. decrease the loss of peripheral BCA rate

    below 10% (from 26% to 9%)

    Measured: 6 months

Secondary outcomes (1)

  1. number and percentage of toxicities with early reinfusion of CAR T cells

    CTCAE Version 5 will be utilized for toxicity evaluation

    Measured: 1 year

Timeline

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

  1. First posted15 Jul 2022
  2. Study start12 Jul 2022
  3. Primary completionJul 2027 (estimated)
  4. Study completionJul 2027 (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 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
30 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT05460533
Sponsor's protocol ID
22-220
Status last verified
Feb 2026
First submitted
13 Jul 2022
Last updated
4 Feb 2026
Results posted
No
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
Yes: Memorial Sloan Kettering Cancer Center supports the international committee of medical journal editors (ICMJE) and the ethical obligation of responsible sharing of data from clinical trials. The protocol summary, a statistical summary, and informed consent form will be made available on clinicaltrials.gov when required as a condition of Federal awards, other agreements supporting the research and/or as otherwise required. Requests for deidentified individual participant data can be made beginning 12 months after publication and for up to 36 months post publication. Deidentified individual participant data reported in the manuscript will be shared under the terms of a Data Use Agreement and may only be used for approved proposals. Requests may be made to: crdatashare@mskcc.org.

Publications

  • Davis KL, Yao CC, Zimmerman JAO, Rau RE. Immunotherapy in B-Cell Acute Lymphoblastic Leukemia. J Natl Compr Canc Netw. 2025 Dec;23(12):e257067. doi: 10.6004/jnccn.2025.7067.

    PubMed 41671463

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Tisagenlecleucel in people with B-cell Acute Lymphoblastic Leukemia, run by Memorial Sloan Kettering Cancer Center at 6 sites 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 28 inclusion and 13 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 4 Feb 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 6 sites 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 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?

decrease the loss of peripheral BCA rate (time frame: 6 months). 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 NCT05460533, which also lists 1 linked publication.

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 4 Feb 2026.

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