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LeukemiaPhase 1CART19-BE-01NCT03144583

Pilot Study on the Infusion of ARI-0001 Cells in Patients With CD19+ Leukemia or Lymphoma Refractory to Therapy

A Phase 1 study testing Adult differentiated autologous T-cells in people with Leukemia and Lymphoma, run by Sara V. Latorre at 2 sites in Spain.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

To assess the infusion of ARI-0001 cells (Adult differentiated autologous T-cells from peripheral blood, expanded and transduced with a lentivirus to express a chimeric antigen receptor with anti-CD19 specificity [A3B1] conjugated with the co-stimulatory regions 4-1BB and CD3z ) safety on patients with leukemia or… Read more about this study

Who is it for?
Ages 2 to 80 years
Conditions the study is for
Leukemia, Lymphoma
What is being tested?
Adult differentiated autologous T-cells
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
50 participants
Where
2 sitesSpain
Study dates
15 Jun 2017to 13 Sep 2022
Phase
Phase 1
Are there results?
None posted in the registry

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 2 to 80 years
  • All sexes
  • No healthy volunteers
  • Child, Adult, Older adult

Who may be able to join

  • Diagnosis of leukemia or CD19 + lymphoma, with a life expectancy less than 2 years that meet the following conditions:- Adult acute lymphoid leukemia in second or third response, not candidate for transplantation due to age, associated diseases or lack of donor, or in relapse post allogeneic transplant.- Pediatric acute lymphoid leukemia in second or third response, refractory or non-transplant candidate due to donor absence, or in relapse post allogeneic transplant, or with minimal residual residual disease (0.1% or greater) after two or more lines of treatment. - Symptomatic follicular lymphoma, which has received at least 2 treatment regimens (one of them including rituximab) and a progression-free interval of less than 2 years. Patients not candidates for transplantation or post-transplant relapse may be included.- Symptomatic chronic lymphocytic leukemia, which has received at least 2 treatment regimens (one of them including rituximab) and a progression-free survival of less than 2 years. Patients with a 17p deletion or TP53 mutation may be included after the first line of treatment. - Mantle cell lymphoma in the first relapse (or higher) when it is not a candidate for transplantation, or second post-transplant relapse (or higher). - Diffuse large cell lymphoma in first relapse (or higher) when it is not a candidate for transplantation, or second post-transplant relapse (or higher).
  • Age greater than 2 years and less than 80.
  • ECOG functional status from 0 to 2
  • Life expectancy of at least 3 months.
  • Appropriate venous access to perform an apheresis procedure. Absence of contraindications for it.
  • Signature of informed consent (patient or legal guardian).

Who cannot join

  • Treatment with any experimental or non-marketed substance within four weeks prior to recruitment, or actively participating in another therapeutic clinical trial.
  • Diagnosis of another past or present neoplasm. Patients may be included in complete remission for more than 3 years, or with a history of non-melanoma skin cancer or completely resected in-situ carcinoma.
  • Central nervous system involvement (CNS-3) at inclusion. Inclusion will be permitted with patients with a lower grade (CNS-2) or with CNS-3 who have responded to intrathecal chemotherapy.
  • Early relapse after allogeneic transplantation (less than 3 months for apheresis of mononuclear cells, less than 6 months for infusion of ARI-0001) or patients on active immunosuppressive therapy for graft-versus-host disease (corticosteroids or other systemic immunosuppressants ).
  • Active infection requiring systemic medical treatment such as chronic kidney infection, chronic lung infection or tuberculosis.
  • HIV infection.
Show all 14Show fewer
  • Concurrent and uncontrolled medical illnesses including cardiac, renal, hepatic, gastrointestinal, endocrine, pulmonary, neurological or psychiatric diseases which in the opinion of the researcher represent a risk to the patient.
  • Positive serology for hepatitis B, defined as a positive test for HBsAg. In addition, if the patient is HBsAg negative but has anti-HBc antibodies it will be necessary to perform a DNA test of the hepatitis B virus, and if the result is positive the patient will be excluded.
  • Positive serology for hepatitis C, defined as a positive test for anti-HCV antibodies confirmed by RIBA.
  • Severe organ failure, defined as a cardiac ejection fraction <40%; DLCO <40%; calculated glomerular filtrate rate <30 ml / min; Or bilirubin> 3 times the upper limit of normal (unless it is due to CLL or Gilbert syndrome).
  • Pregnant or lactating women. Women of childbearing potential should have a negative pregnancy test in the screening phase.
  • Women of childbearing age, including those whose last menstrual cycle was in the year prior to screening, who are unable or unwilling to use highly effective contraceptive methods from the start of the study to the end of the study.
  • Men who are unable or unwilling to use highly effective contraceptive methods from the start of the study to the completion of the study.
  • The need to take glucocorticoids in a chronic manner at doses higher than 10 mg / day of prednisone (or equivalent) or other chronic immunosuppressants. Hormonal contraceptives, intrauterine device, intrauterine systems of hormonal release, sexual abstinence, vasectomy of the couple or bilateral tubal occlusion.

Trial locations and contacts

2 sites in 1 country.

Spain2 sites

  • Hospital Clínic of Barcelona

    Barcelona, 08036

  • Hospital Sant Joan de Deu

    Barcelona, 08950

Other trials in Spain

About this study

Official title: Pilot Study on the Infusion of Differentiated Autologous T-cells From Peripheral Blood, Expanded and Transduced With a Lentivirus to Express a Chimeric Antigen Receptor With Anti-CD19 Specificity Conjugated With the Co-stimulatory Regions 4-1BB and CD3z in Patients With CD19+ Leukemia or Lymphoma Refractory to Therapy

To assess the infusion of ARI-0001 cells (Adult differentiated autologous T-cells from peripheral blood, expanded and transduced with a lentivirus to express a chimeric antigen receptor with anti-CD19 specificity [A3B1] conjugated with the co-stimulatory regions 4-1BB and CD3z ) safety on patients with leukemia or lymphoma CD19+ resistant or refractory to treatment and with a prognosis of less than 2 years.

Conditions
Leukemia, Lymphoma
Being tested
Adult differentiated autologous T-cells
Keywords
CART19
Medical subject headings
Leukemia, Lymphoma

What participants receive

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

  • Adult differentiated autologous T-cellsBiological

    After pretreatment, adult differentiated autologous T-cells with a chimeric antigen receptor with anti-CD19 specificity will be transfused.

Study groups in NCT03144583
Study groupRoleWhat they receive
Adult differentiated autologous T-cellsExperimentalAdult differentiated autologous T-cells from peripheral blood, expanded and transduced with a lentivirus to express a chimeric antigen receptor with anti-CD19 specificity (A3B1) conjugated with the co-stimulatory regions 4-1BB and CD3z. Such cells will be administered in a single infusion intravenously at a total ARI-0001 cell dose of 0.5-10 x 106 / kg body weight.
Receives: Biological: Adult differentiated autologous T-cells

What is being measured

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

  1. Procedure-related mortality (PRM)

    Any death not caused directly by leukemia / lymphoma. For the estimation of PRM, relapse or progression of the disease will be considered as a competitive event.

    Measured: Year 1

  2. Procedure-related mortality (PRM)

    Any death not caused directly by leukemia / lymphoma. For the estimation of PRM, relapse or progression of the disease will be considered as a competitive event.

    Measured: Year 3

  3. Assessment of toxicity

    number of adverse events grade III-IV using CTC (common toxicity criteria)

    Measured: Month 3

  4. Assessment of toxicity

    number of adverse events grade III-IV using CTC (common toxicity criteria)

    Measured: Year 1

Secondary outcomes (7)

  1. Response rate (overall and complete)

    Defined differently for each disease:

    • On chronic lymphoid and acute lymphocytic leukemia, the usual criteria NCCN and IWCLL will be used.
    • On non-Hodgkin's lymphoma, the Lugano criteria will be used-
    • On patients with leukemia will be quantified the persistence of minimal residual disease, in bone marrow and peripheral blood, using multiparametric cytometry and new generation sequencing techniques.

    Measured: Month 3 and Year 1

  2. Progression-free survival

    Time lag between infusion of ARI-0001 and the progression of disease or death. Patients alive and in complete remission will be censored at the last follow-up

    Measured: Year 2 after procedure

  3. Overall survival (OS) at 2 years

    Time lag between the infusion of ARI-0001 and the death of the patient from any cause. Living patients will be censored at the the last follow-up.

    Measured: 3 years

  4. In vivo survival of ARI-0001 cells in peripheral blood, bone marrow and cerebrospinal fluid

    Determined monthly during the first 6 months by flow cytometry and quantitative transgene PCR.

    Measured: months 1,2,3,4,5,6

  5. In vivo survival of ARI-0001 cells in peripheral blood, bone marrow and cerebrospinal fluid

    Determined quarterly from month 6 until the 2 years after infusion, by flow cytometry and quantitative transgene PCR.

    Measured: months 9,12,15,18,21,24

  6. Quality of life of included patients

    Evaluated by a questionnaire completed by patients or their legal guardians

    Measured: Month 3, 6, 12

  7. Toxicity assessment

    defined as number of adverse events of any type occurring throughout the study using the common toxicity criteria

    Measured: Month 3 and year 1

Timeline

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

  1. First posted9 May 2017
  2. Study start15 Jun 2017
  3. Primary completion13 Sep 2022
  4. Study completion13 Sep 2022

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
Primary purpose
Other
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
50 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03144583
Sponsor's protocol ID
CART19-BE-01
Acronym
CART19-BE-01
Status last verified
Aug 2023
First submitted
26 Apr 2017
Last updated
28 Aug 2023
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
No

Publications

  • Krawczyk M, Druzynska M, Bednarska E, Winiarska M. The race between 4-1BB- and CD28-based CD19 CAR-T products in the therapy of B-cell malignancies. Biochim Biophys Acta Rev Cancer. 2026 Feb;1881(1):189519. doi: 10.1016/j.bbcan.2025.189519. Epub 2025 Dec 20.

    PubMed 41429239

  • Bartolo-Ibars A, Aran A, Johansson AM, Ortiz-Maldonado V, Klein-Gonzalez N, Alonso-Saladrigues A, James E, Urbano-Ispizua A, Rives S, Delgado J, Gonzalez-Navarro EA, Kwok WW, Juan M. T-cell responses against CD19-targeted CAR T cells varnimcabtagene autoleucel (ARI-0001): implications for immune response and therapy outcomes. J Immunother Cancer. 2025 Sep 22;13(9):e010792. doi: 10.1136/jitc-2024-010792.

    PubMed 40987492

  • Martinez-Cibrian N, Ortiz-Maldonado V, Espanol-Rego M, Alserawan L, Navarro S, Albiol N, Lozano M, Charry P, Magnano L, Rivero A, Correa JG, Mozas P, Cortes-Bullich A, Jimenez-Vicente C, Gine E, Montoro-Lorite M, Ramos C, Ayora P, Calderon H, Sanchez-Castanon M, Benitez-Ribas D, Mata-Molanes JJ, Rojas K, Setoain X, Rodriguez S, Murias A, Alcubilla P, Varea S, Olesti E, Bachiller M, Calvo-Orteu M, Sans-Pola C, Saez-Penataro J, de Larrea CF, Lopez-Guillermo A, Gonzalez-Navarro EA, Juan M, Delgado J. Efficacy and safety of the academic anti-CD19 chimeric antigen receptor T-cell product varnimcabtagene autoleucel for the treatment of relapsed/refractory follicular lymphoma. Hemasphere. 2025 Sep 11;9(9):e70166. doi: 10.1002/hem3.70166. eCollection 2025 Sep.

    PubMed 40951523

  • Martinez-Cibrian N, Ortiz-Maldonado V, Espanol-Rego M, Blazquez A, Cid J, Lozano M, Magnano L, Gine E, Correa JG, Mozas P, Rodriguez-Lobato LG, Rivero A, Montoro-Lorite M, Ayora P, Navarro S, Alserawan L, Gonzalez-Navarro EA, Castella M, Sanchez-Castanon M, Cabezon R, Benitez-Ribas D, Setoain X, Rodriguez S, Brillembourg H, Varea S, Olesti E, Guillen E, Saez-Penataro J, de Larrea CF, Lopez-Guillermo A, Pascal M, Urbano-Ispizua A, Juan M, Delgado J. The academic point-of-care anti-CD19 chimeric antigen receptor T-cell product varnimcabtagene autoleucel (ARI-0001 cells) shows efficacy and safety in the treatment of relapsed/refractory B-cell non-Hodgkin lymphoma. Br J Haematol. 2024 Feb;204(2):525-533. doi: 10.1111/bjh.19170. Epub 2023 Oct 31.

    PubMed 37905734

  • Ortiz-Maldonado V, Rives S, Castella M, Alonso-Saladrigues A, Benitez-Ribas D, Caballero-Banos M, Baumann T, Cid J, Garcia-Rey E, Llanos C, Torrebadell M, Villamor N, Gine E, Diaz-Beya M, Guardia L, Montoro M, Catala A, Faura A, Gonzalez EA, Espanol-Rego M, Klein-Gonzalez N, Alsina L, Castro P, Jordan I, Fernandez S, Ramos F, Sune G, Perpina U, Canals JM, Lozano M, Trias E, Scalise A, Varea S, Saez-Penataro J, Torres F, Calvo G, Esteve J, Urbano-Ispizua A, Juan M, Delgado J. CART19-BE-01: A Multicenter Trial of ARI-0001 Cell Therapy in Patients with CD19+ Relapsed/Refractory Malignancies. Mol Ther. 2021 Feb 3;29(2):636-644. doi: 10.1016/j.ymthe.2020.09.027. Epub 2020 Sep 20.

    PubMed 33010231

  • Castella M, Caballero-Banos M, Ortiz-Maldonado V, Gonzalez-Navarro EA, Sune G, Antonana-Vidosola A, Boronat A, Marzal B, Millan L, Martin-Antonio B, Cid J, Lozano M, Garcia E, Tabera J, Trias E, Perpina U, Canals JM, Baumann T, Benitez-Ribas D, Campo E, Yague J, Urbano-Ispizua A, Rives S, Delgado J, Juan M. Point-Of-Care CAR T-Cell Production (ARI-0001) Using a Closed Semi-automatic Bioreactor: Experience From an Academic Phase I Clinical Trial. Front Immunol. 2020 Mar 20;11:482. doi: 10.3389/fimmu.2020.00482. eCollection 2020.

    PubMed 32528460

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing Adult differentiated autologous T-cells in people with Leukemia and Lymphoma, run by Sara V. Latorre at 2 sites in Spain.

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 6 inclusion and 14 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: finished, as of the sponsor's last update on 28 Aug 2023. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 2 sites in Spain. 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?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

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?

Procedure-related mortality (PRM) (time frame: Year 1). The study lists 4 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 NCT03144583, which also lists 6 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 28 Aug 2023.

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