# CART19-BE-01: A Multicenter Trial of ARI-0001 Cell Therapy in Patients with CD19(+) Relapsed/Refractory Malignancies

Source: https://onco.cc/key-papers/paper-cart19-be-01-mol-ther-2021/  
OnCo record `paper-cart19-be-01-mol-ther-2021` (Key paper). Data CC BY-NC 4.0, attribute "Data from OnCo (onco.cc)"; commercial use needs a licence.

## TL;DR

The results of the academic CAR-T trial that Spain's regulator used to authorise a hospital-made cell therapy for adult leukaemia, which the AEMPS described as the first CAR-T developed entirely in Europe to be approved by a regulator.

## Summary

Hospital Clínic de Barcelona and Hospital Sant Joan de Déu treated adults and children with relapsed or refractory CD19-positive malignancies with ARI-0001, a CD19 CAR-T cell product made at the hospital, after cyclophosphamide and fludarabine. The dose of 0.4 to 5 x 10^6 cells per kg was given at first as one infusion and later, after an amendment, split into three fractions with the full dose depending on the absence of cytokine release syndrome. Of 58 patients included, 47 were treated: 38 with acute lymphoblastic leukaemia, 8 with non-Hodgkin lymphoma and 1 with chronic lymphocytic leukaemia. The authors conclude that the safety and efficacy results are comparable with other academic or commercially available products. The trial is registered as NCT03144583.

## Fields

- Kind: Key paper
- Last checked: 2026-11-03
- Journal: Molecular Therapy
- Year: 2021
- DOI: 10.1016/j.ymthe.2020.09.027
- Authors: Ortíz-Maldonado V, Rives S, Castellà M, et al.
- Findings: In acute lymphoblastic leukaemia, grade 3 or higher cytokine release syndrome occurred in 13.2% of patients: 26.7% before the fractionation amendment against 4.3% after.; Grade 3 or higher neurotoxicity occurred in 2.6%; procedure-related mortality was 7.9% at day 100, with no procedure-related deaths after the amendment.; The measurable residual disease-negative complete response rate at day 100 was 71.1%.; Progression-free survival at one year was 47% (95% CI 27% to 67%) and overall survival 68.6% (95% CI 49.2% to 88%).
- What it means: This is the evidence behind the AEMPS's February 2021 authorisation of ARI-0001 under the hospital exemption, which let a public hospital make and give its own CAR-T to adults with relapsed acute lymphoblastic leukaemia. The fractionated dosing it introduced is the approach later used for the hospital's BCMA CAR-T.
- Caveats: A single-arm pilot study with 47 treated patients and no comparison group; one-year confidence intervals are wide.; The dosing schedule changed partway through, so the before and after figures come from small subgroups.

## Sources

- Mol Ther 2021: https://doi.org/10.1016/j.ymthe.2020.09.027
- PubMed: https://pubmed.ncbi.nlm.nih.gov/33010231/

## Connected records

- cancers: [Acute lymphoblastic leukaemia](https://onco.cc/cancers/all-leukemia/)
- technologies: [CAR-T cell therapy](https://onco.cc/technologies/car-t/)
- targets: [CD19](https://onco.cc/targets/cd19/)
- drugs: [Varnimcabtagene autoleucel](https://onco.cc/drugs/varnimcabtagene-autoleucel/)
- institutions: [CIBERONC (Centro de Investigación Biomédica en Red de Cáncer)](https://onco.cc/institutions/ciberonc/), [Hospital Clínic de Barcelona / IDIBAPS](https://onco.cc/institutions/hospital-clinic-barcelona/)
- trials: [CART19-BE-01 (ARI-0001 CAR-T, Hospital Clínic de Barcelona)](https://onco.cc/trials/cart19-be-01/)
- people: [Julio Delgado](https://onco.cc/people/julio-delgado/), [Manel Juan](https://onco.cc/people/manel-juan/), [Valentín Ortiz-Maldonado](https://onco.cc/people/valentin-ortiz-maldonado/)

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