Real-life Management of Patients Eligible for CAR-T Cell Therapy

Official title

Evaluation of the Real-life Management of Patients Eligible for CAR-T Cell Therapy for Hematologic Malignancies at Toulouse University Hospital

Overview

Adoptive immunotherapy using CAR-T cells is now one of the Advanced Therapy Medicines routinely used for relapsed or refractory lymphoid hemopathies. In 2023, in France, 5 types of CAR-T cells have marketing authorization for 6 different indications. However, these marketing authorizations are based on clinical trials involving a limited number of selected patients. Real-life data are essential for assessing the post-authorization use of these innovative treatments. The French national DESCAR-T registry, promoted by LYSARC and in which Toulouse University Hospital plays an active role, is an international reference for this real-life evaluation. It does not, however, allow precise evaluation of patient-centered indicators and care pathways. With the increasing number of indications and candidate patients, Toulouse University Hospital, the only healthcare facility authorized in the Western Occitanie region to administer CAR-T cells, is faced with growing hospital needs and longer treatment times. In 2023, this has necessitated the implementation of new ambulatory and inter-facility care pathways in collaboration with the referral centers of the Onco-Occitanie Ouest regional cancer network. The selection of patients for CAR-T cell treatment is based on objective clinical criteria linked to the pathology (histology, morphological localization, size and kinetics of the tumor mass) and the patient (physiological age, performance index, comorbidities, patient choice). Because of their innovative nature, in a difficult psychological and physical context for the patient (refractory disease), CAR-T cell care pathways also need to be evaluated in terms of their "quality of life" dimension. The impact of non-biological determinants (also described as social and territorial inequalities in health) such as place of residence and distance from healthcare provision, marital, economic and social status, has never been explored on the accessibility and progress of the CAR-T cell treatment pathway. The creation of a registry of patients eligible for CAR-T cells at Toulouse University Hospital will enable these lines of research to be explored on the scale of a region with a population of 3 million.

Detailed description

Ambispective observational cohort: This observational study is monocentric. For each patient, data will be collected during 5 years.

Interventions

• Other Patients eligible for CAR-T treatment
  Patients eligible for CAR-T treatment for their hemopathy at TOULOUSE University Hospital under early access or marketing authorization or as part of a clinical trial between 01/01/2019 and 31/12/2028

Primary outcome measures

• Overall survival of patients with hemopathy eligible for CAR-T cell therapy [Time frame: 5 years]

Secondary outcome measures (2)

• Compare progression-free survival and overall survival of patients according to care pathway and type of hemopathy, and investigate clinical and socioeconomic factors associated with better survival [Time frame: 5 years]
• Evaluate the time taken to treat patients with CAR-T cells [Time frame: 5 years]

Eligibility criteria

Inclusion criteria

• Patient eligible for CAR-T treatment for hemopathy at TOULOUSE University Hospital under early access or marketing authorization or as part of a clinical trial between 01/01/2019 and 31/12/2028
• Patient able to understand the purpose and constraints of the research project
• Patient has read the study information leaflet and does not object to the research.

Exclusion criteria

• Patient under guardianship, curatorship or safeguard of justice
• Patient objects to the collection of data concerning him/her

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Observational model

Cohort

Study locations

France · 1 center

• CHU de Toulouse — Toulouse

Identifiers

NCT: NCT06369389 · RC31/23/0417

Primary sources (government registries)

• ClinicalTrials.gov (US NIH)

View this study on ClinicalTrials.gov ↗