An observational study in Relapsed/Refractory Acute Lymphoblastic Leukemia and T-cell Acute Lymphoblastic Leukemia, sponsored by Versailles Hospital. Status unknown at 31 sites in France. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2023-04-27.
Sponsored by Versailles Hospital · Observational
In order to improve the outcome of relapsed and/or refractory T-cell precursor acute lymphoblastic leukemia (T-ALL) patients, and to facilitate the use of oncogenetic targeted therapies in these patients, we set up an observational cohort, collecting clinical and biological information's from patients with T-ALL in relapse or refractory, as well as the use or not of a targeted therapy. The analysis of the cohort will allow us to evaluate the impact of this therapeutic strategies on the patients' fate, and to facilitate access to innovation and personalized medicine for these patients.
Depending on protocol and leukemia subtype, 5-10% of T-ALL patients are primary refractory, and 30-40% of patients will relapse. A new complete remission is attained in 20-40% of patients, but prolonged disease-free survival is observed in only 10-15% of cases. Nelarabine is approved for R/R T-ALL in second relapses, with a CR rate of 36% in the registration study, and an overall survival of 24% at 1 year and 12% at 3 years.
The biological landscape of T-ALL is well characterized, with the identification of at least 10 key recurrently mutated pathways including transcriptional regulation (91% of cases), cell cycle regulation and tumor suppression (84%), NOTCH1 signaling (79%), epigenetic regulation (68%), PI3K-AKT-mTOR signaling (29%), JAK/STAT signaling (25%), RAS signaling (14%), ribosomal function (13%), ubiquitination (9%) and RNA processing (9%). Furthermore, T-ALL cells are dependent upon BCL-XL and upon BCL-2, especially when the T-ALL blasts bear an ETP phenotype. However, genomic data cannot reliably predict the response of leukemic cells to a given treatment, due to interactions of the different cellular pathways affected in a living leukemic cell. Therefore, the combination of genotypic and phenotypic data may overcome this problem.
In France, patients with relapsed or refractory T-ALL (and also T-cell lymphoblastic lymphomas) are already proposed to undergo a genotypic (oncogenetic characterization) and a phenotypic (drug testing assay) characterization as a standard of care procedure. Based on the results obtained in fresh leukemic cells, a national scientific committee may recommend the used of targeted drugs alone or in combination, in the context of a "off-label" or a "compassionate" use (for example : Temsirolimus + Erwiniase + Venetoclax in PI3K-AKT-mTOR mutated ALL / Tofacitinib + Venetoclax in IL7R-JAK-STAT mutated ALL / 5-azacytidine + Venetocax in hypermethylated ALL / ...).
All patients who undergone this procedure will be proposed to be registered in the ALL-Target registry (ALL-target Observatory). After registration, data related to disease history, disease characterization and disease treatment as well as data describing the patient's condition will be collected.
Some patients may receive conventional chemotherapy as a salvage (conventional cohort), others may receive targeted therapy as a salvage (personalized medicine cohort). The aim of the registry is to evaluate the benefit of each treatment strategy in term of response as a primary end point. Comparison between the two cohorts will be performed after adjustment for confounding factors. Results of subgroups will also be reported using descriptive statistics. Secondary endpoints will include safety of the treatment strategy, survival, disease free survival and progression free survival.
5,441 studies on the registry are indexed under Leukemia; 636 are open to participants now.
This study's planned enrollment of 80 is below the median of 120 across 744 observational studies indexed under Leukemia.
Browse Leukemia studies →Versailles Hospital is the lead sponsor of 74 studies on the registry; 17 are open to participants now.
Counted across the registry records on this site, refreshed daily.
Patients 18 years of age or older, with relapsed or refractory T cell precursor-ALL or T-cell Lymphoblastic lymphoma, with oncogenetic analysis performed at diagnosis and/or relapse in the central laboratory.
Exclusion Criteria:
Response rate defined as complete remission and remission without complete hematological recovery (CR + CRi).
Response rate defined as complete remission and remission without complete hematological recovery (CR + CRi).
Time frame: 3 months
Performance Status of patients
Eastern Cooperative Oncology Group (ECOG) - Performans Status Scale PS 0 : Fully active, able to carry on all pre-disease performance without restriction PS 4: Completely disabled; cannot carry on any selfcare; totally confined to bed or chair
Time frame: 3 months
Biological description of T-ALL
Phenotypic and oncogenetic characterization with mutated signaling pathways
Time frame: 3 months
Description of the treatments received
Number and type of treatment lines received, including current treatment
Time frame: 3 months
Description of Overall response rate to treatment
Overall response rate defined by partial response rate + complete response rate
Time frame: 2 years
Description of the relapse rate
Event of relapse
Time frame: 2 years
Description of survival rate
Death
Time frame: 2 years
Duration of response
Response
Time frame: 2 years
Description of Adverse Events
Adverse Events
Time frame: 2 years
This study is status unknown, as verified in Apr 2023. You cannot join it, but the record below documents what was studied.
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Versailles Hospital