Clinical trial NCT03751072: Efficacy and Toxicity of Blinatumomab in the French ATU for Adult BCP-ALL R/R, or With MRD+ (FRENCH-CYTO)
Official title: Efficacy and Toxicity of Blinatumomab in the French Compassionate Use Program (ATU) for Adult Patients With B-cell Precursor Acute Lymphoblastic Leukemia (BCP-ALL) Refractory, in Relapse, or With Positive Minimal Residual Disease.
Study type: OBSERVATIONAL
Overall status: UNKNOWN
Conditions: Acute Lymphoblastic Leukemia, in Relapse
Acute Lymphoblastic Leukemia With Failed Remission
Lead sponsor: Group for Research in Adult Acute Lymphoblastic Leukemia
Sponsor class: OTHER
Collaborators: name: Amgen
First posted: 2018-11-23
Enrollment: 92
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: CASE_ONLY
Time perspective: RETROSPECTIVE
Sampling method: PROBABILITY_SAMPLE
Study population: Adult patients aged 18y+ with R/R or MRD+ BCP-ALL treated in the French GRAALL Network

Brief summary: The outcome of young adults (18-60 years) with ALL has been dramatically improved by the use of pediatric-inspired trials. About 60% of these young adult patients will be cured at 5 years. In this context, early evaluation of minimal residual disease (MRD) at complete remission has been shown to be one of the most powerful prognostic factor, but also predictive of the benefit of allogeneic stem cell transplantation (ASCT). Despite this global improvement, about 30% of patients experience a relapse and will be exposed to be refractory to salvage therapy or to early disease escape. In adult ALL, the most important prognostic factors at relapse are : the time from first CR to relapse, the achievement of a second complete remission (CR), and the feasibility of ASCT.

Blinatumomab is a bispecific T-cell engager that recruits T-cell on CD19 positive blast cells and induces anti-leukemic cytotoxicity. In a phase 3 trial in relapse/refractory Philadelphia-negative (Ph-) ALL, 43% of patients achieved a CR or CR with partial hematological recovery (CRh), with the majority of responses occurring within the first cycle. In patients with positive MRD (MRD+) BCP-ALL, blinatumomab resulted in complete MRD response in 78% of patients after one cycle.

Between 2012 and 2016, blinatumomab was available in France for R/R and MRD+ ALL adult patients through the French Compassionate Use Program. About 92 adult ALL were treated at different stages of the disease in 27 centers.

Detailed description: To evaluate the efficacy of blinatumomab given in the French Compassionate Use Program, in term of overall survival in both R/R (first cohort) and MRD positive (second cohort) patients.

To evaluate the efficacy of blinatumomab given in the French Compassionate Use Program, in term of CR/CRH in R/R patients, To evaluate the efficacy in term of molecular response in both R/R and MRD+ cohorts, To evaluate the efficacy of blinatumomab given in the French Compassionate Use Program, in both Ph+/Ph- ALL patients To evaluate the feasibility and the safety of blinatumomab administration in a multi-center setting.To evaluate the feasibility of allogeneic stem cell transplant after blinatumomab administration in both populations.

Eligibility criteria: Inclusion Criteria:

Patient with Philadelphia-negative or positive (Ph+) ALL in relapse, refractory to salvage therapy, or with MRD positive ALL that received blinatumomab in the French ATU program.,

* Patient treated in the GRAALL network,
* Patient who does'nt object to participate in the study

Exclusion Criteria :

- none

Primary outcomes:
measure: Overall Survival in R/R and MRD+ cohorts
timeFrame: 6 months
Secondary outcomes:
measure: Response rates
timeFrame: 6 months
measure: Adverse events
timeFrame: 6 months
measure: Disease free survival
timeFrame: 6 months
Other outcomes:
Locations:
city: Paris
country: France
facility: CABANNES-HAMY Aurélie
Study officials:
affiliation: 142499643
name: Nicolas BOISSEL, MD
role: PRINCIPAL_INVESTIGATOR
References:
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