Clinical trial NCT03439371: Micro-transplantation in Elderly Patients With Acute Myeloid Leukemia
Official title: A Phase II, Multicenter, Open Label Study Evaluating the Efficacy and the Tolerance of Micro-transplantation in Elderly Patients With Acute Myeloid Leukemia (MTSA)
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: NA
Conditions: Acute Myeloid Leukemia
Keywords: Acute Myeloid Leukemia
Micro-transplantation
Elderly
Graft-versus-leukemia
Lead sponsor: Centre Hospitalier Universitaire de Saint Etienne
Sponsor class: OTHER
Collaborators: name: Institut de Cancérologie de la Loire
First posted: 2018-02-20
Enrollment: 21
Sex: ALL
Minimum age (years): 60
Maximum age (years): 75
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: This study aims at evaluating the safety and the tolerance of the micro-transplantation in elderly patients with acute myeloid leukemia who are ineligible to conventional allogeneic transplantation.

Detailed description: Acute Myeloid Leukemia (AML) is an aggressive hematological malignancy with a median age at diagnosis of 65 years. Outcomes of AML in elderly population remain unsatisfactory with low rates of complete remission, poor disease-free and overall survival. Therapeutic management of older patients with AML deals with patient-related features (i.e. comorbid conditions and performance status) as well as disease-related prognostic factors (i.e. cytogenetics and molecular genetics). Even if allogeneic hematopoietic-cell transplantation provides the strongest antineoplasic effect, this treatment option remains limited for older patients owing to toxicities, the development of significant graft-versus-host disease (GVHD) and logistics of donor availability. More recently, micro-transplantation has emerged as an alternative strategy based on the infusion of mobilized HLA-mismatched related donor cells after induction chemotherapy, thus exerting a graft-versus-leukemia effect without substantial donor engraftment and GVHD. Therefore, there is much of interest in investigating the efficacy and the safety of this method for older patients with AML who are not candidates for allogeneic stem cell transplantation.

Eligibility criteria: Inclusion Criteria:

* Patient affiliated to a social security regimen or beneficiary of the same
* Signed written informed consent form
* Patient, ≥ 60 years-old - \< 75 years-old, with established diagnosis of de novo or secondary AML with intermediate-risk or adverse-risk cytogenetic profile, or with established myelodysplasic syndromes (RAEB), in pathologically confirmed complete remission following anti-leukemic induction therapy (\<5% blasts)
* Contra-indication to conditioning regimen in conventional allogeneic transplantation

Exclusion Criteria:

* Patient with established diagnosis of acute myeloid leukemia with standard-risk cytogenetic profile
* Promyelocytic leukemia t(15;17)
* CBF-AML t(8;21) or inv(16)
* Normal karyotype with a favorable molecular profile: NPM1+ and FLT3-; NPM1+, FLT3- and double mutation CEBPα or chronic myeloid leukemia in blastic phase
* Patient under guardianship or deprived of his liberty or any condition that may affect the patient's ability to understand and sign the informed consent
* Refusing participation

Primary outcomes:
measure: Rate of overall survival
timeFrame: 2 years
Secondary outcomes:
measure: Hematopoietic recovery
timeFrame: 3 months
measure: Hematopoietic recovery
timeFrame: 3 months
measure: Hematopoietic recovery
timeFrame: 3 months
measure: Rate of complete remission
timeFrame: 2 years
measure: GVHD (graft versus host disease)
timeFrame: 2 years
measure: Median overall survival
timeFrame: 2 years
measure: Median progression-free survival
timeFrame: 2 years
measure: Microchimerism
timeFrame: 3 months
Other outcomes:
Locations:
city: Clermont-Ferrand
country: France
facility: CHU Estaing
city: Grenoble
country: France
facility: Centre Hospitalier Universitaire de Grenoble
city: Lille
country: France
facility: CHRU de Lille
city: Lyon
country: France
facility: Centre Hospitalier Lyon Sud
city: Nancy
country: France
facility: Centre Hospitalier Universitaire de Nancy
city: Paris
country: France
facility: Hôpital de la Pitié-Salpêtrière
city: Saint-Étienne
country: France
facility: CHU de Saint-Etienne
Study officials:
affiliation: CHU de Saint-Etienne
name: Jérôme Cornillon, MD
role: PRINCIPAL_INVESTIGATOR
References: