ChEmo-Genomics Based Treatment of Acute Myeloid Leukemia

Study Overview
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Brief Summary
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Adult acute myeloid leukemia (AML) is a heterogeneous hematologic malignancy associated with poor prognosis, especially after relapse. High-throughput genomic studies have highlighted the importance of molecular alteration in the pathophysiology, clinical evolution and treatment response of AML. In addition, identification of specific gene mutation can be targeted by specific inhibitors, opening the way to personalized treatments. However, only a limited number of gene mutations are druggable or actionable, highlighting the need for additional information to guide treatment choices. Among them, new Drug Screening Tests (DST) allow for the screening of library of hundreds of drugs to ex-vivo patient-derived AML cells. Combination of genomic and pharmacologic approaches might therefore improve prediction of drug effects. There is an urgent need to bring these approaches into the clinic but feasibility trials are necessary before incorporating them into treatments strategies.The proposed study is a prospective multicentre feasibility study of a combined chemo-genomic approach in patients with advanced AML.

Official Title
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ChEmo-Genomics Based Treatment of Acute Myeloid Leukemia- CeGAL-IPC 2014-012

Conditions
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Leukemia, Myeloid, Acute

Intervention / Treatment
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* Procedure: Tumor sampling
* Biological: Constiutional DNA sampling


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Age >18 years Diagnosis of acute myeloid leukemia according to WHO classification Refractory or relapsed disease ECOG performance status of <3 Life expectancy >3 months Written informed consent Affiliation to the French Social Security System. Exclusion Criteria: Diagnosis of Acute Promyelocytic Leukemia. Patients deprived of liberty or placed under the authority of a tutor.

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Refractory or relapsed acute myeloid leukemia<br> | Procedure: Tumor sampling<br>* Bone marrow aspirate, blood sampling<br>Biological: Constiutional DNA sampling<br>* Buccal swab or Hair follicles<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| The proportion of patients for whom a treatment tailored according to chemogenomic data could be proposed to the investigator within a 21 days time-frame in at least 30% of cases. |  | 24 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Correlations between genomic alterations (identified by mutatome and transcriptome analyses) and drug sensitivity profiles |  | 24 months | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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acute myeloid leukemia- refractory AML- relapsed AML- precision medecine-