Clinical trial NCT04777916: Prospective Non-interventional Study of Adult Patients With Acute Myeloid Leukemia (AML)
Official title: A Prospective Non-interventional Study Documenting the Management and Outcomes of Adult Patients With Acute Myeloid Leukemia (AML)
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Acute Myeloid Leukemia (AML)
Lead sponsor: Acute Leukemia French Association
Sponsor class: OTHER
First posted: 2021-03-02
Enrollment: 2500
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: PROBABILITY_SAMPLE
Study population: Natural History and assessment of the outcomes of different standard approved treatments in recently diagnosed or relapsed/refractory AML patients

Brief summary: During the last fifteen years, the landscape of AML diagnosis and therapeutical options has markedly evolved. Refined genetic and prognostic characterizations, together with new drug approvals and new allogeneic hematopoietic stem cell transplantation (HSCT) procedures, have increased patient journey diversity.

Detailed description: During the last fifteen years, the landscape of AML diagnosis and therapeutical options has markedly evolved. Refined genetic and prognostic characterizations, together with new drug approvals and new allogeneic hematopoietic stem cell transplantation (HSCT) procedures, have increased patient journey diversity.

I - At initial AML diagnosis, not all newly diagnosed patients are entering clinical trials. A substantial proportion of them are treated with standard therapies outside of any trial. To date, the standard approved frontline treatment options include:

1. Standard intensive 3+7 (anthracycline + cytarabine) chemotherapy ± an approved FLT3 inhibitor (midostaurine, Rydapt®), according to different dose schedules in older versus younger patients
2. Combination of sequential gemtuzumab ozogamicin (GO, Mylotarg®) with 3+7
3. Liposomal formulation of daunorubicin + cytarabine (CPX-351, Vyxeos®)
4. Less intensive chemotherapy with azacytidine or low dose cytarabine (LDAC) in patients considered as not eligible for the more intensive options above

The investigator's choice is guided by AML and patient's characteristics, and by the approved indications for each of these treatment options. This study will thus start including these specific options. Further study amendments might be necessary in case of new standard treatment definition.

II - Secondly, no specific salvage regimen has emerged as a standard in patients with primary refractory or relapsed AML (R/R AML). R/R AML is thus an important field for investigational new drugs (INDs) and precision medicine development. To date, the only IND approved to treat R/R AML is gilteritinib for FLT3-mutated AML patients. The French agency ANSM also allow to use GO for treating R/R AML patients in the frame of a RTU (Recommendation Temporaire d'Utilisation).

In the "real life", because of the multiplicity of treatments used in these patients, some of them being now quite efficient, it has become difficult to accurately describe the general outcome of R/R AML patients.

III - Thirdly, allogeneic HSCT is no more considered at the ultimate and final goal of AML therapy in all patients, as it was in the past. Transplant indications have been better described and HSCT in now evaluated in the context of the whole treatment course, including pre- and post-transplant therapy, as well as pre- and post-transplant minimal residual disease (MRD) levels.

For all these reasons, it is of utmost importance to document the various characteristics, treatments and outcomes of patients treated in the real-life, outside of clinical trials, for 1) real-world treatment evaluation; 2) post-approval use of recently approved drugs; 3) standardization and improvement of routine patient management; and 4) better disease understanding.

Eligibility criteria: Inclusion Criteria:

* Patient aged 18 years old or more
* Patient with newly diagnosed previously untreated de novo, secondary or therapy-related AML
* Patients with R/R de novo, secondary or therapy-related AML
* Patient with Health insurance

Exclusion Criteria:

* Acute promyelocytic leukemia
* AML which is not morphologically proven (patients with granulocytic sarcoma may be included)
* For newly diagnosed AML: previous treatment of leukemia apart from hydroxyurea. Previous anti leukemia treatments are allowed if they were administered before the diagnosis of AML to treat a MDS, MPN, MPN/MDS or CML
* Opposition of the patient to participate to this non-interventional study

More specific eligibility criteria might be requested to enter some study modules

Primary outcomes:
measure: OS
timeFrame: 1 year
measure: OS
timeFrame: 3 years
measure: OS
timeFrame: 5 years
measure: OS
timeFrame: 10 years
measure: EFS
timeFrame: 1 year
measure: EFS
timeFrame: 3 years
measure: EFS
timeFrame: 5 years
measure: EFS
timeFrame: 10 years
Secondary outcomes:
Other outcomes:
Locations:
city: Amiens
country: France
facility: Chu Amiens
city: Argenteuil
country: France
facility: Centre Hospitalier Victor Dupouy
city: Bobigny
country: France
facility: AP-HP-GHU - Hôpital AVICENNE
city: Caen
country: France
facility: CHU de la cote de Nacre
city: Clamart
country: France
facility: Hôpital MILITAIRE PERCY
city: Corbeil-Essonnes
country: France
facility: Centre hospitalier Sud Francilien
city: Créteil
country: France
facility: Hôpital Henri Mondor AP-HP
city: Dijon
country: France
facility: CHU Dijon- François Mitterrand
city: Dunkerque
country: France
facility: Centre Hospitalier de Dunkerque
city: Le Chesnay
country: France
facility: Centre Hospitalier de Versailles André Mignot
city: Lens
country: France
facility: Centre Hospitalier Dr Schaffner
city: Lille
country: France
facility: CHRU de Lille- Hopital C. HURIEZ
city: Lille
country: France
facility: GHICL-Hopital St Vincent de Paul
city: Limoges
country: France
facility: C H U DE LIMOGES- Hopital Dupuytren
city: Marseille
country: France
facility: CHU La Conception
city: Meaux
country: France
facility: Centre Hopsitalier de l'Est Francilien - Site de Meaux
city: Nice
country: France
facility: Centre Antoine Lacassagne
city: Nice
country: France
facility: CHU Nice,Hopital Archet 1
city: Paris
country: France
facility: Hopital Pitié-Salpétrière APHP
city: Paris
country: France
facility: Hôpital Necker - APHP
city: Paris
country: France
facility: Hôpital SAINT ANTOINE-APHP
city: Paris
country: France
facility: Hôpital Saint Louis- APHP
city: Pierre-Bénite
country: France
facility: Centre Hospitalier Lyon Sud
city: Pontoise
country: France
facility: Centre Hospitalier René Dubos
city: Roubaix
country: France
facility: Centre Hospitalier de Roubaix
city: Rouen
country: France
facility: Centre Henri Becquerel
city: Saint-Cloud
country: France
facility: Institut Curie - Hôpital René HUGUENIN
city: Saint-Quentin
country: France
facility: Centre Hospitalier de St Quentin
city: Valenciennes
country: France
facility: Centre Hospitalier Valenciennes
city: Villejuif
country: France
facility: Institut Gustave Roussy
Study officials:
affiliation: Acute Leukemia French Association
name: Hervé DOMBRET, PD-Prof
role: STUDY_CHAIR
References: