Prospective Non-interventional Study of Adult Patients With Acute Myeloid Leukemia (AML)

Acute Myeloid Leukemia (AML) Clinical Trial

— ALFAPPP  

Official title:

A Prospective Non-interventional Study Documenting the Management and Outcomes of Adult Patients With Acute Myeloid Leukemia (AML)

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT04777916
• Other study ID #: ALFA PPP Study
• Status: Recruiting
• Start date: April 14, 2022
• Est. completion date: April 1, 2046

Study information

• Verified date: February 2023
• Source: Acute Leukemia French Association
• Contact: Karine CELLI LEBRAS, Mrs
• Phone: 33 1 57 27 67 17
• Email: karine.celli-lebras[@]aphp.fr
• Is FDA regulated: No
• Study type: Observational [Patient Registry]

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 2500
• Est. completion date: April 1, 2046
• Est. primary completion date: April 1, 2032
• Gender: All
• Age group: 18 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Acute Myeloid Leukemia (AML)
• Leukemia
• Leukemia, Myeloid
• Leukemia, Myeloid, Acute

Locations

Country	Name	City	State
France	Chu Amiens	Amiens
France	Centre Hospitalier Victor Dupouy	Argenteuil
France	AP-HP-GHU - Hôpital AVICENNE	Bobigny
France	CHU de la cote de Nacre	Caen
France	Hôpital MILITAIRE PERCY	Clamart
France	Centre hospitalier Sud Francilien	Corbeil-Essonnes
France	Hôpital Henri Mondor AP-HP	Créteil
France	CHU Dijon- François Mitterrand	Dijon
France	Centre Hospitalier de Dunkerque	Dunkerque
France	Centre Hospitalier de Versailles André Mignot	Le Chesnay
France	Centre Hospitalier Dr Schaffner	Lens
France	CHRU de Lille- Hopital C. HURIEZ	Lille
France	GHICL-Hopital St Vincent de Paul	Lille
France	C H U DE LIMOGES- Hopital Dupuytren	Limoges
France	CHU La Conception	Marseille
France	Centre Hopsitalier de l'Est Francilien - Site de Meaux	Meaux
France	Centre Antoine Lacassagne	Nice
France	CHU Nice,Hopital Archet 1	Nice
France	Hôpital Necker - APHP	Paris
France	Hopital Pitié-Salpétrière APHP	Paris
France	Hôpital SAINT ANTOINE-APHP	Paris
France	Hôpital Saint Louis- APHP	Paris
France	Centre Hospitalier Lyon Sud	Pierre-Bénite
France	Centre Hospitalier René Dubos	Pontoise
France	Centre Hospitalier de Roubaix	Roubaix
France	Centre Henri Becquerel	Rouen
France	Institut Curie - Hôpital René HUGUENIN	Saint-Cloud
France	Centre Hospitalier de St Quentin	Saint-Quentin
France	Centre Hospitalier Valenciennes	Valenciennes
France	Institut Gustave Roussy	Villejuif

Sponsors (1)

Lead Sponsor	Collaborator
Acute Leukemia French Association

Country where clinical trial is conducted

France, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	OS	The primary objective of this multicenter non-interventional study is to record and prospectively evaluate the real-life characteristics, treatments and outcomes of adult patients with newly diagnosed or R/R AML, when managed and treated in the French ALFA centers according to standard practices outside of a clinical trial.; The two co-primary endpoints are event-free (EFS) and overall survival (OS) estimations at 1, 3, 5 and 10 years: From first treatment initiation in patients with newly diagnosed AML; From the date of relapse/refractoriness (R/R) in patients, with R/R AML	1 year
Primary	OS	The primary objective of this multicenter non-interventional study is to record and prospectively evaluate the real-life characteristics, treatments and outcomes of adult patients with newly diagnosed or R/R AML, when managed and treated in the French ALFA centers according to standard practices outside of a clinical trial.; The two co-primary endpoints are event-free (EFS) and overall survival (OS) estimations at 1, 3, 5 and 10 years: From first treatment initiation in patients with newly diagnosed AML; From the date of relapse/refractoriness (R/R) in patients, with R/R AML	3 years
Primary	OS	The primary objective of this multicenter non-interventional study is to record and prospectively evaluate the real-life characteristics, treatments and outcomes of adult patients with newly diagnosed or R/R AML, when managed and treated in the French ALFA centers according to standard practices outside of a clinical trial.; The two co-primary endpoints are event-free (EFS) and overall survival (OS) estimations at 1, 3, 5 and 10 years: From first treatment initiation in patients with newly diagnosed AML; From the date of relapse/refractoriness (R/R) in patients, with R/R AML	5 years
Primary	OS	The primary objective of this multicenter non-interventional study is to record and prospectively evaluate the real-life characteristics, treatments and outcomes of adult patients with newly diagnosed or R/R AML, when managed and treated in the French ALFA centers according to standard practices outside of a clinical trial.; The two co-primary endpoints are event-free (EFS) and overall survival (OS) estimations at 1, 3, 5 and 10 years: From first treatment initiation in patients with newly diagnosed AML; From the date of relapse/refractoriness (R/R) in patients, with R/R AML	10 years
Primary	EFS	The primary objective of this multicenter non-interventional study is to record and prospectively evaluate the real-life characteristics, treatments and outcomes of adult patients with newly diagnosed or R/R AML, when managed and treated in the French ALFA centers according to standard practices outside of a clinical trial.; The two co-primary endpoints are event-free (EFS) and overall survival (OS) estimations at 1, 3, 5 and 10 years: From first treatment initiation in patients with newly diagnosed AML; From the date of relapse/refractoriness (R/R) in patients, with R/R AML	1 year
Primary	EFS	The primary objective of this multicenter non-interventional study is to record and prospectively evaluate the real-life characteristics, treatments and outcomes of adult patients with newly diagnosed or R/R AML, when managed and treated in the French ALFA centers according to standard practices outside of a clinical trial.; The two co-primary endpoints are event-free (EFS) and overall survival (OS) estimations at 1, 3, 5 and 10 years: From first treatment initiation in patients with newly diagnosed AML; From the date of relapse/refractoriness (R/R) in patients, with R/R AML	3 years
Primary	EFS	The primary objective of this multicenter non-interventional study is to record and prospectively evaluate the real-life characteristics, treatments and outcomes of adult patients with newly diagnosed or R/R AML, when managed and treated in the French ALFA centers according to standard practices outside of a clinical trial.; The two co-primary endpoints are event-free (EFS) and overall survival (OS) estimations at 1, 3, 5 and 10 years: From first treatment initiation in patients with newly diagnosed AML; From the date of relapse/refractoriness (R/R) in patients, with R/R AML	5 years
Primary	EFS	The primary objective of this multicenter non-interventional study is to record and prospectively evaluate the real-life characteristics, treatments and outcomes of adult patients with newly diagnosed or R/R AML, when managed and treated in the French ALFA centers according to standard practices outside of a clinical trial.; The two co-primary endpoints are event-free (EFS) and overall survival (OS) estimations at 1, 3, 5 and 10 years: From first treatment initiation in patients with newly diagnosed AML; From the date of relapse/refractoriness (R/R) in patients, with R/R AML	10 years