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

January 30, 2024 updated by: Acute Leukemia French Association

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

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.

Study Overview

Status

Recruiting

Conditions

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.

Study Type

Observational

Enrollment (Estimated)

2500

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact Backup

Study Locations

  • France
      • Amiens, France
        • Recruiting
        • CHU Amiens
        • Contact:
          • Delphine LEBON, MD
      • Argenteuil, France
        • Recruiting
        • Centre Hospitalier Victor Dupouy
        • Contact:
          • Ahmad AL JIJAKLI, MD
      • Bobigny, France
        • Recruiting
        • AP-HP-GHU - Hôpital AVICENNE
        • Contact:
          • Thorsten BRAUN, MD-Prof
      • Caen, France
        • Recruiting
        • CHU de la cote de Nacre
        • Contact:
          • Sylvain CHANTEPIE, MD
      • Clamart, France
        • Recruiting
        • Hôpital MILITAIRE PERCY
        • Contact:
          • Jean-Valère MALFUSON, MD-Prof
      • Corbeil-Essonnes, France
        • Recruiting
        • Centre Hospitalier Sud Francilien
        • Contact:
          • Stéphanie HAIAT, MD
      • Créteil, France
        • Recruiting
        • Hôpital Henri Mondor AP-HP
        • Contact:
          • Cécile PAUTAS, MD
      • Dijon, France, 21000
        • Active, not recruiting
        • CHU Dijon- François Mitterrand
      • Dunkerque, France
        • Recruiting
        • Centre Hospitalier de Dunkerque
        • Contact:
          • Kevin James WATTEBLED, MD
      • Le Chesnay, France
        • Recruiting
        • Centre Hospitalier de Versailles André Mignot
        • Contact:
          • Juliette LAMBERT, MD
      • Lens, France
        • Recruiting
        • Centre Hospitalier Dr Schaffner
        • Contact:
          • Claire BORIES, MD
      • Lille, France
        • Recruiting
        • CHRU de Lille- Hopital C. HURIEZ
        • Contact:
          • Céline BERTHON, MD
      • Lille, France
        • Recruiting
        • GHICL-Hopital St Vincent de Paul
        • Contact:
          • Benjamin CARPENTIER, MD
      • Limoges, France
        • Recruiting
        • C H U DE LIMOGES- Hopital Dupuytren
        • Contact:
          • Pascal TURLURE, MD
      • Marseille, France
        • Recruiting
        • CHU la Conception
        • Contact:
          • Laure FARNAULT, MD
      • Meaux, France
        • Recruiting
        • Centre Hopsitalier de l'Est Francilien - Site de Meaux
        • Contact:
          • Jamilé FRAYFER, MD
      • Nice, France
        • Recruiting
        • Centre Antoine Lacassagne
        • Contact:
          • Anna BORRA, MD
      • Nice, France
        • Recruiting
        • CHU Nice,Hopital Archet 1
        • Contact:
          • Thomas CLUZEAU, MD-Prof
      • Paris, France
        • Recruiting
        • Hopital Pitie-Salpetriere APHP
        • Contact:
          • Madalina UZUNOV, MD
      • Paris, France
        • Recruiting
        • Hôpital Necker - APHP
        • Contact:
          • Ambroise MARCAIS, MD
      • Paris, France
        • Recruiting
        • Hôpital Saint Louis- APHP
        • Contact:
          • Raphaël ITZYKSON, MD-Prof
      • Paris, France
        • Recruiting
        • Hôpital SAINT ANTOINE-APHP
        • Contact:
          • Ollivier LEGRAND, MD-Prof
      • Pierre-Bénite, France
        • Recruiting
        • Centre Hospitalier Lyon Sud
        • Contact:
          • Maël HEIBLIG, MD
      • Pontoise, France
        • Recruiting
        • Centre Hospitalier Rene Dubos
        • Contact:
          • Iona VAIDA, MD
      • Roubaix, France
        • Recruiting
        • Centre Hospitalier de Roubaix
        • Contact:
          • Isabelle PLANTIER, MD
      • Rouen, France
        • Recruiting
        • Centre Henri Becquerel
        • Contact:
          • Emilie LEMASLE, MD
      • Saint-Cloud, France
        • Not yet recruiting
        • Institut Curie - Hôpital René Huguenin
        • Contact:
          • Jacques VARGAFTIG, MD
      • Saint-Quentin, France
        • Recruiting
        • Centre Hospitalier de St Quentin
        • Contact:
          • Reda GARIDI, MD
      • Valenciennes, France
        • Not yet recruiting
        • Centre Hospitalier Valenciennes
        • Contact:
          • Sabine TRICOT, MD
      • Villejuif, France
        • Recruiting
        • Institut Gustave Roussy
        • Contact:
          • Jean-Baptiste MICOL, MD

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

N/A

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

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Observational Models: Cohort
  • Time Perspectives: Prospective

Cohorts and Interventions

Group / Cohort
Standard intensive 3+7 YOUNG OR ELDERLY
Standard intensive 3+7 (anthracycline + cytarabine) chemotherapy ± an approved FLT3 inhibitor (midostaurine, Rydapt®), according to different dose schedules in older versus younger patients
GO, Mylotarg®) with 3+7
Combination of sequential gemtuzumab ozogamicin (GO, Mylotarg®) with 3+7
CPX-351, Vyxeos®)
Liposomal formulation of daunorubicin + cytarabine (CPX-351, Vyxeos®)
Lower intensity chemotherapy with azacytidine or low dose cytarabine (LDAC)
Lower intensity chemotherapy with azacytidine or low dose cytarabine (LDAC) in patients considered as not eligible for the more intensive options above
Refractory or relapsed AML

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.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
OS
Time Frame: 1 year

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
OS
Time Frame: 3 years

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
OS
Time Frame: 5 years

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
OS
Time Frame: 10 years

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
EFS
Time Frame: 1 year

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
EFS
Time Frame: 3 years

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
EFS
Time Frame: 5 years

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
EFS
Time Frame: 10 years

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

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Acute Leukemia French Association

Investigators

  • Study Chair: Hervé DOMBRET, PD-Prof, Acute Leukemia French Association

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

April 14, 2022

Primary Completion (Estimated)

April 1, 2032

Study Completion (Estimated)

April 1, 2046

Study Registration Dates

First Submitted

February 25, 2021

First Submitted That Met QC Criteria

March 1, 2021

First Posted (Actual)

March 2, 2021

Study Record Updates

Last Update Posted (Estimated)

January 31, 2024

Last Update Submitted That Met QC Criteria

January 30, 2024

Last Verified

February 1, 2023

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • ALFA PPP Study

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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