AzTrials
AzTrials

Acute Myeloid Leukemia (AML)ALFAPPPNCT04777916

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

An observational study of Acute Myeloid Leukemia (AML), run by Acute Leukemia French Association at 30 sites in France.

Recruiting

Looking for participants now. Contact the study team or a site below to ask about taking part.

Sites and contacts
Official record
Search all trials

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. Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Acute Myeloid Leukemia (AML)
How many people
5,000 participants
Where
30 sitesFrance
Study dates
14 Apr 2022to 1 Apr 2046
How long will I take part?
15 Years
Phase
Observational
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 years and older
  • All sexes
  • Adult, Older adult

Study population: Natural History and assessment of the outcomes of different standard approved treatments in recently diagnosed or relapsed/refractory AML patients

Who may be able to join

  • 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

Who cannot join

  • 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
  • Patient weighting less than 50 kgs
  • Opposition of the patient to participate to this non-interventional study
  • More specific eligibility criteria might be requested to enter some study modules

Trial locations and contacts

30 sites in 1 country. Contact the study team or a site near you to ask about taking part.

Karine CELLI LEBRAS, Mrs contact

33 1 57 27 67 17

karine.cellilebras@alfa-leukemia.org

Hervé DOMBRET, MD-Prof contact

33 1 57 27 67 17

herve.dombret@mac.com

30 sites in 1 country

France30 sites, 29 recruiting

  • Chu Amiens

    Amiens

    Delphine LEBON, MD

    Recruiting

  • Centre Hospitalier Victor Dupouy

    Argenteuil

    Ahmad AL JIJAKLI, MD

    Recruiting

  • AP-HP-GHU - Hôpital AVICENNE

    Bobigny

    Thorsten BRAUN, MD-Prof

    Recruiting

  • CHU de la cote de Nacre

    Caen

    Sylvain CHANTEPIE, MD

    Recruiting

  • Hôpital MILITAIRE PERCY

    Clamart

    Jean-Valère MALFUSON, MD-Prof

    Recruiting

  • Centre hospitalier Sud Francilien

    Corbeil-Essonnes

    Stéphanie HAIAT, MD

    Recruiting

  • Hôpital Henri Mondor AP-HP

    Créteil

    Mathieu LECLERC, MD

    Recruiting

  • CHU Dijon- François Mitterrand

    Dijon, 21000

    Active, not recruiting

  • Centre Hospitalier de Dunkerque

    Dunkirk

    Adrien DANIEL, MD

    Recruiting

  • Centre Hospitalier de Versailles André Mignot

    Le Chesnay

    Juliette LAMBERT, MD

    Recruiting

  • Centre Hospitalier Dr Schaffner

    Lens

    Claire BORIES, MD

    Recruiting

  • CHRU de Lille- Hopital C. HURIEZ

    Lille

    Céline BERTHON, MD

    Recruiting

  • GHICL-Hopital St Vincent de Paul

    Lille

    Benjamin CARPENTIER, MD

    Recruiting

  • C H U DE LIMOGES- Hopital Dupuytren

    Limoges

    Pascal TURLURE, MD

    Recruiting

  • CHU La Conception

    Marseille

    Laure FARNAULT, MD

    Recruiting

  • Centre Hopsitalier de l'Est Francilien - Site de Meaux

    Meaux

    Jamilé FRAYFER, MD

    Recruiting

  • Centre Antoine Lacassagne

    Nice

    Luca INCHIAPPA, MD

    Recruiting

  • CHU Nice,Hopital Archet 1

    Nice

    Thomas CLUZEAU, MD-Prof

    Recruiting

  • Hopital Pitié-Salpétrière APHP

    Paris

    Madalina UZUNOV, MD

    Recruiting

  • Hôpital Necker - APHP

    Paris

    Ambroise MARCAIS, MD

    Recruiting

  • Hôpital SAINT ANTOINE-APHP

    Paris

    Ollivier LEGRAND, MD-Prof

    Recruiting

  • Hôpital Saint Louis- APHP

    Paris

    Raphaël ITZYKSON, MD-Prof

    Recruiting

  • Centre Hospitalier Lyon Sud

    Pierre-Bénite

    Maël HEIBLIG, MD

    Recruiting

  • Centre Hospitalier René Dubos

    Pontoise

    Iona VAIDA, MD

    Recruiting

  • Centre Hospitalier de Roubaix

    Roubaix

    Isabelle PLANTIER, MD

    Recruiting

  • Centre Henri Becquerel

    Rouen

    Emilie LEMASLE, MD

    Recruiting

  • Institut Curie - Hôpital René HUGUENIN

    Saint-Cloud

    Jacques VARGAFTIG, MD

    Not yet recruiting

  • Centre Hospitalier de St Quentin

    Saint-Quentin

    Reda GARIDI, MD

    Recruiting

  • Centre Hospitalier Valenciennes

    Valenciennes

    Sabine TRICOT, MD

    Not yet recruiting

  • Institut Gustave Roussy

    Villejuif

    Jean-Baptiste MICOL, MD

    Recruiting

Other trials in France

About this study

Official title: 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.

Read the 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®), or ± quizartinib (Vanflyta®) 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.

Medical subject headings
Leukemia, Myeloid, Acute

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

Study groups in NCT04777916
Study groupRoleWhat they receive
Standard intensive 3+7 YOUNG OR ELDERLYStandard intensive 3+7 (anthracycline + cytarabine) chemotherapy ± an approved FLT3 inhibitor (midostaurine, Rydapt® or ± quizartinib, Vanflyta®), according to different dose schedules in older versus younger patients
GO, (Mylotarg®) with 3+7Combination of sequential gemtuzumab ozogamicin (GO, Mylotarg®) with 3+7
CPX-351, (Vyxeos®)Liposomal formulation of daunorubicin + cytarabine (CPX-351, Vyxeos®)
Less intensive chemotherapy with azacytidine combined or not with venetoclax; LDAC; ivosidenibLess intensive chemotherapy with azacytidine either combined or not with venetoclax or low dose cytarabine (LDAC) or in AML bearing an IDH1 somatic mutation, with ivosidenib in newly diagnosed patients considered as not eligible for the more intensive options above
Refractory or relapsed AMLSecondly, 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 being measured

The primary outcome is the main question the study is designed to answer.

  1. 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

    Measured: 1 year

  2. 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

    Measured: 3 years

  3. 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

    Measured: 5 years

  4. 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

    Measured: 10 years

  5. 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

    Measured: 1 year

  6. 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

    Measured: 3 years

  7. 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

    Measured: 5 years

  8. 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

    Measured: 10 years

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted2 Mar 2021
  2. Study start14 Apr 2022
  3. Primary completion1 Apr 2032 (estimated)
  4. Study completion1 Apr 2046 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Observational
Observational model
Cohort
Time perspective
Prospective
Enrolment
5,000 participants (estimated)
Target duration
15 Years
IDs, dates and publications
ClinicalTrials.gov ID
NCT04777916
Sponsor's protocol ID
ALFA PPP Study
Acronym
ALFAPPP
Status last verified
Sep 2025
First submitted
25 Feb 2021
Last updated
3 Oct 2025
Results posted
No
Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No

Questions about this trial

What is this clinical trial studying?

An observational study of Acute Myeloid Leukemia (AML), run by Acute Leukemia French Association at 30 sites in France.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 4 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: looking for participants now, as of the sponsor's last update on 3 Oct 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 30 sites in France. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

Contact Karine CELLI LEBRAS, Mrs (33 1 57 27 67 17, karine.cellilebras@alfa-leukemia.org), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

OS (time frame: 1 year). The study lists 8 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT04777916.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 3 Oct 2025; AzTrials last refreshed its copy on 8 Nov 2026.

AzTrials uses cookies for analytics and advertising. You can accept or reject them; the site works either way. Privacy Policy.

Free account

Log in to keep searching

You’ve used your 3 free searches. AzTrials is free: log in or create a free account to keep searching clinical trials and save the ones you want to follow.

Create a free accountLog in