Acute Myeloid LeukemiaALLO-BESTNCT04822766
A Study Comparing Allogeneic Hematopoietic Cell Transplantation Versus Best Available Standard of Care Therapy in Elderly Patients With Acute Myeloid Leukemia
A study testing Hematopoietic stem cell transplantation and Best chemotherapy treatment in people with Acute Myeloid Leukemia, run by Assistance Publique - Hôpitaux de Paris at 1 site in France.
Trial summary
A subject of major interest for researchers, clinicians, patients, and payers, is the role of allogeneic hematopoietic stem cell transplantation (allo-HSCT) in the treatment of these older patients with AML. Read more about this study
- Who is it for?
- Ages 65 to 75 years
- Conditions the study is for
- Acute Myeloid Leukemia
- What is being tested?
- Hematopoietic stem cell transplantation and Best chemotherapy treatment
- Type of treatment
- Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 24 participants
- Where
- 1 siteFrance
- Study dates
- 31 Dec 2021to Jun 2027
- Phase
- Interventional
- 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 65 to 75 years
- All sexes
- No healthy volunteers
- Older adult
Who may be able to join
- Men and women
- Age ≥ 65 and ≤ 75 years
- Newly diagnosed patients with de novo or secondary AML in first complete remission who are considered as potential candidates and eligible for an allo-HSCT procedure
- Presence of a donor (matched related or unrelated or haplo-mismatched) willing to donate peripheral blood stem cells
- Patient is fit for the allo-HSCT procedure
- Patient is fit for further consolidation therapy (non-transplant arm)
- Written informed consent
Who cannot join
- Acute promyelocytic leukemia (AML FAB M3)
- AML deemed not eligible for allo-HSCT
- Karnofsky score <70%
- HIV positive patient
- Life expectancy less than one month according to the attending physician
- Acute or chronic heart failure (Cardiac ejection fraction < 40%)
Show all 12Show fewer
- Pulmonary function - diffusion capacity < 50% predicted
- Estimated glomerular filtration rate < 50 ml/min (CKD-EPI)
- Severe neurological disorders
- Patient subject to a legal protection measure (guardianship, curatorship and safeguard of justice) or unable to consent
- Patient deprived of their liberty by a judicial or administrative decision
- Patient with severe psychiatric disorders or hospitalized without consent for psychiatric care
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Allogeneic Hematopoietic Cell Transplantation Versus Best Available Standard of Care Therapy in Elderly Patients With Acute Myeloid Leukemia: a Randomized Phase 3 Trial
A subject of major interest for researchers, clinicians, patients, and payers, is the role of allogeneic hematopoietic stem cell transplantation (allo-HSCT) in the treatment of these older patients with AML. With conventional induction chemotherapy or hypomethylating agents, the expected 2-year overall survival (OS) is less than 25% in patients with intermediate- or high-risk disease. The 2-year OS ranges from 50 to 56% with allo-HSCT in AML patients older than 65 years.
Performing an allo-HSCT in older patients is however still controversial because of the higher risk of non-relapse mortality (15 to 35%) and graft-versus-host disease. Depending on the center policy, patients older than 65 years will either be contraindicated for transplant or will receive allo-HSCT.
With a phase III comparative, randomized, controlled, prospective, multicenter study, the trial aim to assess prospectively the outcomes and quality of life of older patients with AML receiving allo-HSCT strategy compared to those receiving a non-transplant approach.
Read the detailed description
Every year, 30,000 patients in Europe and 20,000 in the USA are diagnosed with acute myeloid leukemia (AML). More than half of them are over 65 years old. In this older population, the median overall survival (OS) is only 2 to 8 months. With conventional induction chemotherapy or hypomethylating agents, the expected 2-year OS is less than 25% in patients with intermediate- or high-risk disease.
Performing an allo-HSCT in older patients is however still controversial because of the higher risk of non-relapse mortality (15 to 35%) and graft-versus-host disease. Depending on the center policy, patients older than 65 years will either be contraindicated for transplant or will receive allo-HSCT. Noteworthy, no prospective randomized trial has yet compared allo-HSCT to a non-transplant strategy in older patients with AML. A previous attempt made 10 years ago, by the EBMT to run a slightly similar trial, has failed in France and most European countries, mainly (i) because it mandated the type of transplant procedure to be applied and (ii) because of the absence of novel and effective drugs.
Every year, 30,000 patients in Europe and 20,000 in the USA are diagnosed with acute myeloid leukemia (AML). More than half of them are over 65 years old. In this older population, the median overall survival (OS) is only 2 to 8 months. With conventional induction chemotherapy or hypometylating agents, the expected 2-year OS is less than 25% in patients with intermediate- or high-risk disease.
Performing an allo-HSCT in older patients is however still controversial because of the higher risk of non-relapse mortality (15 to 35%) and graft-versus-host disease. Depending on the center policy, patients older than 65 years will either be contraindicated for transplant or will receive allo-HSCT. Noteworthy, no prospective randomized trial has yet compared allo-HSCT to a non-transplant strategy in older patients with AML. A previous attempt made 10 years ago, by the EBMT to run a slightly similar trial, has failed in France and most European countries, mainly (i) because it mandated the type of transplant procedure to be applied and (ii) because of the absence of novel and effective drugs.
New targeted therapies and treatment strategies are evolving rapidly. A standardized unique treatment administrated to all sub-types of AML is no longer the optimal approach for induction and non-transplant maintenance strategies. No treatment has reached consensus for older patients. For these reasons, this trial will not limit the choices of drugs administered to the patients but compare two strategies allowing patients to receive the best available standard of care.
The trial aim to assess prospectively the outcomes and quality of life of older patients with AML receiving allo-HSCT strategy compared to those receiving a non-transplant approach.
Patients will receive initial treatment with chemotherapy (or other appropriate non-palliative therapy). Once first complete remission is achieved and a donor is identified, patients will be included.
Patients will be randomly assigned (1:1) after inclusion to receive one of the following strategy:
- Allogeneic hematopoietic stem cell transplantation arm: patients will undergo allo-HSCT after consolidation therapy (or completion of other appropriate non-palliative strategy) according to standard procedures of the transplant center (choice of donor, conditioning regimen, GVHD and infection prophylaxis). The use of novel therapies (such as sorafenib, midaustorine, venetoclax, etc.) will be allowed as post-transplantation maintenance strategy
- Non-transplant arm: patients will be treated according to the standard procedures of the treating center for this type of population.
All patients will receive the best available treatments (including additional conventional chemotherapy or other non-palliative therapies such as 5-azacytidine, decitabine, venetoclax, midaustorine, enasidenib, etc.). Supportive care will be performed according to each participating center usual practice.
- Conditions
- Acute Myeloid Leukemia
- Being tested
- Hematopoietic stem cell transplantation, Best chemotherapy treatment
- Keywords
- Allogeneic, hematopoietic cell transplantation, acute myeloid leukemia
- 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.
Hematopoietic stem cell transplantationProcedure or surgery
patients will undergo allo-HSCT after consolidation therapy (or completion of other appropriate non-palliative strategy) according to standard procedures of the transplant center (choice of donor, conditioning regimen, GVHD and infection prophylaxis). The use of novel therapies (such as sorafenib, midaustorine, venetoclax, etc.) will be allowed as post-transplantation maintenance strategy.
Best chemotherapy treatmentDrug
patients will be treated according to the standard procedures of the treating center for this type of population. Patients will receive the best available treatments (including additional conventional chemotherapy or other non-palliative therapies such as 5-azacytidine, decitabine, venetoclax, midaustorine, enasidenib, etc.).
| Study group | Role | What they receive |
|---|---|---|
| Chemotherapy | Active comparator | Not described Receives: Drug: Best chemotherapy treatment |
| Allogeneic Hematopoietic Cell Transplantation | Experimental | Time of transplant procedure The best available treatments of AML Receives: Procedure: Hematopoietic stem cell transplantation |
What is being measured
The primary outcome is the main question the study is designed to answer.
Overall survival
From inclusion (time of identification of potential donor) until death or at 24 months, whichever comes first]
Measured: 2 years after the inclusion
Secondary outcomes (9)
Leukemia free survival
from inclusion (time of identification of potential donor) until relapse and/or death from any cause or at 24 months, whichever comes first
Measured: within the 2 years after inclusion
Assessment of MRD and time to relapse from inclusion up to 2 years
Measured: : time between inclusion and date of relapse or at 24 months, whichever comes first]
Quality of life FACT-BMT
FACT-BMT (Functional Assessment of Cancer Therapy - Bone Marrow Transplant)
Measured: at baseline, 12 and 24 months after inclusion
Quality of life EQ 5D 5L
EQ 5D 5L (EuroQol group)
Measured: at baseline, 12 and 24 months after inclusion
The Incremental cost-effectiveness ratios (ICERs) expressed in cost per quality-adjusted life-year (QALY) gained
from inclusion (time of identification of potential donor) until death from any cause or at 24 months, whichever comes first
Measured: 2 years after inclusion
The Incremental cost-effectiveness ratios (ICERs) expressed in cost per Life Year Gained
from inclusion (time of identification of potential donor) until death from any cause or at 24 months, whichever comes first
Measured: 2 years after inclusion
Non-relapse mortality
from inclusion (time of identification of potential donor) until death without evidence of relapse or at 24 months, whichever comes first
Measured: within the 2 years after inclusion
In allo-HSCT patients only: cumulative incidence of acute and chronic graft-versus-host disease (GVHD)
from transplantation until occurrence of GVHD or death from any cause or at 24 months after inclusion, whichever comes first
Measured: within the 2 years after inclusion
In allo-HSCT patients only: severity of acute and chronic graft-versus-host disease (GVHD)
from transplantation until occurrence of GVHD or death from any cause or at 24 months after inclusion, whichever comes first
Measured: within the 2 years after inclusion
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted30 Mar 2021
- Study start31 Dec 2021
- Primary completionJun 2027 (estimated)
- Study completionJun 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Acute Myeloid LeukemiaStudies of the same condition, open and under way.
- More trials run by Assistance Publique - Hôpitaux de ParisThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 24 participants (actual)
Sponsor and investigators
- Lead sponsor
- Assistance Publique - Hôpitaux de Paris
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Rémy DULERY, MD, Principal investigator, Assistance Publique - Hôpitaux de Paris
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04822766
- Sponsor's protocol ID
- APHP200134
- Other IDs
- 2020-A01456-33
- Acronym
- ALLO-BEST
- Status last verified
- Aug 2025
- First submitted
- 24 Jan 2021
- Last updated
- 13 Aug 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 13 Aug 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Allogeneic Hematopoietic Cell Transplantation Versus Best Available Standard of Care Therapy in Elderly Patients With Acute Myeloid Leukemia: a Randomized Phase 3 Trial
- ClinicalTrials.gov ID
- NCT04822766
- Acronym / sponsor ID
- ALLO-BEST / APHP200134
- Conditions
- Acute Myeloid Leukemia
- Keywords
- Allogeneic, hematopoietic cell transplantation, acute myeloid leukemia
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 24 participants (actual)
- Lead sponsor
- Assistance Publique - Hôpitaux de ParisUniversity, hospital or other
- Study start
- 31 Dec 2021 (actual)
- Primary completion
- Jun 2027 (estimated)
- Study completion
- Jun 2027 (estimated)
- First posted
- 30 Mar 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
A subject of major interest for researchers, clinicians, patients, and payers, is the role of allogeneic hematopoietic stem cell transplantation (allo-HSCT) in the treatment of these older patients with AML. With conventional induction chemotherapy or hypomethylating agents, the expected 2-year overall survival (OS) is less than 25% in patients with intermediate- or high-risk disease. The 2-year OS ranges from 50 to 56% with allo-HSCT in AML patients older than 65 years.
Performing an allo-HSCT in older patients is however still controversial because of the higher risk of non-relapse mortality (15 to 35%) and graft-versus-host disease. Depending on the center policy, patients older than 65 years will either be contraindicated for transplant or will receive allo-HSCT.
With a phase III comparative, randomized, controlled, prospective, multicenter study, the trial aim to assess prospectively the outcomes and quality of life of older patients with AML receiving allo-HSCT strategy compared to those receiving a non-transplant approach.
Detailed description
Every year, 30,000 patients in Europe and 20,000 in the USA are diagnosed with acute myeloid leukemia (AML). More than half of them are over 65 years old. In this older population, the median overall survival (OS) is only 2 to 8 months. With conventional induction chemotherapy or hypomethylating agents, the expected 2-year OS is less than 25% in patients with intermediate- or high-risk disease.
Performing an allo-HSCT in older patients is however still controversial because of the higher risk of non-relapse mortality (15 to 35%) and graft-versus-host disease. Depending on the center policy, patients older than 65 years will either be contraindicated for transplant or will receive allo-HSCT. Noteworthy, no prospective randomized trial has yet compared allo-HSCT to a non-transplant strategy in older patients with AML. A previous attempt made 10 years ago, by the EBMT to run a slightly similar trial, has failed in France and most European countries, mainly (i) because it mandated the type of transplant procedure to be applied and (ii) because of the absence of novel and effective drugs.
Every year, 30,000 patients in Europe and 20,000 in the USA are diagnosed with acute myeloid leukemia (AML). More than half of them are over 65 years old. In this older population, the median overall survival (OS) is only 2 to 8 months. With conventional induction chemotherapy or hypometylating agents, the expected 2-year OS is less than 25% in patients with intermediate- or high-risk disease.
Performing an allo-HSCT in older patients is however still controversial because of the higher risk of non-relapse mortality (15 to 35%) and graft-versus-host disease. Depending on the center policy, patients older than 65 years will either be contraindicated for transplant or will receive allo-HSCT. Noteworthy, no prospective randomized trial has yet compared allo-HSCT to a non-transplant strategy in older patients with AML. A previous attempt made 10 years ago, by the EBMT to run a slightly similar trial, has failed in France and most European countries, mainly (i) because it mandated the type of transplant procedure to be applied and (ii) because of the absence of novel and effective drugs.
New targeted therapies and treatment strategies are evolving rapidly. A standardized unique treatment administrated to all sub-types of AML is no longer the optimal approach for induction and non-transplant maintenance strategies. No treatment has reached consensus for older patients. For these reasons, this trial will not limit the choices of drugs administered to the patients but compare two strategies allowing patients to receive the best available standard of care.
The trial aim to assess prospectively the outcomes and quality of life of older patients with AML receiving allo-HSCT strategy compared to those receiving a non-transplant approach.
Patients will receive initial treatment with chemotherapy (or other appropriate non-palliative therapy). Once first complete remission is achieved and a donor is identified, patients will be included.
Patients will be randomly assigned (1:1) after inclusion to receive one of the following strategy:
- Allogeneic hematopoietic stem cell transplantation arm: patients will undergo allo-HSCT after consolidation therapy (or completion of other appropriate non-palliative strategy) according to standard procedures of the transplant center (choice of donor, conditioning regimen, GVHD and infection prophylaxis). The use of novel therapies (such as sorafenib, midaustorine, venetoclax, etc.) will be allowed as post-transplantation maintenance strategy
- Non-transplant arm: patients will be treated according to the standard procedures of the treating center for this type of population.
All patients will receive the best available treatments (including additional conventional chemotherapy or other non-palliative therapies such as 5-azacytidine, decitabine, venetoclax, midaustorine, enasidenib, etc.). Supportive care will be performed according to each participating center usual practice.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Chemotherapy | Active comparator | Drug: Best chemotherapy treatment |
| Allogeneic Hematopoietic Cell Transplantation | Experimental | Procedure: Hematopoietic stem cell transplantationTime of transplant procedure The best available treatments of AML |
Who may qualify
- Ages
- 65 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Older adult
Key inclusion criteria (7 listed)
- Men and women
- Age ≥ 65 and ≤ 75 years
- Newly diagnosed patients with de novo or secondary AML in first complete remission who are considered as potential candidates and eligible for an allo-HSCT procedure
- Presence of a donor (matched related or unrelated or haplo-mismatched) willing to donate peripheral blood stem cells
- Patient is fit for the allo-HSCT procedure
Key exclusion criteria (12 listed)
- Acute promyelocytic leukemia (AML FAB M3)
- AML deemed not eligible for allo-HSCT
- Karnofsky score <70%
- HIV positive patient
- Life expectancy less than one month according to the attending physician
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Hematopoietic stem cell transplantationpatients will undergo allo-HSCT after consolidation therapy (or completion of other appropriate non-palliative strategy) according to standard procedures of the transplant center (choice of donor, conditioning regimen, GVHD and infection prophylaxis). The use of novel therapies (such as sorafenib, midaustorine, venetoclax, etc.) will be allowed as post-transplantation maintenance strategy. | Procedure or surgery | Allogeneic Hematopoietic Cell Transplantation |
| Best chemotherapy treatmentpatients will be treated according to the standard procedures of the treating center for this type of population. Patients will receive the best available treatments (including additional conventional chemotherapy or other non-palliative therapies such as 5-azacytidine, decitabine, venetoclax, midaustorine, enasidenib, etc.). | Drug | Chemotherapy |
Primary outcome
Overall survival
Time frame: 2 years after the inclusion
From inclusion (time of identification of potential donor) until death or at 24 months, whichever comes first]
9 secondary outcomes
Leukemia free survival
Time frame: within the 2 years after inclusion
Assessment of MRD and time to relapse from inclusion up to 2 years
Time frame: : time between inclusion and date of relapse or at 24 months, whichever comes first]
Quality of life FACT-BMT
Time frame: at baseline, 12 and 24 months after inclusion
Quality of life EQ 5D 5L
Time frame: at baseline, 12 and 24 months after inclusion
The Incremental cost-effectiveness ratios (ICERs) expressed in cost per quality-adjusted life-year (QALY) gained
Time frame: 2 years after inclusion
The Incremental cost-effectiveness ratios (ICERs) expressed in cost per Life Year Gained
Time frame: 2 years after inclusion
Non-relapse mortality
Time frame: within the 2 years after inclusion
In allo-HSCT patients only: cumulative incidence of acute and chronic graft-versus-host disease (GVHD)
Time frame: within the 2 years after inclusion
In allo-HSCT patients only: severity of acute and chronic graft-versus-host disease (GVHD)
Time frame: within the 2 years after inclusion
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 13 Aug 2025.
- Individual participant dataSharing planned: Undecided
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Rémy DULERY, MD principal investigator
Assistance Publique - Hôpitaux de Paris
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Hematopoietic stem cell transplantation and Best chemotherapy treatment in people with Acute Myeloid Leukemia, run by Assistance Publique - Hôpitaux de Paris at 1 site 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 7 inclusion and 12 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: under way, not looking for participants, as of the sponsor's last update on 13 Aug 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site 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?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Overall survival (time frame: 2 years after the inclusion). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 13 Aug 2025.