MDSPhase 2NCT04857645
ASTX727 and Donor Lymphocyte Infusions After Allogenic Stem Cell Transplantation in Very High Risk MDS or AML Patients
A Phase 2 study testing ASTX727 and Donor Lymphocyte Infusions in people with MDS and AML, run by Groupe Francophone des Myelodysplasies at 12 sites in France.
Trial summary
Study of early administration of ASTX727 associated with late Donor Lymphocyte Infusions after allogenic stem cell transplantation in very high risk MDS or AML patients Read more about this study
- Who is it for?
- Ages 18 to 70 years
- What is being tested?
- ASTX727 and Donor Lymphocyte Infusions
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 57 participants
- Where
- 12 sitesFrance
- Study dates
- 22 Jun 2021to 22 Apr 2025
- Phase
- Phase 2
- Are there results?
- None posted in the registry
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 to 70 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Patients aged from 18 to 70 years
- MDS or AML with unfavorable genetics defined as follow:
- 4 or more cytogenetic abnormalities or
- 3 cytogenetic abnormalities and TP53 or other unfavorable mutations (ASXL1, RUNX1) or
- 3 cytogenetic abnormalities and monosomal karyotype or
- mutations involving EVI1
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- AML patients should have received chemotherapy
- Marrow blast < 20% for MDS and < 10% for AML post chemotherapy
- For MDS : Revised IPSS poor or very poor ; For AML : ELN adverse risk
- Non-proliferative disease
- A donor is available (HLA matched or mismatched)
- Adequate contraception in women < 50 years and for men. Subjects must agree to use, and to be able to comply with, effective contraception without interruption, at least the first six months after transplant, throughout the entire duration of study drug therapy and for at least 6 months for women and 3 months for men after the last dose of study drug therapy.
Who cannot join
- ECOG 3 or more
- Cancer less than 2 years before inclusion or cancer not in remission the last 2 years before inclusion (except in situ cancer or baso cellular cancer)
- Cardiac failure with Ejection Fraction < 50%
- Creatininemia level > 150 µmol/L
- Liver enzyme > 3 N
- Conjugated bilirubinemia > 25 µmol/L
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- MDS occurring in patients with Fanconi anemia or congenital dyskeratosis
- Proliferative disease in patients not in remission: White Blood Cell (WBC) > 15 G/L or use of continuous cytotoxic to maintain WBC < 15 G/L
- AML with marrow or peripheral blast count higher than 10% after chemotherapy
- Known allergy or hypersensitivity to the investigational agent or decitabine or its metabolites or formulation excipients
- No contraception
- Pregnant or breastfeeding women
Trial locations and contacts
12 sites in 1 country.
12 sites in 1 country
France12 sites
CHU d'Amiens Picardie - Site sud - Service hématologie clinique et thérapie cellulaire
Amiens, 80054
CHU d'Angers - Service des maladies du sang
Angers, 49933
CHU Estaing - Service hématologie clinique et thérapie cellulaire
Clermont-Ferrand, 63000
CHU de Grenoble - Clinique Universitaire d'hématologie
Grenoble, 38043
Hôpital Saint Eloi - Service hématologie clinique
Montpellier, 34295
CHU Hôtel Dieu - Service hématologie clinique
Nantes, 44093
Hôpital Saint Louis - Service hématologie-greffe
Paris, 75010
CHU de Haut-Lévèque de Bordeaux - Service des maladies du sang
Pessac, 33604
CH Lyon Sud - Servide Hématologie
Pierre-Bénite, 69645
Centre Henri Becquerel - Département d'hématologie
Rouen, 76038
IUCT Oncopole - Département d'hématologie - Service de greffe de cellules souches hématopoïétiques
Toulouse, 31059
CHU Brabois - Service hématologie clinique
Vandœuvre-lès-Nancy, 54511
About this study
Official title: A Phase II Prospective Study "GFM-DACORAL-DLI" ASTX727 and Donor Lymphocyte Infusions After Allogenic Stem Cell Transplantation in Very High Risk MDS or AML Patients
Study of early administration of ASTX727 associated with late Donor Lymphocyte Infusions after allogenic stem cell transplantation in very high risk MDS or AML patients
Read the detailed description
Prospective study of early administration of ASTX727 associated with late Donor Lymphocyte Infusions after allogenic stem cell transplantation in very high risk MDS or AML patients
- Being tested
- ASTX727, Donor Lymphocyte Infusions
- Keywords
- Very high risk MDS, AML, ASTX727, DLI
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ASTX727Drug
Eligible patient started ASTX727 between 40 and 130 days after allogenic stem cell transplantation
Donor Lymphocyte InfusionsOther
Also called DLI
In absence of previous grade 2-4 or chronic graft-versus-host disease (GVHD), DLI will be administered at increasing doses the first day of ASTX727 cycles
| Study group | Role | What they receive |
|---|---|---|
| ASTX727 treatment | Experimental | Not described Receives: Drug: ASTX727; Other: Donor Lymphocyte Infusions |
What is being measured
The primary outcome is the main question the study is designed to answer.
Disease free Survival (DFS) at one year post transplant
Measure of time during which no sign of progression is found
Measured: 1 year post transplant
Secondary outcomes (3)
Overall Survival (OS) at one year post transplant
Measure of time from randomization to death from any cause
Measured: 1 year post transplant
Overall Survival (OS) at two years post transplant
Measure of time from randomization to death from any cause
Measured: 2 years post transplant
Risk factors for DFS, OS and non-relapse mortality at 1 and 2 years
Statistical study of cumulative incidence curves
Measured: 1 and 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Apr 2021
- Study start22 Jun 2021
- Primary completion30 Apr 2024
- Study completion22 Apr 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for MDSStudies of the same condition, open and under way.
- 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 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 57 participants (actual)
Sponsor and investigators
- Lead sponsor
- Groupe Francophone des Myelodysplasies
- Sponsor type
- University, hospital or other
- Collaborators
- Astex Pharmaceuticals, Inc.
- Responsible party
- Sponsor
- Investigators
- Marie ROBIN, MD, Principal investigator, Hôpital Saint Louis - Service hématologie-greffePierre FENAUX, MD, Principal investigator, Hôpital Saint Louis - Service hématologie séniors
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04857645
- Sponsor's protocol ID
- GFM-DACORAL-DLI
- Status last verified
- May 2025
- First submitted
- 19 Apr 2021
- Last updated
- 14 May 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Robin M, D'Aveni M, Stamatoullas A, Raffoux E, Chevallier P, Garnier A, Mediavilla C, Carre M, Himberlin C, Sebert M, Ravinet A, Desseaux K, Labussiere H, Alani M, Rubio MT, Huynh A, Ades L, de Latour RP, Paul F, Chermat F, Petit R, Mokeddem C, Charbonnier A, Thepot S, Chevret S, Fenaux P; Societe Francophone de Greffe de Moelle et de Therapie Cellulaire (SFGM-TC) and the Groupe Francophone des Myelodysplasies (GFM). Oral decitabine and cedazuridine maintenance after haematopoietic stem-cell transplantation in very high-risk acute myeloid leukaemia or myelodysplastic syndrome (GFM-DACORAL-DLI): a multicentre, single-arm, phase 2 trial. Lancet Haematol. 2025 Sep;12(9):e705-e716. doi: 10.1016/S2352-3026(25)00172-3. Epub 2025 Aug 7.
PubMed 40784355
Study at a glance
From the registry record, last updated by the sponsor on 14 May 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase II Prospective Study "GFM-DACORAL-DLI" ASTX727 and Donor Lymphocyte Infusions After Allogenic Stem Cell Transplantation in Very High Risk MDS or AML Patients
- ClinicalTrials.gov ID
- NCT04857645
- Acronym / sponsor ID
- GFM-DACORAL-DLI
- Keywords
- Very high risk MDS, AML, ASTX727, DLI
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 57 participants (actual)
- Lead sponsor
- Groupe Francophone des MyelodysplasiesUniversity, hospital or other
- Collaborators
- Astex Pharmaceuticals, Inc.
- Study start
- 22 Jun 2021 (actual)
- Primary completion
- 30 Apr 2024 (actual)
- Study completion
- 22 Apr 2025 (actual)
- First posted
- 23 Apr 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.
Study of early administration of ASTX727 associated with late Donor Lymphocyte Infusions after allogenic stem cell transplantation in very high risk MDS or AML patients
Detailed description
Prospective study of early administration of ASTX727 associated with late Donor Lymphocyte Infusions after allogenic stem cell transplantation in very high risk MDS or AML patients
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| ASTX727 treatment | Experimental | Drug: ASTX727, Other: Donor Lymphocyte Infusions |
Who may qualify
- Ages
- 18 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (12 listed)
- Patients aged from 18 to 70 years
- MDS or AML with unfavorable genetics defined as follow:
- 4 or more cytogenetic abnormalities or
- 3 cytogenetic abnormalities and TP53 or other unfavorable mutations (ASXL1, RUNX1) or
- 3 cytogenetic abnormalities and monosomal karyotype or
Key exclusion criteria (12 listed)
- ECOG 3 or more
- Cancer less than 2 years before inclusion or cancer not in remission the last 2 years before inclusion (except in situ cancer or baso cellular cancer)
- Cardiac failure with Ejection Fraction < 50%
- Creatininemia level > 150 µmol/L
- Liver enzyme > 3 N
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 |
|---|---|---|
| ASTX727Eligible patient started ASTX727 between 40 and 130 days after allogenic stem cell transplantation | Drug | ASTX727 treatment |
| Donor Lymphocyte InfusionsAlso: DLIIn absence of previous grade 2-4 or chronic graft-versus-host disease (GVHD), DLI will be administered at increasing doses the first day of ASTX727 cycles | Other | ASTX727 treatment |
Primary outcome
Disease free Survival (DFS) at one year post transplant
Time frame: 1 year post transplant
Measure of time during which no sign of progression is found
3 secondary outcomes
Overall Survival (OS) at one year post transplant
Time frame: 1 year post transplant
Overall Survival (OS) at two years post transplant
Time frame: 2 years post transplant
Risk factors for DFS, OS and non-relapse mortality at 1 and 2 years
Time frame: 1 and 2 years
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 14 May 2025.
- Derived from this study: PubMed 40784355Robin M, D'Aveni M, Stamatoullas A, Raffoux E, Chevallier P, Garnier A, Mediavilla C, Carre M, Himberlin C, Sebert M, Ravinet A, Desseaux K, Labussiere H, Alani M, Rubio MT, Huynh A, Ades L, de Latour RP, Paul F, Chermat F, Petit R, Mokeddem C, Charbonnier A, Thepot S, Chevret S, Fenaux P; Societe Francophone de Greffe de Moelle et de Therapie Cellulaire (SFGM-TC) and the Groupe Francophone des Myelodysplasies (GFM). Oral decitabine and cedazuridine maintenance after haematopoietic stem-cell transplantation in very high-risk acute myeloid leukaemia or myelodysplastic syndrome (GFM-DACORAL-DLI): a multicentre, single-arm, phase 2 trial. Lancet Haematol. 2025 Sep;12(9):e705-e716. doi: 10.1016/S2352-3026(25)00172-3. Epub 2025 Aug 7.
- Individual participant dataSharing planned: No
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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.
Marie ROBIN, MD principal investigator
Hôpital Saint Louis - Service hématologie-greffe
Pierre FENAUX, MD principal investigator
Hôpital Saint Louis - Service hématologie séniors
12 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing ASTX727 and Donor Lymphocyte Infusions in people with MDS and AML, run by Groupe Francophone des Myelodysplasies at 12 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 12 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: finished, as of the sponsor's last update on 14 May 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 12 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.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Disease free Survival (DFS) at one year post transplant (time frame: 1 year post transplant). 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 NCT04857645, which also lists 1 linked publication.
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 14 May 2025.