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MDSPhase 1/2NCT05226455

Venetoclax in Patients With MDS or AML in Relapse After AHSCT

A Phase 1/2 study testing venetoclax + azacitidine +/- donor lymphocyte infusion in people with MDS and AML, run by Groupe Francophone des Myelodysplasies at 11 sites in France.

Active, not recruiting

Under way, not looking for participants. Everyone needed has enrolled. The study is still running.

Official record
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Trial summary

Study to assess venetoclax + azacitidine and donor lymphocyte infusion (DLI) in patients with myelodysplastic syndrome (MDS) or acute myeloid leukemia (AML) in relapse after allohematopoietic stem cell transplantation (AHSCT). Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
MDS, AML
What is being tested?
venetoclax + azacitidine +/- donor lymphocyte infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
55 participants
Where
11 sitesFrance
Study dates
23 Nov 2022to 1 Sep 2027
Phase
Phase 1/2
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
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Documented cytologic relapse of MDS according to FAB/WHO classification 2016 (including CMML with WBC < 13000/mm3) or AML, with WBC < 15000/mm3, after allo-SCT.
  • Relapse of MDS or AML is defined as :
  • Return to pretreatment bone marrow blast percentage
  • Decrement of at least 50% from maximum remission
  • Age ≥ 18 years.
  • Eastern Cooperative Oncology Group (ECOG) performance status ≤ 2.
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  • Patient must have adequate organ function:
  • Serum creatinine < 2 mg/dL or calculated creatinine clearance ≥ 30 mL/min for patients with creatinine levels > 1.5 times Upper Limit of Normal
  • Serum total bilirubin ≤ 2.5 times Upper Limit of Normal or direct bilirubin ≤ Upper Limit of Normal for patients with total bilirubin levels ≥ 2 mg/d
  • Aspartate aminotransferase (AST) and Alanine aminotransferase (ALT) ≤ 2.5 times Upper Limit of Normal
  • Alkaline phosphatase ≤ 5 times Upper Limit of Normal (if > 2.5 times Upper Limit of Normal, then liver fraction should be ≤ 2.5 times Upper Limit of Normal).
  • Patient not refractory to platelet transfusions.
  • Female subject of childbearing potential must practice at least one protocol specified method of birth control, starting on Study Day 1 through at least 30 days after the last dose of venetoclax or 6 months after the last dose of azacitidine.
  • Not being of childbearing potential is defined as:
  • Age > 55 years with no menses for 12 or more months without an alternative medical cause, or
  • Age ≤ 55 years with no menses for 12 or more months without an alternative medical cause AND an Follicle Stimulating Hormone (FSH) level > 40 IU/L, or
  • Permanent surgical sterility (bilateral oophorectomy, bilateral salpingectomy or hysterectomy).
  • Female subjects of childbearing potential must have negative results for pregnancy test performed:
  • At Screening with a serum sample obtained within 14 days prior to the first study drug administration, and
  • Prior to dosing with urine sample obtained on Cycle 1 Day 1, if it has been > 7 days since obtaining the serum pregnancy test results.
  • Female subjects who are not of childbearing potential at Screening do not require pregnancy testing.
  • Male subjects sexually active with female partner(s) of childbearing potential, must agree from first dose of study drug(s) through at least 30 days after the last dose of venetoclax or 3 months after the last dose of azacitidine, whichever is later, to practice the protocol specified contraception.
  • Patient is available for periodic blood sampling, study related assessments, and appropriate clinical management at the treating institution for the duration of the study.
  • Patient has the ability to understand and willingness to sign an informed consent form indicating the investigational nature of the study.
  • Patient is able to swallow capsules.

Who cannot join

  • Patient has active and uncontrolled infection.
  • Patient has active acute or chronic Graft-versus-Host-Disease (GVHD).
  • Patient receives more than 1mg/kg/day prednisolone.
  • Patient has uncontrolled intercurrent illness or circumstances that could limit compliance with the study, including but not limited to the following: symptomatic congestive heart failure, unstable angina pectoris, uncontrolled cardiac arrhythmia, pancreatitis, or psychiatric or social conditions that may interfere with patient compliance.
  • Patient is currently participating or has participated in a study with an investigational compound or device within 30 days of initial dosing with study drug.
  • Patient has known human immunodeficiency virus (HIV) infection or HIV-related malignancy.
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  • Patient has clinically active hepatitis B or hepatitis C infection.
  • Patient has a known allergy or hypersensitivity to any component of venetoclax or azacitidine.
  • Patient with a "currently active" second malignancy, other than non-melanoma skin cancer and carcinoma in situ of the cervix, should not be enrolled. Patients are not considered to have a "currently active" malignancy if they have completed therapy for a prior malignancy, are disease free from prior malignancies for > 2 years or are considered by their physician to be at less than 30% risk of relapse.
  • Patient has received growth factors such as erythropoietin alfa (EPO) or granulocyte colony-stimulating factor (G-CSF) or has received non cytotoxic agents (including low dose oral chemotherapy) in the 30 days before inclusion. In case of previous cytotoxic treatment, an interval of 3 months is required.
  • Patient is on any systemic steroids that have not been stabilized to the equivalent of ≤ 10 mg/day prednisone during the 4 weeks prior to the start of the study drugs.
  • Patients with clinical evidence of Central Nervous System leukemia.
  • Patient has a history of Gastrointestinal surgery or other procedures that might interfere with the absorption or swallowing of the study drugs.
  • Subject has received strong or moderate CYP3A (Cytochrome P450, family 3, subfamily A) inhibitors within 3 days prior to the first dose of study drug.
  • Patient is unable to take and/or tolerate oral medications on a continuous basis.
  • Patient is pregnant or breastfeeding within the projected duration of the study.
  • Subject has a malabsorption syndrome or other condition that precludes an enteral route of administration.
  • Absence of social security.

Trial locations and contacts

11 sites in 1 country.

11 sites in 1 country

France11 sites

  • CHU d'Angers

    Angers, 49933

  • CHU de Grenoble

    Grenoble, 38043

  • Hôpital Dupuytren

    Limoges, 87042

  • Hôpital Saint-Eloi

    Montpellier, 34295

  • CHU Hôtel Dieu

    Nantes, 44093

  • Hôpital l'Archet I

    Nice, 06200

  • Hôpital Saint louis

    Paris, 75010

  • CHU de Haut-Lévèque

    Pessac, 33604

  • Centre Hospitalier Lyon-Sud

    Pierre-Bénite, 69495

  • Centre Henri Becquerel

    Rouen, 76038

  • IUCT Oncopole

    Toulouse, 31059

Other trials in France

About this study

Official title: A Phase I-II Study to Assess Venetoclax + Azacitidine and Donor Lymphocyte Infusion in Patients With MDS or AML in Relapse After Allohematopoietic Stem Cell Transplantation

Study to assess venetoclax + azacitidine and donor lymphocyte infusion (DLI) in patients with myelodysplastic syndrome (MDS) or acute myeloid leukemia (AML) in relapse after allohematopoietic stem cell transplantation (AHSCT).

Read the detailed description

A phase I-II study to assess venetoclax + azacitidine and donor lymphocyte infusion (DLI) in patients with myelodysplastic syndrome (MDS) or acute myeloid leukemia (AML) in relapse after allohematopoietic stem cell transplantation (AHSCT).

Conditions
MDS, AML
Being tested
venetoclax + azacitidine +/- donor lymphocyte infusion
Keywords
MDS, AML, AHSCT, Venetoclax, Azacitidine, DLI

What participants receive

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

  • venetoclax + azacitidine +/- donor lymphocyte infusionDrug

    Also called venclyxto, vidaza

    Venetoclax will be given once daily orally on days 1 to 14 for all cycles. Venetoclax + azacitidine +/- donor lymphocyte infusion (12 cycles maximum)

Study groups in NCT05226455
Study groupRoleWhat they receive
venetoclax + azacitidine +/- donor lymphocyte infusionExperimentalVenetoclax + azacitidine +/- donor lymphocyte infusion (maximum 12 cycles). Venetoclax: on D1 to D14 of 28 days cycle ; Phase I: 4 dose levels: 50, 100, 200, 400 mg/d, starting dose at 100 mg ; Phase II: dose defined in the phase I. Azacitidine 75 mg/m²/d or 50 mg/m²/d (if allohematopoietic stem cell transplantation relapse < 4 months) x 5 days (on D1 to D5 of 28 days cycle).
Receives: Drug: venetoclax + azacitidine +/- donor lymphocyte infusion

What is being measured

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

  1. Phase I: Dose-finding study

    Evaluation of Dose-limiting toxicity (DLT) to determine the optimal dose level in terms of both toxicity and safety for venetoclax + azacitidine

    Measured: At the end of cycle 1 of venetoclax + azacitidine (each cycle is 28 days)

  2. Phase II: Overall improvement rate of venetoclax + azacitidine +/- DLI

    Response assessment performed according to modified IWG (International Working Group) 2006 criteria for myelodysplastic syndrome and to European Leukemia Net criteria for acute myeloid leukemia

    Measured: After 8 cycles of venetoclax + azacitidine (each cycle is 28 days)

Secondary outcomes (6)

  1. Toxicity assessment

    Adverse events analyzed by frequency, NCI CTCAE 5.0 grade, and causality

    Measured: At 60 months (at end of study)

  2. Graft-versus-Host-Disease (GVHD) rate

    Acute and chronic graft-versus-host-disease (GVHD) rate assessment (graft-versus-host events analyzed by frequency and grade)

    Measured: At 60 months (at end of study)

  3. Duration of response

    Duration of response defined as time from the date of the first observed response (complete or partial) to the date of first subsequent documented disease progression or relapse or death

    Measured: At 60 months (at end of study)

  4. Overall survival

    Overall Survival defined as time from the date of the first dose of venetoclax to the date of death or end of study

    Measured: At 60 months (at end of study)

  5. Progression-free survival

    Progression-free Survival for patients with at least a partial response defined as time from the date of the first dose of venetoclax to the date of the first documented disease progression or relapse or death

    Measured: At 60 months (at end of study)

  6. Event-free survival

    Event-free Survival defined as time from the date of the first dose of venetoclax to the date of earliest disease progression or death

    Measured: At 60 months (at end of study)

Timeline

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

  1. First posted7 Feb 2022
  2. Study start23 Nov 2022
  3. Primary completion15 Nov 2026 (estimated)
  4. Study completion1 Sep 2027 (estimated)

Resources

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 1/2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
55 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT05226455
Sponsor's protocol ID
VENTOGRAFT
Other IDs
2024-514877-23-00
Status last verified
Mar 2026
First submitted
11 Jan 2022
Last updated
12 Mar 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
No
FDA-regulated device
No

Questions about this trial

What is this clinical trial studying?

A Phase 1/2 study testing venetoclax + azacitidine +/- donor lymphocyte infusion in people with MDS and AML, run by Groupe Francophone des Myelodysplasies at 11 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 25 inclusion and 18 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 12 Mar 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 11 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 combined phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

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?

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?

Phase I: Dose-finding study (time frame: At the end of cycle 1 of venetoclax + azacitidine (each cycle is 28 days)). The study lists 2 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 NCT05226455.

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 12 Mar 2026.

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