AML (Acute Myeloid Leukemia)Phase 2NCT07214064
Venetoclax- Augmented Treosulfan-Based Reduced Intensity Conditioning Before Allogeneic Stem Cell Transplantation
A Phase 2 study testing RIC regimen in people with AML (Acute Myeloid Leukemia) and MDS/AML, run by University Hospital Tuebingen at 1 site in Germany.
Trial summary
Safety and Feasibility of a Venetoclax- Augmented Treosulfan-Based Reduced Intensity Conditioning Before Allogeneic Stem Cell Transplantation in AML, MDS/AML and Higher Risk MDS Read more about this study
- Who is it for?
- Ages 18 to 75 yearsHealthy volunteers can take part
- Conditions the study is for
- AML (Acute Myeloid Leukemia), MDS/AML, MDS (Myelodysplastic Syndrome)
- What is being tested?
- RIC regimen
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 27 participants
- Where
- 1 siteGermany
- Study dates
- Jan 2026to Dec 2027
- Phase
- Phase 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 to 75 years
- All sexes
- Healthy volunteers accepted
- Adult, Older adult
Who may be able to join
- Age between 18 and 75 years at the time of signing the Informed Consent
- Patient is fluent in German
- Signed written Informed Consent with the cognitive ability to understand all consequences of trial participation and to comply with all trial related procedures
- Diagnosis of AML,MDS/AML (according to ICC 20226) or HRMDS (IPSS-R7 >3.5 or IPSS-M8 >0; according to ICC 20226 and IWG 20232)
- Myeloid neoplasm (AML, MDS/AML or HR-MDS according to ICC 20226) under control* at time of screening, defined as one of the following:
- 5.1. AML (ICC 20226):
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- Scheduled for alloHCT after prior Remission
- Induction ± Consolidation:
- Achievement of at least MLFS** (according to ELN 20221 criteria) after up to two cycles of intensive, anthracycline-based induction chemotherapy OR
- Achievement of at least MLFS** (according to ELN 20221 criteria) after intensive, anthracycline-based induction chemotherapy folllowed by up to three cycles of cytostatic consolidation therapy OR
- Achievement of at least MLFS** (according to ELN 20221) after less intensive, HMA-based treatment (up to six cycles) OR
- Achievement of at least MLFS** (according to ELN 20221) after a combination of intensive and less intensive treatments (up to six cycles in total) 5.2. MDS/AML (ICC 20226):• Scheduled for alloHCT after prior Remission
- Induction ± Consolidation:
- Achievement of at least MLFS** (according to ELN 20221 criteria) after up to two cycles of intensive, anthracycline-based induction chemotherapy OR
- Achievement of at least MLFS** (according to ELN 20221 criteria), after intensive, anthracycline-based induction chemotherapy folllowed by up to three cycles of cytostatic consolidation therapy OR
- Achievement of at least MLFS** (according to ELN 20221 criteria) after less intensive, HMA-based treatment (up to six cycles and including HMA monotherapy) OR
- Achievement of at least MLFS (according to ELN 20221 criteria) after a combination of intensive and less intensive treatments (up to six cycles in total)• Scheduled for upfront alloHCT:
- ≤19% bone marrow blastsAND• ≤19% peripheral blood blastsAND• ≤15.000/µl peripheral blood Leukocytes 5.3. HR- MDS (ICC 20226, IWG 20232)
- Scheduled for alloHCT after prior therapy:
- up to six cycles of a less intensive HMAbased treatment or HMA monotherapy AND
- ≤9% bone marrow blastsAND• ≤9% peripheral blood blasts• Scheduled for upfront alloHCT:
- ≤9% bone marrow blasts AND
- ≤9% peripheral blood blasts *The disease is considered clinically controlled, when it is either aggressive but has proven responsive to cytostatic chemotherapy (e.g. AML with achievement of at least MLFS) or slowly progressive ( e.g. suitable for upfront alloHCT in cases of MDS/AML) or is both slowly progressive and responsive to therapy
- a bone marrow assessment is mandatory during screening phase. Patients canonly be enrolled after the according inclusion criteria have been confirmed bybone marrow biopsy. The time window for bone marrow biopsy is day -21 to -14.
- Eligiblity for alloHCT according to a board of experienced haematologists
- Karnofsky Performance Index ≥60%
- Planned alloHCT with Peripheral Blood Stem Cells (PBSC)
- Infusion of allogeneic stem cells schedulded between day 14 and day 28 after Screening
- Availability of a suitable donor, defind as one of the following:
- 10.1. HLA-identical sibling (MSD)OR 10.2. HLA-compatible (9/10 antigens matched for HLA-A, -B, -C, -DRB1, and -DQB1) unrelated donor (MUD) with completed confirmatory typing.
- OR 10.3. Two unrelated donors with >90% probability of a 9/10 match for HLA- A, -B, -C, -DRB1, and -DRQB1, according to OptiMatch list (MUD)
- Documented diffusion lung capacity for carbon monoxide (DLCO) >40% (adjusted for hemoglobin, if available) and FEV1/FVC >50%
- Left ventricular ejection fraction (LVEF) ≥40%
- GFR (CKD-EPI) ≥ 30 ml/min/1,73 m2
- Bilirubin ≤3x ULN and AST ≤5x ULN
- Thoracic imaging (either X-ray or computed tomography (CT)) without evidence of active infection or second malignancy
- Subject (male or female) is willing to use highly effective methods during treatment and for 6 months (male or female) after the end of treatment (adequate: combined hormonal contraception associated with inhibition of ovulation, progestogen-only hormonal contraception associated with inhibition of ovulation, intrauterine device, intrauterine hormone-releasing system, bilateral tubal occlusion, vasectomized partner1, sexual abstinence2). Female participants using homonal contraceptives should use a barrier method as well.1 Vasectomized partner is a highly effective birth control method provided that partner is the sole sexual partner of the FCBP trial participant and that the vasectomized partner has received medical assessment of the surgical success2 In the context of this guidance sexual abstinence is considered a highly effective method only if defined as refraining from heterosexual intercourse during the entire period of risk associated with the study treatments. The reliability of sexual abstinence needs to be evaluated in relation to the duration of the clinical trial and the preferred and usual lifestyle of the subject.
- Absence of pregnancy confirmed by a highly sensitive pregnancy test not older than 3 days at time of screening (only FCBP).
- Subject agrees not to share medication.
Who cannot join
- APL (AML with t(15;17))
- MDS/MPN (ICC 20226)
- Karnofsky Performance Index <60%
- Patient scheduled for haploidentical allogeneic hematopoetic stem cell transplantation or bone marrow stem cell transplantation
- Presence of extramedullary myelosarcoma
- Disease Relapse after prior CRc
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- History of allogeneic hematopoietic stem cell transplantation
- Significant active cardiac disease within 6 months prior to the start of study treatment, including:
- New York Heart Association (NYHA) class III or IV congestive heart failure
- Myocardial infarction
- Unstable angina
- Cerebral apoplexy
- Severe cardiac arrhythmias
- Left ventricular ejection fraction (LVEF) <40% by TTE
- Documented diffusion lung capacity for carbon monoxide (DLCO) ≤40% (adjusted for hemoglobin, if available) and FEV1/FVC ≤50%
- GFR (CKD-EPI) <30 ml/min/1,73 m2
- Bilirubin >3x ULN or AST >5x ULN
- Clinical symptoms suggestive of active central nervous system (CNS) leukemia or known CNS leukemia.
- Active viral infection, including hepatitis B, hepatitis C or Human Immunodeficiency Virus (HIV) infection, that is uncontrolled prior to first dose of study treatment and may interfere with the study objectives or which could expose the patient to undue risk through the participation in the clinical trial. An infection controlled with an approved antiviral treatment is allowed.
- Presence of Proven, Probable or Possible Invasive Fungal Disease (IFD) as defined by EORTC/MSG 20209 Definitions (please consult Appendix)
- Serologies suggestive of recent (<6 months) infection or reactivation with/of Toxoplasma gondii (based on IgG, IgM and Avidity) or of infection with Treponema pallidum (based on TPPA).
- Any clinically uncontrolled infection (bacterial or unknown pathogen), defined as persisting or recurring fever or rising levels of CRP (≥10 mg/dl) despite intravenous antibacterial or antifungal therapy (initiated or escalated at least 72h hours ago)
- Immediate life-threatening, severe complications of leukemia such as uncontrolled bleeding and/or disseminated intravascular coagulation.
- Conditions that limit the ingestion or gastrointestinal absorption of orally administered drugs.
- Patients with a currently active second malignancy. Patients are not considered to have a currently active malignancy, if they have completed therapy and are considered by their physician to be at <30% risk of relapse within one year. However, patients with the following history/concurrent conditions are allowed:
- Basal or squamous cell carcinoma of the skin;
- Carcinoma in situ of the cervix;
- Carcinoma in situ of the breast;
- Incidental histologic finding of prostate cancer.
- Receipt of live, attenuated vaccine within 30 days prior to the study inclusion (NOTE: patients, if enrolled, should not receive live vaccine during the study and until 6 months after the therapy)
- Severe neurological or psychiatric disorder interfering with ability to give an informed consent.
- Women during pregnancy and lactation.
- History of hypersensitivity to the investigational medicinal product or to any drug with similar chemical structure or to any excipient present in the pharmaceutical form of the investigational medicinal product.
- Participation in other trials interfering with the endpoint of this study. Prior trial participation is permitted, provided that treatment with the investigational medicinal product has been completed at least 4 days prior to screening for this trial (at least 10 days before planned study treatment).
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
Germany1 site
University Hospital , Department of Internal Medicine II
Tübingen, Baden-Wurttemberg, 72076
Christoph Faul, Dr., +49 7071 29-84087, Christoph.Faul@med.uni-tuebingen.de
Wolfgang Bethge, Prof. Dr., +49 7071 29-83176, Wolfgang.Bethge@med.uni-tuebingen.de
About this study
Official title: Safety and Feasibility of a Venetoclax- Augmented Treosulfan-Based Reduced Intensity Conditioning Before Allogeneic Stem Cell Transplantation in AML, MDS/AML and Higher Risk MDS
Safety and Feasibility of a Venetoclax- Augmented Treosulfan-Based Reduced Intensity Conditioning Before Allogeneic Stem Cell Transplantation in AML, MDS/AML and Higher Risk MDS
- Being tested
- RIC regimen
- Keywords
- Allogeneic Stem Cell Transplantation, Venetoclax, Venetoclax- Augmented Treosulfan-Based Reduced Intensity Conditioning, RIC
- Medical subject headings
- Leukemia, Myeloid, Acute, Anemia, Refractory, with Excess of Blasts
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
RIC regimenDrug
Venetoclax (Venclyxto®): 400 mg abs./d day -8 to -2, Fludarabine 30 mg/m2 BSA day -6 to day -2, Treosulfan 10 g/m2 BSA day -4 to day -2
| Study group | Role | What they receive |
|---|---|---|
| Reduced Intensity Conditioning before Allogeneic Stem Cell Transplantation | Experimental | Patients with AML, MDS/AML or HR-MDS, who have either responded to remission inducing therapy or are intended for upfront transplantion receive conditioning chemotherapy as follows:
IMPs:
* Venetoclax 400 mg abs. day -8 to day -2
* Fludarabine 30 mg/m2 BSA day -6 to day -2
* Treosulfan 10 g/m2 BSA day -4 to day -2 Receives: Drug: RIC regimen |
What is being measured
The primary outcome is the main question the study is designed to answer.
Overall survival (OS)
Primary objective of this trial is to evaluate the feasibility and safety of the augmentation of a treosulfanbased RIC regimen for alloHCT in AML, MDS/AML and HR-MDS patients with the Bcl-2-inhibitor Venetoclax. As outlined above, the aim is to increase antileukemic acitiviy without increasing morbidity and mortality in a vulnerable patient collective. This is operationalized by the following primary endpoint:
- Overall survival (OS) at day 28 after alloHCT Most adverse events (AEs) due to the IMPs are expected to occur before day 0. Protacted or lateonset toxicities with potentially lethal consequences cannot be ruled out. Therefore, we regard it most suitable to evaluate overall survival at day 28 after alloHCT.
Measured: at day 28 after alloHCT
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Oct 2025
- Study startJan 2026 (estimated)
- Primary completionAug 2027 (estimated)
- Study completionDec 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for AML (Acute Myeloid Leukemia)Studies 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
- 27 participants (estimated)
Sponsor and investigators
- Lead sponsor
- University Hospital Tuebingen
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Christoph Faul, Dr., Principal investigator, Sponsor's Delegate
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07214064
- Sponsor's protocol ID
- VeStAL
- Other IDs
- 2025-521372-62-00, 2025
- Status last verified
- Sep 2025
- First submitted
- 2 Oct 2025
- Last updated
- 9 Oct 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 9 Oct 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Safety and Feasibility of a Venetoclax- Augmented Treosulfan-Based Reduced Intensity Conditioning Before Allogeneic Stem Cell Transplantation in AML, MDS/AML and Higher Risk MDS
- ClinicalTrials.gov ID
- NCT07214064
- Acronym / sponsor ID
- VeStAL
- Keywords
- Allogeneic Stem Cell Transplantation, Venetoclax, Venetoclax- Augmented Treosulfan-Based Reduced Intensity Conditioning, RIC
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 27 participants (estimated)
- Lead sponsor
- University Hospital TuebingenUniversity, hospital or other
- Study start
- Jan 2026 (estimated)
- Primary completion
- Aug 2027 (estimated)
- Study completion
- Dec 2027 (estimated)
- First posted
- 9 Oct 2025 (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.
Safety and Feasibility of a Venetoclax- Augmented Treosulfan-Based Reduced Intensity Conditioning Before Allogeneic Stem Cell Transplantation in AML, MDS/AML and Higher Risk MDS
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Reduced Intensity Conditioning before Allogeneic Stem Cell Transplantation | Experimental | Drug: RIC regimenPatients with AML, MDS/AML or HR-MDS, who have either responded to remission inducing therapy or are intended for upfront transplantion receive conditioning chemotherapy as follows: IMPs: * Venetoclax 400 mg abs. day -8 to day -2 * Fludarabine 30 mg/m2 BSA day -6 to day -2 * Treosulfan 10 g/m2 BSA day -4 to day -2 |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (39 listed)
- Age between 18 and 75 years at the time of signing the Informed Consent
- Patient is fluent in German
- Signed written Informed Consent with the cognitive ability to understand all consequences of trial participation and to comply with all trial related procedures
- Diagnosis of AML,MDS/AML (according to ICC 20226) or HRMDS (IPSS-R7 >3.5 or IPSS-M8 >0; according to ICC 20226 and IWG 20232)
- Myeloid neoplasm (AML, MDS/AML or HR-MDS according to ICC 20226) under control* at time of screening, defined as one of the following:
Key exclusion criteria (34 listed)
- APL (AML with t(15;17))
- MDS/MPN (ICC 20226)
- Karnofsky Performance Index <60%
- Patient scheduled for haploidentical allogeneic hematopoetic stem cell transplantation or bone marrow stem cell transplantation
- Presence of extramedullary myelosarcoma
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 |
|---|---|---|
| RIC regimenVenetoclax (Venclyxto®): 400 mg abs./d day -8 to -2, Fludarabine 30 mg/m2 BSA day -6 to day -2, Treosulfan 10 g/m2 BSA day -4 to day -2 | Drug | Reduced Intensity Conditioning before Allogeneic Stem Cell Transplantation |
Primary outcome
Overall survival (OS)
Time frame: at day 28 after alloHCT
Primary objective of this trial is to evaluate the feasibility and safety of the augmentation of a treosulfanbased RIC regimen for alloHCT in AML, MDS/AML and HR-MDS patients with the Bcl-2-inhibitor Venetoclax. As outlined above, the aim is to increase antileukemic acitiviy without increasing morbidity and mortality in a vulnerable patient collective. This is operationalized by the following primary endpoint: 1) Overall survival (OS) at day 28 after alloHCT Most adverse events (AEs) due to the IMPs are expected to occur before day 0. Protacted or lateonset toxicities with potentially lethal consequences cannot be ruled out. Therefore, we regard it most suitable to evaluate overall survival at day 28 after alloHCT.
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 9 Oct 2025.
- 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.
Christoph Faul, Dr. principal investigator
Sponsor's Delegate
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing RIC regimen in people with AML (Acute Myeloid Leukemia) and MDS/AML, run by University Hospital Tuebingen at 1 site in Germany.
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 39 inclusion and 34 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: opening soon, as of the sponsor's last update on 9 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 1 site in Germany. 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?
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 Christoph Faul, Dr. (+49 7071 29-84087, Christoph.Faul@med.uni-tuebingen.de), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Overall survival (OS) (time frame: at day 28 after alloHCT). 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 9 Oct 2025.