Acute Myeloid Leukemia (AML)Phase 2VENAZA-5SNCT05833438
Venetoclax in Combination With 5 Days Azacitidine in Untreated AML Patients, Not Eligible for Standard Induction Therapy
A Phase 2 study testing VEN+AZA-5 in people with Acute Myeloid Leukemia (AML), run by University of Leipzig at 10 sites in Germany.
Trial summary
Acute myeloid leukemia (AML): continuous oral Venetoclax (VEN) and 7 days of s.c. Azacitidine (AZA) per 28-day cycle = standard of care for intensive induction therapy ineligible AML patients in Germany The VENAZA-5S pilot trial: AZA administration reduced to 5 days within each cycle to improve tolerability and… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Acute Myeloid Leukemia (AML)
- What is being tested?
- VEN+AZA-5
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 45 participants
- Where
- 10 sitesGermany
- Study dates
- 17 May 2023to May 2026
- 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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Confirmed diagnosis of AML by World Health Organization (WHO) criteria 2016
- Ineligible for treatment with a standard cytarabine and anthracycline induction regimen due to age or comorbidities
- Age ≥ 18 years
- Life expectancy of at least 12 weeks
Who cannot join
- Prior treatment for AML or myelodysplastic syndrome (MDS) with one of the following:
- Hypomethylating agent (HMA)
- Chemotherapeutic agent
- Chimeric Antigen Receptor (CAR)-T cell therapy
- Experimental therapies
- Note: Prior use of hydroxyurea is allowed
Show all 9Show fewer
- History of myeloproliferative neoplasm (MPN)
- Diagnosis of acute promyelocytic leukemia (APL)
- Presence of favorable-risk karyotype abnormalities: t(15;17), t(8;21), inv(16) or t(16;16)
Trial locations and contacts
10 sites in 1 country.
10 sites in 1 country
Germany10 sites
Helios Klinikum Berlin-Buch Klinik für Hämatologie und Stammzelltransplantation
Berlin, 13125
Klinikum Chemnitz gGmbH Klinik für lnnere Medizin Ill
Chemnitz, 09116
Carl-Thiem-Klinikum Cottbus gGmbH
Cottbus, 03048
Universitatsklinikum Carl Gustav Carus Dresden an der TU Dresden Medizinische Klinik und Poliklinik 1 Bereich Hamatologie
Dresden, 01307
Universitätsklinikum Heidelberg, Innere Medizin V; Klinik für Hämatologie, Onkologie und Rheumatologie
Heidelberg, 69120
Universitätsklinikum Leipzig, Klinik und Poliklinik für Hämatologie, Zelltherapie und Hämostaseologie
Leipzig
Kliniken Maria Hilf GmbH, Klinik für Hämatologie, Onkologie und Gastroenterologie
Mönchengladbach, 41063
Rotkreuzklinikum München, III. Medizinische Abteilung
München, 80364
Klinikum rechts der lsar der TU München, Klinik und Poliklinik für lnnere Medizin Ill
München, 81675
Kliniken Sindelfingen,Medizinische Klinik I
Sindelfingen, 71065
About this study
Official title: A Single-arm, Pilot Study of Venetoclax in Combination With 5 Days Azacitidine in Treatment-naïve Subjects With Acute Myelogenous Leukemia Who Are ≥18 Years of Age and Not Eligible for Standard Induction Therapy (VENAZA-5S PILOT TRIAL)
Acute myeloid leukemia (AML): continuous oral Venetoclax (VEN) and 7 days of s.c. Azacitidine (AZA) per 28-day cycle = standard of care for intensive induction therapy ineligible AML patients in Germany
The VENAZA-5S pilot trial: AZA administration reduced to 5 days within each cycle to improve tolerability and treatment adherence due to less neutropenic infections, less treatment interruptions and less hospitalizations.
Read the detailed description
Acute myeloid leukemia (AML) is a uniformly fatal disease if untreated. The combination of continuous oral Venetoclax (VEN) and 7 days of s.c. Azacitidine (AZA) per 28-day cycle has recently emerged as the new standard of care for AML patient who are ineligible for intensive induction therapy, and has been widely adopted in Germany.
The VENAZA-5S pilot trial aims to reduce the reported hematological toxicity profile of this currently approved combination, while preserving efficacy, by modifying AZA administration to 5 days within each cycle. The hypothesis is that this modification will not interfere with the response rates achieved by the combination, but will rather improve tolerability and treatment adherence due to less neutropenic infections, less treatment interruptions and hospitalizations, and thus result in better quality of life and favorable long-term outcomes in elderly or comorbid AML patients. This single-arm pilot study is intended to generate first data on the efficacy and toxicity of 5 days AZA + VEN, which will be compared to a historical control cohort treated with the current standard of 7 days AZA + VEN.
- Conditions
- Acute Myeloid Leukemia (AML)
- Being tested
- VEN+AZA-5
- Keywords
- Venclyxto, Venetoclax, Azacitidine, Hematologic Diseases, Acute Myelogenous Leukemia
- Medical subject headings
- Leukemia, Myeloid, Acute, Hematologic Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
VEN+AZA-5Drug
Up to 6 cycles: Azacitidine (AZA) 75 mg/m2, d1-5 of each 28 day cycle (SC) in combination with Venetoclax (VEN): 400 mg daily (orally)
| Study group | Role | What they receive |
|---|---|---|
| VEN+AZA-5 | Experimental | Azacitidine (AZA) 75 mg/m2, d1-5 of each 28 day cycle (SC) in combination with Venetoclax (VEN): 400 mg daily (orally) Receives: Drug: VEN+AZA-5 |
What is being measured
The primary outcome is the main question the study is designed to answer.
The primary outcome measure is the response rate defined as the rate of CR/CRi after up to 6 cycles of therapy (best response).
Bone marrow assessments will be performed at least at screening, at the end of cycle 1, after cycle 4 and after cycle 6 resp. end of treatment (EOT). Criteria for disease status / response assessment follow the ELN-2022 recommendations .
Measured: best response after up to 6 cycles (each cycle is 28 days)
Secondary outcomes (12)
Rate of CR or CRi by the Initiation of Cycle 2
Rate of CR or CRi by the Initiation of Cycle 2. Criteria for disease status / response assessment follow the European LeukemiaNet (ELN) - 2022 recommendations.
Measured: At the end of Cycle 1 (each cycle is 28 days)
Rate of CR with partial hematologic recovery (CRh) after up to 6 cycles of therapy
Rate of CR with partial hematologic recovery (CRh) after up to 6 cycles of therapy. Criteria for disease status / response assessment follow the ELN-2022 recommendations.
Measured: after up to 6 cycles (each cycle is 28 days)
Time from initiation of treatment (C1D1) until achievement of CR or CRi
Time from initiation of treatment (C1D1) until achievement of CR or CRi. Criteria for disease status / response assessment follow the ELN-2022 recommendations.
Measured: from start of treatment (C1D1) until up to 6 cycles (each cycle is 28 days)
Objective response rate
Objective response rate (CR, CRh, CRi, MLFS). Criteria for disease status / response assessment follow the ELN-2022 recommendations.
Measured: at EOT, after up to 6 cycles therapy (each cycle is 28 days)
Event free survival (EFS)
Event free survival (EFS), defined as the number of days from start of treatment to the date of relapse from CR or CRi, treatment failure (i.e., no CR or CRi after 6 cycles (each cycle is 28 days) of therapy or disease progression requiring treatment discontinuation), or death from any cause.
Measured: From start of treatment to the date of relapse from CR or CRi, treatment failure (i.e., no CR or CRi after up to 6 cycles (each cycle is 28 days) of therapy or disease progression requiring treatment discontinuation), or death from any cause.
Overall survival (OS)
Overall survival (OS), defined as the number of days from start of treatment to death from any cause.
After EOT, patients will be further followed up for survival until the end of the trial is reached (= last patient out, i.e. when the last surviving patient has reached the 3-month follow-up visit after EOT). Therefore, the duration of follow-up can differ between patients but is at least 3 months after EOT.
Measured: From start of treatment to date of death from any cause. OS will be assessed until 3 months after end of treatment of the last patient on study.
Descriptive assessment of measurable residual disease (MRD) levels on study treatment, determined by quantitative PCR or targeted next-generation sequencing
Determined by quantitative PCR or targeted next-generation sequencing. Molecular profiling and MRD assessment of all patients will be carried out centrally (Hämatologisches Diagnostiklabor, Universitätsklinikum Leipzig), at least at screening, at the end of cycle 1, after cycle 4 and after cycle 6 resp. end of treatment (EOT).
Measured: From Screening until EOT (after up to 6 cycles (each cycle is 28 days))
Time to treatment discontinuation
Time to treatment discontinuation, defined as the number of days from start of treatment to premature stop of treatment. The stop date is the date the first cycle that was not given should have started as scheduled. This endpoint will be analyzed with death and progression or relapse as competing risk.
Measured: From start of treatment to day of treatment discontinuation (within up to 6 cycles (each cycle is 28 days))
Rate of patients with at least one treatment interruption
Rate of patients with at least one treatment interruption, i.e. a delay of the next cycle, and duration of treatment interruptions.
Measured: From start of treatment until EOT (after up to 6 cycles (each cycle is 28 days))
Delay of subsequent cycles, dose reductions or shortening/interruption of study drug administration
Delay of subsequent cycles, dose reductions or shortening/interruption of study drug administration
Measured: From start of treatment until EOT (after up to 6 cycles (each cycle is 28 days))
Duration of patient hospitalization
Duration of patient hospitalization, defined as days in hospital from start of treatment until EOT.
Measured: From start of treatment until EOT (after up to 6 cycles (each cycle is 28 days))
Quality of life (QoL)
The established cancer-specific Core Quality of Life questionnaire QLQ-C30 from the EORTC (European Organisation for Research) will be used for QoL measurement, at screening and at the beginning of each cycle and at EOT.
Measured: From Screening until EOT (after up to 6 cycles (each cycle is 28 days))
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Apr 2023
- Study start17 May 2023
- Primary completion16 Nov 2025
- Study completionMay 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Acute Myeloid Leukemia (AML)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
- 45 participants (estimated)
Sponsor and investigators
- Lead sponsor
- University of Leipzig
- Sponsor type
- University, hospital or other
- Collaborators
- AbbVie, University Hospital Leipzig, Hematology Diagnostics Laboratory, University of Leipzig, Clinical Trial Centre (ZKS)
- Responsible party
- Principal investigator, Klaus Metzeler, University of Leipzig
- Investigators
- Klaus Metzeler, Prof. Dr., Principal investigator, Universitätsklinikum Leipzig, Klinik und Poliklinik für Hämatologie, Zelltherapie und Hämostaseologie
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05833438
- Sponsor's protocol ID
- VENAZA-5S
- Other IDs
- 2022-501537-23-00
- Acronym
- VENAZA-5S
- Status last verified
- Apr 2026
- First submitted
- 4 Mar 2023
- Last updated
- 1 May 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: Individual participant data that underlie the results reported in publications of this study, after deidentification
Study at a glance
From the registry record, last updated by the sponsor on 1 May 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Single-arm, Pilot Study of Venetoclax in Combination With 5 Days Azacitidine in Treatment-naïve Subjects With Acute Myelogenous Leukemia Who Are ≥18 Years of Age and Not Eligible for Standard Induction Therapy (VENAZA-5S PILOT TRIAL)
- ClinicalTrials.gov ID
- NCT05833438
- Acronym / sponsor ID
- VENAZA-5S / VENAZA-5S
- Conditions
- Acute Myeloid Leukemia (AML)
- Keywords
- Venclyxto, Venetoclax, Azacitidine, Hematologic Diseases, Acute Myelogenous Leukemia
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 45 participants (estimated)
- Lead sponsor
- University of LeipzigUniversity, hospital or other
- Collaborators
- AbbVie, University Hospital Leipzig, Hematology Diagnostics Laboratory, University of Leipzig, Clinical Trial Centre (ZKS)
- Study start
- 17 May 2023 (actual)
- Primary completion
- 16 Nov 2025 (actual)
- Study completion
- May 2026 (estimated)
- First posted
- 27 Apr 2023 (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.
Acute myeloid leukemia (AML): continuous oral Venetoclax (VEN) and 7 days of s.c. Azacitidine (AZA) per 28-day cycle = standard of care for intensive induction therapy ineligible AML patients in Germany
The VENAZA-5S pilot trial: AZA administration reduced to 5 days within each cycle to improve tolerability and treatment adherence due to less neutropenic infections, less treatment interruptions and less hospitalizations.
Detailed description
Acute myeloid leukemia (AML) is a uniformly fatal disease if untreated. The combination of continuous oral Venetoclax (VEN) and 7 days of s.c. Azacitidine (AZA) per 28-day cycle has recently emerged as the new standard of care for AML patient who are ineligible for intensive induction therapy, and has been widely adopted in Germany.
The VENAZA-5S pilot trial aims to reduce the reported hematological toxicity profile of this currently approved combination, while preserving efficacy, by modifying AZA administration to 5 days within each cycle. The hypothesis is that this modification will not interfere with the response rates achieved by the combination, but will rather improve tolerability and treatment adherence due to less neutropenic infections, less treatment interruptions and hospitalizations, and thus result in better quality of life and favorable long-term outcomes in elderly or comorbid AML patients. This single-arm pilot study is intended to generate first data on the efficacy and toxicity of 5 days AZA + VEN, which will be compared to a historical control cohort treated with the current standard of 7 days AZA + VEN.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| VEN+AZA-5 | Experimental | Drug: VEN+AZA-5Azacitidine (AZA) 75 mg/m2, d1-5 of each 28 day cycle (SC) in combination with Venetoclax (VEN): 400 mg daily (orally) |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- Confirmed diagnosis of AML by World Health Organization (WHO) criteria 2016
- Ineligible for treatment with a standard cytarabine and anthracycline induction regimen due to age or comorbidities
- Age ≥ 18 years
- Life expectancy of at least 12 weeks
Key exclusion criteria (9 listed)
- Prior treatment for AML or myelodysplastic syndrome (MDS) with one of the following:
- History of myeloproliferative neoplasm (MPN)
- Diagnosis of acute promyelocytic leukemia (APL)
- Presence of favorable-risk karyotype abnormalities: t(15;17), t(8;21), inv(16) or t(16;16)
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 |
|---|---|---|
| VEN+AZA-5Up to 6 cycles: Azacitidine (AZA) 75 mg/m2, d1-5 of each 28 day cycle (SC) in combination with Venetoclax (VEN): 400 mg daily (orally) | Drug | VEN+AZA-5 |
Primary outcome
The primary outcome measure is the response rate defined as the rate of CR/CRi after up to 6 cycles of therapy (best response).
Time frame: best response after up to 6 cycles (each cycle is 28 days)
Bone marrow assessments will be performed at least at screening, at the end of cycle 1, after cycle 4 and after cycle 6 resp. end of treatment (EOT). Criteria for disease status / response assessment follow the ELN-2022 recommendations .
12 secondary outcomes
Rate of CR or CRi by the Initiation of Cycle 2
Time frame: At the end of Cycle 1 (each cycle is 28 days)
Rate of CR with partial hematologic recovery (CRh) after up to 6 cycles of therapy
Time frame: after up to 6 cycles (each cycle is 28 days)
Time from initiation of treatment (C1D1) until achievement of CR or CRi
Time frame: from start of treatment (C1D1) until up to 6 cycles (each cycle is 28 days)
Objective response rate
Time frame: at EOT, after up to 6 cycles therapy (each cycle is 28 days)
Event free survival (EFS)
Time frame: From start of treatment to the date of relapse from CR or CRi, treatment failure (i.e., no CR or CRi after up to 6 cycles (each cycle is 28 days) of therapy or disease progression requiring treatment discontinuation), or death from any cause.
Overall survival (OS)
Time frame: From start of treatment to date of death from any cause. OS will be assessed until 3 months after end of treatment of the last patient on study.
Descriptive assessment of measurable residual disease (MRD) levels on study treatment, determined by quantitative PCR or targeted next-generation sequencing
Time frame: From Screening until EOT (after up to 6 cycles (each cycle is 28 days))
Time to treatment discontinuation
Time frame: From start of treatment to day of treatment discontinuation (within up to 6 cycles (each cycle is 28 days))
Rate of patients with at least one treatment interruption
Time frame: From start of treatment until EOT (after up to 6 cycles (each cycle is 28 days))
Delay of subsequent cycles, dose reductions or shortening/interruption of study drug administration
Time frame: From start of treatment until EOT (after up to 6 cycles (each cycle is 28 days))
Duration of patient hospitalization
Time frame: From start of treatment until EOT (after up to 6 cycles (each cycle is 28 days))
Quality of life (QoL)
Time frame: From Screening until EOT (after up to 6 cycles (each cycle is 28 days))
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 1 May 2026.
- Individual participant dataSharing planned: Yes. Individual participant data that underlie the results reported in publications of this study, after deidentification
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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.
Klaus Metzeler, Prof. Dr. principal investigator
Universitätsklinikum Leipzig, Klinik und Poliklinik für Hämatologie, Zelltherapie und Hämostaseologie
10 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 VEN+AZA-5 in people with Acute Myeloid Leukemia (AML), run by University of Leipzig at 10 sites 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 4 inclusion and 9 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 1 May 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 10 sites 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?
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?
The primary outcome measure is the response rate defined as the rate of CR/CRi after up to 6 cycles of therapy (best response). (time frame: best response after up to 6 cycles (each cycle is 28 days)). 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 1 May 2026.