Myelodysplastic Syndromes (MDS)Phase 1NCT02942290
A Study Evaluating Venetoclax in Combination With Azacitidine in Participants With Treatment-Naïve Higher-Risk Myelodysplastic Syndromes (MDS)
A Phase 1 study testing Azacitidine and Venetoclax in people with Myelodysplastic Syndromes (MDS), run by AbbVie at 37 sites in 7 countries.
Trial summary
This is a Phase 1b, open-label, non-randomized, multicenter, dose-finding study evaluating venetoclax in combination with azacitidine in participants with treatment-naïve higher-risk MDS comprising a dose-escalation portion and a safety expansion portion. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Myelodysplastic Syndromes (MDS)
- What is being tested?
- Azacitidine and Venetoclax
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 134 participants
- Where
- 37 sitesin 7 countries
- Study dates
- 12 Jan 2017to 15 Apr 2026
- Phase
- Phase 1
- 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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Participant must have documented diagnosis of untreated de novo MDS with:
- International Prognostic Scoring System (IPSS) risk categories Int-2 or High (minimum IPSS overall score of 1.5) OR Revised IPSS (IPSS-R) categories intermediate, high or very high (score of > 3) and
- Presence of less than 20% bone marrow blasts per bone marrow biopsy/aspirate.
- Eastern Cooperative Oncology Group (ECOG) performance score of less than or equal to 2.
Who cannot join
- Participant has received prior therapy for MDS. (Prior supportive care in form of transfusions or growth factors, etc., is not considered prior therapy).
- Participant has received prior therapy with a BCL-2 Homology 3 (BH3) mimetic.
- Participant has a diagnosis other than previously untreated de novo MDS (as defined in the protocol) including:
- MDS with IPSS risk categories Low or Int-1 (overall IPSS score < 1.5)
- Therapy-related MDS (t-MDS).
- MDS evolving from a pre-existing myeloproliferative neoplasm (MPN).
Show all 9Show fewer
- MDS/MPN including chronic myelomonocytic leukemia (CMML), atypical chronic myeloid leukemia (CML), juvenile myelomonocytic leukemia (JMML) and unclassifiable MDS/MPN.
- Participant has received allogeneic Hematopoietic Stem Cell Transplantation (HSCT) or solid organ transplantation.
- Participant has received a live attenuated vaccine within 4 weeks prior to the first dose of study drug.
Trial locations and contacts
37 sites in 7 countries.
37 sites in 7 countries
United States13 sites
University of Maryland, Baltimore /ID# 153669
Baltimore, Maryland, 21201
Tufts Medical Center /ID# 153672
Boston, Massachusetts, 02111-1552
Dana-Farber Cancer Institute /ID# 152735
Boston, Massachusetts, 02215
The University of Chicago Medical Center /ID# 153673
Chicago, Illinois, 60637-1443
UT MD Anderson Cancer Center /ID# 153809
Houston, Texas, 77030
Tennessee Oncology-Nashville Centennial /ID# 222769
Nashville, Tennessee, 37203-1632
Vanderbilt University Medical Center /ID# 152738
Nashville, Tennessee, 37232-0011
Columbia University Medical Center /ID# 153661
New York, New York, 10032-3729
Weill Cornell Medical College /ID# 155524
New York, New York, 10065
University of Pittsburgh MC /ID# 153662
Pittsburgh, Pennsylvania, 15260
Oregon Medical Research Center /ID# 152734
Portland, Oregon, 97239
Washington University-School of Medicine /ID# 153671
St Louis, Missouri, 63110
Duplicate_University of Arizona Cancer Center - North Campus /ID# 154155
Tucson, Arizona, 85719-1478
Australia9 sites
Concord Repatriation General Hospital /ID# 154958
Concord, New South Wales, 2139
Duplicate_St. Vincent's Hospital, Darlinghurst /ID# 222846
Darlinghurst, New South Wales, 2010
Austin Health /ID# 154955
Heidelberg, Victoria, 3084
St George Hospital /ID# 154954
Kogarah, New South Wales, 2217
Liverpool Hospital /ID# 222410
Liverpool, New South Wales, 2170
The Alfred Hospital /ID# 154956
Melbourne, Victoria, 3004
Fiona Stanley Hospital /ID# 222847
Murdoch, Western Australia, 6150
Calvary Mater Newcastle /ID# 154957
Waratah, New South Wales, 2298
Duplicate_Princess Alexandra Hospital /ID# 154990
Woolloongabba, Queensland, 4102
Germany6 sites
Universitaetsklinikum Koeln /ID# 153141
Cologne, North Rhine-Westphalia, 50937
Duplicate_Universitaetsklinikum Carl Gus /ID# 153958
Dresden, Saxony, 01307
Universitaetsklinikum Halle (Saale) /ID# 153760
Halle, 06120
Universitaetsklinikum Leipzig /ID# 153142
Leipzig, Saxony, 04103
Universitatsklinikum Mannheim /ID# 153140
Mannheim, Baden-Wurttemberg, 68167
Klinikum rechts der Isar /ID# 153139
Munich, Bavaria, 81675
United Kingdom4 sites
University Hospitals Birmingham NHS Foundation Trust /ID# 158810
Birmingham, B15 2TH
King's College Hospital NHS Foundation Trust /ID# 156489
London, SE5 9RS
Norfolk and Norwich University Hospitals NHS Foundation Trust /ID# 156492
Norwich, Norfolk, NR4 7UY
Oxford University Hospitals NHS Foundation Trust /ID# 222567
Oxford, Oxfordshire, OX3 9DU
France2 sites
Centre Hospitalier Universitaire de Nantes, Hotel Dieu -HME /ID# 153828
Nantes, Pays de la Loire Region, 44000
Hôpital Saint-Louis /ID# 153827
Paris, 75010
Italy2 sites
Duplicate_IRCCS AOU di Bologna - Policlinico Sant'Orsola-Malpighi /ID# 153763
Bologna, Emilia-Romagna, 40138
Duplicate_Azienda Ospedaliero-Universitaria Policlinico Umberto I /ID# 153764
Rome, Roma, 00161
About this study
Official title: A Phase 1b Dose Escalation Study Evaluating the Safety and Pharmacokinetics of Venetoclax in Combination With Azacitidine in Subjects With Treatment-Naïve Higher-Risk Myelodysplastic Syndromes (MDS)
This is a Phase 1b, open-label, non-randomized, multicenter, dose-finding study evaluating venetoclax in combination with azacitidine in participants with treatment-naïve higher-risk MDS comprising a dose-escalation portion and a safety expansion portion.
- Conditions
- Myelodysplastic Syndromes (MDS)
- Being tested
- Azacitidine, Venetoclax
- Keywords
- Higher-Risk (HR) Myelodysplastic Syndromes (MDS), Pharmacokinetic, Venetoclax, Azacitidine, Acute Myelogenous Leukemia, Myelodysplastic Syndromes (MDS), Treatment-Naïve Higher-Risk
- Medical subject headings
- Myelodysplastic Syndromes, Leukemia, Myeloid, Acute
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AzacitidineDrug
Powder for injection; taken subcutaneously (SC) or intravenous (IV); Administered on Days 1-7 of 28 days cycle or Days 1-5 of Week 1 \& Days 1-2 of Week 2 of 28 day cycle.
VenetoclaxDrug
Also called ABT-199, GDC-0199, VENCLEXTA
Oral; Tablet
| Study group | Role | What they receive |
|---|---|---|
| Venetoclax + Azacitidine | Experimental | Not described Receives: Drug: Azacitidine; Drug: Venetoclax |
What is being measured
The primary outcome is the main question the study is designed to answer.
AUCt for Azacitidine
Area under the plasma concentration-time curve (AUC) from 0 to the time of the last measurable concentration (AUCt) for azacitidine.
Measured: Up to 32 days
Cmax of venetoclax
Maximum plasma concentration (Cmax) of venetoclax.
Measured: Up to 32 days
AUCt for venetoclax
Area under the plasma concentration-time curve (AUC) from 0 to the time of the last measurable concentration (AUCt) for venetoclax.
Measured: Up to 32 days
Tmax of venetoclax
Time to Cmax (peak time, Tmax) of venetoclax.
Measured: Up to 32 days
AUC[0 to infinity] for azacitidine
Area under the plasma concentration-time curve from Time 0 to infinite time.
Measured: Up to 32 days
Recommended Phase 2 dose (RPTD) and dosing schedule of venetoclax in combination with azacitidine
The RPTD of venetoclax [co-administered venetoclax and azacitidine] will be determined during the dose escalation phase of the study. RPTD will be determined using available safety and pharmacokinetics data [upon completion of the dose escalation phase].
Measured: Measured from Day 1 until Day 28 per dose level.
Half-life (t[1/2]) for azacitidine
Terminal elimination half-life (t[1/2]) for azacitidine.
Measured: Up to 32 days
Cmax for azacitidine
Maximum plasma concentration (Cmax) of azacitidine.
Measured: Up to 32 days
AUC[0-24] for venetoclax
AUC over a 24-hour dose interval (AUC[0-24]) for venetoclax.
Measured: Up to 32 days
Clearance (CL) for azacitidine
Clearance is defined as the volume of plasma cleared of the drug per unit time.
Measured: Up to 32 days
Tmax for azacitidine
Time to Cmax (peak time, Tmax) of azacitidine.
Measured: Up to 32 days
Complete Remission (CR) Rate
Complete remission rate will be defined as the proportion of participants who achieved a complete response per the International Working Group (IWG) 2006 criteria for Myelodysplastic Syndromes (MDS).
Measured: Measured from Cycle 1 Day 1 as long as the participant continues to benefit, or until the occurrence of unacceptable toxicity, death, exercise of investigator discretion, or withdrawal of consent, and for an anticipated maximum duration of 24 months.
Secondary outcomes (18)
Rate of red blood cell (RBC) transfusion independence
Percentages of participants who become RBC transfusion-independent.
Measured: Measured from Cycle 1 Day 1 as long as the participant continues to benefit, or until the occurrence of unacceptable toxicity, death, exercise of investigator discretion, or withdrawal of consent, and for an anticipated maximum duration of 24 months.
Progression-Free Survival (PFS)
PFS is defined as the number of days from the date of the first dose of study drug to the date of earliest disease progression or death due to disease progression or febrile neutropenia.
Measured: Measured from the date of first dose of study drug to the date of earliest disease progression or death due to disease progression or febrile neutropenia, and for an anticipated maximum duration of 24 months.
Overall Survival (OS)
OS is defined as number of days from the date of first dose of the study drug to the date of death of any cause.
Measured: Measured from the date of first dose of study drug to the date of death, and for up to 5 years after the last participant is enrolled.
Hematologic Improvement (HI) rate
Percentages of participants with HI (erythroid/platelet/neutrophil responses).
Measured: Measured from Cycle 1 Day 1 as long as the participant continues to benefit, or until the occurrence of unacceptable toxicity, death, exercise of investigator discretion, or withdrawal of consent, and for an anticipated maximum duration of 24 months.
Rate of platelet (PLT) transfusion independence
Percentages of participants who become platelet transfusion-independent.
Measured: Measured from Cycle 1 Day 1 as long as the participant continues to benefit, or until the occurrence of unacceptable toxicity, death, exercise of investigator discretion, or withdrawal of consent, and for an anticipated maximum duration of 24 months.
Event-Free Survival (EFS)
Event-free survival (EFS) will be defined as the number of days from the date of the first dose of study drug to the date of earliest disease progression or death of any cause.
Measured: Measured from the date of the first dose of study drug to the date of earliest disease progression, death of any cause and for up to 5 yrs after the last participant is enrolled
Time to transformation to acute myeloid leukemia (AML)
Defined as the number of days from the date of the first dose of study drug to the date of documented AML transformation.
Measured: Measured from the date of first dose of study drug to date of documented AML transformation, defined as the presence of blast count greater than or equal to 20% in either peripheral blood or bone marrow for an anticipated maximum duration of 24 months.
Overall Response Rate (OR)
OR (equals the rates of complete remission [CR] + partial remission [PR]) of venetoclax in combination with azacitidine.
Measured: Measured from Cycle 1 Day 1 as long as the participant continues to benefit, or until the occurrence of unacceptable toxicity, death, exercise of investigator discretion, or withdrawal of consent and for an anticipated maximum duration of 24 months.
Time to next treatment (TTNT)
Time to next treatment (TTNT) will be defined as the time from the first dose of study drug to start of new non-protocol specified MDS therapy or death from any cause.
Measured: Measured from the first dose of study drug to start of new non-protocol specified MDS therapy, and for up to 5 years after the last participant is enrolled.
Marrow Complete Remission (mCR) Rate
Defined as the proportion of participants who achieved a marrow complete response with or without hematological improvement per the International Working Group (IWG) 2006 criteria for Myelodysplastic Syndromes.
Measured: Measured from Cycle 1 Day 1 as long as the participant continues to benefit, or until the occurrence of unacceptable toxicity, death, exercise of investigator discretion, or withdrawal of consent, and for an anticipated maximum duration of 24 months.
Modified Overall Response Rate (mOR)
mOR (equals CR + PR + mCR) of venetoclax in combination with azacitidine.
Measured: Measured from Cycle 1 Day 1 as long as the participant continues to benefit, or until the occurrence of unacceptable toxicity, death, exercise of investigator discretion, or withdrawal of consent and for an anticipated maximum duration of 24 months.
Duration of CR
Duration of CR will be defined as the number of days from the date of first response CR to the earliest documentation of progressive disease or death of any cause, whichever occurs earlier.
Measured: Measured from Cycle 1 Day 1 as long as the participant continues to benefit, or until the occurrence of unacceptable toxicity, death, exercise of investigator discretion, or withdrawal of consent, and for an anticipated maximum duration of 24 months.
Duration of mOR
Duration of response (mOR) will be defined as the number of days from the date of first response (CR, PR or mCR) to the earliest documentation of progressive disease or death of any cause, whichever occurs earlier.
Measured: Measured from Cycle 1 Day 1 as long as the participant continues to benefit, or until the occurrence of unacceptable toxicity, death, exercise of investigator discretion, or withdrawal of consent and for an anticipated maximum duration of 24 months.
Duration of OR
Duration of response (OR) will be defined as the number of days from the date of first response (CR or PR) to the earliest documentation of progressive disease or death of any cause, whichever occurs earlier.
Measured: Measured from Cycle 1 Day 1 as long as the participant continues to benefit, or until the occurrence of unacceptable toxicity, death, exercise of investigator discretion, or withdrawal of consent and for an anticipated maximum duration of 24 months.
Rate of AML transformation
The AML transformation rate is defined as the proportion of participants transformed to Acute Myelogenous Leukemia.
Measured: Measured from the date of first dose of study drug to date of documented AML transformation, defined as the presence of blast count greater than or equal to 20% in either peripheral blood or bone marrow for an anticipated maximum duration of 24 months.
Time to First Response (CR)
Time to first response (CR) will be defined as the number of days from the date of first dose of the study drug to the date of the first response of CR.
Measured: Measured from Cycle 1 Day 1 as long as the participant continues to benefit, or until the occurrence of unacceptable toxicity, death, exercise of investigator discretion, or withdrawal of consent, and for an anticipated maximum duration of 24 months.
Time to First Response (mOR)
Time to first response (mOR) will be defined as the number of days from the date of first dose of the study drug to the date of the first response of (CR, PR, or mCR).
Measured: Measured from Cycle 1 Day 1 as long as the participant continues to benefit, or until the occurrence of unacceptable toxicity, death, exercise of investigator discretion, or withdrawal of consent and for an anticipated maximum duration of 24 months.
Time to First Response (OR)
Time to first response (OR) will be defined as the number of days from the date of first dose of the study drug to the date of the first response of (CR or PR).
Measured: Measured from Cycle 1 Day 1 as long as the participant continues to benefit, or until the occurrence of unacceptable toxicity, death, exercise of investigator discretion, or withdrawal of consent and for an anticipated maximum duration of 24 months.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Oct 2016 (estimated)
- Study start12 Jan 2017
- Primary completion15 Apr 2026
- Study completion15 Apr 2026
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 Myelodysplastic Syndromes (MDS)Studies of the same condition, open and under way.
- More trials run by AbbVieThe 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 1
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 134 participants (actual)
Sponsor and investigators
- Lead sponsor
- AbbVie
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Genentech, Inc.
- Responsible party
- Sponsor
- Investigators
- ABBVIE INC., Study director, AbbVie
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02942290
- Sponsor's protocol ID
- M15-531
- Other IDs
- 2016-001657-41, 2023-507154-32-00, 2023, 2016
- Status last verified
- Aug 2025
- First submitted
- 18 Oct 2016
- Last updated
- 12 Aug 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Garcia JS, Platzbecker U, Odenike O, Fleming S, Fong CY, Borate U, Jacoby MA, Nowak D, Baer MR, Peterlin P, Chyla B, Wang H, Ku G, Hoffman D, Potluri J, Garcia-Manero G. Efficacy and safety of venetoclax plus azacitidine for patients with treatment-naive high-risk myelodysplastic syndromes. Blood. 2025 Mar 13;145(11):1126-1135. doi: 10.1182/blood.2024025464.
PubMed 39652823
Study at a glance
From the registry record, last updated by the sponsor on 12 Aug 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1b Dose Escalation Study Evaluating the Safety and Pharmacokinetics of Venetoclax in Combination With Azacitidine in Subjects With Treatment-Naïve Higher-Risk Myelodysplastic Syndromes (MDS)
- ClinicalTrials.gov ID
- NCT02942290
- Acronym / sponsor ID
- M15-531
- Conditions
- Myelodysplastic Syndromes (MDS)
- Keywords
- Higher-Risk (HR) Myelodysplastic Syndromes (MDS), Pharmacokinetic, Venetoclax, Azacitidine, Acute Myelogenous Leukemia, Myelodysplastic Syndromes (MDS), Treatment-Naïve Higher-Risk
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 134 participants (actual)
- Lead sponsor
- AbbVieIndustry (pharma, biotech, devices)
- Collaborators
- Genentech, Inc.
- Study start
- 12 Jan 2017 (actual)
- Primary completion
- 15 Apr 2026 (actual)
- Study completion
- 15 Apr 2026 (actual)
- First posted
- 24 Oct 2016 (estimated)
- 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.
This is a Phase 1b, open-label, non-randomized, multicenter, dose-finding study evaluating venetoclax in combination with azacitidine in participants with treatment-naïve higher-risk MDS comprising a dose-escalation portion and a safety expansion portion.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Venetoclax + Azacitidine | Experimental | Drug: Azacitidine, Drug: Venetoclax |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- Participant must have documented diagnosis of untreated de novo MDS with:
- Eastern Cooperative Oncology Group (ECOG) performance score of less than or equal to 2.
Key exclusion criteria (9 listed)
- Participant has received prior therapy for MDS. (Prior supportive care in form of transfusions or growth factors, etc., is not considered prior therapy).
- Participant has received prior therapy with a BCL-2 Homology 3 (BH3) mimetic.
- Participant has a diagnosis other than previously untreated de novo MDS (as defined in the protocol) including:
- Participant has received allogeneic Hematopoietic Stem Cell Transplantation (HSCT) or solid organ transplantation.
- Participant has received a live attenuated vaccine within 4 weeks prior to the first dose of study drug.
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 |
|---|---|---|
| AzacitidinePowder for injection; taken subcutaneously (SC) or intravenous (IV); Administered on Days 1-7 of 28 days cycle or Days 1-5 of Week 1 \& Days 1-2 of Week 2 of 28 day cycle. | Drug | Venetoclax + Azacitidine |
| VenetoclaxAlso: ABT-199, GDC-0199, VENCLEXTAOral; Tablet | Drug | Venetoclax + Azacitidine |
Primary outcome
AUCt for Azacitidine
Time frame: Up to 32 days
Area under the plasma concentration-time curve (AUC) from 0 to the time of the last measurable concentration (AUCt) for azacitidine.
Cmax of venetoclax
Time frame: Up to 32 days
Maximum plasma concentration (Cmax) of venetoclax.
AUCt for venetoclax
Time frame: Up to 32 days
Area under the plasma concentration-time curve (AUC) from 0 to the time of the last measurable concentration (AUCt) for venetoclax.
Tmax of venetoclax
Time frame: Up to 32 days
Time to Cmax (peak time, Tmax) of venetoclax.
AUC[0 to infinity] for azacitidine
Time frame: Up to 32 days
Area under the plasma concentration-time curve from Time 0 to infinite time.
Recommended Phase 2 dose (RPTD) and dosing schedule of venetoclax in combination with azacitidine
Time frame: Measured from Day 1 until Day 28 per dose level.
The RPTD of venetoclax [co-administered venetoclax and azacitidine] will be determined during the dose escalation phase of the study. RPTD will be determined using available safety and pharmacokinetics data [upon completion of the dose escalation phase].
Half-life (t[1/2]) for azacitidine
Time frame: Up to 32 days
Terminal elimination half-life (t[1/2]) for azacitidine.
Cmax for azacitidine
Time frame: Up to 32 days
Maximum plasma concentration (Cmax) of azacitidine.
AUC[0-24] for venetoclax
Time frame: Up to 32 days
AUC over a 24-hour dose interval (AUC[0-24]) for venetoclax.
Clearance (CL) for azacitidine
Time frame: Up to 32 days
Clearance is defined as the volume of plasma cleared of the drug per unit time.
Tmax for azacitidine
Time frame: Up to 32 days
Time to Cmax (peak time, Tmax) of azacitidine.
Complete Remission (CR) Rate
Time frame: Measured from Cycle 1 Day 1 as long as the participant continues to benefit, or until the occurrence of unacceptable toxicity, death, exercise of investigator discretion, or withdrawal of consent, and for an anticipated maximum duration of 24 months.
Complete remission rate will be defined as the proportion of participants who achieved a complete response per the International Working Group (IWG) 2006 criteria for Myelodysplastic Syndromes (MDS).
18 secondary outcomes
Rate of red blood cell (RBC) transfusion independence
Time frame: Measured from Cycle 1 Day 1 as long as the participant continues to benefit, or until the occurrence of unacceptable toxicity, death, exercise of investigator discretion, or withdrawal of consent, and for an anticipated maximum duration of 24 months.
Progression-Free Survival (PFS)
Time frame: Measured from the date of first dose of study drug to the date of earliest disease progression or death due to disease progression or febrile neutropenia, and for an anticipated maximum duration of 24 months.
Overall Survival (OS)
Time frame: Measured from the date of first dose of study drug to the date of death, and for up to 5 years after the last participant is enrolled.
Hematologic Improvement (HI) rate
Time frame: Measured from Cycle 1 Day 1 as long as the participant continues to benefit, or until the occurrence of unacceptable toxicity, death, exercise of investigator discretion, or withdrawal of consent, and for an anticipated maximum duration of 24 months.
Rate of platelet (PLT) transfusion independence
Time frame: Measured from Cycle 1 Day 1 as long as the participant continues to benefit, or until the occurrence of unacceptable toxicity, death, exercise of investigator discretion, or withdrawal of consent, and for an anticipated maximum duration of 24 months.
Event-Free Survival (EFS)
Time frame: Measured from the date of the first dose of study drug to the date of earliest disease progression, death of any cause and for up to 5 yrs after the last participant is enrolled
Time to transformation to acute myeloid leukemia (AML)
Time frame: Measured from the date of first dose of study drug to date of documented AML transformation, defined as the presence of blast count greater than or equal to 20% in either peripheral blood or bone marrow for an anticipated maximum duration of 24 months.
Overall Response Rate (OR)
Time frame: Measured from Cycle 1 Day 1 as long as the participant continues to benefit, or until the occurrence of unacceptable toxicity, death, exercise of investigator discretion, or withdrawal of consent and for an anticipated maximum duration of 24 months.
Time to next treatment (TTNT)
Time frame: Measured from the first dose of study drug to start of new non-protocol specified MDS therapy, and for up to 5 years after the last participant is enrolled.
Marrow Complete Remission (mCR) Rate
Time frame: Measured from Cycle 1 Day 1 as long as the participant continues to benefit, or until the occurrence of unacceptable toxicity, death, exercise of investigator discretion, or withdrawal of consent, and for an anticipated maximum duration of 24 months.
Modified Overall Response Rate (mOR)
Time frame: Measured from Cycle 1 Day 1 as long as the participant continues to benefit, or until the occurrence of unacceptable toxicity, death, exercise of investigator discretion, or withdrawal of consent and for an anticipated maximum duration of 24 months.
Duration of CR
Time frame: Measured from Cycle 1 Day 1 as long as the participant continues to benefit, or until the occurrence of unacceptable toxicity, death, exercise of investigator discretion, or withdrawal of consent, and for an anticipated maximum duration of 24 months.
Duration of mOR
Time frame: Measured from Cycle 1 Day 1 as long as the participant continues to benefit, or until the occurrence of unacceptable toxicity, death, exercise of investigator discretion, or withdrawal of consent and for an anticipated maximum duration of 24 months.
Duration of OR
Time frame: Measured from Cycle 1 Day 1 as long as the participant continues to benefit, or until the occurrence of unacceptable toxicity, death, exercise of investigator discretion, or withdrawal of consent and for an anticipated maximum duration of 24 months.
Rate of AML transformation
Time frame: Measured from the date of first dose of study drug to date of documented AML transformation, defined as the presence of blast count greater than or equal to 20% in either peripheral blood or bone marrow for an anticipated maximum duration of 24 months.
Time to First Response (CR)
Time frame: Measured from Cycle 1 Day 1 as long as the participant continues to benefit, or until the occurrence of unacceptable toxicity, death, exercise of investigator discretion, or withdrawal of consent, and for an anticipated maximum duration of 24 months.
Time to First Response (mOR)
Time frame: Measured from Cycle 1 Day 1 as long as the participant continues to benefit, or until the occurrence of unacceptable toxicity, death, exercise of investigator discretion, or withdrawal of consent and for an anticipated maximum duration of 24 months.
Time to First Response (OR)
Time frame: Measured from Cycle 1 Day 1 as long as the participant continues to benefit, or until the occurrence of unacceptable toxicity, death, exercise of investigator discretion, or withdrawal of consent and for an anticipated maximum duration of 24 months.
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 12 Aug 2026.
- Derived from this study: PubMed 39652823Garcia JS, Platzbecker U, Odenike O, Fleming S, Fong CY, Borate U, Jacoby MA, Nowak D, Baer MR, Peterlin P, Chyla B, Wang H, Ku G, Hoffman D, Potluri J, Garcia-Manero G. Efficacy and safety of venetoclax plus azacitidine for patients with treatment-naive high-risk myelodysplastic syndromes. Blood. 2025 Mar 13;145(11):1126-1135. doi: 10.1182/blood.2024025464.
- This clinical study may be evaluating a usage that is not currently FDA approved. Please see US Prescribing Information for approved uses.Link provided by the sponsor.
- Individual participant dataSharing planned: No
Related trials for Myelodysplastic Syndromes (MDS)
RecruitingPhase 2
Testing New Therapies for Patients With Acute Myeloid Leukemia Who Are Newly Diagnosed and Have Not Yet Started Treatment (A MyeloMATCH Treatment Trial)
Acute Myeloid Leukemia, Acute Myeloid Leukemia Arising From Previous Myelodysplastic/Myeloproliferative Neoplasm
Active, not recruitingPhase 1/2
Omacetaxine + Azacitidine in Untreated Patients With High Grade MDS
High Grade Myelodysplastic Syndromes
RecruitingPhase 1/2
Pacritinib, a Kinase Inhibitor of CSF1R, IRAK1, JAK2, and FLT3, in Adults and Pediatric Participants 12 Years of Age or Older With Myelodysplastic Syndromes or Myelodysplastic/Myeloproliferative Neoplasms
Myelodysplastic Syndromes
Active, not recruitingPhase 2
Clofarabine and Melphalan Before Donor Stem Cell Transplant in Treating Patients With Myelodysplasia, Acute Leukemia in Remission, or Chronic Myelomonocytic Leukemia
Adult Acute Lymphoblastic Leukemia in Remission, Acute Myeloid Leukemia Arising From Previous Myelodysplastic Syndrome
Not yet recruitingPhase 1
ATRN-119 in Combination With Decitabine in Patients With TP53-Mutated AML or Higher-Risk MDS
Acute Myeloid Leukemia, High-risk Myelodysplastic Syndrome
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.
ABBVIE INC. study director
AbbVie
37 sites in 7 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Azacitidine and Venetoclax in people with Myelodysplastic Syndromes (MDS), run by AbbVie at 37 sites in 7 countries.
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: finished, as of the sponsor's last update on 12 Aug 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 37 sites in United States, Australia, Germany, United Kingdom, France and 2 more countries. 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 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.
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?
AUCt for Azacitidine (time frame: Up to 32 days). The study lists 12 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 NCT02942290, 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 12 Aug 2026.