A Study Evaluating Venetoclax in Combination With Azacitidine in Participants With Treatment-Naïve Higher-Risk Myelodysplastic Syndromes (MDS)

NCT ID: NCT02942290

Last Updated: 2025-08-05

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

ACTIVE_NOT_RECRUITING

Clinical Phase

PHASE1

Total Enrollment

129 participants

Study Classification

INTERVENTIONAL

Study Start Date

2017-01-12

Study Completion Date

2027-01-31

Brief Summary

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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.

Detailed Description

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Conditions

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Myelodysplastic Syndromes (MDS)

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Venetoclax + Azacitidine

Group Type EXPERIMENTAL

Azacitidine

Intervention Type DRUG

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.

Venetoclax

Intervention Type DRUG

Oral; Tablet

Interventions

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Azacitidine

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.

Intervention Type DRUG

Venetoclax

Oral; Tablet

Intervention Type DRUG

Other Intervention Names

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ABT-199 GDC-0199 VENCLEXTA

Eligibility Criteria

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Inclusion Criteria

* 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.

Exclusion Criteria

* 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).
* 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.
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Genentech, Inc.

INDUSTRY

Sponsor Role collaborator

AbbVie

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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ABBVIE INC.

Role: STUDY_DIRECTOR

AbbVie

Locations

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Duplicate_University of Arizona Cancer Center - North Campus /ID# 154155

Tucson, Arizona, United States

Site Status

The University of Chicago Medical Center /ID# 153673

Chicago, Illinois, United States

Site Status

University of Maryland, Baltimore /ID# 153669

Baltimore, Maryland, United States

Site Status

Tufts Medical Center /ID# 153672

Boston, Massachusetts, United States

Site Status

Dana-Farber Cancer Institute /ID# 152735

Boston, Massachusetts, United States

Site Status

Washington University-School of Medicine /ID# 153671

St Louis, Missouri, United States

Site Status

Columbia University Medical Center /ID# 153661

New York, New York, United States

Site Status

Weill Cornell Medical College /ID# 155524

New York, New York, United States

Site Status

Oregon Medical Research Center /ID# 152734

Portland, Oregon, United States

Site Status

University of Pittsburgh MC /ID# 153662

Pittsburgh, Pennsylvania, United States

Site Status

Tennessee Oncology-Nashville Centennial /ID# 222769

Nashville, Tennessee, United States

Site Status

Vanderbilt University Medical Center /ID# 152738

Nashville, Tennessee, United States

Site Status

UT MD Anderson Cancer Center /ID# 153809

Houston, Texas, United States

Site Status

Concord Repatriation General Hospital /ID# 154958

Concord, New South Wales, Australia

Site Status

Duplicate_St. Vincent's Hospital, Darlinghurst /ID# 222846

Darlinghurst, New South Wales, Australia

Site Status

St George Hospital /ID# 154954

Kogarah, New South Wales, Australia

Site Status

Liverpool Hospital /ID# 222410

Liverpool, New South Wales, Australia

Site Status

Calvary Mater Newcastle /ID# 154957

Waratah, New South Wales, Australia

Site Status

Duplicate_Princess Alexandra Hospital /ID# 154990

Woolloongabba, Queensland, Australia

Site Status

Austin Health /ID# 154955

Heidelberg, Victoria, Australia

Site Status

The Alfred Hospital /ID# 154956

Melbourne, Victoria, Australia

Site Status

Fiona Stanley Hospital /ID# 222847

Murdoch, Western Australia, Australia

Site Status

Juravinski Cancer Centre /ID# 152947

Hamilton, Ontario, Canada

Site Status

Centre Hospitalier Universitaire de Nantes, Hotel Dieu -HME /ID# 153828

Nantes, Pays de la Loire Region, France

Site Status

Hôpital Saint-Louis /ID# 153827

Paris, , France

Site Status

Universitatsklinikum Mannheim /ID# 153140

Mannheim, Baden-Wurttemberg, Germany

Site Status

Klinikum rechts der Isar /ID# 153139

Munich, Bavaria, Germany

Site Status

Universitaetsklinikum Koeln /ID# 153141

Cologne, North Rhine-Westphalia, Germany

Site Status

Duplicate_Universitaetsklinikum Carl Gus /ID# 153958

Dresden, Saxony, Germany

Site Status

Universitaetsklinikum Leipzig /ID# 153142

Leipzig, Saxony, Germany

Site Status

Universitaetsklinikum Halle (Saale) /ID# 153760

Halle, , Germany

Site Status

Duplicate_IRCCS AOU di Bologna - Policlinico Sant'Orsola-Malpighi /ID# 153763

Bologna, Emilia-Romagna, Italy

Site Status

Duplicate_Azienda Ospedaliero-Universitaria Policlinico Umberto I /ID# 153764

Rome, Roma, Italy

Site Status

Norfolk and Norwich University Hospitals NHS Foundation Trust /ID# 156492

Norwich, Norfolk, United Kingdom

Site Status

Oxford University Hospitals NHS Foundation Trust /ID# 222567

Oxford, Oxfordshire, United Kingdom

Site Status

University Hospitals Birmingham NHS Foundation Trust /ID# 158810

Birmingham, , United Kingdom

Site Status

King's College Hospital NHS Foundation Trust /ID# 156489

London, , United Kingdom

Site Status

Countries

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United States Australia Canada France Germany Italy United Kingdom

References

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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.

Reference Type DERIVED
PMID: 39652823 (View on PubMed)

Related Links

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http://rxabbvie.com/

This clinical study may be evaluating a usage that is not currently FDA approved. Please see US Prescribing Information for approved uses.

Other Identifiers

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2016-001657-41

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

2023-507154-32-00

Identifier Type: OTHER

Identifier Source: secondary_id

M15-531

Identifier Type: -

Identifier Source: org_study_id

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