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A Phase 1b Study Evaluating the Safety, Pharmacokinetics and Efficacy of Venetoclax as a Single-Agent and in Combination with Azacitidine in Subjects with Higher-Risk Myelodysplatic Syndromes after Hypomethyllating Agent-failure

Status: Active (Recruitment Closed)

Purpose / Objectives

Primary Outcome

  • Assess the safety profile and PK of venetoclax as a single-agent and in combination with azacitidine
  • Determine the recommended dose (RD) and dosing schedule of venetoclax as a single-agent and in combination with azacitidine
  • Determine the overall response rate (ORR = sum of rates of complete remission [CR]

Secondary Outcomes

  • Rate of complete remission (CR)
  • Rate of hematologic improvement (HI: erythroid/platelet/neutrophil responses)
  • Rate of red blood cell (RBC) transfusion independence
  • Rate of platelet (PLT) transfusion independence
  • Rate of cytogenetic response
  • Rate of bone marrow blast response
  • Time to transformation to acute myelogenous leukemia (AML)
  • Additional secondary outcome measures (duration of response [DOR], overall survival [OS],
    progression-free survival [PFS], time to next MDS treatment [TTNT], event-free survival
  • [...]


high risk MDs

Patient attributes


after HMA failure



Inclusion criteria

  • Subject must voluntarily sign and date an informed consent, approved by an Independent Ethics Committee (IEC)/Institutional Review Board (IRB), prior to the initiation of any screening or studyspecific procedures.
  • Subject must be ≥ 18 years of age.
  • Subject must have documented diagnosis and classification of MDS with application of the International Prognostic Scoring System (IPSS) as HR MDS at the time of first diagnosis with IPSS risk categories Int-2 or High (IPSS overall score ≥ 1.5).
  • Subjects must have presence of ≥ 5% and < 30% bone marrow blasts per bone marrow biopsy/aspirate at screening.
  • Subject must have an Eastern Cooperative Oncology Group (ECOG) performance score of ≤ 2.
  • Subject must have failure of prior therapy with HMA (azacitidine or decitabine) as first-line MDS treatment.
  • [...]

Exclusion criteria

  • Subject had lower-risk MDS with IPSS risk categories Low or Int-1 (overall IPSS score < 1.5) at the time of first diagnosis.
  • Subject has therapy-related MDS (t-MDS).
  • Subject has MDS evolving from a pre-existing myeloproliferative neoplasm (MPN).
  • Subject has MDS/MPN including chronic myelomonocytic leukemia (CMML), atypical chronic myelogenous leukemia (CML), juvenile myelomonocytic leukemia (JMML) and unclassifiable MDS/MPN.
  • Subject has received more than 1prior therapy. Note: Supportive care for MDS (e.g., transfusions, growth factors, agents intended to improve blood count) is not considered a prior therapy.
  • Subject has received therapy other than azacitidine or decitabine for the treatment of MDS.
  • Subject has received prior therapy with a BH3 mimetic.
  • Subject has received hematopoietic growth factors, agents intended to improve blood counts (e.g., erythropoietin, eltrombopag), or corticosteroids within 14 days prior to the first dose of study drug.
  • [...]

Trial design

  • Phase I
  • Multicenter
  • Prospective
  • Cohort
  • Open Label


Venetoclax 400 mg qd, ramp-up from Day 1 to Day 3 of Cycle 1

  • Cycle 1: Day 1, 100 mg; Day 2, 200 mg; Days 3 – 28, 400 mg
  • Cycle 2 through treatment discontinuation: 400 mg qd

Venetoclax 800 mg qd, ramp-up from Day 1 to Day 4 of Cycle 1

  • Cycle 1: Day 1, 100 mg; Day 2, 200 mg; Day 3, 400 mg;Days 4 – 28, 800 mg
  • Cycle 2 through treatment discontinuation: 800 mg qd

Documents (password protected)

Responsibilities in overall trial

Abbvie Deutschland GmbH & Co.KG

National Coordinating Investigator

Prof. Dr. med. Katharina Götze

Study Sites

Klinik I für Innere Medizin

Study office

  • Klinisches Studienzentrum der Klinik I für Innere Medizin Köln


Active (Recruitment Closed)

Principal Investigator

Prof. Dr. med. Karl-Anton Kreuzer

Deputy of Principal Investigator

  • Dr. med. Joanna Schiller


  • Dr. med. Janine Böll, geb. Schwamb
  • Dr. med. Birgit Cremer
  • Dr.med. Vangica Galkin geb.Ristovska
  • Valeska Möntenich
  • Prof. Dr. med. Christian Pallasch
  • PD Dr. med. Stephanie Sasse
  • Dr. med. Armin Tuchscherer
  • Univ.-Prof. Dr. med. Dr. rer.-nat. Roland Ullrich
  • Priv.-Doz. Dr. med. Bastian von Tresckow
  • PD Dr. med. Thomas Zander
  • Dr. med. Anja Lohneis

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