Phase 1-2 Study of Low Dose ASTX727 (ASTX727 LD) in Lower Risk MDS

Completed · Phase 1/Phase 2

Conditions studied: Myelodysplastic Syndromes

In brief

Multicenter, open-label study of various ASTX727 LD doses and schedules to assess safety, pharmacodynamics, pharmacokinetics, and hematologic response in subjects with International Prognostic Scoring System (IPSS) risk category of low-risk or Intermediate-1 MDS. This study will be conducted in two phases. In phase 1 subjects will be randomized into 3 cohorts in a 28-day cycles. Phase 2, 80 new subjects will be randomized in a 1:1 ratio into 2 doses/schedules.

Key facts

Study ID

NCT03502668

Run by

Taiho Oncology, Inc.

People needed

160

Starts

2018-07-27

Expected to finish

2026-01-13

Last updated by the study team

2026-04-03

Who can join

Age: 18 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Able to understand and comply with the study procedures, understand the risks involved in the study, and provide written informed consent before the first study-specific procedure.
• Men or women ≥18 years with IPSS low risk or Int-1 MDS (all subjects). Subjects must have had at least 1 of the following disease-related criteria during the 8 weeks before randomization:
• Red blood cell (RBC) transfusion dependence of 2 or more units of RBC transfusions (RBC transfusion administered for hemoglobin (Hb) levels ≤9.0 g/dL are counted).
• Hb of <9.0 g/dL in at least 2 blood counts prior to randomization or in 1 blood count if RBC transfusion was received.
• Absolute Neutrophil Count (ANC) of <0.5 × 10\^9/L in at least 2 blood counts prior to randomization.
• Platelet counts of <50 × 10\^9/L in at least 2 blood counts prior to randomization.
• Eastern Cooperative Oncology Group (ECOG) performance status of 0 to 2.
• Adequate organ function.
• Women of child-bearing potential (according to recommendations of the Clinical Trial Facilitation Group [CTFG]) must not be pregnant or breastfeeding and must have a negative pregnancy test at screening.
• Women of child-bearing potential must agree to use contraceptive measures of birth control for 6 months after completing treatment; men must use contraceptive measures and agree not to father a child for at least 3 months after completing treatment.

You may not qualify if…

• Treatment with any investigational drug or therapy within 2 weeks before study treatment.
• Treatments for MDS must be concluded 1 month prior to study treatment.
• Prior treatment with azacitidine, decitabine, or guadecitabine.
• Diagnosis of chronic myelomonocytic leukemia (CMML).
• Poor medical risk because of other conditions such as uncontrolled systemic diseases or active uncontrolled infections.
• Prior malignancy, except for adequately treated basal cell or squamous cell skin cancer, in situ cervical cancer, prostate cancer or breast cancer under control with hormone therapy, or other cancer from which the subject has been disease free for at least 1 year.
• Known active infection with human immunodeficiency virus or hepatitis viruses.

Where it is running

• The University of Alabama at Birmingham — Birmingham, Alabama, United States
• University of Colorado, Anschutz Cancer Pavilion — Aurora, Colorado, United States
• Yale Cancer Center — New Haven, Connecticut, United States
• Mayo Clinic Florida — Jacksonville, Florida, United States
• BRCR Medical Center Inc. — Plantation, Florida, United States
• Moffitt Cancer Center Site#507 — Tampa, Florida, United States
• The University of Chicago — Chicago, Illinois, United States
• Indiana University Health Hospital - Simon Cancer Center — Indianapolis, Indiana, United States
• University of Kansas Clinical Research Center — Westwood, Kansas, United States
• The Center for Cancer and Blood Disorders (RCCA MD LLC - Maryland Division) — Bethesda, Maryland, United States
• Mayo Clinic — Rochester, Minnesota, United States
• University of Nebraska Medical Center — Omaha, Nebraska, United States
• Roswell Park Comprehensive Cancer Center — Buffalo, New York, United States
• Oregon Health and Science University Knight Cancer Institute — Portland, Oregon, United States
• Sarah Cannon Research Institute — Nashville, Tennessee, United States
• Vanderbilt University Medical Center - Hematology-Oncology — Nashville, Tennessee, United States
• The University of Texas MD Anderson Cancer Center — Houston, Texas, United States
• Texas Oncology - Tyler — Tyler, Texas, United States
• ZNA - Campus Middelheim — Antwerp, Belgium
• Az St-Jan Brugge-Oostende A.V. — Bruges, Belgium
• London Regional Cancer Center — London, Ontario, Canada
• University of Alberta Hospital - Hematology Research — Edmonton, Canada
• Universitaetsklinikum Freiburg Site#703 — Freiburg im Breisgau, Germany
• Universitätsklinikum Halle — Halle, Germany
• Universita degli Studi di Firenze — Florence, Italy

Full record on ClinicalTrials.gov

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