Phase 1-2 Study of Low Dose ASTX727 (ASTX727 LD) in Lower Risk MDS
NCT ID: NCT03502668
Last Updated: 2025-12-29
Study Results
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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ACTIVE_NOT_RECRUITING
PHASE1/PHASE2
160 participants
INTERVENTIONAL
2018-07-27
2026-01-31
Brief Summary
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Detailed Description
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Phase 1: In Stage A, subjects will be randomized into 3 cohorts of 6 subjects each testing different doses of oral decitabine with cedazuridine in 28-day cycles. When safety has been established in Phase 1 Stage A, Phase 1 Stage B will open, wherein additional 30 subjects will be randomized in a 1:1:1 ratio into 3 cohorts of 10 subjects.
Phase 2: Using 2 doses/schedules one of which will be selected from Phase 1, 40 additional subjects per dose/schedule will be randomized in a 1:1 ratio. The selected doses/schedules will be evaluated for safety (drug-related AEs), efficacy (including hematologic response), PD (long interspersed nucleotide element-1 (LINE-1 methylation, and fetal hemoglobin as fraction of total hemoglobin), and PK.
Conditions
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Keywords
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Study Design
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RANDOMIZED
SEQUENTIAL
TREATMENT
NONE
Study Groups
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Phase 1 Stage A
3 cohorts of 6 subjects each in a schedule in 28-day cycles of ASTX727 LD
ASTX727 LD
oral decitabine (LD) + cedazuridine (E7727)
Phase 1 Stage B
3 cohorts of 10 subjects each in 28-day cycles of ASTX727 LD
ASTX727 LD
oral decitabine (LD) + cedazuridine (E7727)
Phase 2
80 additional subjects randomized in a 1:1 ratio studying two different doses
ASTX727 LD
oral decitabine (LD) + cedazuridine (E7727)
ASTX727 SD
oral decitabine (SD) + cedazuridine (E7727)
Interventions
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ASTX727 LD
oral decitabine (LD) + cedazuridine (E7727)
ASTX727 SD
oral decitabine (SD) + cedazuridine (E7727)
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
2. 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:
1. 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).
2. 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.
3. Absolute Neutrophil Count (ANC) of \<0.5 × 10\^9/L in at least 2 blood counts prior to randomization.
4. Platelet counts of \<50 × 10\^9/L in at least 2 blood counts prior to randomization.
3. Eastern Cooperative Oncology Group (ECOG) performance status of 0 to 2.
4. Adequate organ function.
5. 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.
6. 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.
Exclusion Criteria
2. Treatments for MDS must be concluded 1 month prior to study treatment.
3. Prior treatment with azacitidine, decitabine, or guadecitabine.
4. Diagnosis of chronic myelomonocytic leukemia (CMML).
5. Poor medical risk because of other conditions such as uncontrolled systemic diseases or active uncontrolled infections.
6. 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.
7. Known active infection with human immunodeficiency virus or hepatitis viruses.
18 Years
ALL
No
Sponsors
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Taiho Oncology, Inc.
INDUSTRY
Responsible Party
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Locations
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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
Hospital Universitario Vall d Hebron
Barcelona, , Spain
Institut Català d'Oncologia Badalona Hospital Universitari Germans Trias i Pujol
Barcelona, , Spain
Hospital General Universitario Gregorio Marañón
Madrid, , Spain
Hospital Univeristario y Politecnico La Fe Servicio de Hematologia
Valencia, , Spain
Countries
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Other Identifiers
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ASTX727-03
Identifier Type: -
Identifier Source: org_study_id