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Myelodysplastic SyndromesPhase 1/2NCT03502668

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

A Phase 1/2 study testing ASTX727 LD and ASTX727 SD in people with Myelodysplastic Syndromes, run by Taiho Oncology, Inc. at 29 sites in 6 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
Search all trials

Trial summary

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. Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Myelodysplastic Syndromes
What is being tested?
ASTX727 LD and ASTX727 SD
Type of treatment
Pill or oral medicine
Could I receive a placebo?
No, everyone receives an active treatment
How many people
160 participants
Where
29 sitesin 6 countries
Study dates
27 Jul 2018to 13 Jan 2026
Phase
Phase 1/2
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

  • 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.
Show all 10Show fewer
  • 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.

Who cannot join

  • 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.

Trial locations and contacts

29 sites in 6 countries.

29 sites in 6 countries

United States18 sites

  • University of Colorado, Anschutz Cancer Pavilion

    Aurora, Colorado, 80045

  • The Center for Cancer and Blood Disorders (RCCA MD LLC - Maryland Division)

    Bethesda, Maryland, 20817

  • The University of Alabama at Birmingham

    Birmingham, Alabama, 35233

  • Roswell Park Comprehensive Cancer Center

    Buffalo, New York, 14263

  • The University of Chicago

    Chicago, Illinois, 60637

  • The University of Texas MD Anderson Cancer Center

    Houston, Texas, 77030

  • Indiana University Health Hospital - Simon Cancer Center

    Indianapolis, Indiana, 46202

  • Mayo Clinic Florida

    Jacksonville, Florida, 32224

  • Sarah Cannon Research Institute

    Nashville, Tennessee, 37203

  • Vanderbilt University Medical Center - Hematology-Oncology

    Nashville, Tennessee, 37232

  • Yale Cancer Center

    New Haven, Connecticut, 06510

  • University of Nebraska Medical Center

    Omaha, Nebraska, 68198

  • BRCR Medical Center Inc.

    Plantation, Florida, 33324

  • Oregon Health and Science University Knight Cancer Institute

    Portland, Oregon, 97239

  • Mayo Clinic

    Rochester, Minnesota, 55905

  • Moffitt Cancer Center Site#507

    Tampa, Florida, 33612

  • Texas Oncology - Tyler

    Tyler, Texas, 75702

  • University of Kansas Clinical Research Center

    Westwood, Kansas, 66205

Other trials in United States

Spain4 sites

  • Hospital Universitario Vall d Hebron

    Barcelona

  • Institut Català d'Oncologia Badalona Hospital Universitari Germans Trias i Pujol

    Barcelona

  • Hospital General Universitario Gregorio Marañón

    Madrid

  • Hospital Univeristario y Politecnico La Fe Servicio de Hematologia

    Valencia, 46026

Other trials in Spain

Belgium2 sites

  • ZNA - Campus Middelheim

    Antwerp

  • Az St-Jan Brugge-Oostende A.V.

    Bruges

Other trials in Belgium

Canada2 sites

  • University of Alberta Hospital - Hematology Research

    Edmonton, T6G 2B7

  • London Regional Cancer Center

    London, Ontario, N6A 5W9

Other trials in Canada

Germany2 sites

  • Universitaetsklinikum Freiburg Site#703

    Freiburg im Breisgau, 79106

  • Universitätsklinikum Halle

    Halle, 06120

Other trials in Germany

Italy1 site

  • Universita degli Studi di Firenze

    Florence

Other trials in Italy

About this study

Official title: A Randomized, Open-Label, Phase 1-2 Study of ASTX727 Low Dose (ASTX727 LD) Extended Schedule in Subjects With Lower Risk (IPSS Low or Intermediate-1) Myelodysplastic Syndromes (MDS)

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.

Read the detailed description

A Phase 1-2, multicenter, open-label study of various ASTX727 LD doses and schedules to assess the safety, pharmacodynamics (PD), pharmacokinetics (PK), and hematologic response in subjects with IPSS risk category of low-risk or Intermediate-1 MDS. The study will be conducted in 2 phases.

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.

Being tested
ASTX727 LD, ASTX727 SD
Keywords
low risk myelodysplastic syndromes, MDS, ASTX727
Medical subject headings
Myelodysplastic Syndromes

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • ASTX727 LDDrug

    Also called oral decitabine (LD) + cedazuridine (E7727)

    oral decitabine (LD) + cedazuridine (E7727)

  • ASTX727 SDDrug

    Also called oral decitabine (SD) + cedazuridine (E7727)

    oral decitabine (SD) + cedazuridine (E7727)

Study groups in NCT03502668
Study groupRoleWhat they receive
Phase 1 Stage AExperimental3 cohorts of 6 subjects each in a schedule in 28-day cycles of ASTX727 LD
Receives: Drug: ASTX727 LD
Phase 1 Stage BExperimental3 cohorts of 10 subjects each in 28-day cycles of ASTX727 LD
Receives: Drug: ASTX727 LD
Phase 2Experimental80 additional subjects randomized in a 1:1 ratio studying two different doses
Receives: Drug: ASTX727 LD; Drug: ASTX727 SD

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Incidence of drug-related Grade ≥3 Adverse Events (AEs) or dose-limiting toxicities (DLTs) (if any) for each cohort dose/schedule

    Phase 1: Safety

    Measured: 18-24 months

  2. Hematologic response based on normalization of conversion of any baseline cytopenia or anemia (hemoglobin response, neutrophil response, platelet response, transfusion independence)

    Phase 2: Efficacy

    Measured: 18-24 months

Secondary outcomes (9)

  1. %LINE-1 methylation change from baseline

    pharmacodynamics

    Measured: 18-24 months

  2. Area under the curve (AUC)

    pharmacokinetics parameter

    Measured: 18-24 months

  3. Maximum plasma concentration (Cmax)

    pharmacokinetics parameter

    Measured: 18-24 months

  4. Time to reach maximum concentration (Tmax)

    pharmacokinetics parameter

    Measured: 18-24 months

  5. Half life (t1/2)

    pharmacokinetics parameter

    Measured: 18-24 months

  6. Hematologic response (Phase 1 only) based on normalization of conversion of any baseline cytopenia or anemia (hemoglobin response, neutrophil response, platelet response, transfusion independence)

    Phase 1: Efficacy

    Measured: 18-24 months

  7. Time to bone marrow blasts >5%

    Number of days from the date of randomization to the date when bone marrow blasts are >5% and increased by ≥50%.

    Measured: 18-24 months

  8. Leukemia-free survival

    Number of days from the date of randomization to the date when bone marrow or peripheral blood blasts reach ≥20%, or death from any cause

    Measured: 18-24 months

  9. Overall survival

    Number of days from the date of randomization to the date of death from any cause

    Measured: 18-24 months

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted19 Apr 2018
  2. Study start27 Jul 2018
  3. Primary completion13 Jan 2026
  4. Study completion13 Jan 2026

Resources

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 1/2
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Sequential
Model description
Multicenter, open label
Masking
None (open label)
Enrolment
160 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03502668
Sponsor's protocol ID
ASTX727-03
Status last verified
Apr 2026
First submitted
11 Apr 2018
Last updated
3 Apr 2026
Results posted
No
Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No

Questions about this trial

What is this clinical trial studying?

A Phase 1/2 study testing ASTX727 LD and ASTX727 SD in people with Myelodysplastic Syndromes, run by Taiho Oncology, Inc. at 29 sites in 6 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 10 inclusion and 7 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 3 Apr 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 29 sites in United States, Spain, Belgium, Canada, Germany and 1 more country. 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 combined 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.

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

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?

Incidence of drug-related Grade ≥3 Adverse Events (AEs) or dose-limiting toxicities (DLTs) (if any) for each cohort dose/schedule (time frame: 18-24 months). The study lists 2 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 NCT03502668.

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 3 Apr 2026.

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