Lower-risk MyelodysplasticPhase 1NCT03906695
Phase 1 Trial of ASTX727 in Subjects With Lower-risk Myelodysplastic Syndromes
A Phase 1 study testing ASTX727 in people with Lower-risk Myelodysplastic, run by Otsuka Pharmaceutical Co., Ltd. at 1 site in Japan.
Trial summary
To investigate the tolerability and safety of ASTX727 in Japanese subjects with lower-risk MDS. Read more about this study
- Who is it for?
- Ages 20 years and older
- Conditions the study is for
- Lower-risk Myelodysplastic
- What is being tested?
- ASTX727
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 30 participants
- Where
- 1 siteJapan
- Study dates
- 15 Mar 2019to 31 Jan 2026
- Phase
- Phase 1
- Are there results?
- Not yet: the study is under way
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 20 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Subjects with a definitive diagnosis of MDS and classified as low or Intermediate-1 risk by the International Prognostic Scoring System (IPSS) risk category
- Subjects meeting at least one of the disease-related criteria for Red blood cell (RBC) transfusion, hemoglobin (Hb) ,Absolute neutrophil count,Platelet count within 8 weeks prior to initial administration of IMP
- Subjects with Eastern Cooperative Oncology Group Performance Status (ECOG PS) of 0 or 1
- Adequate hepatic and renal function
- Sexually active men with reproductive capacity (except those who have undergone bilateral orchidectomy) must agree to use 2 effective contraceptive measures or remain abstinent during the trial and for 3 months after final administration of IMP. Sexually active women of child-bearing potential must agree to use 2 effective contraceptive measures or remain abstinent during the trial and for 6 months after final administration of IMP.
- Subjects who have provided written informed consent using the form approved by the institutional review board
Who cannot join
- Subjects who have received cytokine therapy, immunosuppressant therapy, or chemotherapy within 4 weeks prior to initial investigational medicinal product (IMP) administration
- Subjects who have received any other IMP or privately-imported medicine within 2 weeks prior to initial IMP administration
- Subjects with deletion 5q who are to be treated with lenalidomide
- Subjects with current or previous bone marrow blast percentage of >10%
- Subjects with a diagnosis of chronic myelomonocytic leukemia
- Subjects with heart disease of New York Heart Association (NYHA) Functional Class 3 or 4
Show all 18Show fewer
- Subjects with an uncontrolled systemic disease or active uncontrolled infection
- Subjects with diabetes mellitus requiring medical treatment
- Subjects with a life-threatening illness, medical condition or multiple organ dysfunction, or other reason, including laboratory abnormalities, which in the investigator's or subinvestigator's opinion could compromise the subject's safety, interfere with the absorption or metabolism of IMP, or compromise the integrity of the trial outcome
- Subjects with prior malignancy
- Subjects who test positive for human immunodeficiency virus antibody, hepatitis B virus DNA, or hepatitis C virus antibody
- Subjects with a history of surgical gastrectomy
- Subjects with previous organ transplantation
- Subjects with a ≥Grade 2 AE attributable to treatment of underlying disease, excluding the AEs
- Subjects who have undergone an invasive and extensive operation within 2 weeks prior to initial IMP administration
- Subjects with hypersensitivity to the IMPs or their excipients
- Subjects with known significant mental illness or other condition, such as active alcohol or other substance abuse or addiction, that in the opinion of the investigator or subinvestigator predisposes the subject to high risk of noncompliance with the protocol
- Female subjects who are pregnant, breast-feeding, or who test positive for pregnancy at screening
Trial locations and contacts
1 site in 1 country.
About this study
Official title: A Multicenter, Open-label, Dose-escalation, Phase 1 Trial to Investigate the Tolerability and Safety of ASTX727 in Subjects With Lower-risk Myelodysplastic Syndromes
To investigate the tolerability and safety of ASTX727 in Japanese subjects with lower-risk MDS.
- Conditions
- Lower-risk Myelodysplastic
- Being tested
- ASTX727
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ASTX727Drug
oral decitabine 5mg + cedazuridine
ASTX727Drug
oral decitabine 5mg + cedazuridine
ASTX727Drug
oral decitabine 10mg + cedazuridine
ASTX727Drug
oral decitabine 20mg + cedazuridine
ASTX727Drug
oral decitabine 10mg + cedazuridine
| Study group | Role | What they receive |
|---|---|---|
| 10-Day Schedule | Experimental | 10-Day Schedule
Investigational Medicinal Products (IMP) will be administered for 10 days in total per 4 weeks, i.e. a 28-day cycle. Receives: Drug: ASTX727 |
| 5-Day Schedule A | Experimental | 5-Day Schedule A
IMP will be administered for 5 days in total per 4 weeks, i.e. a 28-day cycle. Receives: Drug: ASTX727 |
| 5-Day Schedule B | Experimental | 5-Day Schedule B
IMP will be administered for 5 days in total per 4 weeks, i.e. a 28-day cycle. Receives: Drug: ASTX727 |
| 5-Day Schedule C | Experimental | 5-Day Schedule C
IMP will be administered for 5 days in total per 4 weeks, i.e. a 28-day cycle. Receives: Drug: ASTX727 |
| 7-Day Schedule | Experimental | 7-Day Schedule
IMP will be administered for 7 days in total per 4 weeks, i.e. a 28-day cycle. Receives: Drug: ASTX727 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Dose Limiting Toxicity
Measured: 28days
Secondary outcomes (2)
Area under the curve (AUC)
pharmacokinetics parameter
Measured: Pre-dose, 15 min, 30 min, 60 min, 90 min, 2 h, 3 h, 4 h, 6 h, 8 h, 24 h after dosing
Maximum plasma concentration (Cmax)
pharmacokinetics parameter
Measured: Pre-dose, 15 min, 30 min, 60 min, 90 min, 2 h, 3 h, 4 h, 6 h, 8 h, 24 h after dosing
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Apr 2019
- Study start15 Mar 2019
- Primary completion31 Jan 2026 (estimated)
- Study completion31 Jan 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Lower-risk MyelodysplasticStudies of the same condition, open and under way.
- More trials run by Otsuka Pharmaceutical Co., Ltd.The sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
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
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 30 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Otsuka Pharmaceutical Co., Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Nobuhito Sanada, Study director, Otsuka Pharmaceutical Co., Ltd.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03906695
- Sponsor's protocol ID
- 393-102-00002
- Other IDs
- JapicCTI-194654
- Status last verified
- Mar 2025
- First submitted
- 5 Apr 2019
- Last updated
- 20 Mar 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No: The focus of this study is a rare disease.
Study at a glance
From the registry record, last updated by the sponsor on 20 Mar 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multicenter, Open-label, Dose-escalation, Phase 1 Trial to Investigate the Tolerability and Safety of ASTX727 in Subjects With Lower-risk Myelodysplastic Syndromes
- ClinicalTrials.gov ID
- NCT03906695
- Acronym / sponsor ID
- 393-102-00002
- Conditions
- Lower-risk Myelodysplastic
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 30 participants (estimated)
- Lead sponsor
- Otsuka Pharmaceutical Co., Ltd.Industry (pharma, biotech, devices)
- Study start
- 15 Mar 2019 (actual)
- Primary completion
- 31 Jan 2026 (estimated)
- Study completion
- 31 Jan 2026 (estimated)
- First posted
- 8 Apr 2019 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
To investigate the tolerability and safety of ASTX727 in Japanese subjects with lower-risk MDS.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 5
| Arm | Type | Receives |
|---|---|---|
| 10-Day Schedule | Experimental | Drug: ASTX72710-Day Schedule Investigational Medicinal Products (IMP) will be administered for 10 days in total per 4 weeks, i.e. a 28-day cycle. |
| 5-Day Schedule A | Experimental | Drug: ASTX7275-Day Schedule A IMP will be administered for 5 days in total per 4 weeks, i.e. a 28-day cycle. |
| 5-Day Schedule B | Experimental | Drug: ASTX7275-Day Schedule B IMP will be administered for 5 days in total per 4 weeks, i.e. a 28-day cycle. |
| 5-Day Schedule C | Experimental | Drug: ASTX7275-Day Schedule C IMP will be administered for 5 days in total per 4 weeks, i.e. a 28-day cycle. |
| 7-Day Schedule | Experimental | Drug: ASTX7277-Day Schedule IMP will be administered for 7 days in total per 4 weeks, i.e. a 28-day cycle. |
Who may qualify
- Ages
- 20 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- Subjects with a definitive diagnosis of MDS and classified as low or Intermediate-1 risk by the International Prognostic Scoring System (IPSS) risk category
- Subjects meeting at least one of the disease-related criteria for Red blood cell (RBC) transfusion, hemoglobin (Hb) ,Absolute neutrophil count,Platelet count within 8 weeks prior to initial administration of IMP
- Subjects with Eastern Cooperative Oncology Group Performance Status (ECOG PS) of 0 or 1
- Adequate hepatic and renal function
- Sexually active men with reproductive capacity (except those who have undergone bilateral orchidectomy) must agree to use 2 effective contraceptive measures or remain abstinent during the trial and for 3 months after final administration of IMP. Sexually active women of child-bearing potential must agree to use 2 effective contraceptive measures or remain abstinent during the trial and for 6 months after final administration of IMP.
Key exclusion criteria (18 listed)
- Subjects who have received cytokine therapy, immunosuppressant therapy, or chemotherapy within 4 weeks prior to initial investigational medicinal product (IMP) administration
- Subjects who have received any other IMP or privately-imported medicine within 2 weeks prior to initial IMP administration
- Subjects with deletion 5q who are to be treated with lenalidomide
- Subjects with current or previous bone marrow blast percentage of >10%
- Subjects with a diagnosis of chronic myelomonocytic leukemia
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| ASTX727oral decitabine 5mg + cedazuridine | Drug | 10-Day Schedule |
| ASTX727oral decitabine 5mg + cedazuridine | Drug | 5-Day Schedule A |
| ASTX727oral decitabine 10mg + cedazuridine | Drug | 5-Day Schedule B |
| ASTX727oral decitabine 20mg + cedazuridine | Drug | 5-Day Schedule C |
| ASTX727oral decitabine 10mg + cedazuridine | Drug | 7-Day Schedule |
Primary outcome
Dose Limiting Toxicity
Time frame: 28days
2 secondary outcomes
Area under the curve (AUC)
Time frame: Pre-dose, 15 min, 30 min, 60 min, 90 min, 2 h, 3 h, 4 h, 6 h, 8 h, 24 h after dosing
Maximum plasma concentration (Cmax)
Time frame: Pre-dose, 15 min, 30 min, 60 min, 90 min, 2 h, 3 h, 4 h, 6 h, 8 h, 24 h after dosing
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 20 Mar 2025.
- Individual participant dataSharing planned: No. The focus of this study is a rare disease.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Nobuhito Sanada study director
Otsuka Pharmaceutical Co., Ltd.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing ASTX727 in people with Lower-risk Myelodysplastic, run by Otsuka Pharmaceutical Co., Ltd. at 1 site in Japan.
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 6 inclusion and 18 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: under way, not looking for participants, as of the sponsor's last update on 20 Mar 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Japan. 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 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.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
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?
Dose Limiting Toxicity (time frame: 28days). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 20 Mar 2025.