Myelodysplastic SyndromesPhase 1/2NCT06091267
PK/Efficacy Bridging Study of ASTX727 in Chinese Subjects With Myelodysplastic Syndromes
A Phase 1/2 study testing IV Decitabine, Decitabine and cedazuridine and only Decitabine and cedazuridine in people with Myelodysplastic Syndromes, run by Otsuka Beijing Research Institute at 1 site in China.
Trial summary
This is an Open-Label, Crossover, Pharmacokinetic and Efficacy Bridging Study of Oral ASTX727 versus IV Decitabine in Chinese Subjects with Myelodysplastic Syndromes 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?
- IV Decitabine, Decitabine and cedazuridine and only Decitabine and cedazuridine
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 72 participants
- Where
- 1 siteChina
- Study dates
- 16 Oct 2023to 30 Jun 2027
- Phase
- Phase 1/2
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
- Agree to participate in this trial and voluntarily sign the informed consent form.
- Men or women ≥ 18 years at the time of signing the informed consent form.
- Subjects with MDS previously treated or untreated with de novo or secondary MDS.
- Eastern Cooperative Oncology Group (ECOG) performance status of 0 to 1 at screening.
Who cannot join
- Prior treatment with more than 1 cycle of azacitidine or decitabine.
- Cytotoxic chemotherapy or prior azacitidine or decitabine within 4 weeks of first dose of study treatment.
- Conditions as judged by the investigator to be inappropriate for participation in the clinical trial.
- Previous diagnosis of malignant tumor.
- History of immune deficiency.
- Acute myeloid leukemia (AML) with bone marrow or peripheral blast count ≥ 20% or other malignant hematological diseases.
Trial locations and contacts
1 site in 1 country.
China1 site
The First Affiliated Hospital,Zhejiang University School of Medicine
Hangzhou, Zhejiang, 310003
About this study
Official title: An Open-label, Crossover, Pharmacokinetic and Efficacy Bridging Study of Oral ASTX727 Versus IV Decitabine in Chinese Subjects With Myelodysplastic Syndromes
This is an Open-Label, Crossover, Pharmacokinetic and Efficacy Bridging Study of Oral ASTX727 versus IV Decitabine in Chinese Subjects with Myelodysplastic Syndromes
- Conditions
- Myelodysplastic Syndromes
- Being tested
- IV Decitabine, Decitabine and cedazuridine, only Decitabine and cedazuridine
- Medical subject headings
- Myelodysplastic Syndromes
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
IV DecitabineDrug
The subjects will receive decitabine 20 mg/m^2 IV daily × 5 days in 28-day cycles.
Decitabine and cedazuridineDrug
subjects will receive treatment with ASTX727, 1 tablet/day for 5 consecutive days, in 28-day cycles.
only Decitabine and cedazuridineDrug
subjects will receive treatment with ASTX727, 1 tablet/day for 5 consecutive days, in 28-day cycles, until disease progression, unacceptable toxicity, or the subject/investigator decides that the subject should discontinue treatment or withdraw from the trial.
| Study group | Role | What they receive |
|---|---|---|
| ASTX727 and IV Decitabine | Experimental | Cycle1：ASTX727 tablets, oral, 1 tablet/day for 5 days；Cycle2：IV Decitabine, 20 mg/m^2, is administered for 1 hour at a time for 5 days；≥ Cycle 3：ASTX727 tablets, oral, 1 tablet/day for 5 days Receives: Drug: IV Decitabine; Drug: Decitabine and cedazuridine; Drug: only Decitabine and cedazuridine |
| IV Decitabine and ASTX727 | Active comparator | Cycle1：IV Decitabine, 20 mg/m^2, is administered for 1 hour at a time for 5 days； Cycle2：ASTX727 tablets, oral, 1 tablet/day for 5 days；≥ Cycle 3：ASTX727 tablets, oral, 1 tablet/day for 5 days Receives: Drug: IV Decitabine; Drug: Decitabine and cedazuridine; Drug: only Decitabine and cedazuridine |
| ASTX727 | Experimental | ASTX727 tablets, oral, 1 tablet/day for 5 days； Receives: Drug: only Decitabine and cedazuridine |
What is being measured
The primary outcome is the main question the study is designed to answer.
Complete Response Rate
Assess efficacy [Complete Response Rate (CR)] of treatment with ASTX727 in Chinese subjects with myelodysplastic syndromes (MDS);
Measured: An analysis is planned when the last enrolled patient have completed Follow-up 12 months.
5day_AUC0-τ
Assess pharmacokinetic (PK) parameters (Total 5-day AUC exposures of decitabine) after treatment with ASTX727 (oral) versus decitabine for IV infusion for 5 days;
Measured: An analysis is planned when the last enrolled patient have completed the treatment with ASTX727 (oral) versus decitabine for IV infusion for 5 day.
Secondary outcomes (16)
Objective Response Rate
Objective Response Rate (ORR): The proportion of subjects who achieve CR and partial response (PR) based on IWG 2006 criteria;
Measured: through study completion, an average of 1 year.
Clinical Response Rate
Clinical Response Rate: The proportion of subjects who achieve CR, PR, marrow complete response (mCR), and hematologic improvement (HI) based on IWG 2006 criteria.
Measured: through study completion, an average of 1 year.
Rate of transfusion independence
Rate of transfusion independence: The proportion of subjects who had no blood transfusion of 2 or more units of PRBCs for 56 days or more after treatment;
Measured: through study completion, an average of 1 year.
disease progression
Time to progression to acute myeloid leukemia (AML);
Measured: through study completion, an average of 1 year.
Overall survival
Overall survival (OS).
Measured: through study completion, an average of 1 year.
Safety assessment
Safety as assessed by adverse events (AEs), concomitant medications, physical examination, clinical laboratory tests (hematology , serum chemistry and urinalysis), vital signs, Eastern Cooperative Oncology Group (ECOG) performance status, and electrocardiogram (ECG).
Measured: through study completion, an average of 1 year.
peak concentration (Cmax)
Decitabine PK parameters: peak concentration (Cmax).
Measured: through study completion, an average of 1 year.
time to peak concentration (Tmax)
Decitabine PK parameters: time to peak concentration (Tmax).
Measured: through study completion, an average of 1 year.
area under the plasma concentration-time curve over a dosing interval (AUC0-τ).
Decitabine PK parameters: area under the plasma concentration-time curve over a dosing interval (AUC0-τ).
Measured: through study completion, an average of 1 year.
accumulation ratio based on AUC0-τ (Rac_AUC0-τ).
Decitabine PK parameters: accumulation ratio based on AUC0-τ (Rac_AUC0-τ).
Measured: through study completion, an average of 1 year.
accumulation ratio based on Cmax (Rac_Cmax).
Decitabine PK parameters: accumulation ratio based on Cmax (Rac_Cmax).
Measured: through study completion, an average of 1 year.
Cmax
PK parameters of E7727 and E7727-epimer: Cmax.
Measured: through study completion, an average of 1 year.
Tmax
PK parameters of E7727 and E7727-epimer: Tmax.
Measured: through study completion, an average of 1 year.
AUC0-τ
PK parameters of E7727 and E7727-epimer: area under the plasma concentration-time curve over a dosing interval .
Measured: through study completion, an average of 1 year.
Rac_AUC0-τ
PK parameters of E7727 and E7727-epimer: accumulation ratio based on AUC0-τ.
Measured: through study completion, an average of 1 year.
Rac_Cmax
PK parameters of E7727 and E7727-epimer: accumulation ratio based on Cmax.
Measured: through study completion, an average of 1 year.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Oct 2023
- Study start16 Oct 2023
- Primary completion22 May 2026
- Study completion30 Jun 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Myelodysplastic SyndromesStudies of the same condition, open and under way.
- 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 1/2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- None (open label)
- Enrolment
- 72 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Otsuka Beijing Research Institute
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06091267
- Sponsor's protocol ID
- 393-403-00072
- Status last verified
- Oct 2026
- First submitted
- 22 Sep 2023
- Last updated
- 16 Oct 2026
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No