Myelodysplastic Syndrome (MDS)Phase 1NCT04608110
A Phase 1 Trial of ASTX030 in Patients With Myelodysplastic Syndrome
A Phase 1 study testing ASTX030 (AZA + CED) in people with Myelodysplastic Syndrome (MDS), run by Taiho Pharmaceutical Co., Ltd. at 10 sites in Japan.
Trial summary
[Tolerability Assessment Part] The purpose is to identify the doses of the oral azacitidine(AZA) formulations and cedazuridine (CED) tablets which achieve a total AUC for AZA comparable to that for AZA injection at 75 mg/m2. Read more about this study
- Who is it for?
- Ages 20 years and older
- Conditions the study is for
- Myelodysplastic Syndrome (MDS)
- What is being tested?
- ASTX030 (AZA + CED)
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 59 participants
- Where
- 10 sitesJapan
- Study dates
- 30 Oct 2020to Jan 2028
- Phase
- Phase 1
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
- [Tolerability Assessment Part】
- Patients aged 20 years or older
- Patients with a diagnosis of MDS (refractory anemia [RA], refractory anemia with ringed sideroblasts [RARS], refractory anemia with excess blasts [RAEB], refractory anemia with excess blasts in transformation [RAEB-T], or chronic myelomonocytic leukemia [CMML]) according to the French-American-British (FAB) classification. Low-risk patients who fall under the risk category of low or intermediate-1 (Int-1) based on the International Prognostic Scoring System (IPSS) can be enrolled only if they are unlikely to respond to any other treatment or if they are currently being treated with AZA injection.
- Patients with an ECOG PS score of 0 or 1
- Patients with adequate organ function as indicated below
- 1. Hepatic function: All of the following criteria must be satisfied
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- Total bilirubin ≤ 2.0 × upper limit of normal (ULN)
- Aspartate aminotransferase (AST) ≤ 2.5 × ULN
- Alanine aminotransferase (ALT) ≤ 2.5 × ULN 2. Renal function: The following criteria must be satisfied
- Creatinine clearance or glomerular filtration rate ≥ 50 mL/min 3. Respiratory function: percutaneous arterial oxygen saturation (SpO₂) ≥ 90%
- Patients who are expected to survive for at least 3 months
- Patients who give written consent to participate in the trial using the informed consent form approved by the institutional review board
- [Expansion Assessment Part]
- Patients aged 20 years or older
- Patients with a diagnosis of MDS (RA, RARS, RAEB, RAEB-T, or CMML) according to the FAB classification, who are categorized as Int-2 or high risk according to the IPSS
- Patients with an ECOG PS score of 0 or 1
- Patients with adequate organ function as indicated below
- 1. Hepatic function: All of the following criteria must be satisfied
- Total bilirubin ≤ 2.0 ×ULN
- AST ≤ 2.5 × ULN
- ALT ≤ 2.5 × ULN 2. Renal function: The following criteria must be satisfied
- Creatinine clearance or glomerular filtration rate ≥ 50 mL/min 3. Respiratory function: percutaneous arterial oxygen saturation (SpO₂) ≥ 90%
- Patients who are expected to survive for at least 3 months
- Patients who give written consent to participate in the trial using the informed consent form approved by the institutional review board
Who cannot join
- [Tolerability Assessment Part]
- Patients who are unlikely to respond to AZA
- Patients who have received chemotherapy, hormone therapy, antibody therapy, radiotherapy, or other exploratory anti-cancer treatments for the primary disease within 3 weeks prior to the first administration of the investigational medicinal product (IMP)
- Patients who have used any other IMP or any privately imported medicine within 4 weeks prior to the first administration of IMP
- Patients with heart disease of Class 3 or 4 according to the New York Heart Association classification
- Patients with uncontrolled systemic disease or active infection
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- Patients with uncontrolled gastric or duodenal ulcer
- Patients with prior or current interstitial lung disease
- Patients with a history of surgical gastrectomy
- Patients with life-threatening conditions/symptoms, multiple organ failure, or other factors (including laboratory abnormalities) that, in the opinion of the investigator, are likely to affect their safety or the absorption and metabolism of AZA and CED, or influence the trial evaluation
- Patients with other malignancies (except appropriately treated basal cell carcinoma, squamous cell carcinoma, or cervical carcinoma in situ; prostate or breast cancer stabilized by endocrine therapies; and malignancies that have not relapsed for at least 1 year since the last successful treatment)
- Patients who are positive for HIV antibody, HBV-DNA, or HCV antibody
- Patients with any ≥ Grade 2 AE (except alopecia) associated with prior treatment of the primary disease. However, the parameters defined in inclusion criterion 4) above are excluded.
- Patients who have undergone a highly invasive and extensive surgical procedure within 4 weeks prior to the first administration of IMP
- Patients who previously underwent hematopoietic stem cell transplantation and have evidence of graft-versus-host disease (GVHD), or who received immunotherapy within 3 weeks before the start of study treatment.
- Patients with a history of hypersensitivity to the active ingredient or any excipient of IMP
- Patients who are, in the opinion of the investigator, at high risk for being unable to comply with the trial protocol because of mental disorders or other medical conditions (alcohol/substance abuse or addiction)
- Pregnant or nursing female patients, or female patients with a positive pregnancy test at screening
- Sexually active males (except those with a history of bilateral orchiectomy) or females of childbearing potential who do not agree to practice 2 different methods of birth control or remain abstinent during the trial and for 3 months (males) and 6 months (females) after the last dose of IMP
- Patients who, in the opinion of the investigator, are otherwise ineligible to participate in the trial
- [ Expansion Assessment Part]
- Patients who are unlikely to respond to AZA
- Patients who have received chemotherapy, hormone therapy, antibody therapy, radiotherapy, or other exploratory anti-cancer treatments for the primary disease within 3 weeks prior to the first administration of the IMP
- Patients who have received prior treatment with DNA methylation inhibitors, such as decitabine or AZA
- Patients who have used any other IMP or any privately imported medicine within 4 weeks prior to the first administration of IMP
- Patients with heart disease of Class 3 or 4 according to the New York Heart Association classification
- Patients with uncontrolled systemic disease or active infection
- Patients with uncontrolled gastric or duodenal ulcer
- Patients with prior or current interstitial lung disease
- Patients with a history of surgical gastrectomy
- Patients with life-threatening conditions/symptoms, multiple organ failure, or other factors (including laboratory abnormalities) that, in the opinion of the investigator, are likely to affect their safety or the absorption and metabolism of AZA and CED, or influence the trial evaluation
- Patients with other malignancies (except appropriately treated basal cell carcinoma, squamous cell carcinoma, or cervical carcinoma in situ; prostate or breast cancer stabilized by endocrine therapies; and malignancies that have not relapsed for at least 1 year since the last successful treatment)
- Patients who are positive for HIV antibody, HBV-DNA, or HCV antibody
- Patients with any ≥ Grade 2 AE (except alopecia) associated with prior treatment of the primary disease. However, the parameters defined in inclusion criterion 4) above are excluded.
- Patients who have undergone a highly invasive and extensive surgical procedure within 4 weeks prior to the first administration of IMP
- Patients who previously underwent hematopoietic stem cell transplantation and have evidence of GVHD, or who received immunotherapy within 3 weeks before the start of study treatment
- Patients with a history of hypersensitivity to the active ingredient or any excipient of IMP
- Patients who are, in the opinion of the investigator, at high risk for being unable to comply with the trial protocol because of mental disorders or other medical conditions (alcohol/substance abuse or addiction)
- Pregnant or nursing female patients, or female patients with a positive pregnancy test at screening
- Sexually active males (except those with a history of bilateral orchiectomy) or females of childbearing potential who do not agree to practice 2 different methods of birth control or remain abstinent during the trial and for 3 months (males) and 6 months (females) after the last dose of IMP
- Patients who, in the opinion of the investigator, are otherwise ineligible to participate in the trial
Trial locations and contacts
10 sites in 1 country. Contact the study team or a site near you to ask about taking part.
10 sites in 1 country
Japan10 sites, 10 recruiting
- Recruiting
Nippon Medical School Hospital
Bunkyō City
Hematology, +81-3-3822-2131
- Recruiting
Fukushima Medical University Hospital
Fukushima
Hematology, +81-24-547-1111
- Recruiting
Saitama Medical University Hospital
Iruma
Hematology, +81-49-276-1111
- Recruiting
University Hospital, Kyoto Prefectural University of Medicine
Kyoto
Hematology and Oncology, +81-75-251-5111
- Recruiting
Nagasaki University Hospital
Nagasaki
Hematology, +81-95-819-7200
- Recruiting
Osaka City General Hospital
Osaka
Hematology, +81-6-6929-1221
- Recruiting
Kindai University Hospital
Sakai
Hematology and Rheumatology, +81-72-288-7222
- Recruiting
NTT Medical Center Tokyo
Shinagawa-Ku
Hematology, +81-3-3448-6111
- Recruiting
Tokyo Medical University Hospital
Shinjuku-Ku
Hematology, +81-3-3342-6111
- Recruiting
Yamagata University Hospital
Yamagata
Hematology, +81-23-633-1122
About this study
Official title: A Phase 1, Multi-center, Open-label, Uncontrolled, Dose-escalation Study to Evaluate the Pharmacokinetics of ASTX030 in Patients With Myelodysplastic Syndrome (MDS)
[Tolerability Assessment Part] The purpose is to identify the doses of the oral azacitidine(AZA) formulations and cedazuridine (CED) tablets which achieve a total AUC for AZA comparable to that for AZA injection at 75 mg/m2.
[Expansion Assessment Part] The purpose is to compare the total AUC for AZA after administration of ASTX030 with that after administration of AZA injection at 75 mg/m2.
- Conditions
- Myelodysplastic Syndrome (MDS)
- Being tested
- ASTX030 (AZA + CED)
- Medical subject headings
- Myelodysplastic Syndromes
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ASTX030 (AZA + CED)Drug
In Cycle 1 (28 days per cycle), subcutaneous (SC) AZA will be administered on Day 1, followed by oral AZA formulations and CED tablets on Days 2-7. In Cycle 2 and onward, oral AZA formulations and CED tablets will be administered on Day 1-7.
ASTX030 (AZA + CED)Drug
In Cycle 1 (28 days per cycle), ASTX030 will be administered on Days 1-7. In Cycle 2, SC AZA will be administered on Days 1-7 according to a crossover design. From Cycle 3 onward, ASTX030 will be administered on Days 1-7.
ASTX030 (AZA + CED)Drug
In Cycle 1 (28 days per cycle), SC AZA will be administered on Days 1-7. In Cycle 2, ASTX030 will be administered on Days 1-7 according to a crossover design. From Cycle 3 onward, ASTX030 will be administered on Days 1-7.
| Study group | Role | What they receive |
|---|---|---|
| Tolerability Assessment Part | Experimental | Not described Receives: Drug: ASTX030 (AZA + CED) |
| Expansion Assessment Part Sequence A | Experimental | Not described Receives: Drug: ASTX030 (AZA + CED) |
| Expansion Assessment Part Sequence B | Experimental | Not described Receives: Drug: ASTX030 (AZA + CED) |
What is being measured
The primary outcome is the main question the study is designed to answer.
[Tolerability Assessment Part] AUC ratio of AZA after administration of oral AZA formulations in combination with CED tablets compared with SC administration of AZA injection.
Measured: Up to 1 month
[Expansion Assessment Part] AUC ratio of AZA after administration of ASTX030 compared with SC administration of AZA injection
Measured: Up to 2month
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Oct 2020
- Study start30 Oct 2020
- Primary completionDec 2027 (estimated)
- Study completionJan 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Myelodysplastic Syndrome (MDS)Studies 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
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- None (open label)
- Enrolment
- 59 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Taiho Pharmaceutical Co., Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Taiho Pharmaceutical Co., Ltd., Study director, Taiho Pharmaceutical Co., Ltd.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04608110
- Sponsor's protocol ID
- 418-102-00001
- Status last verified
- Aug 2026
- First submitted
- 23 Oct 2020
- Last updated
- 10 Aug 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: Anonymized Individual participant data (IPD) that underlie the results of this study will be shared with researchers to achieve aims pre-specified in a methodologically sound research proposal.