Actively Recruiting
A Phase 1, Multi-center, Open-label, Uncontrolled, Dose-escalation Study to Evaluate the Pharmacokinetics of ASTX030 in Patients With Myelodysplastic Syndrome (MDS)
Led by Taiho Pharmaceutical Co., Ltd. · Updated on 2025-12-03
40
Participants Needed
10
Research Sites
4 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are studying the doses of oral azacitidine formulations combined with cedazuridine tablets to achieve a total drug exposure similar to that of azacitidine injection in patients with Myelodysplastic Syndrome (MDS). This Phase 1, multi-center, open-label study aims to evaluate the pharmacokinetics of ASTX030, focusing on how the body absorbs and processes the drugs. The study is sponsored by Taiho Pharmaceutical Co., Ltd. Participants receive ASTX030, which includes oral azacitidine formulations and cedazuridine tablets. In the first 28-day cycle, a single oral dose of azacitidine is given on day -3, followed by a subcutaneous azacitidine injection on day 1. From day 2 to 7, participants take oral azacitidine and cedazuridine tablets. In subsequent cycles, the oral formulations and cedazuridine tablets are administered from day 1 to 7 each cycle. During the study, researchers monitor the drug levels in the body by measuring the area under the curve (AUC) of azacitidine after oral and injectable administration, focusing on the first month. Participants undergo regular assessments to evaluate safety, organ function, and overall health status. The study includes screening for eligibility, and participants provide written consent. The trial lasts as long as treatment continues, with close observation of response and any side effects.
CONDITIONS
Brief Title
A Phase 1 Trial of ASTX030 in Patients With Myelodysplastic Syndrome
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Patients aged 20 years or older
- Diagnosis of Myelodysplastic Syndrome (MDS) according to the French-American-British classification
- Low-risk or intermediate-1 risk patients unlikely to respond to other treatments or currently treated with azacitidine injection
- ECOG performance status score of 0 or 1, or 2 if due to primary disease
- Adequate hepatic function: total bilirubin 2.0 ULN, AST 2.5 ULN, ALT 2.5 ULN
- Adequate renal function: serum creatinine 1.5 ULN or creatinine clearance/glomerular filtration rate 50 mL/min
- Adequate respiratory function: oxygen saturation 90%
- Expected survival of at least 3 months
- Provided written informed consent
You will not qualify if you...
- Unlikely to respond to azacitidine
- Received chemotherapy, hormone therapy, antibody therapy, radiotherapy, or other anti-cancer treatments within 3 weeks prior to study
- Used any other investigational medicinal product or privately imported medicine within 4 weeks prior to study
- Heart disease classified as Class 3 or 4 by New York Heart Association
- Uncontrolled systemic disease or active infection
- Uncontrolled gastric or duodenal ulcer
- Prior or current interstitial lung disease
- History of surgical gastrectomy
- Life-threatening conditions, multiple organ failure, or other safety-affecting factors
- Other malignancies except certain stabilized or treated cancers
- Positive for HIV antibody, HBV-DNA, or HCV antibody
- Grade 2 or higher adverse events from prior treatment except alopecia
- Had highly invasive surgery within 4 weeks prior to study
- Previous or planned hematopoietic stem cell transplantation
- Hypersensitivity to study drug or excipients
- Mental disorders or substance abuse affecting compliance
- Pregnant or nursing females or positive pregnancy test at screening
- Sexually active males or females of childbearing potential not agreeing to two birth control methods or abstinence
- Otherwise deemed ineligible by investigator
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
screening and enrollment visit
Duration - Repeated 28-day cycles
Participants receive oral and subcutaneous azacitidine formulations combined with cedazuridine tablets according to a dosing schedule.
Visits on days -3, 1, and 2 to 7 in Cycle 1; days 1 to 7 in subsequent cycles
Trial Site Locations
Total: 10 locations
1
Nippon Medical School Hospital
Bunkyō City, Japan
Actively Recruiting
2
Fukushima Medical University Hospital
Fukushima, Japan
Actively Recruiting
3
Saitama Medical University Hospital
Iruma, Japan
Actively Recruiting
4
University Hospital, Kyoto Prefectural University of Medicine
Kyoto, Japan
Actively Recruiting
5
Nagasaki University Hospital
Nagasaki, Japan
Actively Recruiting
6
Osaka City General Hospital
Osaka, Japan
Actively Recruiting
7
Kindai University Hospital
Sakai, Japan
Actively Recruiting
8
NTT Medical Center Tokyo
Shinagawa-Ku, Japan
Actively Recruiting
9
Tokyo Medical University Hospital
Shinjuku-Ku, Japan
Actively Recruiting
10
Yamagata University Hospital
Yamagata, Japan
Actively Recruiting
Research Team
D
Drug Information Center
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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