A Phase 1 Trial of ASTX030 in Patients With Myelodysplastic Syndrome

NCT04608110 | Recruiting Phase 1 DMC

• 1 other identifier: 418-102-00001
• Study Type: interventional
• Target: 59
• Locations: 1 country
• Sites: 10

Brief 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. \[Expansion Assessment Part\] 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)

Outcome Measures

Primary Outcomes (2)

• [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.

  Up to 1 month

• [Expansion Assessment Part] AUC ratio of AZA after administration of ASTX030 compared with SC administration of AZA injection

  Up to 2month

Study Arms (3)

Tolerability Assessment Part

EXPERIMENTAL

Drug: ASTX030 (AZA + CED)

Expansion Assessment Part Sequence A

EXPERIMENTAL

Drug: ASTX030 (AZA + CED)

Expansion Assessment Part Sequence B

EXPERIMENTAL

Drug: ASTX030 (AZA + CED)

Interventions

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.

Tolerability Assessment Part

Eligibility Criteria

• Age: 20 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• \[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
• \. Hepatic function: All of the following criteria must be satisfied
• 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

You may not qualify if:

• \[Tolerability Assessment Part\]
• 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
• 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 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

Sponsors & Collaborators

• Taiho Pharmaceutical Co., Ltd.: lead

Study Sites (10)

Nippon Medical School Hospital

Bunkyō City, Japan

RECRUITING

Fukushima Medical University Hospital

Fukushima, Japan

RECRUITING

Saitama Medical University Hospital

Iruma, Japan

RECRUITING

University Hospital, Kyoto Prefectural University of Medicine

Kyoto, Japan

RECRUITING

Nagasaki University Hospital

Nagasaki, Japan

RECRUITING

Osaka City General Hospital

Osaka, Japan

RECRUITING

Kindai University Hospital

Sakai, Japan

RECRUITING

NTT Medical Center Tokyo

Shinagawa-Ku, Japan

RECRUITING

Tokyo Medical University Hospital

Shinjuku-Ku, Japan

RECRUITING

Yamagata University Hospital

Yamagata, Japan

RECRUITING

MeSH Terms

Conditions

Myelodysplastic Syndromes

Condition Hierarchy (Ancestors)

Bone Marrow Diseases; Hematologic Diseases; Hemic and Lymphatic Diseases

Study Officials

• Taiho Pharmaceutical Co., Ltd.

  Taiho Pharmaceutical Co., Ltd.

  STUDY DIRECTOR

Central Study Contacts

Drug Information Center

CONTACT

+81-3-3294-4527; ke-watanabe@taiho.co.jp

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 23, 2020
• First Posted: October 29, 2020
• Study Start: October 30, 2020
• Primary Completion (Estimated): December 1, 2027
• Study Completion (Estimated): January 1, 2028
• Last Updated: August 10, 2026

Record last verified: 2026-08

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP
• Time Frame: Data will be available after marketing approval in global markets, or beginning 1-3 years following article Publication. There is no end date to the availability of the data.
• Access Criteria: Otsuka will share data on an Otsuka-owned remotely accessible data sharing platform with Python and R analytical software. Research requests should be directed to clinicaltransparency@Otsuka-us.com.

Locations

JP (10)