A Phase 1 Trial of ASTX030 in Patients With Myelodysplastic Syndrome

ConditionMyelodysplastic Syndrome (MDS)

Trial statusRecruiting

Trial phasePhase 1

Trial typeInterventional

Biological sexAll

Age20+

SponsorTaiho Pharmaceutical Co., Ltd.

About this trial

\[Tolerability Assessment Part\] 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.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

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

Disqualifiers

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

Trial design

Design model

Crossover

Treatments tested in this trial

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.

Treatment groups

59 Participants

are divided into 3 treatment groups

Group A: Tolerability Assessment PartExperimental treatment 1 intervention

Group B: Expansion Assessment Part Sequence AExperimental treatment 1 intervention

Group C: Expansion Assessment Part Sequence BExperimental treatment 1 intervention

Trial outcomes

Primary outcomes

[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.

Time frame

Up to 1 month

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

Time frame

Up to 2month

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Taiho Pharmaceutical Co., Ltd.

Lead sponsor

Source ClinicalTrials.gov

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