Low Dose ASTX727 for Myelodysplastic Syndrome

Recruiting at 42 trial locations

Age: 18+

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 1 & 2

Waitlist Available

Sponsor: Taiho Oncology, Inc.

No Placebo Group

Trial Summary

What is the purpose of this trial?

Multicenter, open-label study of various ASTX727 LD doses and schedules to assess safety, pharmacodynamics, pharmacokinetics, and hematologic response in subjects with International Prognostic Scoring System (IPSS) risk category of low-risk or Intermediate-1 MDS. This study will be conducted in two phases. In phase 1 subjects will be randomized into 3 cohorts in a 28-day cycles. Phase 2, 80 new subjects will be randomized in a 1:1 ratio into 2 doses/schedules.

Research Team

Yuri Sano, MD, PhD

Principal Investigator

Astex Pharmaceuticals, Inc.

Eligibility Criteria

This trial is for adults with low-risk or Intermediate-1 Myelodysplastic Syndrome who have specific blood count criteria, can follow study procedures, and are not pregnant. They must agree to use birth control during and after the trial. People with certain other conditions or treatments, like recent investigational drugs or prior malignancies (with some exceptions), cannot join.

Inclusion Criteria

Able to understand and comply with the study procedures, understand the risks involved in the study, and provide written informed consent before the first study-specific procedure

Subjects must have had at least 1 of the following disease-related criteria during the 8 weeks before randomization: Red blood cell (RBC) transfusion dependence of 2 or more units of RBC transfusions, Hb of <9.0 g/dL in at least 2 blood counts prior to randomization or in 1 blood count if RBC transfusion was received, Absolute Neutrophil Count (ANC) of <0.5 × 10^9/L in at least 2 blood counts prior to randomization, Platelet counts of <50 × 10^9/L in at least 2 blood counts prior to randomization, Eastern Cooperative Oncology Group (ECOG) performance status of 0 to 2, Adequate organ function, Women of child-bearing potential must not be pregnant or breastfeeding and must have a negative pregnancy test at screening, Women of child-bearing potential must agree to use contraceptive measures of birth control for 6 months after completing treatment; men must use contraceptive measures and agree not to father a child for at least 3 months after completing treatment

I am 18 or older with low to intermediate-1 risk MDS.

Exclusion Criteria

I do not have an active HIV or hepatitis infection.

I have been treated with azacitidine, decitabine, or guadecitabine before.

I haven't taken any experimental drugs in the last 2 weeks.

Treatment Details

Interventions

• ASTX727 LD (Anti-metabolites)

Trial Overview The study tests different doses of ASTX727 LD in people with MDS over two phases: Phase 1 randomizes participants into three groups for a 28-day cycle; Phase 2 involves 80 new subjects split evenly between two dose schedules to evaluate safety and effectiveness.

Participant Groups

Group I: Phase 2 Experimental Treatment 2 Interventions

80 additional subjects randomized in a 1:1 ratio studying two different doses

Group II: Phase 1 Stage B Experimental Treatment 1 Intervention

3 cohorts of 10 subjects each in 28-day cycles of ASTX727 LD

Group III: Phase 1 Stage A Experimental Treatment 1 Intervention

3 cohorts of 6 subjects each in a schedule in 28-day cycles of ASTX727 LD

Who Is Running the Clinical Trial?

Taiho Oncology, Inc.

Lead Sponsor

Trials

79

Recruited

12,700+

Tim Whitten

Taiho Oncology, Inc.

Chief Executive Officer since 2018

MBA and Pharmacy degree

Harold Keer

Taiho Oncology, Inc.

Chief Medical Officer

MD, PhD

Astex Pharmaceuticals, Inc.

Lead Sponsor

Trials

97

Recruited

7,400+

Dr. Harren Jhoti

Astex Pharmaceuticals, Inc.

Chief Executive Officer since 2007

PhD in Biochemistry from Birkbeck College, London

Dr. Harold N. Keer

Astex Pharmaceuticals, Inc.

Chief Medical Officer since 2020

MD