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Combined Evaluation of Epigenetic and Sensitising Therapy in AML and MDS

NCT05636514 · Status: RECRUITING · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 12

Last updated 2026-03-18

No results posted yet for this study

Summary

The goal of this project is to see if two new potential treatments (defactinib and the combination tablet of decitabine/cedazuridine) can safely be combined to improve outcomes in people with high-risk myelodysplastic syndrome (MDS), certain forms of Acute Myeloid Leukaemia (AML), and Chronic Myelomonocytic Leukaemia (CMML). Decitabine/cedazuridine is approved for use by the Australian Therapeutics Goods Administration (TGA) as treatment for MDS. Defactinib is an experimental treatment. This means it is not an approved treatment for MDS in Australia. So far it has been given to over 625 patients in studies across the world.

All study participants will receive active treatment, there is no placebo. Participants will take the decitabine/cedazuridine treatment once a day for 5 days in a row (day 1 to day 5) on its own for the first month (cycle). From month 2 participants will take the decitabine/cedazuridine treatment and will also take the defactinib treatment, both for 5 days in a row on days 1 to day 5 each month (cycle). Defactinib is taken twice a day.

Conditions

Interventions

DRUG

Decitabine/Cedazuridine 35 Mg-100 Mg ORAL TABLET

Fixed dose treatment cycles 1 to 6. Cycle 1 is monotherapy, cycles 2 to 6 combination therapy with defactinib.

DRUG

Defactinib

Defactinib treatment will commence with at the starting dose (dose level 1) from cycle 2 to 6. Dose escalation/de-escalation will proceed based on the MTD determination. Dose Level 1: 200mg Defactinib twice daily (starting dose level) Dose Level 2: 400mg defactinib twice daily Dose Level -1: 200mg Defactinib daily

Sponsors & Collaborators

  • The University of New South Wales

    collaborator OTHER

  • Australian National University

    collaborator OTHER

  • Clinical Hub for Interventional Research (CHOIR)

    lead OTHER_GOV

Principal Investigators

  • John Pimanda, Professor · University of New South Wales

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2022-12-14
Primary Completion
2026-08-30
Completion
2027-12-30
FDA Drug
Yes

Countries

  • Australia

Study Locations

More Related Trials

Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT05636514 on ClinicalTrials.gov