Combined Evaluation of Epigenetic and Sensitising Therapy in AML and MDS

Sponsor
Clinical Hub for Interventional Research (CHOIR)
Study ID
NCT05636514
Phase
PHASE1
Status
Recruiting
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Conditions

Eligibility Criteria

Sex
ALL
Age
18 Years - N/A
Healthy Volunteers
Not accepted

Interventions

  • Decitabine/Cedazuridine 35 Mg-100 Mg ORAL TABLET — DRUG
    Fixed dose treatment cycles 1 to 6. Cycle 1 is monotherapy, cycles 2 to 6 combination therapy with defactinib.
  • Defactinib — DRUG
    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

Study Details

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.

Key Dates

Start date
Dec 14, 2022
Status verified
Mar 2026
Primary completion
Aug 30, 2026
Completion
Dec 30, 2027

Study Design

Enrollment
12 participants (estimated)
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Experimental: Decitabine/cedazuridine + defactinib
    Decitabine/cedazuridine taken days 1-5 of each 28 day treatment cycle, cycle 1 to 6 Defactinib taken on days 1-5 of each 28 treatment day cycle from cycle 2 to cycle 6.

Primary Outcome Measure

Maximum tolerated dose [ Time Frame: Once 3 participants have completed 2 cycles, assessed at approximately 2 months (each cycle is 28 days) ]

Central Contacts

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