Purpose of this Study
We are doing this study to find out if adding a drug called enasidenib (the study drug) to the standard treatment for myelodysplastic syndrome (MDS) can improve the likelihood of achieving complete remission. The study drug is approved for acute myeloid leukemia (AML), but is not approved for the treatment of MDS.
Who Can Participate?
Eligibility
Adults ages 18+ who:
- Are diagnosed with MDS that has changes to the IDH2 gene
- Are enrolled in the myeloMATCH Master Screening and Reassessment Protocol
- Have not received prior anti-cancer therapy for AML or MDS
Age Range
18-110
Sex/Genders
Male (cisgender)
Female (cisgender)
Non-binary or gender fluid
Transgender male
Transgender female
Looking for Healthy Participants
No
What is Involved?
Description
If you choose to join this study, you will get a random assignment (like a coin flip) to 1 of 2 groups.
Group 1: If you are in this group, you will get ASTX727 as a pill you take by mouth once a day for the first 5 days of the cycle. Each cycle lasts 28 days. You will continue on treatment indefinitely if you achieve a remission or are having clinical benefit and do not go on another myeloMATCH treatment study or onto receive an allogeneic stem cell transplant. ASTX727 is a standard, approved drug for the treatment of MDS.
If your disease does not have a complete remission after 6 cycles, you may be offered the opportunity to begin taking the study drug along with ASTX727. If you do take part, you will get ASTX727 as pills you take by mouth once a day for the first 5 days of the cycle and enasidenib as pills you take by mouth once a day for 28 days. Each cycle lasts for 28 days
Group 1: If you are in this group, you will get the study drug plus ASTX727. You will get ASTX727 as pills you take by mouth once a day for the first 5 days of the cycle and the study drug as pills you take by mouth once a day for 28 days. Each cycle lasts 28 days. You will continue on treatment indefinitely if you achieve a remission or are having clinical benefit and do not go on another myeloMATCH treatment study or onto receive an allogeneic stem cell transplant.
Locations
Duke University Hospital
Visit Timing
Weekdays
Compensation
No
Spanish Materials Available
Yes
Study Details
Full Title
MM1MDS-A01, A Randomized Phase II Trial of Enasidenib-Based Therapies Versus Cedazuridine-Decitabine in Higher-Risk IDH2-Mutated Myelodysplastic Syndrome: A MyeloMATCH Sub-Study,(NCT06577441)
Principal Investigator
Harry
Erba
Protocol Number
PRO00118285
NCT ID
NCT06577441
Phase
II
Enrollment Status
Open to Enrollment