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Testing the Addition of an IDH2 Inhibitor, Enasidenib, to Usual Treatment (Cedazuridine-Decitabine) for Higher-Risk Myelodysplastic Syndrome (MDS) With IDH2 Mutation (A MyeloMATCH Treatment Trial)

ConditionMyelodysplastic Syndrome
Trial statusRecruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age18+
SponsorNational Cancer Institute (NCI)

About this trial

This phase II MyeloMATCH treatment trial compares the usual treatment of cedazuridine-decitabine (ASTX727) to the combination treatment of ASTX727 and enasidenib in treating patients with higher-risk, IDH2-mutated myelodysplastic syndrome (MDS). ASTX727 is a combination of two drugs, decitabine and cedazuridine. Cedazuridine is in a class of medications called cytidine deaminase inhibitors. It prevents the breakdown of decitabine, making it more available in the body so that decitabine will have a greater effect. Decitabine is in a class of medications called hypomethylation agents. It works by helping the bone marrow produce normal blood cells and by killing abnormal cells in the bone marrow. Enasidenib is an enzyme inhibitor that may stop the growth of cells by blocking some of the enzymes needed for cell growth. Giving ASTX727 in combination with enasidenib may be effective in treating patients with higher-risk IDH2-mutated MDS.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

Patients must be registered to the Master Screening and Reassessment Protocol (MYELOMATCH) and assigned to this protocol by the MATCHBox Treatment Verification Team.

Participants must not have received prior anti-cancer therapy for AML or MDS.

Note: Hydroxyurea to control the white blood cell count (WBC) is allowed.

Note: Prior erythroid stimulating agent (ESA) is not considered prior therapy for the purposes of eligibility.

Disqualifiers

None

Trial design

Design model

Parallel

Treatments tested in this trial

  • Biospecimen Collection

    Procedure/Surgery

    Undergo buccal swab and blood sample collection

  • Bone Marrow Aspiration

    Procedure/Surgery

    Undergo bone marrow aspiration

  • Bone Marrow Biopsy

    Procedure/Surgery

    Undergo bone marrow biopsy

  • Decitabine and Cedazuridine

    Drug

    Given PO

  • Enasidenib

    Drug

    Given PO

Treatment groups

54 Participants
are divided into 2 treatment groups
Group A: Regimen 1 (ASTX727)Active comparator 4 interventions
Group B: Regimen 2 (ASTX727, enasidenib)Experimental treatment 5 interventions

Trial outcomes

Primary outcomes

1

Complete response (CR) rate

Will be assessed using the International Working Group 2023 (IWG2023) criteria. Will compare the CR rate between the two treatment arms to determine if patients treated with enasidenib + ASTX727 have a statistically significantly higher CR rate than patients treated with the ASTX727 monotherapy.

Time frame
Up to 4 cycles of treatment

Secondary outcomes

1

Event-free survival (EFS)

Sensitivity analyses will be conducted. Will use the methods of Kaplan-Meier as well as Cox regression models.

Time frame
Time from randomization to either a failure to achieve a CR, CR with limited count recovery (CRL), or CR with partial count recovery (CRh) after four cycles of treatment, relapse, or death due to any cause, assessed up to 18 months
2

Overall survival (OS)

Will use the methods of Kaplan-Meier as well as Cox regression models.

Time frame
Time from randomization to death due to any cause, assessed up to 18 months
3

Time to response

Will be assessed only among patients who achieve a response (CR, CRL, or CRh). Will utilize the methods of Kaplan-Meier as well as Cox regression models.

Time frame
Time from randomization to documented response, assessed up to 5 years
4

Duration of response

Will be assessed only among patients who achieve a response (CR, CRL, or CRh). Will use the methods of Kaplan-Meier as well as Cox regression models.

Time frame
From patient first achieves a response until either progression or death, assessed up to 5 years

Other outcomes

Sponsors and contacts

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

National Cancer Institute (NCI)

Lead sponsor

Source ClinicalTrials.gov
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