A Phase 2/3, Multicenter, Randomized, Dose Optimization (Part I), Double-blind (Part II) Study to Compare the Efficacy and Safety of Oral Azacitidine (Oral-Aza, ONUREG®) plus Best Supportive Care (BSC) versus Placebo plus BSC in Participants with IPSS-R Low- or Intermediate-risk Myelodysplastic Syndrome (MDS)

Conditions

International Prognostic Scoring System-Revised (IPSS-R) Low - or Intermediate - risk Myelodysplastic Syndrome (MDS)

Brief summary

For Part I (Phase 2) - AEs evaluated using NCI CTCAE criteria, v.5.0, including TEAEs, laboratory assessments, and vital signs, For Part I (Phase 2) - Achievement of CR per IWG 2006 criteria within 6 cycles, For Part II (Phase 3) - Achievement of CR within 6 cycles

Detailed description

For Part I (Phase 2) - Achievement of OR within 6 cycles (CR, PR, mCR, HI-E, HI-P, H-IN per IWG 2006 criteria), best OR; OR duration, For Part I (Phase 2) - CR duration, For Part I (Phase 2) - Achievement of 84-day pRBC-TI within 6 cycles; pRBC-TI duration, For Part I (Phase 2) - Achievement of 84-day PLT-TI within 6 cycles; PLT-TI duration, For Part II (Phase 3) - Achievement of 84-day pRBC-TI within 6 cycles, For Part II (Phase 3) - pRBC-TI duration, For Part II (Phase 3) - Achievement of 84-day platelet-TI within 6 cycles, For Part II (Phase 3) - Platelet-TI duration, For Part II (Phase 3) - Achievement of pRBC transfusion reduction; pRBC transfusion reduction duration, For Part II (Phase 3) - CR duration, For Part II (Phase 3) - Achievement of OR within 6 cycles (CR, PR, mCR, HI-E, HI-P, H-IN per IWG 2006 criteria), best OR; OR duration, For Part II (Phase 3) - OS, EFS (event: death, AML, MDS-EB 2), time to AML, time to subsequent therapy, For Part II (Phase 3) - Iron parameters, For Part II (Phase 3) - AEs evaluated using NCI CTCAE criteria, v.5.0, including TEAEs, laboratory assessments and vital signs, For Part II (Phase 3) - Change from baseline in the FACT-An, QUALMS, and EQ-5D-5L, For Part II (Phase 3) - Evaluation of healthcare resource use (eg, hospitalization) associated with IP during study

Related papers

No related papers found yet.

Interventions

DRUGazacitidine

DRUGAzacitidine 200 mg tablet

DRUGAzacitidine 300 mg tablet - describe tablets

Eligibility

Sex/Gender

All

Age

18 Years to No maximum

Design outcomes

Primary

Measure	Time frame
For Part I (Phase 2) - AEs evaluated using NCI CTCAE criteria, v.5.0, including TEAEs, laboratory assessments, and vital signs, For Part I (Phase 2) - Achievement of CR per IWG 2006 criteria within 6 cycles, For Part II (Phase 3) - Achievement of CR within 6 cycles	—

Secondary

Measure	Time frame
For Part I (Phase 2) - Achievement of OR within 6 cycles (CR, PR, mCR, HI-E, HI-P, H-IN per IWG 2006 criteria), best OR; OR duration, For Part I (Phase 2) - CR duration, For Part I (Phase 2) - Achievement of 84-day pRBC-TI within 6 cycles; pRBC-TI duration, For Part I (Phase 2) - Achievement of 84-day PLT-TI within 6 cycles; PLT-TI duration, For Part II (Phase 3) - Achievement of 84-day pRBC-TI within 6 cycles, For Part II (Phase 3) - pRBC-TI duration, For Part II (Phase 3) - Achievement of 84-day platelet-TI within 6 cycles, For Part II (Phase 3) - Platelet-TI duration, For Part II (Phase 3) - Achievement of pRBC transfusion reduction; pRBC transfusion reduction duration, For Part II (Phase 3) - CR duration, For Part II (Phase 3) - Achievement of OR within 6 cycles (CR, PR, mCR, HI-E, HI-P, H-IN per IWG 2006 criteria), best OR; OR duration, For Part II (Phase 3) - OS, EFS (event: death, AML, MDS-EB 2), time to AML, time to subsequent therapy, For Part II (Phase 3) - Iron parameter	—

Measure

Time frame

For Part I (Phase 2) - Achievement of OR within 6 cycles (CR, PR, mCR, HI-E, HI-P, H-IN per IWG 2006 criteria), best OR; OR duration, For Part I (Phase 2) - CR duration, For Part I (Phase 2) - Achievement of 84-day pRBC-TI within 6 cycles; pRBC-TI duration, For Part I (Phase 2) - Achievement of 84-day PLT-TI within 6 cycles; PLT-TI duration, For Part II (Phase 3) - Achievement of 84-day pRBC-TI within 6 cycles, For Part II (Phase 3) - pRBC-TI duration, For Part II (Phase 3) - Achievement of 84-day platelet-TI within 6 cycles, For Part II (Phase 3) - Platelet-TI duration, For Part II (Phase 3) - Achievement of pRBC transfusion reduction; pRBC transfusion reduction duration, For Part II (Phase 3) - CR duration, For Part II (Phase 3) - Achievement of OR within 6 cycles (CR, PR, mCR, HI-E, HI-P, H-IN per IWG 2006 criteria), best OR; OR duration, For Part II (Phase 3) - OS, EFS (event: death, AML, MDS-EB 2), time to AML, time to subsequent therapy, For Part II (Phase 3) - Iron parameter

—

Countries

Austria, Czechia, Denmark, France, Germany, Greece, Italy, Poland, Spain, Sweden

Outcome results

None listed