A Study to Compare the Efficacy and Safety of Oral Azacitidine (Oral-Aza, ONUREG) plus BSC versus Placebo plus BSC in Participants with IPSS-R Low- or Intermediate-risk MDS

JRCT ID: jRCT2071220092

Registered date:18/01/2023

Basic Information

• Recruitment status: Not Recruiting
• Health condition(s) or Problem(s) studied: Myelodysplastic Syndrome
• Date of first enrollment: 12/04/2023
• Target sample size: 5
• Countries of recruitment: US,Japan,Canada,Japan,Argentina,Japan,Australia,Japan,Korea Republic,Japan,Germany,Japan,France,Japan,Italy,Japan,Spain,Japan,Denmark,Japan,Switzerland,Japan,Czech Republic,Japan,Poland,Japan,Austria,Japan,Sweden,Japan,Israel,Japan,Turkey,Japan
• Study type: Interventional

Intervention(s)

- Phase2-Oral azacitidine fix dose per protocol
- Phase3: Arm A: Oral Azacitidine dose determined according to Phase 2 and Arm B: Pracebo

Outcome(s)

Primary Outcome

- Phase2-Number of participants with Adverse Events (AEs) evaluated using the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) criteria v.5.0
- Phase2/3:Number of participants who achieved complete remission (CR) per International Working Group (IWG) 2006 criteria within 6 cycles

Secondary Outcome

- Phase2/3:Number of participants who achieved Overall Response (OR) per IWG 2006 criteria within 6 cycles
- Phase2/3:Number of participants who achieved 84-day packed red blood cells transfusion independence (pRBC-TI)

Key inclusion & exclusion criteria

• Age minimum: >= 20age old
• Age maximum: Not applicable
• Gender: Both

Include criteria

- Participant has a documented diagnosis of MDS according to WHO 2016 classification that meets International Prognostic Scoring System Revised (IPSS-R) classification 17 of low- or intermediate-risk disease (IPSS-R score between 1.5 and 4.5).
- Participant must have an Eastern Cooperative Oncology Group (ECOG) performance status of 0, 1, or 2.

Exclude criteria

- Participants with prior malignancies must have an expected median life expectancy of at least 12 months at the time of inclusion and no active treatment of any sort for at least 24 weeks prior to randomization (including but not limited to immunotherapy or targeted therapy)
- Hypoplastic Myelodysplastic Syndrome (MDS) with a marrow cellularity of <- 10%
- Participants diagnosed with MDS with excess blasts-2 (MDS-EB2)
- Prior treatment with azacitidine (any formulation), decitabine, or other hypomethylating agent

Related Information

• Primary Sponsor: Carlos Vigil
• Secondary ID(s): NCT05469737