A Study to Compare the Efficacy and Safety of Oral Azacitidine Plus Best Supportive Care (BSC) Versus Placebo Plus BSC in Participants With International Prognostic Scoring System Revised (IPSS-R) Low- or Intermediate-risk Myelodysplastic Syndrome (MDS)
Myelodysplastic Syndromes
About this trial
This is an interventional treatment trial for Myelodysplastic Syndromes focused on measuring Anemia, Thrombocytopenia
Eligibility Criteria
Inclusion 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).
MDS diagnosis, WHO classification, and IPSS-R risk classification will be prospectively determined by independent central pathology and cytogenetics review, and applicable central laboratory results.
• Participant must have an Eastern Cooperative Oncology Group (ECOG) performance status of 0, 1, or 2.
Exclusion 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
Other protocol-defined inclusion/exclusion criteria apply
Sites / Locations
- University of Miami Hospital and Clinics, Sylvester Cancer Center
- Advanced Research
- Moffitt Cancer Center
- Henry Ford Hospital
- Rutgers Cancer Institute of New Jersey
- Hematology-Oncology Associates of Central New York, PCRecruiting
- Memorial Sloan Kettering Cancer Center
- Novant Health Presbyterian Medical Center
- Oregon Health and Science University
- AHN West Penn HospitalRecruiting
- Charleston Oncology, P.A.
- Houston Methodist Hospital
- University of Texas MD Anderson Cancer CenterRecruiting
- Huntsman Cancer Institute
- Inova Schar Cancer InstituteRecruiting
- Local Institution - 0070Recruiting
- Local Institution - 0039Recruiting
- Local Institution - 0021
- Local Institution - 0016Recruiting
- Local Institution - 0022Recruiting
- Local Institution - 0050Recruiting
- Local Institution - 0144
- Local Institution - 0145
- Local Institution - 0146
- Local Institution - 0006Recruiting
- Local Institution - 0069
- Local Institution - 0018Recruiting
- Local Institution - 0004Recruiting
- Local Institution - 0003Recruiting
- Local Institution - 0034
- Local Institution - 0029
- Local Institution - 0074
- Local Institution - 0091
- Local Institution - 0008Recruiting
- Local Institution - 0015Recruiting
- Local Institution - 0090Recruiting
- Local Institution - 0171
- Local Institution - 0167
- Local Institution - 0156
- Local Institution - 0157
- Local Institution - 0168
- Local Institution - 0162
- Local Institution - 0163
- Local Institution - 0179
- Local Institution - 0166
- Local Institution - 0160
- Local Institution - 0169
- Local Institution - 0164
- Local Institution - 0165
- Local Institution - 0141
- Local Institution - 0161
- Local Institution - 0170
- Local Institution - 0009
- Local Institution - 0060Recruiting
- Local Institution - 0045
- Local Institution - 0115Recruiting
- Local Institution - 0116
- Local Institution - 0114
- Centre Hospitalier Universitaire de Nice - Hôpital l'Archet
- CHU Bordeaux Haut-LevequeRecruiting
- Centre Hospitalier Régional Universitaire de Tours - Hôpital BretonneauRecruiting
- Centre Hospitalier Universitaire d'AngersRecruiting
- Hopital Claude Huriez - CHU de LilleRecruiting
- Gustave RoussyRecruiting
- Hôpital Saint-LouisRecruiting
- Local Institution - 0059
- Local Institution - 0081Recruiting
- Local Institution - 0128Recruiting
- Local Institution - 0055Recruiting
- Local Institution - 0037Recruiting
- Local Institution - 0007Recruiting
- Local Institution - 0076
- Local Institution - 0028Recruiting
- Local Institution - 0125Recruiting
- Local Institution - 0129Recruiting
- Local Institution - 0127Recruiting
- Local Institution - 0178
- Fondazione Policlinico Tor VergataRecruiting
- Local Institution - 0088
- Local Institution - 0052Recruiting
- Local Institution - 0075Recruiting
- Azienda Ospedale - Università Padova
- IRCCS Azienda Ospedaliero-Universitaria di Bologna, Policlinico di Sant'OrsolaRecruiting
- Local Institution - 0067
- Local Institution - 0053
- Local Institution - 0143
- Local Institution - 0136Recruiting
- Local Institution - 0154Recruiting
- Local Institution - 0153
- Local Institution - 0130Recruiting
- Local Institution - 0135Recruiting
- Local Institution - 0150Recruiting
- Local Institution - 0124Recruiting
- Local Institution - 0058Recruiting
- Local Institution - 0048Recruiting
- Local Institution - 0036Recruiting
- Local Institution - 0012Recruiting
- Local Institution - 0051Recruiting
- Local Institution - 0044
- Local Institution - 0097Recruiting
- Local Institution - 0043
- Local Institution - 0105
- Local Institution - 0106
- Local Institution - 0112Recruiting
- Local Institution - 0109Recruiting
- Local Institution - 0107Recruiting
- Local Institution - 0111Recruiting
- Local Institution - 0110Recruiting
- Local Institution - 0108Recruiting
- Local Institution - 0117
- Karolinska Universitetssjukhuset HuddingeRecruiting
- Local Institution - 0113
- Local Institution - 0119Recruiting
- Local Institution - 0077
- Local Institution - 0066
- Local Institution - 0139
- Local Institution - 0138
- Local Institution - 0140
Arms of the Study
Arm 1
Arm 2
Arm 3
Arm 4
Experimental
Experimental
Experimental
Experimental
Part I - Oral-Aza (Dose 1)
Part I - Oral-Aza (Dose 2)
Part II - Oral-Aza (RP3D)
Part II - Placebo
RP3D: Recommended Phase 3 Dose