The Efficacy and Safety of Oral Azacitidine Plus Best Supportive Care Versus Placebo and Best Supportive Care in Subjects With Red Blood Cell (RBC) Transfusion-Dependent Anemia and Thrombocytopenia Due to International Prognostic Scoring System (IPSS) Low Risk Myelodysplastic Syndrome (MDS)
CompletedPhase 3Results postedLast updated 7 January 2025
What this trial tests
Phase 3 trial testing Oral Azacitidine in Myelodysplastic Syndrome in 216 participants. Completed in 21 December 2023.
18 and older, any sex, with Myelodysplastic Syndrome. Patients with the condition only — healthy volunteers not accepted.
Results — posted to ClinicalTrials.gov
Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.
Percentage of Participants Who Achieved Red Blood Cell (RBC) Transfusion Independence for ≥ 56 DaysPrimary· Each participant was assessed for at least 56 days or more; from the date of randomization of study drug up to the data cut-off date of 25 January 2019, approximately 5 months.
RBC transfusion (tfx) independence was defined as the absence of any RBC transfusion during any consecutive "rolling" 56 days within the treatment period. Participants who did not receive any RBC transfusion during a consecutive rolling 56 days (i.e., day 1 to day 56, day 2 to day 57) were considered as a 56-day RBC transfusion independent responder.
Group
Value
95% CI
Oral Azacitidine and Best Supportive Care
30.8
22.1 – 39.6
Placebo Plus Best Supportive Care
11.9
5.8 – 18.0
Duration of RBC Transfusion Independence Among Participants Who Achieved RBC Transfusion Independence for at Least 56 DaysSecondary· From the date of randomization of study drug up to the data cut-off date of 25 January 2019
Duration of RBC transfusion independence was analyzed only for participants who achieved RBC transfusion independence of ≥ 56 days on treatment. Duration of RBC transfusion independence was defined as the time from the date transfusion independence is first observed (day 1 of a ≥ 56 days period without a transfusion) until the date the participants had a subsequently documented RBC transfusion. In the event a participant had more than one ≥56 days rolling periods which met the RBC independence criteria, the duration with the longest rolling period was used in the analysis. Participants who mai
Group
Value
95% CI
Oral Azacitidine and Best Supportive Care
11.1
8.2 – 26.0
Placebo Plus Best Supportive Care
12.0
2.3 – NA
Time to RBC Transfusion Independence for at Least 56 Days Among Participants Who Achieved RBC Transfusion Independence for at Least 56 DaysSecondary· From the date of randomization of study drug up to the data cut-off date of 25 January 2019; median duration of treatment to oral azacitidine was 5.29 months and 5.36 months for placebo
Time to RBC transfusion independence of ≥ 56 days was defined as the time between randomization and the date onset of transfusion independence was first observed (ie, Day 1 of 56 without any RBC transfusions).
Group
Value
95% CI
Oral Azacitidine and Best Supportive Care
2.37
0.0 – 10.9
Placebo Plus Best Supportive Care
2.04
0.0 – 14.3
Duration of RBC Transfusion Reduction for Participants Who Achieved RBC Transfusion Reduction of at Least 4 Units of RBCs for at Least 8 WeeksSecondary· From the date of randomization of study drug up to the treatment period; up to the data cut-off date of 25 January 2019; median duration of treatment to oral azacitidine was 5.29 months and 5.36 months for placebo
A participant was considered as a RBC transfusion reduction responder if the participant had at least 4 units reduction in transfusion units over any consecutive 56 days period compared to the baseline transfusion units in 56 days.
Group
Value
95% CI
Oral Azacitidine and Best Supportive Care
10.0
7.1 – 13.3
Placebo Plus Best Supportive Care
2.3
2.0 – 5.0
Percentage of Participants Who Achieved RBC Transfusion Independence for ≥ 84 DaysSecondary· From the date of randomization of study drug up to the treatment period; up to the data cut-off date of 25 January 2019; median duration of treatment to oral azacitidine was 5.29 months and 5.36 months for placebo
RBC transfusion independence was defined as the absence of any red blood cell (RBC) transfusion during any consecutive "rolling" 84 days within the treatment period. Participants who did not receive any RBC transfusion during a consecutive rolling 84 days (i.e., day 1 to day 84, day 2 to day 85) were considered as a 84-day RBC transfusion independent responder.
Group
Value
95% CI
Oral Azacitidine and Best Supportive Care
28.0
19.5 – 36.5
Placebo Plus Best Supportive Care
6.4
1.8 – 11.0
Duration of RBC Transfusion Independence Among Participants Who Achieved RBC Transfusion Independence for at Least 84 DaysSecondary· From the date of randomization of study drug up to the treatment period; up to the data cut-off date of 25 January 2019; median duration of treatment to oral azacitidine was 5.29 months and 5.36 months for placebo
Duration of RBC transfusion independence was analyzed only for participants who achieved RBC transfusion independence of ≥ 84 days on treatment. Duration of RBC transfusion independence was defined as the time from the date transfusion independence is first observed (day 1 of a ≥ 84 days period without a transfusion) until the date the participants had a subsequently documented RBC transfusion. In case a participant had more than one ≥84 days rolling periods which met the RBC independence criteria, the duration with the longest rolling period was used in the analysis.
Group
Value
95% CI
Oral Azacitidine and Best Supportive Care
11.1
8.2 – 26.0
Placebo Plus Best Supportive Care
NA
5.0 – NA
Time to RBC Transfusion Independence for at Least 84 Days Among Participants Who Achieved RBC Transfusion Independence for at Least 84 DaysSecondary· From the date of randomization of study drug up to the treatment period; up to the data cut-off date of 25 January 2019; median duration of treatment to oral azacitidine was 5.29 months and 5.36 months for placebo
Time to RBC transfusion independence of ≥ 84 days was defined as the time between randomization and the date onset of transfusion independence was first observed (ie, Day 1 of 84 without any RBC transfusions).
Group
Value
95% CI
Oral Azacitidine and Best Supportive Care
2.64
0.0 – 9.9
Placebo Plus Best Supportive Care
4.01
0.5 – 14.3
Percentage of Participants With an Erythroid Hematological Improvement (HI-E) Response According to 2006 IWG CriteriaSecondary· From the date of randomization of study drug up to the treatment period; up to the data cut-off date of 25 January 2019; median duration of treatment to oral azacitidine was 5.29 months and 5.36 months for placebo
Erythroid HI-E improvement was defined as a hemoglobin increase of ≥ 1.5 g/dL; or a reduction in units of RBC transfusions by an absolute number of at least 4 RBC transfusions/8 weeks compared with the pretreatment transfusion number in the previous 8 weeks. Only RBC transfusions given for a hemoglobin of ≤ 9.0 g/dL on treatment were counted in the RBC transfusion response evaluation.
HI-E Response
Group
Value
95% CI
Oral Azacitidine and Best Supportive Care
43.0
33.6 – 52.4
Placebo Plus Best Supportive Care
32.1
23.3 – 40.9
≥ 1.5 g/dL Hemoglobin Increase
Group
Value
95% CI
Oral Azacitidine and Best Supportive Care
23.4
15.3 – 31.4
Placebo Plus Best Supportive Care
5.5
1.2 – 9.8
RBC Transfusion Reduction
Group
Value
95% CI
Oral Azacitidine and Best Supportive Care
42.1
32.7 – 51.4
Placebo Plus Best Supportive Care
31.2
22.5 – 39.9
Percentage of Participants With a Hematological Improvement Response in Platelets (HI-P) According to 2006 IWG CriteriaSecondary· From the date of randomization of study drug up to the treatment period; up to the data cut-off date of 25 January 2019; median duration of treatment to oral azacitidine was 5.29 months and 5.36 months for placebo
HI-P response was defined according to IWG 2006 criteria (Cheson, 2006) and as: 1. Absolute increase of ≥ 30 X 10\^9/L for participants\^ starting with \> 20 X 10\^9/L platelets; 2. Increase from \< 20 X 10\^9/L to \> 20 X 10\^9/L and by at least 100%. HI-P must have lasted at least 8 weeks.
Group
Value
95% CI
Oral Azacitidine and Best Supportive Care
24.3
16.2 – 32.4
Placebo Plus Best Supportive Care
7.3
2.4 – 12.2
Percentage of Participants Who Achieved Platelet Transfusion Independence With a Duration of ≥ 8 Weeks (56 Days)Secondary· From the date of randomization of study drug up to the treatment period; up to the data cut-off date of 25 January 2019; median duration of treatment to oral azacitidine was 5.29 months and 5.36 months for placebo
Platelet transfusion independence was defined as the absence of any platelet transfusion during any consecutive "rolling" 56 days during the treatment period, (ie, Day 1 to 56, Day 2 to 57, Days 3 to 58, etc.). Participants were considered platelet transfusion dependent at baseline if they had received ≥ 2 platelet transfusions during the 56 days immediately preceding randomization and had no consecutive 28-day period during which no platelet transfusions were administered.
Group
Value
95% CI
Oral Azacitidine and Best Supportive Care
16.7
Placebo Plus Best Supportive Care
14.3
Time to Platelet Transfusion IndependenceSecondary· From the date of randomization of study drug up to the treatment period; up to the data cut-off date of 25 January 2019; median duration of treatment to oral azacitidine was 5.29 months and 5.36 months for placebo
Time to platelet transfusion independence was defined as the time between randomization and the first documented date of onset of transfusion independence (ie, Day 1 of 56 without any platelet transfusions).
Group
Value
95% CI
Oral Azacitidine and Best Supportive Care
9.6
9.6 – 10.9
Placebo Plus Best Supportive Care
NA
NA – NA
Overall Survival (OS)Secondary· From randomization up to death from any cause; up to a maximum of approximately 10 years on study; median duration of treatment to oral azacitidine was 5.29 months and 5.36 months for placebo
Overall survival was defined as the time from randomization to death from any cause and was calculated using randomization date and date of death, or date of last follow-up for censored participants. All subjects were followed until drop out (withdrawal of consent from further data collection or lost to follow-up), death, or study closure. Participants who dropped out or were alive at study closure (or at the time of the interim analysis) had their OS times censored at the time of last contact, as appropriate.
Group
Value
95% CI
Oral Azacitidine and Best Supportive Care
17.3
12.9 – 20.8
Placebo Plus Best Supportive Care
16.7
12.8 – 24.0
Adverse events — posted to ClinicalTrials.gov
Time frame: Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months).
Reporting threshold: 5%.
Adverse-event reports describe events observed during the trial — not all are caused by the drug.
Oral Azacitidine and Best Supportive Care
Serious: 83/107 (78%)
Deaths: 83/107
Placebo Plus Best Supportive Care
Serious: 69/109 (63%)
Deaths: 86/109
Serious adverse events (192 terms)
Reaction
System
Oral Azacitidine and Best …
Placebo Plus Best Supporti…
Febrile neutropenia
Blood and lymphatic system disorders
—
—
Pneumonia
Infections and infestations
—
—
Pyrexia
General disorders
—
—
Sepsis
Infections and infestations
—
—
Anaemia
Blood and lymphatic system disorders
—
—
Septic shock
Infections and infestations
—
—
Transformation to acute myeloid leukaemia
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
—
—
Thrombocytopenia
Blood and lymphatic system disorders
—
—
Neutropenic sepsis
Infections and infestations
—
—
Lower respiratory tract infection
Infections and infestations
—
—
Upper respiratory tract infection
Infections and infestations
—
—
Fall
Injury, poisoning and procedural complications
—
—
Cardiac failure congestive
Cardiac disorders
—
—
Abdominal pain
Gastrointestinal disorders
—
—
Diarrhoea
Gastrointestinal disorders
—
—
General physical health deterioration
General disorders
—
—
Myelodysplastic syndrome
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Evaluation of the Efficacy and Safety of Oral Azacitidine plus Best Supportive care versus Placebo and Best Supportive care in subjects with red blood cell (RBC) transfusion-dependent anemia and thrombocytopenia due to International Prognostic Scoring System (IPSS) lower risk myelodysplastic syndromes (MDS).
Publications & conference data
8 peer-reviewed publications reference this trial (live from Europe PMC):
NCT06370000 — Oral Azacitidine in Transplant-Eligible Patients With Acute Myeloid Leukemia (AML) Suffering From Health-Inequality
· Phase 4
· recruiting
NCT05469737 — A Study to Compare the Efficacy and Safety of Oral Azacitidine Plus Best Supportive Care (BSC) Versus Placebo Plus BSC i
· Phase 2, PHASE3
· active not recruiting
NCT05141682 — Oral Azacitidine for the Treatment of Relapsed or Refractory T-cell Large Granular Lymphocytic Leukemia
· Phase 1, PHASE2
· active not recruiting
NCT05197426 — A Study to Compare the Efficacy and Safety of Oral Azacitidine Plus Best Supportive Care Versus Best Supportive Care as
· Phase 2
· active not recruiting
NCT05162976 — CC-486 and Nivolumab for the Treatment of Hodgkin Lymphoma Refractory to PD-1 Therapy or Relapsed
· Phase 1
· active not recruiting
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Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
Sponsor: as reported to ClinicalTrials.gov by Celgene
Last refreshed: 7 January 2025
Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT01566695.