DrugEvidence

Drugs / Azacitidine

Azacitidine

Drug

Azacitidine is an antineoplastic nucleoside analogue medicine used in myelodysplastic syndrome and myeloid leukemias. It is marketed under brands including Vidaza and Onureg. Its activity includes hypomethylating and cytotoxic effects depending on dosing context.

Trials
745
Recruiting
161
Completed
246
With results
199

Last revised

Pipeline

Phase by status

PhaseRecruitingNot yetBy invitationActiveCompletedTerminatedWithdrawnSuspendedUnknownApproved for marketingNo longer availableTotal
Early Phase 1 331 7
Phase 1 2842138239742 181
Phase 1/2 2651736271329 135
Phase 2 72192239441832 291
Phase 2/3 721222 16
Phase 3 14915191314 66
Phase 4 22212 9
Not applicable 7315110 27
Unknown 2232211 13

Trials started per year

2021
12
2022
52
2023
42
2024
70
2025
44
2026
65
2027
1

Conditions studied, by phase

ConditionEarly Phase 1Phase 1Phase 1/2Phase 2Phase 2/3Phase 3Phase 4Not applicableUnknownTotal
Leukemia, Myeloid, Acute 11026714911455128 400
Myelodysplastic Syndromes 2674882514325 228
Leukemia, Myelomonocytic, Chronic 123262114 76
Leukemia 171424271 65
Recurrence 1191222 36
Myeloproliferative Disorders 7108 25
Neoplasms 67521 21
Hematologic Neoplasms 97211 20
Carcinoma, Non-Small-Cell Lung 6371 17
Multiple Myeloma 8531 17

Most active sponsors

#SponsorTrialsPhase 3
1M.D. Anderson Cancer Center651
2National Cancer Institute (NCI)372
3Celgene367
4The First Affiliated Hospital of Soochow University295
5Institute of Hematology & Blood Diseases Hospital, China194
6AbbVie136
7Groupe Francophone des Myelodysplasies120
8Novartis Pharmaceuticals122
9Bristol-Myers Squibb91
10Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins90

Most studied interventions

#InterventionTrials
1Azacitidine745
2venetoclax219
3Cytarabine93
4Decitabine54
5cc-48637
6Daunorubicin37
7Lenalidomide31
8Idarubicin27
9N-(2-amino-5-fluorobenzyl)-4-(N-(pyridine-3-acrylyl)aminomethyl)benzamide21
10Specimen Handling19

Evidence

Results reported
80.9%199 of 246 completed
Published
9.8%24 of 246 completed
Primary endpoint met
36.5%19 of 52 analyses
Median months to report
12

Documents disclosed

Protocol available
129
Statistical analysis plan
128
PubMed citations
59

Trial profile

Median enrollment
41
Median duration
47.2months
Countries
58
Study sites
8095

Who is enrolled

Adults
729
Children
83
Older adults
705
Healthy volunteers
4

Median enrollment by phase

Early Phase 1
6
Phase 1
30
Phase 1/2
45
Phase 2
40
Phase 2/3
230
Phase 3
243
Phase 4
44
Not applicable
35
Unknown
112

Where trials run

United States
440
China
162
France
110
Spain
84
Germany
81
Canada
66
Australia
65
Italy
65
United Kingdom
63
Belgium
40

Busiest cities

#CityCountryTrials
1HoustonUnited States186
2New YorkUnited States118
3ChicagoUnited States93
4BostonUnited States83
5Los AngelesUnited States74
6ParisFrance74
7BarcelonaSpain66
8BaltimoreUnited States65
9ClevelandUnited States62
10DallasUnited States61

Terminations

Terminated
121
Withdrawn
33
Suspended
6
Stopped for recruitment
4.3%32 of 745 trials

Stated reasons

Other / unclear
65
Recruitment
32
Business decision
23
Lack of efficacy
15
Funding
13
Safety
7
Regulatory
5

Reasons are grouped from the sponsor's own wording with a rule-based classifier; the original text is shown below.

Recently stopped

TrialStatusStated reasonStopped
NCT06694454 Suspended Suspended Placed on voluntary hold to new recruitment pending modification to combination monoclonal antibody treatment. 2034-12-31
NCT05766514 Withdrawn Withdrawn Study disapproved by internal scientific review committee on re-review; study will no longer move forward 2030-12-31
NCT06454409 Suspended Suspended resources 2030-09-06
NCT05367401 Withdrawn Withdrawn waiting for update from Study Lead 2029-10-26
NCT06598332 Withdrawn Withdrawn There is no funding. 2029-05-01
NCT06180863 Withdrawn Withdrawn Lack of funding. 2029-04-30
NCT07388563 Suspended Suspended New recruitment on hold for a protocol design amendment/modification. 2027-12-31
NCT06514261 Suspended Suspended Other - Pending amendment for study expansion. 2027-09-16
NCT06466798 Withdrawn Withdrawn Inability to find participants meeting eligibility critieria 2027-07-01
NCT06903702 Withdrawn Withdrawn Lack of funding 2027-06-30

Trial records