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Azacitidine

  • Prescription medicine
  • Given by a clinician
  • Identity checked
  • Sources last checked 2026-09-04

This page shows what was measured, who it was measured in, and what that does not settle.

What Azacitidine does in the body

ONUREG is indicated for continued treatment of adult patients with acute myeloid leukemia who achieved first complete remission (CR) or complete remission with incomplete blood count recovery (CRi) following intensive induction chemotherapy and are not able to complete intensive curative therapy.

From the FDA-approved label: Azacitidine is a pyrimidine nucleoside analog of cytidine that inhibits DNA/RNA methyltransferases. Azacitidine is incorporated into DNA and RNA following cellular uptake and enzymatic biotransformation to nucleotide triphosphates. Incorporation of azacitidine into the DNA of cancer cells in vitro, including acute myeloid leukemia cells, inhibited DNA methyltransferases, reduced DNA methylation and altered gene expression, including re-expression of genes regulating tumor suppression and cell differentiation.

Why people take it. ONUREG is indicated for continued treatment of adult patients with acute myeloid leukemia who achieved first complete remission (CR) or complete remission with incomplete blood count recovery (CRi) following intensive induction chemotherapy and are not able to complete intensive curative therapy.

What happened in people

RNAWiki has not yet published a reviewed conclusion for this use.

A fixed RNAWiki sentence

Where this came from

Wording RNAWiki always uses, not a finding about this substance.

No reviewed claim names a result for any goal on this record.

No source is stored against this line.

The limit that matters most

Not recorded.

Where it acts
Not recorded.
Kind of result
No result is published, so no kind of result applies yet
Supervision
Professional supervision is normally required. A regulator classifies this substance in a way that restricts how it is supplied.

What the registries record it as

  • The substance registry classes this as chemical.

    FDA substance registry · M801H13NRU · read 2026-08-29

  • Its recorded molecular formula is C8H12N4O5·Th, weighing 244.20.

    US prescribing information · 2d15b3ec-ed08-48c6-a400-34e55974afad · read 2026-08-30

Where each sentence above came from

The use the label states, quoted from it. No plain-language version of this sentence has been written.

The use the label states, quoted from it. It is written for a clinician, not for a reader without medical training.

No statement of the main limit is recorded.

The four opening statements run to 162 words.

Words this page uses

Four words worth knowing first

Chosen from what this page shows, with each one explained before the word it depends on.

Biomarker

A biomarker is a number from a test that stands in for something about health.

A picture of it, and where the picture fails

A biomarker is like a fuel gauge.

Where that stops being true. A gauge is wired to the tank. Many biomarkers are only loosely tied to health.

What people get wrong. A better number is read as a better life. Several medicines improved a number and helped nobody.

A measurable indicator used as a substitute for a clinical outcome of interest.

Placebo

A placebo is a dummy treatment given so the real one can be compared with it.

A picture of it, and where the picture fails

A placebo is like a blank control in an experiment.

Where that stops being true. A blank does nothing. People given a placebo often do get better.

What people get wrong. A placebo effect is read as imaginary. The improvement is measured and real.

An inactive intervention matched in appearance to the test intervention, used to control for non-specific effects.

Comparator

A comparator is whatever the treatment was measured against.

A picture of it, and where the picture fails

It is like the other runner in a race.

Where that stops being true. A race has one winner. A study can show both arms improved.

What people get wrong. Results are read without asking what the other group got. Beating nothing is not beating a treatment.

The control condition against which the experimental intervention is assessed.

What happened in peopleRead from sources, not yet reviewed

What happened in people

Each card is one study: the exact question it asked, who was in it, and whether it showed what it set out to show.

Proportion of patients for whom molecular, immunophenotypic, and/or biochemical studies are completed in < 7 calendar days for assignment of treatment

The study did not show it

Who was studied
NCT03013998
How many people
3000
Study design
Phase 1/Phase 2
Compared against
Not recorded for this study
Kind of result
A number that stands in for health
What was found
Result not recorded on this page
Repeated elsewhere
Unreplicated

What this does not prove. A study that did not show an effect does not show that no effect exists anywhere.

The limits of this study, and where it came from

Main limit. No limitation is recorded for this study.

How far it carries. This study is recorded from the curated record, not from the registry match.

Form and route. Intravenous, subcutaneous

Interval reported. Not recorded in this summary

Written into the record, not signed off as a reviewed claim.

  • openFDA Drugs@FDA · a recorded source, not a stored snapshot
  • openFDA NDC Directory · a recorded source, not a stored snapshot
  • openFDA SPL label · a recorded source, not a stored snapshot
  • PubChem PUG-REST · a recorded source, not a stored snapshot

Timing of treatment Substudy or Tier Advancement Pathway (TAP) assignment

The study did not show it

Who was studied
NCT05564390
How many people
2000
Study design
Phase 2
Compared against
Not recorded for this study
Kind of result
A number that stands in for health
What was found
Result not recorded on this page
Repeated elsewhere
Unreplicated

What this does not prove. A study that did not show an effect does not show that no effect exists anywhere.

The limits of this study, and where it came from

Main limit. No limitation is recorded for this study.

How far it carries. This study is recorded from the curated record, not from the registry match.

Form and route. Intravenous, subcutaneous

Interval reported. Not recorded in this summary

Written into the record, not signed off as a reviewed claim.

  • openFDA Drugs@FDA · a recorded source, not a stored snapshot
  • openFDA NDC Directory · a recorded source, not a stored snapshot
  • openFDA SPL label · a recorded source, not a stored snapshot
  • PubChem PUG-REST · a recorded source, not a stored snapshot

Overall survival

The study did not show it

Who was studied
NCT00454480
How many people
2000
Study design
Phase 2/Phase 3
Compared against
Not recorded for this study
Kind of result
Living longer, or avoiding a major event
What was found
Result not recorded on this page
Repeated elsewhere
Unreplicated

What this does not prove. A study that did not show an effect does not show that no effect exists anywhere.

The limits of this study, and where it came from

Main limit. No limitation is recorded for this study.

How far it carries. This study is recorded from the curated record, not from the registry match.

Form and route. Intravenous, subcutaneous

Interval reported. Not recorded in this summary

Written into the record, not signed off as a reviewed claim.

  • openFDA Drugs@FDA · a recorded source, not a stored snapshot
  • openFDA NDC Directory · a recorded source, not a stored snapshot
  • openFDA SPL label · a recorded source, not a stored snapshot
  • PubChem PUG-REST · a recorded source, not a stored snapshot

Overall survival

The study did not show it

Who was studied
NCT02272478
How many people
1600
Study design
Phase 2/Phase 3
Compared against
Not recorded for this study
Kind of result
Living longer, or avoiding a major event
What was found
Result not recorded on this page
Repeated elsewhere
Unreplicated

What this does not prove. A study that did not show an effect does not show that no effect exists anywhere.

The limits of this study, and where it came from

Main limit. No limitation is recorded for this study.

How far it carries. This study is recorded from the curated record, not from the registry match.

Form and route. Intravenous, subcutaneous

Interval reported. Not recorded in this summary

Written into the record, not signed off as a reviewed claim.

  • openFDA Drugs@FDA · a recorded source, not a stored snapshot
  • openFDA NDC Directory · a recorded source, not a stored snapshot
  • openFDA SPL label · a recorded source, not a stored snapshot
  • PubChem PUG-REST · a recorded source, not a stored snapshot

Apyrexia at day 4

The study did not show it

Who was studied
NCT07483736
How many people
1526
Study design
Phase 3
Compared against
Not recorded for this study
Kind of result
A number that stands in for health
What was found
Result not recorded on this page
Repeated elsewhere
Unreplicated

What this does not prove. A study that did not show an effect does not show that no effect exists anywhere.

The limits of this study, and where it came from

Main limit. No limitation is recorded for this study.

How far it carries. This study is recorded from the curated record, not from the registry match.

Form and route. Intravenous, subcutaneous

Interval reported. Not recorded in this summary

Written into the record, not signed off as a reviewed claim.

  • openFDA Drugs@FDA · a recorded source, not a stored snapshot
  • openFDA NDC Directory · a recorded source, not a stored snapshot
  • openFDA SPL label · a recorded source, not a stored snapshot
  • PubChem PUG-REST · a recorded source, not a stored snapshot

Nonintensive Therapy Study: (Primary Endpoint for all countries): Overall survival (OS)

The study did not show it

Who was studied
NCT07007312
How many people
1300
Study design
Phase 3
Compared against
Not recorded for this study
Kind of result
Living longer, or avoiding a major event
What was found
Result not recorded on this page
Repeated elsewhere
Unreplicated

What this does not prove. A study that did not show an effect does not show that no effect exists anywhere.

The limits of this study, and where it came from

Main limit. No limitation is recorded for this study.

How far it carries. This study is recorded from the curated record, not from the registry match.

Form and route. Intravenous, subcutaneous

Interval reported. Not recorded in this summary

Written into the record, not signed off as a reviewed claim.

  • openFDA Drugs@FDA · a recorded source, not a stored snapshot
  • openFDA NDC Directory · a recorded source, not a stored snapshot
  • openFDA SPL label · a recorded source, not a stored snapshot
  • PubChem PUG-REST · a recorded source, not a stored snapshot
What we know
RNAWiki holds 6 written-up human studies for this substance, each with the question it asked.
How we know it
Each card names the study, the number of people and what the study set out to measure.
What this does not prove
These summaries were written by a person and have not been signed off as reviewed claims.
Why it matters
A study that failed is as informative as one that worked, and both are here.
What we still do not know
No reviewed claim exists, so no exact population, effect size or interval is published yet.

What is missing or unclearRead from sources, not yet reviewed

Were people like you studied?

RNAWiki cannot tell whether a study fits you. It can show who was in it, and where the result stops carrying.

Who was studied

People similar to this profile were included.

  • & USAGE Azacitidine for injection (Azacitidine), is a nucleoside metabolic inhibitor indicated for the treatment of: •Adult patients with the following FAB myelodysplastic syndrome (MDS) subtypes: Refractory anemia (RA) or refractory anemia with ringed sideroblasts (RARS) (if accompanied by neutropenia or thrombocytopenia or requiring transfusions), refractory anemia with excess blasts (RAEB), refractory anemia with…

Who is missing from the studies

  • Which groups were under-represented in the studies has not been recorded for this substance. Pregnancy, breastfeeding, children, older adults, and people with liver or kidney conditions are commonly excluded from trials; nothing here says whether they were.

What the label states about particular groups

  • On pediatric, the label states: “Safety and effectiveness of azacitidine in pediatric patients with MDS have not been established.”

    US prescribing information · 2d15b3ec-ed08-48c6-a400-34e55974afad · read 2026-08-30

  • On older people, the label states: “Of the total number of patients in Studies 1, 2 and 3, 62% were 65 years and older and 21% were 75 years and older.”

    US prescribing information · 2d15b3ec-ed08-48c6-a400-34e55974afad · read 2026-08-30

  • On people who are pregnant, the label states: “Risk Summary Based on its mechanism of action and findings in animals, azacitidine can cause fetal harm when administered to a pregnant woman [ see Clinical Pharmacology (12.1)].”

    US prescribing information · 2d15b3ec-ed08-48c6-a400-34e55974afad · read 2026-08-30

  • On people who are breastfeeding, the label states: “Risk Summary There is no information regarding the presence of azacitidine in human milk, the effects of azacitidine on the breastfed infant, or the effects of azacitidine on milk production.”

    US prescribing information · 2d15b3ec-ed08-48c6-a400-34e55974afad · read 2026-08-30

Where the result stopped carrying

  • This is a scope explorer, not a diagnosis engine.
  • It shows who was studied so you can see whether people similar to you were included.

RNAWiki cannot tell whether a study fits you. It can show who was in it.

What it would be like to takeRead from sources, not yet reviewed

Why it might seem to do nothing

A real effect and a noticed effect are different things. These are the recorded reasons the two come apart.

It was studied in different people

The people in the studies were not much like the reader.

On this record: Which groups were under-represented in the studies has not been recorded for this substance. Pregnancy, breastfeeding, children, older adults, and people with liver or kidney conditions are commonly excluded from trials; nothing here says whether they were.

The evidence may simply be wrong

Small early studies often do not hold up.

On this record: RNAWiki has not yet published a reviewed conclusion for this use.

Other reasons RNAWiki checked and found nothing for (11)
It was studied for a different goal
The studies measured something else entirely. Nothing in this record points to this reason.
A different form was studied
The studied form is not the form on the shelf. Nothing in this record points to this reason.
There was nothing to correct
Where a level is already normal, topping it up may change nothing. Nothing in this record points to this reason.
Something else had to happen too
In the studies it was paired with training, a diet or another treatment. Nothing in this record points to this reason.
The change is too small to feel
A real change can still sit below what a person notices. Nothing in this record points to this reason.
Day-to-day swing hides it
Sleep, food, stress and time of day move most numbers more than this would. Nothing in this record points to this reason.
Not enough time yet
The studies ran longer than the person has waited. Nothing in this record points to this reason.
It was not taken as studied
Missed days change the result, and studies count them. Nothing in this record points to this reason.
Something else changed at the same time
Two changes at once cannot be told apart afterwards. Nothing in this record points to this reason.
The product may not be what it says
Contents of a sold product are not always what the label states. Nothing in this record points to this reason.
No recorded reason
RNAWiki has nothing stored that would explain it. Nothing in this record points to this reason.

None of these is a reason to take more. Taking more is not a step this page ever suggests.

What it would be like to takeRead from sources, not yet reviewed

What taking it involves

The recorded facts about getting hold of it and using it. Nothing here is a suggestion about what you should do.

How it is supplied

Given by a clinician

Quoted from a source

Where this came from

Copied from a source RNAWiki stored, with the source named.

Read from the recorded approval status.

No source is stored against this line.

Form and route

Intravenous, subcutaneous

Source-linked record

Where this came from

A person wrote this into the record with the study named beside it. No reviewer has signed it off.

The form and route recorded on this substance.

No source is stored against this line.

Who oversees it

Professional supervision is normally required. A regulator classifies this substance in a way that restricts how it is supplied.

Quoted from a source

Where this came from

Copied from a source RNAWiki stored, with the source named.

Suppression classes recorded: S1, S3, S4.

No source is stored against this line.

What is in the pack

Sold as powder, tablet, injection, powder, lyophilized, for solution, tablet, film coated, given by the intravenous, subcutaneous, oral, intravenous route.

Source-linked record

Where this came from

A person wrote this into the record with the study named beside it. No reviewer has signed it off.

Recorded notes on how this is sold and what that means for what is in the pack.

No source is stored against this line.

Where it is registered

Regulatory records are listed in the technical disclosure at the foot of this page.

Counted from records

Where this came from

A count of rows RNAWiki holds. It describes our records, not your body.

Register entries are stored per jurisdiction and shown with their dates.

No source is stored against this line.

Why people stop

Not recorded.

Nothing recorded

Where this came from

RNAWiki holds nothing here and says so rather than guessing.

No record of why people stopped taking it is stored for this substance.

No source is stored against this line.

What it would be like to takeNothing found in the sources checked

What can go wrong

Sorted by where each line came from. A regulator's label and a report somebody sent in are not the same kind of fact.

Nothing found in the sources checked

No harm is recorded against this substance in the sources RNAWiki checked. Finding nothing is not the same as showing there is nothing.

The sources listed were searched and held nothing. That is not the same as nothing existing.

Over a long time. No completed tested study window is recorded, so long-term effects are not established by the registry record.

Groups the studies covered thinly. Which groups were under-represented in the studies has not been recorded for this substance. Pregnancy, breastfeeding, children, older adults, and people with liver or kidney conditions are commonly excluded from trials; nothing here says whether they were.

What is missing or unclearRead from sources, not yet reviewed

Does the exact form matter?

Evidence belongs to the exact thing that was tested. A different salt, a different mixture or a different preparation is a different question.

The form that was studied

Intravenous, subcutaneous

Source-linked record

Where this came from

A person wrote this into the record with the study named beside it. No reviewer has signed it off.

The form used in the studies cited on this page.

No source is stored against this line.

What is sold

Not recorded.

Nothing recorded

Where this came from

RNAWiki holds nothing here and says so rather than guessing.

Nothing further is recorded about which forms are sold.

No source is stored against this line.

What is recorded as being sold

  • 47 products list this as an active ingredient in the United States drug directory. 47 of them contain it and nothing else.

    FDA National Drug Code directory · 42385-312 · read 2026-08-29

  • They are sold as injection, powder, for solution, injection, powder, lyophilized, for solution, powder and tablet, film coated, taken intravenous, oral and subcutaneous.

    FDA National Drug Code directory · 42385-312 · read 2026-08-29

  • The regulator's established pharmacologic class for it is nucleic acid synthesis inhibitors [moa] and nucleoside metabolic inhibitor [epc].

    FDA National Drug Code directory · 42385-312 · read 2026-08-29

  • 27 published labels name it as an active ingredient. 27 of them describe this substance alone, which is where its own label text on this page comes from.

    US prescribing information · 2d15b3ec-ed08-48c6-a400-34e55974afad · read 2026-08-29

  • Those labels are classed as human prescription drug.

    US prescribing information · 2d15b3ec-ed08-48c6-a400-34e55974afad · read 2026-08-29

  • Azacitidine is intravenous at 3 DOSAGE FORMS AND STRENGTHS Azacitidine for injection is supplied as lyophilized cake or powder in 100 mg single-dose vials., recorded as fda label in effect 2025-08-25 in the United States.

    US prescribing information · 2d15b3ec-ed08-48c6-a400-34e55974afad · read 2026-08-30

Evidence carries across two names for one substance. It does not carry across a salt, a mirror form or a mixture.

What it would be like to takeRead from sources, not yet reviewed

What you could measure

This one is decided with a clinician, so this section holds questions to ask rather than a plan to run.

Self-experiment planning is not offered for a prescription or clinician-supervised medicine. The questions below are for a clinician or pharmacist.

Questions worth asking

  • Which of the trials of Azacitidine studied people like me?
  • What was measured, and for how long?
  • Was the result a laboratory value or a health outcome?
  • What would we watch for, and when would we stop?

Tracking can show whether something changed for you. It cannot show what caused it.

RNAWiki records evidence. It does not say whether this substance is right for you.

What is missing or unclearRead from sources, not yet reviewed

What nobody knows yet

Open questions, each with why it is open and what would close it.

Missing populations

Which groups were under-represented in the studies has not been recorded.

Why it matters. Recorded by the evidence model as a gap on this record.

What would answer it

A study that measures it, and a reviewer to sign it off.

Last checked 2026-09-04. Searched: The sources listed in the receipts at the foot of this page.

Missing long-term data

No completed tested study window is recorded.

Why it matters. Recorded by the evidence model as a gap on this record.

What would answer it

A study that measures it, and a reviewer to sign it off.

Last checked 2026-09-04. Searched: The sources listed in the receipts at the foot of this page.

No reviewed conclusion

RNAWiki has not yet published a reviewed conclusion for this use.

Why it matters. Recorded by the evidence model as a gap on this record.

What would answer it

A study that measures it, and a reviewer to sign it off.

Last checked 2026-09-04. Searched: The sources listed in the receipts at the foot of this page.

Missing interaction studies

No interaction was found in the registers checked. Not finding one is not the same as showing there is none.

Why it matters. Recorded by the evidence model as a gap on this record.

What would answer it

A study that measures it, and a reviewer to sign it off.

Last checked 2026-09-04. Searched: The sources listed in the receipts at the foot of this page.

Formulation uncertainty

Several salts, forms or products of Azacitidine are recorded. Results from one form may not transfer to another.

Why it matters. Recorded by the evidence model as a gap on this record.

What would answer it

A study that measures it, and a reviewer to sign it off.

Last checked 2026-09-04. Searched: The sources listed in the receipts at the foot of this page.

Mechanism not reviewed

No reviewed mechanism story exists for this substance.

Why it matters. Recorded by the evidence model as a gap on this record.

What would answer it

A study that measures it, and a reviewer to sign it off.

Last checked 2026-09-04. Searched: The sources listed in the receipts at the foot of this page.

What happened in peopleRead from sources, not yet reviewed

Check any of this yourself

Every line above traced back to the study it came from. This is the technical layer, and the vocabulary changes here.

How many documents were read

  • 26 documents were read for this substance.

    RNAWiki source record

Where else this substance is registered

FDA substance identifier (UNII)
M801H13NRU
RxNorm concept
485246

Checks this page had to pass

  • Passed

    Identity resolved

    no open identity hold

  • Passed

    No unresolved merge across substance families

    no quarantine open

  • Passed

    Every public sentence names a source

    The opening statement carries the origin: Quoted from a stored source.

  • Not passed

    Trial roles classified for highlighted evidence

    No registered study is classified as testing this substance.

  • Passed

    No internal keys in reader text

    enforced by the copy-contract test over the rendered page

  • Passed

    Safety mode resolved

    Suppression classes recorded: S1, S3, S4.

  • Passed

    Canonical metadata present

    slug and display name present

What is missing or unclearRead from sources, not yet reviewed

How this medicine reached us

Kept near the foot of the page. A historical event moves up only when it changes something about this substance today.

What the approval register records

  • 19 approved applications cover products containing this substance. The earliest was NDA050794, approved 20040519 to BRISTOL-MYERS.

    Drugs@FDA application register · NDA050794 · read 2026-08-29

  • Marketing status on the register: discontinued, none (tentative approval) and prescription.

    Drugs@FDA application register · NDA050794 · read 2026-08-29

  • The earliest marketing start date recorded for a listed product is 20040429.

    FDA National Drug Code directory · 42385-312 · read 2026-08-29

What is missing or unclearRead from sources, not yet reviewed

What to learn next

Ordered by what would most change how you read this page, starting with what is easiest to misunderstand.

This order is fixed in code and does not count clicks or time on the page.

What is not here

9 questions this page could not answer

These sections were prepared and left out because there was nothing to put in them. They are listed rather than hidden, so the gaps in this record are visible.

  • What was measured, goal by goal — found nothing in the sources checked.
  • How close this is to real life — found nothing in the sources checked.
  • The path through the body — found nothing in the sources checked.
  • Felt, measured, or meaningful — found nothing in the sources checked.
  • How long anything takes — found nothing in the sources checked.
  • What it may clash with — found nothing in the sources checked.
  • Other ways to the same goal — found nothing in the sources checked.
  • Claims that go past the evidence — found nothing in the sources checked.
  • What changed on this page — found nothing in the sources checked.

The record as stored

The full record, for auditing

Everything above is built from what is below. This layer keeps the technical vocabulary, record identifiers and every stored row, so a reader who wants to check the page can.

Recorded evidence blocks (11)

On the Azacitidine label: indicated for what?

"ONUREG is indicated for continued treatment of adult patients with acute myeloid leukemia who achieved first complete remission (CR) or complete remission with incomplete blood count recovery (CRi) following intensive induction chemotherapy and are not able to complete intensive curative therapy. ONUREG is a…": indications and usage on Azacitidine's label. DailyMed label · 0e95e33f-8aba-4f19-b332-2416580d358b · 2026-06-12

615 registered trials of Azacitidine — at which phases?

Registered studies posting no result
432 of 615

615 registered studies of Azacitidine: 355 phase2, 267 phase1, 70 phase3, 21 na, 11 na or unstated, 7 phase4, 3 early phase1. CLINICALTRIALS_SNAPSHOT · 2026-09-01

361 with a PubMed record

Show the evidence
  • phase2
    355
  • phase1
    267
  • phase3
    70
  • na
    21
  • na or unstated
    11
  • phase4
    7
12 more recorded rows
  • early phase1
    3
  • completed
    214
  • recruiting
    126
  • terminated
    115
  • active not recruiting
    55
  • unknown
    43
  • not yet recruiting
    28
  • withdrawn
    24
  • suspended
    6
  • enrolling by invitation
    2
  • approved for marketing
    1
  • no longer available
    1

recorded 2026-09-01 · last checked 2026-09-04

126 of Azacitidine's trials stopped: safety, futility/efficacy, accrual/recruitment, funding/business, sponsor decision unspecified, other?

safety (11), futility/efficacy (13), accrual/recruitment (30), funding/business (26), sponsor decision unspecified (8) and other (38): Azacitidine's stop wording, clustered. CLINICALTRIALS_SNAPSHOT · 2026-09-01

"Due to limited resources available to conduct study"; 126 of 615 registered studies

Show the evidence

Trial

  • NCT00118196
    terminated; "Due to limited resources available to conduct study"
  • NCT00234000
    terminated; "The study was closed due to poor enrollment"
  • NCT00379912
    terminated; "Insufficient response rate"
  • NCT00381693
    terminated; "Due to lack of accrual and trial has demonstrated too little clinical benefit"
  • NCT00412919
    terminated; "Lack of efficacy"
  • NCT00413478
    terminated; "Slow accrual."
14 further recorded trials
  • NCT00443261
    terminated; "Accrual problems"
  • NCT00503984
    terminated; "Withdrawal of Funding"
  • NCT00543582
    terminated; "Celgene terminated its collaboration agreement with MethylGene for the development of MGCD0103. All Celgene-sponsored trials with MGCD0103 will be closed."
  • NCT00666497
    terminated; "Celgene terminated its collaboration agreement with MethylGene for the development of MGCD0103. All Celgene-sponsored trials with MGCD0103 with be closed."
  • NCT00842582
    withdrawn; "No enrollment. Unable to recruit due to lack of eligible patients."
  • NCT00901537
    terminated; "Sponsor terminated due to no enrollments within last 4 months."
  • NCT00923234
    terminated; "The primary objective has already been answered with the number of recruited patients."
  • NCT00997243
    terminated; "Drug no longer being supplied by sponsor"
  • NCT01011283
    terminated; "The study was stopped due to insufficient enrollment."
  • NCT01067274
    withdrawn; "Study abandoned"
  • NCT01121757
    terminated; "The study closed temporarily in February 2012 pending analysis of samples collected. In October, 2012, Celgene requested closure of the study."
  • NCT01167816
    terminated; "The study was terminated as the Prinicipal Investigator left the site- all previous subjects enrolled are deceased"
  • NCT01200004
    terminated; "Slow Accrual."
  • NCT01207726
    terminated; "Slow Accrual due to patients not wanting daily clinic administration of 5AZA and/or 6 months of treatment after surgery."

recorded 2026-09-01 · last checked 2026-09-04

Human studies of Azacitidine used Azacitidine 30mg/m2 — over how long?

studies of Azacitidine used the recorded amount. ClinicalTrials.gov · 2026-09-01

3 recorded entries; human; also "Azacitidine 30mg/m2", "Azacitidine 40mg/m2", "Single use vials containing 100 mg azacitidine/vials,.The formulation contained mannitol as an excipient. Lot 090035…"

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human

  • NCT00387465
    Azacitidine 30mg/m2
  • NCT00387465
    Azacitidine 40mg/m2
  • NCT01290302
    Single use vials containing 100 mg azacitidine/vials,.The formulation contained mannitol as an excipient. Lot 090035 and Lot 120011

recorded 2026-09-01 · last checked 2026-09-04

Azacitidine's half-life is 0.5 hours — which schedules were studied?

0.5 hours, the half-life Azacitidine's label states: "Elimination The mean (CV%) terminal half-life is approximately 0.5 hours (27%) and the apparent clearance (CL/F) is 1240 L/hour (64%)." DailyMed label · 0e95e33f-8aba-4f19-b332-2416580d358b · 2026-06-12

tmax 1 hour; bioavailability 11 %.

Show the evidence
  • half life pharmacokinetics
    0.5 hours; Elimination The mean (CV%) terminal half-life is approximately 0.5 hours (27%) and the apparent clearance (CL/F) is 1240 L/hour (64%).
  • tmax pharmacokinetics
    1 hour; The median time to peak plasma concentration of azacitidine is 1 hour.
  • bioavailability pharmacokinetics
    11 %; Absorption The mean oral bioavailability is approximately 11% relative to subcutaneous administration.
  • metabolism pharmacokinetics
    Metabolism Azacitidine undergoes spontaneous hydrolysis and deamination mediated by cytidine deaminase.

recorded 2026-06-12 · last checked 2026-09-04

Which running trial of Azacitidine could settle lifespan?

NCT07458542 measures Relapse-free survival (RFS), reading out 2026-03-31.

35 open trials; n 44; "Effectiveness and Safety of ONUREG (Oral Azacitidine) in Chinese Patients With Acute Myeloid Leukemia"

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Trial

  • NCT07458542
    "Effectiveness and Safety of ONUREG (Oral Azacitidine) in Chinese Patients With Acute Myeloid Leukemia"; n 44; "Relapse-free survival (RFS)"; 2026-03-31
  • NCT05197426
    "A Study to Compare the Efficacy and Safety of Oral Azacitidine Plus Best Supportive Care Versus Best Supportive Care as Maintenance Therapy in Japanese Participants With Acute Myeloid Leukemia (AML) in Complete Remission"; n 19; "Recurrence Free Survival (RFS)"; 2026-04-30
  • NCT02993523
    "A Study of Venetoclax in Combination With Azacitidine Versus Azacitidine in Treatment Naïve Participants With Acute Myeloid Leukemia Who Are Ineligible for Standard Induction Therapy"; n 443; "Overall Survival (OS)"; 2026-06-15
  • NCT03173248
    "Study of AG-120 (Ivosidenib) vs. Placebo in Combination With Azacitidine in Participants With Previously Untreated Acute Myeloid Leukemia With an IDH1 Mutation"; n 146; "Event-Free Survival (EFS)"; 2026-06-30
  • NCT04090736
    "Study to Compare Azacitidine Plus Pevonedistat Versus Azacitidine in Patients With Acute Myeloid Leukemia Not Eligible for Standard Chemotherapy"; n 302; "Overall survival (OS)"; 2026-07-30
  • NCT05413018
    "An Efficacy and Safety Study of Oral Azacitidine (CC-486) as Maintenance Therapy in Chinese Participants With Acute Myeloid Leukemia in Complete Remission"; n 34; "Relapse-free survival (RFS)"; 2026-10-31
14 further recorded trials
  • NCT04128501
    "Venetoclax and Azacitidine for the Treatment of Acute Myeloid Leukemia in the Post-Transplant Setting"; n 100; "Relapse-free survival (RFS) time"; 2026-12-31
  • NCT06754540
    "Azacitidine Combined with Donor Lymphocyte Infusion for Acute Myeloid Leukemia Post-transplant Relapse Prevention."; n 51; "Leukemia-free survival (LFS) time"; 2027-01-01
  • NCT06384261
    "A Study Comparing Venetoclax and Azacitidine Plus Cusatuzumab to Venetoclax and Azacitidine in Newly Diagnosed AML Ineligible for Intensive Therapy"; n 140; "Overall survival"; 2027-06
  • NCT04401748
    "Study Of Venetoclax Tablet With Intravenous or Subcutaneous Azacitidine to Assess Change in Disease Activity In Adult Participants With Newly Diagnosed Higher-Risk Myelodysplastic Syndrome"; n 531; "Overall survival (OS)"; 2027-07
  • NCT05365035
    "A Phase II Study of Cladribine and Low Dose Cytarabine in Combination With Venetoclax, Alternating With Azacitidine and Venetoclax, in Patients With Higher-risk Myeloproliferative Chronic Myelomonocytic Leukemia or Higher-risk Myelodysplastic Syndromes With Excess Blasts"; n 60; "To establish the overall survival (OS)."; 2027-12-31
  • NCT07392242
    "A Study of the Combination of Ivosidenib, Azacitidine, and Venetoclax Followed by Ivosidenib Alone in People With Acute Myeloid Leukemia"; n 45; "Event free survival"; 2028-01
  • NCT04801797
    "Venetoclax + Azacitidine vs. Induction Chemotherapy in AML"; n 172; "Event free survival"; 2028-01-01
  • NCT06744556
    "HAV Versus DAV/IAV Induction Regimen in Elderly Patients With AML"; n 41; "Event-free survival (EFS)"; 2028-06-01
  • NCT07132684
    "Comparison of VA and D/IA Induction Regimens in Elderly Fit Acute Myeloid Leukemia Patients"; n 240; "Overall Survival (OS)"; 2028-08-30
  • NCT06635681
    "Efficacy of Venetoclax Combined With Intensive Chemotherapy in Different Subgroups of AML"; n 380; "Event-free survival (EFS)"; 2028-10-01
  • NCT07131059
    "MRD-positive AML Clinical Study"; n 537; "relapse-free survival rate"; 2028-10-30
  • NCT06557421
    "De-Escalation Study Evaluating Venetoclax and Azacitidine Discontinuation in AML Responding Patients"; n 50; "Disease-Free Survival, measured from inclusion (VEN-AZA de-escalation) to the date of morphologic or measurable residual disease relapse or death from any cause, whichever occurs first."; 2028-11-01
  • NCT06389292
    "A Pivotal Study of APG-2575 (Lisaftoclax) Combined With Azacitidine in the Treatment of Acute Myeloid Leukemia"; n 486; "Overall Survival（OS）"; 2029-03-26
  • NCT07047183
    "Clinical Study of Venetoclax Combined With Azacitidine as Bridging Therapy Prior to Hematopoietic Stem Cell Transplantation in Patients With Higher-Risk Myelodysplastic Syndromes"; n 46; "2-Year Relapse-Free Survival(2-RFS)"; 2029-06-30

Which 93 trials of Azacitidine posted no result?

Posted no result
93 of 93 completed trials
Registrations
NCT00005598, NCT00006019, NCT00005639, NCT00004062, NCT00545519 and NCT00496444, and 87 more
Completion dates
oldest 2002-03; newest 2024-07-02
Show the evidence

Trial

  • NCT00005598
    2002-03
  • NCT00006019
    2003-08
  • NCT00005639
    2005-07
  • NCT00004062
    2006-08
  • NCT00545519
    2008-03
  • NCT00496444
    2009-10
14 further recorded trials
  • NCT00398450
    2010-04
  • NCT00350818
    2010-08
  • NCT00481273
    2010-08-31
  • NCT00795548
    2011-08
  • NCT00996515
    2011-09
  • NCT00741234
    2012-03
  • NCT01048034
    2012-08
  • NCT00529022
    2012-10
  • NCT00739388
    2012-11
  • NCT00915252
    2012-12
  • NCT01129180
    2012-12
  • NCT01571648
    2013-01-01
  • NCT01301820
    2013-02
  • NCT00351975
    2013-03

At the median, Azacitidine's trials enrolled 43 people — anything larger?

Median enrolment
43
Largest enrolment
3000
Registered trials counted
608

What do 3974 spontaneous reports say about Azacitidine — and not say?

These are reports people sent to a regulator. They do not show the medicine caused the reaction. Nobody counted how many people took the medicine and reported nothing. The same event can be reported more than once, and many reports are incomplete. News coverage, lawsuits and new warnings change how often people report. A count is not a rate and not a risk.

Azacitidine appears in spontaneous reports to regulators. Across the 10 most-reported reaction terms, 3974 reaction mentions were counted: febrile neutropenia 1034; pyrexia 451; pneumonia 416; anaemia 392. FAERS via Open Targets · CHEMBL1489 · 2026-06-24

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  • febrile neutropenia
    1034
  • pyrexia
    451
  • pneumonia
    416
  • anaemia
    392
  • neutropenia
    386
  • thrombocytopenia
    365
4 more recorded rows
  • platelet count decreased
    259
  • sepsis
    250
  • pancytopenia
    226
  • acute myeloid leukaemia
    195

recorded 2026-06-24 · last checked 2026-09-04

Which 10 reactions does Azacitidine's label not list?

acute myeloid leukaemia, anaemia and febrile neutropenia and 7 more reported for Azacitidine, absent from its label. FAERS via Open Targets · CHEMBL1489 · 2026-06-24

2 label terms; 10 reported and unlisted; 0e95e33f-8aba-4f19-b332-2416580d358b

Show the evidence
  • acute myeloid leukaemia
    count not stated
  • anaemia
    count not stated
  • febrile neutropenia
    count not stated
  • neutropenia
    count not stated
  • pancytopenia
    count not stated
  • platelet count decreased
    count not stated
4 more recorded rows
  • pneumonia
    count not stated
  • pyrexia
    count not stated
  • sepsis
    count not stated
  • thrombocytopenia
    count not stated

recorded 2026-06-24 · last checked 2026-09-04

Azacitidine and CYP1A2, P-GP and TRANSPORTER: shared by which compounds?

CYP1A2, P-GP and TRANSPORTER appear in Azacitidine's recorded interaction sentences, 8 in all. DailyMed label · 0e95e33f-8aba-4f19-b332-2416580d358b · 2026-06-12

CYP1A2, CYP1A2, CYP2B6, CYP2C19, CYP2C19, CYP2C8; 19 shared nodes; pharmacokinetics, clinical_pharmacology

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Interaction statement

  • pharmacokinetics
    In Vitro Studies Cytochrome P450 (CYP) Enzymes: Azacitidine does not inhibit CYP1A2, CYP2B6, CYP2C8, CYP2C9, CYP2C19, CYP2D6, CYP3A4, or CYP2E1 at clinically relevant concentrations.
  • pharmacokinetics
    Azacitidine is not an inducer of CYP1A2, CYP2C19, or CYP3A.
  • pharmacokinetics
    Transporter Systems: Azacitidine is not a substrate of P-glycoprotein (P-gp).
  • pharmacokinetics
    Azacitidine does not inhibit P-gp, breast cancer resistance protein (BCRP), organic anion transporters (OAT) OAT1 and OAT3, organic anion transporting polypeptides (OATP) OATP1B1 and OATP1B3, or organic cation transporter (OCT) OCT2 at clinically relevant concentrations.
  • clinical_pharmacology
    In Vitro Studies Cytochrome P450 (CYP) Enzymes: Azacitidine does not inhibit CYP1A2, CYP2B6, CYP2C8, CYP2C9, CYP2C19, CYP2D6, CYP3A4, or CYP2E1 at clinically relevant concentrations.
  • clinical_pharmacology
    Azacitidine is not an inducer of CYP1A2, CYP2C19, or CYP3A.
2 more recorded rows
  • Interaction statement clinical_pharmacology
    Transporter Systems: Azacitidine is not a substrate of P-glycoprotein (P-gp).
  • Interaction statement clinical_pharmacology
    Azacitidine does not inhibit P-gp, breast cancer resistance protein (BCRP), organic anion transporters (OAT) OAT1 and OAT3, organic anion transporting polypeptides (OATP) OATP1B1 and OATP1B3, or organic cation transporter (OCT) OCT2 at clinically relevant concentrations.

CYP1A2

  • FINGOLIMOD LAURYL SULFATE, FOSAPREPITANT DIMEGLUMINE, TAFAMIDIS MEGLUMINE, Arimoclomol, Rasagiline, Sofpironium, Golodirsen, Tinidazole
  • FINGOLIMOD LAURYL SULFATE, FOSAPREPITANT DIMEGLUMINE, TAFAMIDIS MEGLUMINE, Arimoclomol, Rasagiline, Sofpironium, Golodirsen, Tinidazole
  • CYP2B6
    FINGOLIMOD LAURYL SULFATE, TAFAMIDIS MEGLUMINE, Arimoclomol, Sofpironium, Bupropion, Golodirsen, Tinidazole, Naldemedine

CYP2C19

  • FINGOLIMOD LAURYL SULFATE, FOSAPREPITANT DIMEGLUMINE, TAFAMIDIS MEGLUMINE, Arimoclomol, Rasagiline, Golodirsen, Naldemedine, Etravirine
  • FINGOLIMOD LAURYL SULFATE, FOSAPREPITANT DIMEGLUMINE, TAFAMIDIS MEGLUMINE, Arimoclomol, Rasagiline, Golodirsen, Naldemedine, Etravirine
  • CYP2C8
    FINGOLIMOD LAURYL SULFATE, TAFAMIDIS MEGLUMINE, Arimoclomol, Golodirsen, Naldemedine, Methylnaltrexone, Lapatinib, Tivozanib
  • CYP2C9
    FINGOLIMOD LAURYL SULFATE, FOSAPREPITANT DIMEGLUMINE, TAFAMIDIS MEGLUMINE, Arimoclomol, Rasagiline, Golodirsen, Tinidazole, Naldemedine
  • CYP2D6
    FINGOLIMOD LAURYL SULFATE, Darunavir Propylene Glycolate, FOSAPREPITANT DIMEGLUMINE, TAFAMIDIS MEGLUMINE, Arimoclomol, Rasagiline, Sofpironium, Fluoxetine
  • CYP2E1
    FINGOLIMOD LAURYL SULFATE, FOSAPREPITANT DIMEGLUMINE, Rasagiline, Tinidazole, Naldemedine, Methylnaltrexone, Alosetron, Metaxalone
  • CYP3A
    FINGOLIMOD LAURYL SULFATE, Darunavir Propylene Glycolate, Vincristine, Naldemedine, Pemetrexed, Eravacycline, Rasburicase, Repotrectinib
  • CYP3A4
    FINGOLIMOD LAURYL SULFATE, ERYTHROMYCIN LACTOBIONATE, FOSAPREPITANT DIMEGLUMINE, TAFAMIDIS MEGLUMINE, Arimoclomol, Beclometasone, Rasagiline, Sofpironium
6 more recorded rows
  • BCRP
    TAFAMIDIS MEGLUMINE, Arimoclomol, Ceftobiprole Medocaril, Deoxycholic acid, Golodirsen, Naldemedine, Eravacycline, Omadacycline
  • OAT1
    TAFAMIDIS MEGLUMINE, Arimoclomol, Ceftobiprole Medocaril, Deoxycholic acid, Golodirsen, Naldemedine, Eravacycline, Prucalopride
  • OAT3
    TAFAMIDIS MEGLUMINE, Arimoclomol, Ceftobiprole Medocaril, Deoxycholic acid, Golodirsen, Naldemedine, Pemetrexed, Eravacycline
  • OATP1B1
    FINGOLIMOD LAURYL SULFATE, TAFAMIDIS MEGLUMINE, Arimoclomol, Ceftobiprole Medocaril, Deoxycholic acid, Golodirsen, Naldemedine, Eravacycline
  • OATP1B3
    FINGOLIMOD LAURYL SULFATE, TAFAMIDIS MEGLUMINE, Arimoclomol, Ceftobiprole Medocaril, Deoxycholic acid, Golodirsen, Naldemedine, Eravacycline
  • OCT2
    TAFAMIDIS MEGLUMINE, Arimoclomol, Ceftobiprole Medocaril, Deoxycholic acid, Sofpironium, Golodirsen, Naldemedine, Eravacycline

P-gp

  • FINGOLIMOD LAURYL SULFATE, ERYTHROMYCIN LACTOBIONATE, Darunavir Propylene Glycolate, FOSAPREPITANT DIMEGLUMINE, TAFAMIDIS MEGLUMINE, Arimoclomol, Ceftobiprole Medocaril, Vincristine
  • FINGOLIMOD LAURYL SULFATE, ERYTHROMYCIN LACTOBIONATE, Darunavir Propylene Glycolate, FOSAPREPITANT DIMEGLUMINE, TAFAMIDIS MEGLUMINE, Arimoclomol, Ceftobiprole Medocaril, Vincristine

recorded 2026-06-12 · last checked 2026-09-04

Where it is registered
Identifiers, relations and other names

The exact record

ChEMBL id
CHEMBL1489
PubChem CID
9444
CAS number
320-67-2
RxCUI
1251
InChIKey
NMUSYJAQQFHJEW-KVTDHHQDSA-N
Also called
5-azacitidine, Azacitidina, Ladakamycin, 5-aza, 5-azacytidine, 5ac, 5azacitidine, aza, azactidine, azacytidine, azc, cc-486
Trade name
Azacitidine accord, Azacitidine betapharm, Azacitidine celgene, Azacitidine kabi, Azacitidine mylan, Onureg, Vidaza, AZACITIDINE AZACITIDINE, Onureg / Vidaza, Azacitidine Adienne
Development code
NSC-102816, U-18,496, U-18496
Salt form
oral azacitidine, oral-aza, AZACITIDINE FOR
Sources (6)

Sources

ChEMBL 37 — CC BY-SA 3.0 Unported · ChEMBL ATC CC BY-SA · ClinicalTrials.gov — US Government work · Open Targets 26.06 — CC0 · mixed register set; per-register licences in docs/specs/corpus-20k-sources.md · openFDA / DailyMed — US Government work

How these records are assembled · Which registers were checked

Index-quality checks
  • identity passed: no open identity hold
  • required summary fields resolved: 4 required field(s) not terminal: Why people use it, Best-supported result, Biggest unanswered question, Human evidence
  • public claims reviewed: 0 reviewed claim(s); drafts are never rendered
  • source coverage passed: 6 source rows
  • no critical contamination: no quarantine open
  • canonical metadata passed: slug and display name present
  • no raw internal fields: enforced by the copy-contract test over the rendered page

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