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Hydroxycarbamide

  • Prescription medicine
  • Prescription only
  • 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 Hydroxycarbamide does in the body

The frequency of painful crises and to reduce the need for blood transfusions

From the FDA-approved label: The precise mechanism by which hydroxyurea produces its antineoplastic effects cannot, at present, be described. However, the reports of various studies in tissue culture in rats and humans lend support to the hypothesis that hydroxyurea causes an immediate inhibition of DNA synthesis by acting as a ribonucleotide reductase inhibitor, without interfering with the synthesis of ribonucleic acid or of protein. This hypothesis explains why, under certain conditions, hydroxyurea may induce teratogenic effects.

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 · X6Q56QN5QC · read 2026-08-29

  • Its recorded molecular formula is CH4N2O2, weighing 76.05.

    US prescribing information · a9494409-3571-4a81-9ad3-15ef7cb636a2 · read 2026-08-30

Where each sentence above came from

No recorded sentence describes the change this makes in a way that stands on its own. The page opens with what it is taken for instead, and the recorded explanation follows below.

Shown as the opening line on this page.

No statement of the main limit is recorded.

The four opening statements run to 102 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.

Access to sickle cell disease screening and care

The study did not show it

Who was studied
NCT06563440
How many people
24000
Study design
Not applicable
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. Oral

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

Improved number and timeliness of refferal of infants with sickle cell disease for care

The study did not show it

Who was studied
NCT07610239
How many people
24000
Study design
Not applicable
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. Oral

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

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. Oral

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

Cumulative incidence of death from vascular origin and other thrombotic and hemorrhagic events (combined endpoint)

The study did not show it

Who was studied
NCT02611973
How many people
2250
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. Oral

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

Survival

The study did not show it

Who was studied
NCT00005823
How many people
2000
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. Oral

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
NCT00055874
How many people
1551
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. Oral

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.

  • SIKLOS ® is indicated to reduce the frequency of painful crises and to reduce the need for blood transfusions in adult and pediatric patients, 2 years of age and older, with sickle cell anemia with recurrent moderate to severe painful crises SIKLOS is an antimetabolite, indicated to reduce the frequency of painful crises and to reduce the need for blood transfusions in adult and pediatric patients, 2 years of age…

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 in pediatric patients have not been established.”

    US prescribing information · a9494409-3571-4a81-9ad3-15ef7cb636a2 · read 2026-08-30

  • On older people, the label states: “Clinical studies of DROXIA did not include sufficient numbers of subjects aged 65 and over to determine whether they respond differently from younger subjects.”

    US prescribing information · a9494409-3571-4a81-9ad3-15ef7cb636a2 · read 2026-08-30

  • On people who are pregnant, the label states: “Risk Summary DROXIA can cause fetal harm based on findings from animal studies and the drug's mechanism of action [see Clinical Pharmacology (12.1) ] .”

    US prescribing information · a9494409-3571-4a81-9ad3-15ef7cb636a2 · read 2026-08-30

  • On people who are breastfeeding, the label states: “Risk Summary Hydroxyurea is excreted in human milk.”

    US prescribing information · a9494409-3571-4a81-9ad3-15ef7cb636a2 · read 2026-08-30

  • On people with reduced liver function, the label states: “There are no data that support specific guidance for dosage adjustment in patients with hepatic impairment.”

    US prescribing information · a9494409-3571-4a81-9ad3-15ef7cb636a2 · read 2026-08-30

  • On people with reduced kidney function, the label states: “The exposure to hydroxyurea is higher in patients with creatinine clearance of less than 60 mL/min.”

    US prescribing information · a9494409-3571-4a81-9ad3-15ef7cb636a2 · 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

Prescription only

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

Oral

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, S6.

No source is stored against this line.

What is in the pack

Sold as tablet, capsule, solution, tablet, film coated, given by the oral 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

Oral

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

  • 31 products list this as an active ingredient in the United States drug directory. 31 of them contain it and nothing else.

    FDA National Drug Code directory · 80725-830 · read 2026-08-29

  • They are sold as capsule, powder, solution and tablet, film coated, taken oral.

    FDA National Drug Code directory · 80725-830 · read 2026-08-29

  • The regulator's established pharmacologic class for it is antimetabolite [epc] and urea [cs].

    FDA National Drug Code directory · 80725-830 · read 2026-08-29

  • 16 published labels name it as an active ingredient. 16 of them describe this substance alone, which is where its own label text on this page comes from.

    US prescribing information · 9e1bdd4b-043a-4cf6-b1ff-8de49e02b7a6 · read 2026-08-29

  • Those labels are classed as human prescription drug.

    US prescribing information · 9e1bdd4b-043a-4cf6-b1ff-8de49e02b7a6 · read 2026-08-29

  • DROXIA is oral at 3 DOSAGE FORMS AND STRENGTHS Capsules: ​​200 mg opaque blue-green capsules, imprinted with black ink "DROXIA" and "200". 300 mg opaque purple capsules, imprinted with black ink "DROXIA" and "300". 400 mg opaque reddish-…, recorded as fda label in effect 2026-01-26 in the United States.

    US prescribing information · a9494409-3571-4a81-9ad3-15ef7cb636a2 · read 2026-08-30

  • Recorded price in US: 0.18676 USD per one unit as the pricing file counts it — a tablet, capsule, patch or single item, across 7 priced products whose strengths and pack forms differ, as of 2026-08-26, paid by retail pharmacies buying the product, as surveyed by the Centers for Medicare & Medicaid Services. It is not a list price, an insurance price, or what anyone pays at a counter..

    Recorded source · 2026-08-26 · read 2026-08-28

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 Hydroxycarbamide 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 Hydroxycarbamide 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.

Where else this substance is registered

FDA substance identifier (UNII)
X6Q56QN5QC
RxNorm concept
197797

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: Written into the record, not signed off.

  • 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, S6.

  • 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

  • 10 approved applications cover products containing this substance. The earliest was NDA016295, approved 19671207 to WAYLIS THERAP.

    Drugs@FDA application register · NDA016295 · read 2026-08-29

  • Marketing status on the register: discontinued and prescription.

    Drugs@FDA application register · NDA016295 · read 2026-08-29

  • The earliest marketing start date recorded for a listed product is 19981019.

    FDA National Drug Code directory · 80725-830 · 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 (9)

On the Hydroxycarbamide label: indicated for what?


"Hydroxyurea capsules are indicated for the treatment of: • Resistant chronic myeloid leukemia. • Locally advanced squamous cell carcinomas of the head and neck (excluding the lip) in combination with chemoradiation.": indications and usage on Hydroxycarbamide's label. DailyMed label · e76fd60e-7644-48c5-9857-3608a045000b · 2026-05-27

186 registered trials of Hydroxycarbamide — at which phases?


Registered studies posting no result
138 of 186

186 registered studies of Hydroxycarbamide: 91 phase2, 41 phase3, 37 phase1, 14 na, 12 na or unstated, 9 phase4, 3 early phase1. CLINICALTRIALS_SNAPSHOT · 2026-09-01

762 with a PubMed record

Show the evidence
  • phase2
    91
  • phase3
    41
  • phase1
    37
  • na
    14
  • na or unstated
    12
  • phase4
    9
8 more recorded rows
  • early phase1
    3
  • completed
    106
  • recruiting
    22
  • terminated
    20
  • unknown
    17
  • active not recruiting
    12
  • withdrawn
    6
  • not yet recruiting
    3

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

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


safety (2), futility/efficacy (3), accrual/recruitment (10), funding/business (1), sponsor decision unspecified (2) and other (3): Hydroxycarbamide's stop wording, clustered. CLINICALTRIALS_SNAPSHOT · 2026-09-01

"Institutional Review Board requested termination - all patients deceased and no new accrual."; 21 of 186 registered studies

Show the evidence

Trial

  • NCT00004897
    terminated; "Institutional Review Board requested termination - all patients deceased and no new accrual."
  • NCT00004933
    terminated; "Poor accrual"
  • NCT00122980
    terminated; "The study has been stopped due to safety and futility concerns."
  • NCT00290771
    terminated; "Lack of efficacy"
  • NCT00350844
    terminated; "Low subject accrual"
  • NCT00532883
    terminated; "Enrollment has been terminated due to a slow rate of enrollment."
14 further recorded trials
  • NCT00978146
    withdrawn; "Study was administratively withdrawn by the IRB; no subjects were enrolled"
  • NCT01103583
    terminated; "No efficacy (interim.analysis)"
  • NCT01425307
    terminated; "The study was stopped early due to successfully meeting the primary endpoint"
  • NCT01521299
    withdrawn; "Insufficient population of eligible patients."
  • NCT01531387
    terminated; "inability to reach a satisfactory endpoint with respect to adequate recruitment"
  • NCT01645124
    terminated; "Low accrual rate not allowing planned sample size leads to a futility condition"
  • NCT01964755
    terminated; "Investigator Decision"
  • NCT02090296
    withdrawn; "Participant withdrew consent"
  • NCT02336373
    terminated; "low enrollment"
  • NCT02640573
    terminated; "Insufficient accrual"
  • NCT03825341
    terminated; "The study terminated early due to challenges with recruitment during the COVID pandemic."
  • NCT03860844
    terminated; "Study was prematurely stopped due to sponsor decision (stage 2 efficacy criteria not met); not due to safety concerns."
  • NCT05005182
    terminated; "Slow accrual"
  • NCT05853458
    terminated; "Sponsor decision"

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

Human studies of Hydroxycarbamide used Oral Hydroxyurea (100 mg/mL) Solution — over how long?


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

5 recorded entries; human; oral; also "Oral Hydroxyurea (100 mg/mL) Solution", "Hydroxycarbamide 500 Mg Oral Capsule+L-Carnitine", "Hydroxycarbamide 500 Mg Oral Capsule"

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human

  • NCT03763656
    Oral Hydroxyurea (100 mg/mL) Solution
  • NCT05081349
    Hydroxycarbamide 500 Mg Oral Capsule+L-Carnitine
  • NCT05081349
    Hydroxycarbamide 500 Mg Oral Capsule
  • NCT06606886
    Cap. Hydrea 500mg
  • NCT06923111
    oral; hydroxycarbamide oral solution 100mg/ml

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

Which running trial of Hydroxycarbamide could settle lifespan?


NCT01962415 measures Post-transplant treatment-related mortality (TRM), reading out 2027-11.

4 open trials; n 100; "Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT"

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Trial

  • NCT01962415
    "Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT"; n 100; "Post-transplant treatment-related mortality (TRM)"; 2027-11
  • NCT06326190
    "177Lu-DOTATATE for Recurrent Meningioma"; n 136; "Progression Free Survival (PFS)"; 2028-12-22
  • NCT04116502
    "MITHRIDATE: Ruxolitinib Versus Hydroxycarbamide or Interferon as First Line Therapy in High Risk Polycythemia Vera"; n 586; "Event Free Survival (EFS)"; 2030-04-01
  • NCT07616154
    "Haploidentical Donor Hematopoietic Cell Transplant for Sickle Cell Disease"; n 45; "GVHD-free and rejection free survival (GRFS)"; 2035-09

Which 61 trials of Hydroxycarbamide posted no result?


Posted no result
61 of 61 completed trials
Registrations
NCT00000586, NCT00000602, NCT00000939, NCT00001074, NCT00002176 and NCT00002177, and 55 more
Completion dates
oldest 1994-06; newest 2024-01-22
Show the evidence

Trial

  • NCT00000586
    1994-06
  • NCT00000602
    1997-03
  • NCT00000939
    2000-01
  • NCT00001074
    2000-01
  • NCT00002176
    2000-03
  • NCT00002177
    2000-03
14 further recorded trials
  • NCT00002674
    2000-03
  • NCT00019474
    2000-08
  • NCT00005018
    2000-10
  • NCT00004492
    2001-09
  • NCT00001818
    2001-11
  • NCT00001958
    2002-02
  • NCT00002940
    2002-03
  • NCT00004089
    2002-04
  • NCT00004404
    2002-09
  • NCT00000919
    2002-11
  • NCT00004901
    2004-07
  • NCT00000916
    2004-08
  • NCT00003172
    2004-09
  • NCT01693718
    2004-09

At the median, Hydroxycarbamide's trials enrolled 70 people — anything larger?


Median enrolment
70
Largest enrolment
2093
Registered trials counted
171

What do 892 spontaneous reports say about Hydroxycarbamide — 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.

Hydroxycarbamide appears in spontaneous reports to regulators. Across the 10 most-reported reaction terms, 892 reaction mentions were counted: pyrexia 150; anaemia 146; thrombocytopenia 133; skin ulcer 100. FAERS via Open Targets · CHEMBL467 · 2026-06-24

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  • pyrexia
    150
  • anaemia
    146
  • thrombocytopenia
    133
  • skin ulcer
    100
  • pancytopenia
    79
  • white blood cell count increased
    70
4 more recorded rows
  • febrile neutropenia
    63
  • platelet count increased
    54
  • splenomegaly
    52
  • squamous cell carcinoma
    45

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

Which 10 reactions does Hydroxycarbamide's label not list?


anaemia, febrile neutropenia and pancytopenia and 7 more reported for Hydroxycarbamide, absent from its label. FAERS via Open Targets · CHEMBL467 · 2026-06-24

2 label terms; 10 reported and unlisted; e76fd60e-7644-48c5-9857-3608a045000b

Show the evidence
  • anaemia
    count not stated
  • febrile neutropenia
    count not stated
  • pancytopenia
    count not stated
  • platelet count increased
    count not stated
  • pyrexia
    count not stated
  • skin ulcer
    count not stated
4 more recorded rows
  • splenomegaly
    count not stated
  • squamous cell carcinoma
    count not stated
  • thrombocytopenia
    count not stated
  • white blood cell count increased
    count not stated

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

Where it is registered
Identifiers, relations and other names

The exact record

ChEMBL id
CHEMBL467
PubChem CID
3657
CAS number
127-07-1
RxCUI
5552
InChIKey
VSNHCAURESNICA-UHFFFAOYSA-N
Also called
HYDROXYUREA, Hidroxicarbamida, Xromi, hc, hu, hydroxiurea, hydroxy urea, HYDROXYCARBAMIDE [EMA EPAR], HYDROXYCARBAMIDE [EP MONOGRAPH], HYDROXYCARBAMIDE [JAN], HYDROXYCARBAMIDE [MART.], HYDROXYUREA [HSDB]
Trade name
Droxia, Hydrea, Siklos, Droxia / Siklos / Hydrea / Xromi
Development code
NSC-32065, SQ 1089
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

This is a record of evidence. It is not medical advice, and it does not say this substance suits you. Nothing here says any substance on RNAWiki is appropriate for a child.