This page shows what was measured, who it was measured in, and what that does not settle.
What Mitapivat does in the body
Hemolytic anemia
From the FDA-approved label: Mitapivat is a pyruvate kinase activator that acts by allosterically binding to the pyruvate kinase tetramer and increasing pyruvate kinase (PK) activity. Imbalances in globin chain production during erythropoiesis result in increased oxidative stress, which leads to ineffective erythropoiesis and hemolysis. In nonclinical models of beta-thalassemia, mitapivat improved energy homeostasis, RBC longevity, ineffective erythropoiesis, and hemolysis by increasing PK activity.
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 · 2WTV10SIKH · read 2026-08-29
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 79 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.
Stand-in result
A stand-in result is a number measured because the real result takes too long.
A picture of it, and where the picture fails
It is like judging a journey by the speedometer rather than by arriving.
Where that stops being true. Speed does predict arrival. Many stand-in results do not predict the real one.
What people get wrong. A stand-in result is often reported as the result itself.
A surrogate endpoint substituted for a clinical endpoint, valid only where the substitution has been shown to hold.
Enzyme
An enzyme is a protein that speeds up one chemical change.
A picture of it, and where the picture fails
An enzyme is like a machine on a production line doing one cut.
Where that stops being true. A machine is switched on and off by a person. Enzymes are controlled by the cell.
What people get wrong. Enzymes are thought to be used up. They are not; they work again and again.
A catalytic protein that lowers the activation energy of a specific reaction.
Receptor
A receptor is a part of a cell that a signal fits into.
A picture of it, and where the picture fails
A receptor is like a lock waiting for one key.
Where that stops being true. A lock either opens or does not. A receptor can be half-triggered, or worn out.
What people get wrong. Fitting a receptor is read as causing a benefit. It causes a step, and nothing more.
A protein that binds a specific ligand and converts that binding into a cellular response.
Pathway
A pathway is a chain of steps inside a cell, each one setting off the next.
A picture of it, and where the picture fails
A pathway is like a row of dominoes.
Where that stops being true. Dominoes fall one way. Pathways loop back and can switch themselves off.
What people get wrong. Changing one step is read as changing the outcome. Other steps often absorb it.
An ordered series of molecular interactions producing a defined cellular change.
What happened in people◇Read 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.
Phase 2: Percentage of Participants With Hemoglobin (Hb) Response
✗ The study did not show it
Who was studied
NCT05031780
How many people
286
Study design
Phase 2/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. 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
Double-blind Period: Percentage of Participants Who Achieved Transfusion Reduction Response (TRR)
✗ The study did not show it
Who was studied
NCT04770779
How many people
258
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. 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
Double-Blind Period: Percentage of Participants Who Achieved Hemoglobin (Hb) Response From Week 12 Through Week 24 Compared With Baseline
✗ The study did not show it
Who was studied
NCT04770753
How many people
194
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. 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
Percentage of Subjects who are Transfusion Free From Week 4 Through Week 52
✗ The study did not show it
Who was studied
NCT07656415
How many people
159
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. 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
All Cohorts: Number of Participants With at Least One Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, Related TEAEs and TEAEs With Severity Greater Than or Equal to Grade 3
✗ The study did not show it
Who was studied
NCT03853798
How many people
90
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. 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
Percentage of Participants Achieving a Hemoglobin (Hb) Response (HR)
✗ The study did not show it
Who was studied
NCT03548220
How many people
80
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. 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 happened in people◇Read from sources, not yet reviewed
How close this is to real life
The top step is something a person would feel or care about. The bottom is a guess from software. Filled steps are where evidence exists for this substance.
□Living longer, or avoiding a major eventNo evidence recorded. Death, a heart attack, a stroke, a hospital stay.No registered study measures this.
□What a body can do day to dayNo evidence recorded. Walking, dressing, breathing, recovering.No registered study measures this.
□Measured performanceNo evidence recorded. How much was lifted, how far was run, how fast.No registered study measures this.
□Symptoms and quality of lifeNo evidence recorded. Pain, tiredness, mood, sleep, as the person rated it.No registered study measures this.
■A number that stands in for healthEvidence recorded. Cholesterol, blood sugar, bone density. A stand-in, not the thing itself.2 registered measures of this kind.
■A step measured inside a personEvidence recorded. Something measured in human tissue or human cells.A human record sits on the stored organism ladder.
■AnimalsEvidence recorded. Mice, rats, dogs. Useful for ideas, not proof about people.Stored records in Mouse. A result in animals says what to test next. It does not say what happens in people.
□Cells in a dishNo evidence recorded. Cells or chemistry on a bench, far from a whole body.No cell or bench record is stored.
□A guess from softwareNo evidence recorded. Nobody measured it. RNAWiki never publishes one as a finding.RNAWiki stores no prediction for this record. Software can suggest a link. RNAWiki does not publish one as a finding.
Higher on these steps means closer to something a person would feel. It does not mean better done.
What it would be like to take◇Read from sources, not yet reviewed
Felt, measured, or meaningful
Three different things that get called the same word. Registered studies measured all three, and mixing them is how a blood test becomes a health claim.
Felt
Things a person could notice without a test.
No registered study measured anything of this kind.
Measured
Things only a test, a scale or a device shows.
achieving a hemoglobin response
maximum observed plasma concentration of midazolam
Meaningful
Things that change how a life goes, not only a number.
No registered study measured anything of this kind.
A registered study says what someone planned to measure. It does not say what they found. A number moving is not the same as a life going better. The two are shown apart here.
Felt, but not shown to help. A person notices a change. No study showed it leads anywhere good.
Measured, but not felt. A number moves. The person notices nothing. Both can be true.
Measured, but not shown to matter. A number moves in the good direction. Nobody showed that lives went better.
Matters, but takes years. The result that counts may take longer than anyone would keep watching.
Names that fit none of the three (3)
safety and tolerability
incidence of adverse events
cmax
These are harms, study-process measures, or names the rules did not recognise. They are shown rather than dropped.
What is missing or unclear◇Read 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.
PYRUKYND is indicated for the treatment of hemolytic anemia in adults with pyruvate kinase (PK) deficiency. PYRUKYND is a pyruvate kinase activator indicated for the treatment of hemolytic anemia in adults with pyruvate kinase (PK) deficiency. ( 1 )
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 · 1d4e2654-1636-0d01-e063-6394a90af303 · read 2026-08-30
On older people, the label states: “Clinical studies of AQVESME did not include sufficient numbers of subjects aged 65 years and over to determine whether they respond differently from younger subjects.”
US prescribing information · 1d4e2654-1636-0d01-e063-6394a90af303 · read 2026-08-30
On people who are pregnant, the label states: “Risk Summary Available data from clinical trials of AQVESME are insufficient to evaluate for a drug-associated risk of major birth defects, miscarriage or other adverse maternal or fetal outcomes.”
US prescribing information · 1d4e2654-1636-0d01-e063-6394a90af303 · read 2026-08-30
On people who are breastfeeding, the label states: “Risk Summary There are no data on the presence of AQVESME or its metabolites in human or animal milk, the effects on the breastfed child, or the effects on milk production.”
US prescribing information · 1d4e2654-1636-0d01-e063-6394a90af303 · read 2026-08-30
On people with reduced liver function, the label states: “Avoid use in patients with cirrhosis (Child-Pugh Class A, B, or C) due to an increased risk of severe outcomes if hepatocellular injury occurs [see Warnings and Precautions (5.1) ] .”
US prescribing information · 1d4e2654-1636-0d01-e063-6394a90af303 · 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 take◇Read 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 for a different goal
The studies measured something else entirely.
On this record: Some registered studies measured things that match no goal on this page.
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 (10)
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 take◇Read 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: S5, S6.
No source is stored against this line.
What is in the pack
Sold as tablet, 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 take∅Nothing 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 unclear◇Read 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
7 products list this as an active ingredient in the United States drug directory. 7 of them contain it and nothing else.
FDA National Drug Code directory · 71334-205 · read 2026-08-29
They are sold as powder and tablet, film coated, taken oral.
FDA National Drug Code directory · 71334-205 · read 2026-08-29
The regulator's established pharmacologic class for it is cytochrome p450 2b6 inducers [moa], cytochrome p450 2c19 inducers [moa] and cytochrome p450 2c8 inducers [moa].
FDA National Drug Code directory · 71334-205 · read 2026-08-29
2 published labels name it as an active ingredient. 2 of them describe this substance alone, which is where its own label text on this page comes from.
US prescribing information · 4ccee896-e313-4c6c-9674-b4746ada0a90 · read 2026-08-29
Those labels are classed as human prescription drug.
US prescribing information · 4ccee896-e313-4c6c-9674-b4746ada0a90 · read 2026-08-29
AQVESME is oral at 3 DOSAGE FORMS AND STRENGTHS 100 mg tablets: oblong, blue, film-coated tablets with "M100" printed on both sides., recorded as fda label in effect 2026-04-22 in the United States.
US prescribing information · 1d4e2654-1636-0d01-e063-6394a90af303 · 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 take◇Read 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 Mitapivat 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 unclear◇Read 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 Mitapivat 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 people◇Read 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
2 documents were read for this substance.
RNAWiki source record
2 of them state the same bioavailability, and they agree.
RNAWiki source record
2 of them state the same tMax, and they agree.
RNAWiki source record
2 of them state the same volumeOfDistribution, and they agree.
RNAWiki source record
Where else this substance is registered
FDA substance identifier (UNII)
2WTV10SIKH
RxNorm concept
2594473
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: S5, S6.
✓ Passed
Canonical metadata present
slug and display name present
What is missing or unclear◇Read 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
1 approved application covers products containing this substance. The earliest was NDA216196, approved 20220217 to AGIOS PHARMS INC.
This order is fixed in code and does not count clicks or time on the page.
What is not here
7 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.
The path through the body — 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)
Q2
What did Mitapivat's largest trial (286 people) and its longest (12 years) measure?
286 people in Mitapivat's largest registered study, 12 years in its longest registered window, measuring Area Under the Concentration-Time Curve From Time Zero to the Last Non-Zero Concentration [AUC(0- t)]. ClinicalTrials.gov · 2026-09-01
11 phase1, 11 phase3, 7 phase2, 1 early phase1, 1 phase4; NCT03692052; 2030-09-30; no ageing endpoint recorded. Last human test completed 2025, NCT02476916.
Interpretation These counts include studies where Mitapivat was a comparison treatment, a background treatment or an exposure in an observational study, and the longest window may be a planned end date. Trial roles have not yet been classified for this record.
Show the evidence
phase1
11
phase3
11
phase2
7
early phase1
1
phase4
1
Last recorded human testNCT02476916
2025-04-02
recorded 2026-09-01 · last checked 2026-09-04
Q3
From mouse to human: where has Mitapivat shown biomarker?
Percentage of Participants Achieving a Hemoglobin Response (HR); Primary outcome measure: Long-term safety and tolerability of mitapivat in subjects with stable sickle cell disease; latest 2032-06
Show the evidence
Trial
NCT03692052
"A Study to Determine the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of AG-348 in Adult Participants With Non-transfusion-dependent Thalassemia"; n 20; "Percentage of Participants Achieving a Hemoglobin Response (HR)"; 2030-09-30
NCT04610866
"Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Long-term Mitapivat Dosing in Subjects With Stable Sickle Cell Disease: An Extension of a Phase I Pilot Study of Mitapivat"; n 15; "Primary outcome measure: Long-term safety and tolerability of mitapivat in subjects with stable sickle cell disease"; 2028-02-28
NCT04770753
"A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Non-Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-NTDT)"; n 194; "Double-Blind Period: Percentage of Participants Who Achieved Hemoglobin (Hb) Response From Week 12 Through Week 24 Compared With Baseline"; 2028-12
NCT04770779
"A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-TDT)"; n 258; "Double-blind Period: Percentage of Participants Who Achieved Transfusion Reduction Response (TRR)"; 2029-06
NCT05031780
"A Study Evaluating the Efficacy and Safety of Mitapivat (AG-348) in Participants With Sickle Cell Disease (RISE UP)"; n 286; "Phase 2: Percentage of Participants With Hemoglobin (Hb) Response"; 2030-02
NCT05144256
"A Study to Evaluate the Efficacy and Safety of Mitapivat in Pediatric Participants With Pyruvate Kinase Deficiency (PKD) Who Are Regularly Transfused, Followed by a 5-Year Extension Period"; n 49; "Percentage of Participants Achieving Transfusion Reduction Response (TRR)"; 2029-06
7 further recorded trials
NCT05175105
"A Study to Evaluate the Efficacy and Safety of Mitapivat in Pediatric Participants With Pyruvate Kinase Deficiency (PKD) Who Are Not Regularly Transfused, Followed by a 5-Year Extension Period"; n 30; "Percentage of Participants Achieving a Hemoglobin (Hb) Response"; 2030-01
NCT05777993
"A Study to Provide Access to Mitapivat for Participants Who Previously Participated in an Agios-Sponsored Mitapivat Study"; n 59; "Not applicable as this protocol is to provide an option for continued access to mitapivat for participants who have previously participated in an Agios-sponsored mitapivat study"; 2029-08
NCT05935202
"Safety and Efficacy of Mitapivat Sulfate in Adult Patients With Erythrocyte Membranopathies"; n 25; "Safety and tolerability of mitapivat in adult patients with erythrocyte membranopathies"; 2026-12-15
NCT07055243
"To Assess Safety of Mitapivat and Provide Proof of Concept of the Efficacy of the Drug in Patients With RBC Membranopathies or CDAII."; n 9; "To evaluate safety of mitapivat"; 2027-06-30
NCT07506863
"A Study to Investigate the Efficacy, Pharmacokinetics, and Safety of Mitapivat in Pediatric Participants With Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-TDT)"; n 54; "Percentage of Participants Who Achieved Transfusion Reduction Response (TRR) Through Week 48"; 2032-06
NCT07517133
"A Study to Investigate the Efficacy, Pharmacokinetics, and Safety of Mitapivat in Pediatric Participants With α- or β-Non-Transfusion-Dependent Thalassemia"; n 45; "Percentage of Participants Who Achieved a Hemoglobin (Hb) Response"; 2032-03
NCT07656415
"A Study to Investigate the Effect of Mitapivat on Transfusion Burden in Subjects With Sickle Cell Disease (SCD)"; n 159; "Percentage of Subjects who are Transfusion Free From Week 4 Through Week 52"; 2030-08
recorded 2026-09-01 · last checked 2026-09-04
Q8
Which 9 trials of Mitapivat posted no result?
Posted no result
9 of 9 completed trials
Registrations
NCT02108106, NCT02149966, NCT03250598, NCT03703505, NCT03991312 and NCT04472832, and 3 more
Completion dates
oldest 2014-08; newest 2023-07-21
Show the evidence
Trial
NCT02108106
2014-08
NCT02149966
2014-11
NCT03250598
2017-10-09
NCT03703505
2018-06-04
NCT03991312
2019-09-10
NCT04472832
2020-11-23
3 further recorded trials
NCT04565678
2020-12-03
NCT04696393
2021-03-19
NCT05610657
2023-07-21
Q9
At the median, Mitapivat's trials enrolled 32 people — anything larger?
Median enrolment
32
Largest enrolment
286
Registered trials counted
29
Q10
Mitapivat and CYP3A4, P-GP and CYP2B6: shared by which compounds?
CYP3A4, P-GP and CYP2B6 appear in Mitapivat's recorded interaction sentences, 6 in all. openfda-label+europepmc · 2026-08-30
Interpretation pharmacokinetics
Show the evidence
CYP2B6pharmacokinetics
In vitro Studies CYP450 and UGT Enzymes Mitapivat induces CYP2B6, CYP2C8, CYP2C9, CYP2C19, and UGT1A1 .
CYP2C19pharmacokinetics
In vitro Studies CYP450 and UGT Enzymes Mitapivat induces CYP2B6, CYP2C8, CYP2C9, CYP2C19, and UGT1A1 .
CYP2C8pharmacokinetics
In vitro Studies CYP450 and UGT Enzymes Mitapivat induces CYP2B6, CYP2C8, CYP2C9, CYP2C19, and UGT1A1 .
CYP2C9pharmacokinetics
In vitro Studies CYP450 and UGT Enzymes Mitapivat induces CYP2B6, CYP2C8, CYP2C9, CYP2C19, and UGT1A1 .
CYP3A4
pharmacokinetics
Metabolism In vitro studies showed that mitapivat is primarily metabolized by CYP3A4.
pharmacokinetics
Effect of Moderate CYP3A Inducers on AQVESME Efavirenz (a moderate CYP3A4 inducer) decreased mitapivat AUC 0-12 and C max by 52% and 21%, respectively, following AQVESME 100 mg twice daily.
recorded 2026-08-30 · last checked 2026-09-04
Where it is registeredIdentifiers, relations and other names
ClinicalTrials.gov — US Government work · Europe PMC — metadata only, under the recorded legal gate · derived from this page's own recorded fields; no external licence · mixed register set; per-register licences in docs/specs/corpus-20k-sources.md · openFDA / DailyMed — US Government work
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