This page shows what was measured, who it was measured in, and what that does not settle.
What Vamorolone does in the body
Duchenne muscular dystrophy
From the FDA-approved label: Vamorolone is a corticosteroid that acts through the glucocorticoid receptor to exert anti-inflammatory and immunosuppressive effects. The precise mechanism by which vamorolone exerts its effect in patients with DMD is unknown.
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
RNAWiki has not recorded a supervision or regulatory status for this substance.
What the registries record it as
The substance registry classes this as chemical.
FDA substance registry · 8XP29XMB43 · read 2026-08-29
Its recorded molecular formula is C22H28O4, weighing 356.46 g/mol.
US prescribing information · fb376f09-cf9c-42b5-b4dd-02d5bcd73211 · 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 51 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.
Efficacy Measured by Time to Stand Test (TTSTAND) Velocity in Rises/Second Change From Baseline
✗ The study did not show it
Who was studied
NCT03439670
How many people
121
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. 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 SPL label · a recorded source, not a stored snapshot
PubChem PUG-REST · a recorded source, not a stored snapshot
FDA label (openFDA SPL) · a recorded source, not a stored snapshot
Number of vertebral fractures per 1000 person-years based on X-ray central reading.
✗ The study did not show it
Who was studied
NCT06713135
How many people
80
Study design
Phase 4
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 SPL label · a recorded source, not a stored snapshot
PubChem PUG-REST · a recorded source, not a stored snapshot
FDA label (openFDA SPL) · a recorded source, not a stored snapshot
Number of Participants With Any Adverse Events as Assessed by Common Terminology Criteria for Adverse Events Version 4.03 (CTCAE v4.03)
✗ The study did not show it
Who was studied
NCT05185622
How many people
54
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. 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 SPL label · a recorded source, not a stored snapshot
PubChem PUG-REST · a recorded source, not a stored snapshot
FDA label (openFDA SPL) · a recorded source, not a stored snapshot
Overall Summary of Adverse Events as Assessed by CTCAE Version 4.03
✗ The study did not show it
Who was studied
NCT02760264
How many people
48
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. 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 SPL label · a recorded source, not a stored snapshot
PubChem PUG-REST · a recorded source, not a stored snapshot
FDA label (openFDA SPL) · a recorded source, not a stored snapshot
Number of Participants With Adverse Events as Assessed by CTCAE Version 4.03
✗ The study did not show it
Who was studied
NCT02760277
How many people
48
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. 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 SPL label · a recorded source, not a stored snapshot
PubChem PUG-REST · a recorded source, not a stored snapshot
FDA label (openFDA SPL) · a recorded source, not a stored snapshot
Safety as measured by Treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs) by system organ class (SOC and PT)
✗ The study did not show it
Who was studied
NCT05166109
How many people
46
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. 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 SPL label · a recorded source, not a stored snapshot
PubChem PUG-REST · a recorded source, not a stored snapshot
FDA label (openFDA SPL) · 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.1 registered measure 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.
determination of the of sodium to potassium in urine
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 (5)
height from baseline to week 12
weight from baseline to week 12
height z
bmi z
cmax of midazolam
These are harms, study-process measures, or names the rules did not recognise. They are shown rather than dropped.
What it would be like to take◇Read from sources, not yet reviewed
How long anything takes
Nine different lengths of time that get confused with each other. None of them is worked out from another.
Before anything is noticed.RNAWiki does not store this separately, and never works it out from another figure on this page.
Before a test result moves.RNAWiki does not store this separately, and never works it out from another figure on this page.
Before performance moves.RNAWiki does not store this separately, and never works it out from another figure on this page.
How long the result was watched.No finished study window is recorded for a study that tested this substance.
How long people took it.How long people actually took it is not stored. The study window is not the same thing.
How long people were followed.Follow-up length is not stored separately. It is never read off the study window, which would be a different thing.
How fast the body clears it. 2 hours hours
Read from the label, which states: “The terminal elimination half-life of vamorolone is approximately 2 hours.”
How long effects linger.RNAWiki does not store this separately, and never works it out from another figure on this page.
Beyond the studies. Nothing is recorded about the long term.
The longest finished study sets the edge of what anyone measured.
A study window is not how long people took it, and neither is how long they were followed. Where RNAWiki holds only one of the three, it shows one.
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.
AGAMREE is indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients 2 years of age and older. AGAMREE is a corticosteroid indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients 2 years of age and older. ( 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: “The safety and effectiveness in pediatric patients below the age of 2 years have not been established.”
US prescribing information · fb376f09-cf9c-42b5-b4dd-02d5bcd73211 · read 2026-08-30
On older people, the label states: “DMD is largely a disease of children and young adults; therefore, there is no geriatric experience with AGAMREE.”
US prescribing information · fb376f09-cf9c-42b5-b4dd-02d5bcd73211 · read 2026-08-30
On people who are pregnant, the label states: “Risk Summary AGAMREE is indicated for use for the treatment of DMD, which is a disease of young male patients.”
US prescribing information · fb376f09-cf9c-42b5-b4dd-02d5bcd73211 · read 2026-08-30
On people who are breastfeeding, the label states: “Risk Summary There are no data on the presence of vamorolone in human milk or the effects on milk production.”
US prescribing information · fb376f09-cf9c-42b5-b4dd-02d5bcd73211 · read 2026-08-30
On people with reduced liver function, the label states: “Moderate hepatic impairment increases vamorolone exposure [see Clinical Pharmacology ( 12.3 )].”
US prescribing information · fb376f09-cf9c-42b5-b4dd-02d5bcd73211 · 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
RNAWiki has not recorded a supervision or regulatory status for this substance.
❝ Quoted from a source
Where this came from
Copied from a source RNAWiki stored, with the source named.
No register row and no identity class settled the question.
No source is stored against this line.
What is in the pack
Sold as suspension, 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
2 products list this as an active ingredient in the United States drug directory. 2 of them contain it and nothing else.
FDA National Drug Code directory · 62450-003 · read 2026-08-29
They are sold as powder and suspension.
FDA National Drug Code directory · 62450-003 · read 2026-08-29
1 published label names it as an active ingredient. 1 of them describe this substance alone, which is where its own label text on this page comes from.
US prescribing information · fb376f09-cf9c-42b5-b4dd-02d5bcd73211 · read 2026-08-29
Those labels are classed as human prescription drug.
US prescribing information · fb376f09-cf9c-42b5-b4dd-02d5bcd73211 · read 2026-08-29
AGAMREE is oral at 3 DOSAGE FORMS AND STRENGTHS Oral Suspension: 40 mg/mL orange flavored white to off-white suspension Oral Suspension: 40 mg/mL ( 3 ), recorded as fda label in effect 2026-01-22 in the United States.
US prescribing information · fb376f09-cf9c-42b5-b4dd-02d5bcd73211 · 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.
RNAWiki could not confidently classify this substance, so no self-experiment plan is offered.
Questions worth asking
Which of the trials of Vamorolone 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 Vamorolone 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.
Where else this substance is registered
FDA substance identifier (UNII)
8XP29XMB43
CAS registry number
13209-41-1
PubChem compound
3035000
ChEMBL
CHEMBL3707311
WHO international nonproprietary name list entry
10419
EMA substance identifier
100000174660
European Chemicals Agency number
236-177-8
DrugBank
DB15114
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
✗ Not passed
Safety mode resolved
No register row and no identity class settled the question.
✓ 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 NDA215239, approved 20231026 to CATALYST PHARMS.
This order is fixed in code and does not count clicks or time on the page.
What is not here
6 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.
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)
Q1
What did Vamorolone's largest trial (250 people) and its longest (7.4 years) measure?
250 people in Vamorolone's largest registered study, 7.4 years in its longest registered window, measuring Safety: concentration of blood laboratory biomarkers as assessed by standardized clinical laboratory reference ranges. ClinicalTrials.gov · 2026-09-01
3 phase2, 2 na or unstated, 2 phase1, 1 phase4; NCT06564974; 2032-02; no ageing endpoint recorded. Last human test completed 2025, NCT05166109.
Interpretation These counts include studies where Vamorolone 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
phase2
3
na or unstated
2
phase1
2
phase4
1
Last recorded human testNCT05166109
2025-08-18
recorded 2026-09-01 · last checked 2026-09-04
Q2
From mouse to human: where has Vamorolone shown biomarker?
Interpretation Safety: concentration of blood laboratory biomarkers as assessed by standardized clinical laboratory reference ranges — the recorded outcome words.
Show the evidence
mouse
mechanism-only
humanNCT05166109
biomarker; Safety: concentration of blood laboratory biomarkers as assessed by standardized clinical laboratory reference ranges; 8
recorded 2026-09-01 · last checked 2026-09-04
Q3
Human studies of Vamorolone used vamorolone 40 mg/mL oral suspension — over how long?
Vamorolone's half-life is 2 hours — which schedules were studied?
2 hours, the half-life Vamorolone's label states. openfda-label · fb376f09-cf9c-42b5-b4dd-02d5bcd73211 · 2026-08-30
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half life
2 hours hours; The terminal elimination half-life of vamorolone is approximately 2 hours.
tmax
Effect of Food Co-administration of vamorolone (2 mg/kg) with a high-fat/high-calorie meal reduced C max by 18%, increased AUC by 13%, and delayed T max by one hour.
metabolism
Metabolism Vamorolone is metabolized via multiple Phase I and Phase II metabolic pathways, such as glucuronidation, hydroxylation, and reduction.
recorded 2026-08-30 · last checked 2026-09-04
Q5
Could one person measure Vamorolone's effect on height from baseline to week 12?
Height from baseline to week 12: measured in Vamorolone's trials.
height from baseline to week 12 is the recorded endpoint.
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biomarkers
height from baseline to week 12; 2026-09-01
weight from baseline to week 12; 2026-09-01
height z; 2026-09-01
bmi z; 2026-09-01
determination of the of sodium to potassium in urine; 2026-09-01
cmax of midazolam; 2026-09-01
half life
2026-09-04; halfLife; hours; 2 hours; 2026-08-30
human trials at or under30
2
smallest human trial
18; NCT06689527; PHASE1; the smallest recorded human enrolment at or below 30; the registry states no direction of its result; COMPLETED
Q6
Which of bmi z, cmax of midazolam and determination of the of sodium to potassium in urine did Vamorolone's trials measure?
bmi z, cmax of midazolam and determination of the of sodium to potassium in urine lead 6 outcome terms across Vamorolone's trials. ClinicalTrials.gov · 2026-09-01
Interpretation bmi z, determination of the of sodium to potassium in urine and cmax of midazolam follow.
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height from baseline to week 12
1
weight from baseline to week 12
1
height z
1
bmi z
1
determination of the of sodium to potassium in urine
1
cmax of midazolam
1
recorded 2026-09-01 · last checked 2026-09-04
Q7
Which of Vamorolone's 3 ongoing trials reports first?
Change in Height z-score; Number of vertebral fractures per 1000 person-years based on X-ray central reading.; latest 2032-02
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Trial
NCT03863119
"Expanded Access Protocol for Boys With Duchenne Muscular Dystrophy"
NCT06564974
"Registry Study to Observe Long-term Safety of Vamorolone (AGAMREE®) in Patients With Duchenne Muscular Dystrophy-SUMMIT"; n 250; "Change in Height z-score"; 2032-02
NCT06713135
"A Study on Safety and Effectiveness of Long-term Treatment With Vamorolone in Boys With Duchenne Muscular Dystrophy"; n 80; "Number of vertebral fractures per 1000 person-years based on X-ray central reading."; 2028-09
recorded 2026-09-01 · last checked 2026-09-04
Q8
At the median, Vamorolone's trials enrolled 54 people — anything larger?
Median enrolment
54
Largest enrolment
250
Registered trials counted
7
Q9
Vamorolone and CYP3A4, BCRP and BSEP: shared by which compounds?
CYP3A4, BCRP and BSEP appear in Vamorolone's recorded interaction sentences, 7 in all. openfda-label+europepmc · 2026-08-30
Interpretation pharmacokinetics
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CYP2C8pharmacokinetics
Metabolism of vamorolone is mediated through CYP3A4/5, CYP2C8, UGT1A3, UGT2B7, and UGT2B17.
CYP3A4
pharmacokinetics
Metabolism of vamorolone is mediated through CYP3A4/5, CYP2C8, UGT1A3, UGT2B7, and UGT2B17.
pharmacokinetics
In Vitro Studies Metabolizing Enzymes Vamorolone induces CYP3A4 in vitro.
pharmacokinetics
Drug Interaction Studies Effect of Strong CYP3A4 Inhibitors on Vamorolone Compared to administration of vamorolone alone, administration of vamorolone following multiple doses of a strong CYP3A4 inhibitor (itraconazole) increased vamorolone C max and AUC by 8% and 44%, respectively [see Drug Interactions ( 7.1 )].
pharmacokinetics
There is no experience with vamorolone when coadministered with moderate and weak CYP3A4 inhibitors.
pharmacokinetics
Other compounds that are substrates of CYP3A4 may have decreased plasma concentrations when co-administered with AGAMREE.
pharmacokinetics
However, no clinical drug interaction studies with CYP3A4 substrates were conducted.
recorded 2026-08-30 · last checked 2026-09-04
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