This page shows what was measured, who it was measured in, and what that does not settle.
What Lumasiran does in the body
Used for an inherited disorder that causes kidney stones and kidney failure.
A broken liver enzyme lets a chemical called glyoxylate turn into oxalate, which the kidneys cannot handle and which forms stones and scarring. Lumasiran does not fix the broken enzyme. It switches off an earlier one, so far less glyoxylate is produced in the first place and there is less raw material to turn into oxalate. Urine oxalate dropped into the normal range for most patients within a few months.
What happened in people
The waste chemical that causes the disease fell by about two thirds after six months.
✓ Reviewed first-read answer
Where this came from
A person wrote this and a reviewer approved it against this exact record. It carries no effect size.
A reviewer approved this against this record. The reviewed-claim record carrying the exact population and effect size does not exist yet.
No source is stored against this line.
The limit that matters most
The study did not show whether lumasiran prevents kidney stones or kidney failure.
Where it acts
Hepatocyte peroxisome and cytoplasm (liver)
Kind of result
The kind of result is not recorded
Supervision
Professional supervision is normally required. It is a prescription or clinician-administered medicine where it is approved.
What the registries record it as
The substance registry classes this as nucleicacid.
FDA substance registry · 67P6XH37HD · 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.
The limit a reviewer approved as the one that matters most here.
The four opening statements run to 99 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.
Protein
A protein is a folded chain your body builds to do a specific job.
A picture of it, and where the picture fails
A protein is like a tool bent into one shape for one task.
Where that stops being true. A tool keeps its shape. A protein can change shape and stop working.
What people get wrong. Protein in food and a protein in the body are related but not the same thing.
A polymer of amino acids folded into a defined structure that determines its function.
Gene
A gene is a stretch of instructions for building one protein.
A picture of it, and where the picture fails
A gene is like one page of a building plan.
Where that stops being true. A page is read the same way every time. A gene can be read more or less often.
What people get wrong. Having a gene is often read as having a trait. Whether it is used matters as much.
A segment of DNA that encodes a functional product, usually a protein.
Messenger RNA
Messenger RNA is a working copy of one gene, carried to where proteins are built.
A picture of it, and where the picture fails
It is like a photocopy of one plan page, taken to the workshop.
Where that stops being true. A photocopy lasts. This copy is destroyed soon after use, on purpose.
What people get wrong. It is often thought to change the gene. It is a copy, and it does not alter the original.
A single-stranded transcript of a gene that ribosomes translate into a protein.
Small interfering RNA
A small interfering RNA is a short piece that makes a cell destroy one working copy.
A picture of it, and where the picture fails
It is like a note telling the workshop to shred one plan page.
Where that stops being true. A note is read once. This keeps working for months after one injection.
What people get wrong. It is often described as gene editing. It leaves the gene untouched.
A short double-stranded RNA that directs the RNA-induced silencing complex to cleave a complementary transcript.
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.
Percent change in 24-hour urinary oxalate excretion from baseline to month 6
✓ The study showed what it set out to show
Who was studied
ILLUMINATE-A (NCT03681184)
How many people
39
Study design
Phase 3
Compared against
Not recorded for this study
Kind of result
A number that stands in for health
What was found
P < 0.001
Repeated elsewhere
Unreplicated
What this does not prove. Meeting one endpoint in one population does not carry to other goals or other people.
The limits of this study, and where it came from
Main limit. Mild transient injection-site reactions in 38% of treated patients. The randomised comparison ends at month 6; everything after that is open-label.
How far it carries. This study is recorded from the curated record, not from the registry match.
Form and route. GalNAc-conjugated siRNA, subcutaneous injection
Interval reported. Not recorded in this summary
Written into the record, not signed off as a reviewed claim.
ILLUMINATE-A (NCT03681184) · a recorded source, not a stored snapshot
OXLUMO (lumasiran) injection, US prescribing information — DailyMed (https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=16985a31-f5e4-4557-9266-fc78d4b… · a recorded source, not a stored snapshot
What we know
RNAWiki holds 1 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 it changes in the body◇Read from sources, not yet reviewed
The path through the body
From what a person takes to what changes. A solid line is a step measured in people. A dashed line is a step nobody has measured that way.
Start
Lumasiran
What a person takes: GalNAc-conjugated siRNA, subcutaneous injection.
The measurement behind this step
A preservative-free solution containing 94.5 mg of lumasiran in 0.5 mL. Weight-banded dosing: three monthly loading doses, then quarterly maintenance for patients weighing 20 kg or more, with different bands below that weight.
Getting in
Weight-based subcutaneous dose with a liver tag
An injection under the skin, dosed by weight, monthly at first and then usually once every three months. The sugar tag means it goes to the liver and nowhere else that matters.
──Measured in people. Written by a person from the study named beside the step. Not signed off as a reviewed claim.
The measurement behind this step
Triantennary N-acetylgalactosamine binds hepatocyte ASGPR. The label gives a three-dose monthly loading phase followed by quarterly maintenance for patients at 20 kg and above, with different bands below that.
ILLUMINATE-A (NCT03681184) · a recorded source, not a stored snapshot
Reaching the cell
Uptake into the hepatocyte
Liver cells pull the drug inside. A portion escapes into the cell body and forms a reservoir that lasts for months.
╌╌Described, with no measurement named. No measurement is named for this step, so it is drawn as an unconfirmed link.
The measurement behind this step
ASGPR-mediated endocytosis delivers the duplex to the endosome; a fraction escapes into the cytoplasm and the remainder forms the slow-release depot responsible for the quarterly dosing interval.
ILLUMINATE-A (NCT03681184) · a recorded source, not a stored snapshot
What it acts on
RISC loading
One of the two strands is loaded into the cell's own silencing machinery; the other is discarded.
╌╌Described, with no measurement named. No measurement is named for this step, so it is drawn as an unconfirmed link.
The measurement behind this step
The 23-nucleotide antisense strand loads into Argonaute 2. Lumasiran is one of the three approved siRNAs whose guide binds its target with complete base-pair complementarity.
ILLUMINATE-A (NCT03681184) · a recorded source, not a stored snapshot
The change it makes
HAO1 messenger RNA is cut and glycolate oxidase disappears
The complex destroys the instructions for glycolate oxidase, the enzyme that makes the raw material for oxalate.
╌╌Described, with no measurement named. No measurement is named for this step, so it is drawn as an unconfirmed link.
The measurement behind this step
Argonaute 2 cleaves HAO1 mRNA in its 3' untranslated region. Loss of hepatic glycolate oxidase reduces conversion of glycolate to glyoxylate, so less substrate reaches the lactate dehydrogenase step that produces oxalate. This is substrate reduction therapy: the deficient enzyme AGT is never touched.
ILLUMINATE-A (NCT03681184) · a recorded source, not a stored snapshot
What that does for a person
Urinary oxalate falls into the normal range
Less oxalate is made, so less arrives at the kidney. In the trial 84% of treated patients reached a normal or near-normal urine level within six months.
──Measured in people. Written by a person from the study named beside the step. Not signed off as a reviewed claim.
The measurement behind this step
Reduced hepatic oxalate synthesis lowers 24-hour urinary oxalate by a placebo-adjusted 53.5 percentage points and plasma oxalate by 39.5 percentage points, with glycolate rising as the substrate accumulates behind the block. Whether that translates into fewer stones and preserved eGFR has not been tested against a control.
ILLUMINATE-A (NCT03681184) · a recorded source, not a stored snapshot
No suggested links are held for this record, so nothing is hidden from this path.
What we know
The record describes 5 steps from taking it to an effect in a person.
How we know it
Each step names the study behind it in the technical detail beside it.
What this does not prove
A change inside the body is a reason to look. It is not a result in a person.
Why it matters
A reader can see exactly where the chain stops being measured in people.
What we still do not know
No reviewed claim binds any of these steps to a named population.
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.
Children and adults with genetically confirmed PH1. Lumasiran is dosed by body weight, with a monthly loading phase followed by quarterly maintenance in most weight bands.
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 of OXLUMO have been established in pediatric patients aged birth and older.”
US prescribing information · 16985a31-f5e4-4557-9266-fc78d4bc5055 · read 2026-08-30
On older people, the label states: “Clinical studies of OXLUMO did not include sufficient numbers of patients aged 65 and over to determine whether they respond differently from younger patients.”
US prescribing information · 16985a31-f5e4-4557-9266-fc78d4bc5055 · read 2026-08-30
On people who are pregnant, the label states: “Risk Summary There are no available data with the use of OXLUMO in pregnant women to evaluate a drug-associated risk of major birth defects, miscarriage, or adverse maternal or fetal outcomes.”
US prescribing information · 16985a31-f5e4-4557-9266-fc78d4bc5055 · read 2026-08-30
On people who are breastfeeding, the label states: “Risk Summary There are no data on the presence of OXLUMO in human milk, the effects on the breastfed child, or the effects of the drug on milk production.”
US prescribing information · 16985a31-f5e4-4557-9266-fc78d4bc5055 · read 2026-08-30
On people with reduced liver function, the label states: “No dose adjustment is recommended for patients with mild [total bilirubin > upper limit of normal (ULN) to 1.5 × ULN or AST > ULN] or moderate hepatic impairment (total bilirubin > 1.5 to 3 × ULN with any AST).”
US prescribing information · 16985a31-f5e4-4557-9266-fc78d4bc5055 · read 2026-08-30
On people with reduced kidney function, the label states: “No dose adjustment is necessary in patients with renal impairment including patients with kidney failure treated with hemodialysis [see Clinical Pharmacology (12.3) ] .”
US prescribing information · 16985a31-f5e4-4557-9266-fc78d4bc5055 · read 2026-08-30
Where the result stopped carrying
Pyridoxine, the prior pharmacological option, works only in a minority of AGXT genotypes and leaves the rest with fluid and citrate management alone
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 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 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
Given by a clinician
❝ Quoted from a source
Where this came from
Copied from a source RNAWiki stored, with the source named.
Read from the recorded approval status.
No source is stored against this line.
Form and route
GalNAc-conjugated siRNA, subcutaneous injection
✎ 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. It is a prescription or clinician-administered medicine where it is approved.
❝ Quoted from a source
Where this came from
Copied from a source RNAWiki stored, with the source named.
The identity record classes it as siRNA (Small Interfering RNA).
No source is stored against this line.
What is in the pack
5 mL. Weight-banded dosing: three monthly loading doses, then quarterly maintenance for patients weighing 20 kg or more, with different bands below that weight.
✎ 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◇Read from sources, not yet reviewed
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.
·Worth asking a clinician aboutWritten into the record from the studies named on this page
The label carries no boxed warning and no warnings-and-precautions section. The only adverse reaction reported in at least 20% of patients is injection-site reaction, and in the pivotal trial these were mild and transient.
Nobody counted how many people took this and were fine, so this cannot be turned into a rate.
ILLUMINATE-A (NCT03681184) · a recorded source, not a stored snapshot
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
GalNAc-conjugated siRNA, subcutaneous injection
✎ 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
5 mL. Weight-banded dosing: three monthly loading doses, then quarterly maintenance for patients weighing 20 kg or more, with different bands below that weight.
✎ 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 which forms are sold and how they compare.
No source is stored against this line.
What is recorded as being sold
3 products list this as an active ingredient in the United States drug directory. 3 of them contain it and nothing else.
FDA National Drug Code directory · 82613-001 · read 2026-08-29
They are sold as injection, solution and powder, taken subcutaneous.
FDA National Drug Code directory · 82613-001 · 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 · 16985a31-f5e4-4557-9266-fc78d4bc5055 · read 2026-08-29
Those labels are classed as human prescription drug.
US prescribing information · 16985a31-f5e4-4557-9266-fc78d4bc5055 · read 2026-08-29
OXLUMO is subcutaneous at 3 DOSAGE FORMS AND STRENGTHS Injection: 94.5 mg/0.5 mL clear, colorless-to-yellow solution in a single-dose vial., recorded as fda label in effect 2025-12-01 in the United States.
US prescribing information · 16985a31-f5e4-4557-9266-fc78d4bc5055 · 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 Lumasiran 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
Claims that go past the evidence
Things said about this substance that sound reasonable, and the exact step that is missing between the evidence and the claim.
✗Goes past the evidence
That lowering urinary oxalate prevents kidney stones — no randomised trial has counted stone events
Why it sounds right. It follows from something real on this page: a body step, an animal result or a related finding.
The missing step. Nobody measured this in people for this use, or the study that did measure it did not show it.
What would change this
A study in people, for this exact use, measuring this exact thing, that a reviewer signs off.
RNAWiki has not yet published a reviewed conclusion for this use.
✗Goes past the evidence
That lowering urinary oxalate preserves kidney function — eGFR was not a powered endpoint
Why it sounds right. It follows from something real on this page: a body step, an animal result or a related finding.
The missing step. Nobody measured this in people for this use, or the study that did measure it did not show it.
What would change this
A study in people, for this exact use, measuring this exact thing, that a reviewer signs off.
RNAWiki has not yet published a reviewed conclusion for this use.
✗Goes past the evidence
That the 39-patient effect size transfers unchanged across all PH1 genotypes and ages
Why it sounds right. It follows from something real on this page: a body step, an animal result or a related finding.
The missing step. Nobody measured this in people for this use, or the study that did measure it did not show it.
What would change this
A study in people, for this exact use, measuring this exact thing, that a reviewer signs off.
RNAWiki has not yet published a reviewed conclusion for this use.
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.
How much did people take in the studies?
The sources RNAWiki checked hold nothing for this field.
Why it matters. A result belongs to an amount. Without the amount the result floats free.
What would answer it
A stored source that records 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 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 Lumasiran 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.
Every line above can be traced to the study named beside it. Follow the link and read it.
ILLUMINATE-A: 53.5 percentage-point reduction in 24-hour urinary oxalate
In plain words
Urinary oxalate fell by about two-thirds on lumasiran and barely moved on placebo, and 84% of treated patients reached a normal or near-normal level by six months against none on placebo.
What was measured
Percent change in 24-hour urinary oxalate excretion at month 6
Effect estimate
No reviewed effect estimate exists for this record.
Limits
See the technical detail.
From the source
Double-blind phase 3, 39 patients aged 6 and over randomised 2:1. Least-squares mean difference in change in 24-hour urinary oxalate excretion, lumasiran minus placebo, was -53.5 percentage points (P<0.001), with a 65.4% reduction in the lumasiran group. Plasma oxalate difference -39.5 percentage points (P<0.001). 84% of lumasiran patients versus 0% of placebo patients had 24-hour urinary oxalate no higher than 1.5 times the upper limit of normal at month 6 (P<0.001). Mild transient injection-site reactions in 38%.
Written into the record, not signed off as a reviewed claim
The indication is written as a chemistry, because the chemistry is what was measured
In plain words
The FDA licensed lumasiran "to lower urinary and plasma oxalate levels". It did not license it to prevent kidney stones or kidney failure, because the trial did not measure those.
What was measured
That normalising urinary oxalate prevents stones, nephrocalcinosis and progression to kidney failure
Effect estimate
No reviewed effect estimate exists for this record.
Limits
See the technical detail.
From the source
The approved indication reads: "for the treatment of primary hyperoxaluria type 1 (PH1) to lower urinary and plasma oxalate levels in pediatric and adult patients". Urinary oxalate is a surrogate: it is the mechanistic cause of stone formation and nephrocalcinosis, and it is strongly associated with progression, but the pivotal trial's 6-month randomised period was never powered for stone events or for eGFR decline. Longer-term data are open-label extensions without a concurrent control.
Source
OXLUMO US prescribing information, section 1
Role in the trial
Not matched to a registered study
Identity check
no open identity hold
Audit mark
caution
Review state
Written into the record, not signed off as a reviewed claim
Sixty-month follow-up sustained the biochemical effect without a control group
In plain words
Patients followed for five years kept the low oxalate levels. Everyone in that extension was on the drug, so there is nothing to compare against.
What was measured
Not recorded
Effect estimate
No reviewed effect estimate exists for this record.
Limits
See the technical detail.
From the source
ILLUMINATE-A ran a 6-month double-blind placebo-controlled period followed by an extension of up to 54 months in which all patients received lumasiran. The final report describes sustained reductions in urinary and plasma oxalate and "encouraging clinical outcomes", with mild injection-site reactions the most common adverse event. From month 6 onward there was no randomised comparator.
Written into the record, not signed off as a reviewed claim
The drug blocks a step and the substrate backs up behind it
In plain words
Because lumasiran turns off an enzyme partway down the pathway, the chemical feeding that enzyme accumulates instead. Rising glycolate is the expected signature of the drug working.
What was measured
Plasma and urinary glycolate as a target-engagement marker
Effect estimate
No reviewed effect estimate exists for this record.
Limits
See the technical detail.
From the source
Silencing HAO1 removes glycolate oxidase, which converts glycolate to glyoxylate. Glyoxylate flux to oxalate therefore falls and plasma and urinary glycolate rise. Isolated glycolate accumulation has no known pathological consequence, and elevated glycolate is used as a pharmacodynamic confirmation of target engagement rather than treated as an adverse finding.
Written into the record, not signed off as a reviewed claim
Thirty-nine patients is the entire randomised evidence base
In plain words
The pivotal trial enrolled 39 people, 26 of them on the drug. In a disease this rare that is a reasonable trial, but it is a small number to carry a licence.
What was measured
That effect sizes from 26 treated patients transfer confidently to every PH1 genotype and age band
Effect estimate
No reviewed effect estimate exists for this record.
Limits
See the technical detail.
From the source
ILLUMINATE-A randomised 39 patients aged 6 and over, 26 to lumasiran and 13 to placebo. Subsequent studies — ILLUMINATE-B in infants and young children and ILLUMINATE-C in advanced kidney disease — were single-arm. No randomised trial of lumasiran has been powered for a clinical event.
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.
How close this is to real life — 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.
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.
The older medicine-wide conclusion held in this record
Written for a clinical reader, and about the medicine as a whole rather than about one indication, population and set of trials. RNAWiki does not treat it as a programme conclusion and no reviewer has signed it off in that form. It is here word for word because it is what the record holds.
Silences glycolate oxidase so the liver diverts glyoxylate away from oxalate, cutting 24-hour urinary oxalate by 53.5 percentage points against placebo in 39 patients — a biochemical endpoint, which is exactly what the FDA indication says it is licensed for.
Recorded evidence blocks (4)
Q1
On the Lumasiran label: indicated for what?
"OXLUMO is indicated for the treatment of primary hyperoxaluria type 1 (PH1) to lower urinary and plasma oxalate levels in pediatric and adult patients [see Clinical Pharmacology (12.1) , Clinical Studies (14.1 , 14.2 , 14.3) ] . OXLUMO is a HAO1 -directed small interfering ribonucleic acid (siRNA) indicated for the…": indications and usage on Lumasiran's label. DailyMed label · 16985a31-f5e4-4557-9266-fc78d4bc5055 · 2025-12-01
Q2
8 registered trials of Lumasiran — at which phases?
ChEMBL ATC CC BY-SA · ClinicalTrials.gov — US Government work · mixed register set; per-register licences in docs/specs/corpus-20k-sources.md · openFDA / DailyMed — US Government work
✗ 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: 4 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.