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Condition (MeSH)

Porphyria, Variegate

ClinicalTrials.gov lists 8 trials for Porphyria, Variegate or a narrower condition beneath it. Alnylam Pharmaceuticals has the most among industry and government lead sponsors, with 3.

MeSH D046350 Digestive System Diseases C06 C16 C17 C18 Listed by Orphanet as a rare disease
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Phase 2 Phase 2 2 Phase 3 Phase 3 1 Observational Observational 4 Expanded access Expanded access 1 Phase 2 Phase 2 2 Phase 3 Phase 3 1 Observational Observational 4 Expanded access Expanded access 1

Trials per phase

8 of the 8 trials are tagged with Porphyria, Variegate itself; the rest are tagged with a narrower condition beneath it.

Get the Porphyria, Variegate report as a PDF

The PDF carries up to 50 rows of each section, where the page shows the first few, with the sources listed. Enter a work email and confirm it from the message we send, and the report follows by email.

When did Porphyria, Variegate trials start? (7)

Shown in this order: most recent first, 1990 onwards. 2026 is partial: trials registered with a 2026 start date so far, not a full year.

Trials over time, by phase

  • Phase 2
  • Phase 3
  • Observational

Top of scale: 1

2011: 1 trials (Observational 1) 2014: 1 trials (Observational 1) 2015: 1 trials (Phase 2 1) 2017: 1 trials (Phase 3 1) 2018: 1 trials (Observational 1) 2019: 1 trials (Observational 1) 2023: 1 trials (Phase 2 1)

Trials registered with a start date in each year, 1990 onward, stacked by phase (complete years only; the current partial year is in the table's notes). The table below lists the same counts, most recent years first.

Trials started per year
Year Trials started
2023 1
2019 1
2018 1
2017 1
2015 1
2014 1
2011 1

No forecast is made for this condition: it averages 0.2 trial starts a year over the five years to September, under the 5 a year the method needs.

Recruitment duration, a model prediction: none of the 8 trials evaluated at the 2026-10-09 snapshot is predicted and still running. Source: ClinicalTrials.gov / AACT (CTTI), public domain.

What phase are Porphyria, Variegate trials in? (4)

Shown in this order: phase order.

Show the trials by phase as a table
Trials by phase
Phase Trials
Phase 2 2
Phase 3 1
Observational 4
Expanded access 1

How many Porphyria, Variegate trials are recruiting? (4)

Shown in this order: the order a trial moves through, from not yet recruiting to unknown.

Trials by recruitment status
Status Trials
Active, not recruiting 1
Completed 4
Approved for marketing 1
Terminated 2

Status as registered on ClinicalTrials.gov. Each trial is counted once.

Which lead sponsors run the most Porphyria, Variegate trials? (2)

Shown in this order: most trials first. Lead sponsors the registry classes as industry, NIH, US federal, other government or network. A hospital or university the registry files as government can appear here. Sponsors it classes as academic or other are shown on the full page, because that class cannot separate an institution from a named investigator.

Lead sponsors
Sponsor Trials
Alnylam Pharmaceuticals 3
Clinuvel Pharmaceuticals Limited 1

Which serious adverse events do Porphyria, Variegate trials report? (10)

Shown in this order: most subjects affected first. Subjects affected summed across trials, a cross-trial signal, not a rate.

Serious adverse events in this condition's trials
Adverse event Subjects affected Trials
Pulmonary embolism 8 1
Chronic kidney disease 6 1
Device related infection 5 1
Blood homocysteine increased 4 1
COVID-19 pneumonia 4 1

Which publications cover Porphyria, Variegate? (1)

Shown in this order: major-topic papers first, then newest, then most linked to trials.

Publications
Publication Journal Year Major topic
Acute porphyrias in the USA: features of 108 subjects from porphyrias consortium. The American journal of medicine 2014 Yes

Prevalence (66)

Shown in this order: jurisdiction, then disease name. Orphanet observation for the mapped ORPHA concept; the mapping relation column says whether that concept is exactly this condition or broader or narrower.

Prevalence
Orphanet disease Type Class Geography Jurisdiction Mapping relation
Variegate porphyria Annual incidence <1 / 1 000 000 United Kingdom GB Exact
Variegate porphyria Point prevalence 1-9 / 1 000 000 United Kingdom GB Exact
Variegate porphyria Point prevalence 1-9 / 1 000 000 United Kingdom GB Exact
Variegate porphyria Annual incidence <1 / 1 000 000 United Kingdom GB Unspecified
Variegate porphyria Point prevalence 1-9 / 1 000 000 United Kingdom GB Unspecified

Rare-disease identity (2)

Shown in this order: exact, broader, narrower, other.

Rare-disease identity
Orphanet disease ORPHA code Relation Source
Variegate porphyria 79473 Exact Orphanet Rare Disease Ontology (ORDO)
Variegate porphyria 79473 Exact Orphadata Science knowledge files, Product 1

Not on this page

Not measured: delivery record.

Checked, with nothing recorded for this condition: sub-conditions in mesh.

Not computed for conditions with fewer than 10 trials: drugs tested.

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