Condition (MeSH)
Porphyria, Acute Intermittent
ClinicalTrials.gov lists 21 trials for Porphyria, Acute Intermittent or a narrower condition beneath it. Alnylam Pharmaceuticals has the most among industry and government lead sponsors, with 6.
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Trials per phase
21 of the 21 trials are tagged with Porphyria, Acute Intermittent itself; the rest are tagged with a narrower condition beneath it.
When did Porphyria, Acute Intermittent trials start? (13)
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 1
- Phase 1/2
- Phase 2
- Phase 2/3
- Phase 3
- Not applicable
- Observational
Top of scale: 2
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.
| Year | Trials started |
|---|---|
| 2024 | 1 |
| 2022 | 1 |
| 2019 | 2 |
| 2018 | 2 |
| 2017 | 1 |
| 2016 | 2 |
| 2015 | 2 |
| 2014 | 2 |
| 2012 | 2 |
| 2011 | 2 |
Showing 10 of 13 earlier years.
No forecast is made for this condition: it averages 0.4 trial starts a year over the five years to September, under the 5 a year the method needs.
Recruitment duration, a model prediction: 1 trial still running at the 2026-10-09 snapshot, fewer than the 5 needed for a median. Source: ClinicalTrials.gov / AACT (CTTI), public domain.
What phase are Porphyria, Acute Intermittent trials in? (8)
Shown in this order: phase order.
Show the trials by phase as a table
| Phase | Trials |
|---|---|
| Phase 1 | 4 |
| Phase 1/2 | 2 |
| Phase 2 | 2 |
| Phase 2/3 | 1 |
| Phase 3 | 1 |
| Not applicable | 1 |
| Observational | 9 |
| Expanded access | 1 |
How many Porphyria, Acute Intermittent trials are recruiting? (6)
Shown in this order: the order a trial moves through, from not yet recruiting to unknown.
| Status | Trials |
|---|---|
| Recruiting | 3 |
| Active, not recruiting | 1 |
| Completed | 13 |
| Approved for marketing | 1 |
| Terminated | 2 |
Showing 5 of 6 statuses.
Status as registered on ClinicalTrials.gov. Each trial is counted once.
Which lead sponsors run the most Porphyria, Acute Intermittent trials? (5)
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.
| Sponsor | Trials |
|---|---|
| Alnylam Pharmaceuticals | 6 |
| Digna Biotech S.L. | 2 |
| Cycle Pharmaceuticals Ltd. | 1 |
| National Center for Research Resources (NCRR) | 1 |
| Zymenex A/S | 1 |
Which drugs are being tested for Porphyria, Acute Intermittent? (6)
Shown in this order: most trials first. One row per drug, matched to a single molecule under all its names, ranked by number of trials.
| Drug | Trials |
|---|---|
| Givosiran | 5 |
| Hemin | 2 |
| tin mesoporphyrin | 1 |
| recombinant human porphobilinogen deaminase (Porphozym) | 1 |
| Heme arginate | 1 |
Showing 5 of 6 drugs.
Interventions as named in the registry (3)
Shown in this order: most trials first.
| Drug | Trials |
|---|---|
| Hemin for injection | 1 |
| Anhydrous glucose | 1 |
| 5-probe cocktail | 1 |
These are the names sponsors entered on ClinicalTrials.gov that we have not matched to a single molecule. Some are drugs under a brand or code name; others are procedures, doses or arm labels.
Which serious adverse events do Porphyria, Acute Intermittent trials report? (10)
Shown in this order: most subjects affected first. Subjects affected summed across trials, a cross-trial signal, not a rate.
| Adverse event | Subjects affected | Trials |
|---|---|---|
| Pulmonary embolism | 8 | 1 |
| Chronic kidney disease | 6 | 1 |
| Device related infection | 5 | 1 |
| Pyrexia | 5 | 2 |
| Abdominal pain | 4 | 2 |
Showing 5 of 10 adverse events.
Which publications cover Porphyria, Acute Intermittent? (12)
Shown in this order: major-topic papers first, then newest, then most linked to trials.
| Publication | Journal | Year | Major topic |
|---|---|---|---|
| Long-term follow-up of givosiran treatment in patients with acute intermittent porphyria from a phase 1/2, 48-month open-label extension study. | Orphanet journal of rare diseases | 2024 | Yes |
| Cardiovascular abnormalities in patients with acute intermittent porphyria: the AIPRACUS study. | Polish archives of internal medicine | 2024 | Yes |
| Disease burden in patients with acute hepatic porphyria: experience from the phase 3 ENVISION study. | Orphanet journal of rare diseases | 2022 | Yes |
| Phase 3 Trial of RNAi Therapeutic Givosiran for Acute Intermittent Porphyria. | The New England journal of medicine | 2020 | Yes |
| Brain ventricular enlargement in human and murine acute intermittent porphyria. | Human molecular genetics | 2020 | Yes |
Showing 5 of 12 publications.
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.
| Orphanet disease | Type | Class | Geography | Jurisdiction | Mapping relation |
|---|---|---|---|---|---|
| Acute intermittent porphyria | Point prevalence | 1-9 / 1 000 000 | United Kingdom | GB | Exact |
| Acute intermittent porphyria | Annual incidence | <1 / 1 000 000 | United Kingdom | GB | Exact |
| Acute intermittent porphyria | Annual incidence | <1 / 1 000 000 | United Kingdom | GB | Exact |
| Acute intermittent porphyria | Annual incidence | <1 / 1 000 000 | United Kingdom | GB | Unspecified |
| Acute intermittent porphyria | Point prevalence | 1-9 / 1 000 000 | United Kingdom | GB | Unspecified |
Showing 5 of 66 prevalence observations.
Rare-disease identity (2)
Shown in this order: exact, broader, narrower, other.
| Orphanet disease | ORPHA code | Relation | Source |
|---|---|---|---|
| Acute intermittent porphyria | 79276 | Exact | Orphanet Rare Disease Ontology (ORDO) |
| Acute intermittent porphyria | 79276 | 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.