Condition (MeSH)
Porphyrias, Hepatic
ClinicalTrials.gov lists 62 trials for Porphyrias, Hepatic or a narrower condition beneath it. Alnylam Pharmaceuticals has the most among industry and government lead sponsors, with 8.
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Trials per phase
2 of the 62 trials are tagged with Porphyrias, Hepatic itself; the rest are tagged with a narrower condition beneath it.
When did Porphyrias, Hepatic trials start? (24)
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: 6
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 |
|---|---|
| 2026 (partial year) | 3 |
| 2025 | 2 |
| 2024 | 2 |
| 2023 | 4 |
| 2022 | 5 |
| 2021 | 3 |
| 2019 | 2 |
| 2018 | 3 |
| 2017 | 3 |
| 2016 | 3 |
Showing 10 of 24 earlier years.
No forecast is made for this condition: it averages 3.2 trial starts a year over the five years to September, under the 5 a year the method needs.
Recruitment duration, a model prediction: 3 trials 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 Porphyrias, Hepatic trials in? (8)
Shown in this order: phase order.
Show the trials by phase as a table
| Phase | Trials |
|---|---|
| Phase 1 | 5 |
| Phase 1/2 | 3 |
| Phase 2 | 11 |
| Phase 2/3 | 4 |
| Phase 3 | 9 |
| Not applicable | 7 |
| Observational | 21 |
| Expanded access | 2 |
How many Porphyrias, Hepatic trials are recruiting? (9)
Shown in this order: the order a trial moves through, from not yet recruiting to unknown.
| Status | Trials |
|---|---|
| Recruiting | 6 |
| Enrolling by invitation | 1 |
| Active, not recruiting | 3 |
| Available | 1 |
| Completed | 40 |
Showing 5 of 9 statuses.
Status as registered on ClinicalTrials.gov. Each trial is counted once.
Which lead sponsors run the most Porphyrias, Hepatic trials? (7)
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 | 8 |
| Clinuvel Pharmaceuticals Limited | 6 |
| Disc Medicine, Inc | 4 |
| Portal Therapeutics, Inc. | 3 |
| Digna Biotech S.L. | 2 |
Showing 5 of 7 sponsors.
Which drugs are being tested for Porphyrias, Hepatic? (5)
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 |
|---|---|
| Afamelanotide | 6 |
| RO4917838 | 5 |
| Givosiran | 5 |
| Hemin | 2 |
| cysteine hydrochloride | 2 |
Interventions as named in the registry (10)
Shown in this order: most trials first.
| Drug | Trials |
|---|---|
| PORT-77 | 2 |
| Exercise | 2 |
| Deferasirox FCT | 2 |
| Oral Iron | 1 |
| Isoniazida | 1 |
Showing 5 of 10 interventions.
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 Porphyrias, Hepatic 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 | 9 | 2 |
| Chronic kidney disease | 6 | 1 |
| Abdominal pain | 5 | 3 |
| Device related infection | 5 | 1 |
| Pyrexia | 5 | 2 |
Showing 5 of 10 adverse events.
Which publications cover Porphyrias, Hepatic? (4)
Shown in this order: major-topic papers first, then newest, then most linked to trials.
| Publication | Journal | Year | Major topic |
|---|---|---|---|
| A randomized, placebo-controlled study of givosiran in patients with acute hepatic porphyrias (ENVISION): Final (36-month) analysis of the Taiwan Cohort. | Journal of the Formosan Medical Association = Taiwan yi zhi | 2024 | Yes |
| Disease burden in patients with acute hepatic porphyria: experience from the phase 3 ENVISION study. | Orphanet journal of rare diseases | 2022 | Yes |
| Hepatocellular Carcinoma in Acute Hepatic Porphyrias: Results from the Longitudinal Study of the U.S. Porphyrias Consortium. | Hepatology (Baltimore, Md.) | 2021 | Yes |
| Liver transplantation for porphyria: who, when, and how? | Liver transplantation : official publication of the American Association for the Study of Liver Diseases and the International Liver Transplantation Society | 2007 | Yes |
Sub-conditions in MeSH (5)
MeSH descendants one level down with at least one trial, most trials first.
Not on this page
Not measured: delivery record.
Checked, with nothing recorded for this condition: prevalence and rare-disease identity.