Condition (MeSH)
Protoporphyria, Erythropoietic
ClinicalTrials.gov lists 29 trials for Protoporphyria, Erythropoietic or a narrower condition beneath it. Clinuvel Pharmaceuticals Limited has the most among industry and government lead sponsors, with 5.
Search trials, drugs, companies and investigators, and follow each one to the others. Individual plan, $249/seat/mo. Card required.
Trials per phase
29 of the 29 trials are tagged with Protoporphyria, Erythropoietic itself; the rest are tagged with a narrower condition beneath it.
When did Protoporphyria, Erythropoietic trials start? (15)
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 1/2
- Phase 2
- Phase 2/3
- Phase 3
- Not applicable
- Observational
Top of scale: 3
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 | 1 |
| 2023 | 3 |
| 2022 | 3 |
| 2018 | 1 |
| 2017 | 1 |
| 2016 | 1 |
| 2012 | 3 |
| 2011 | 3 |
Showing 10 of 15 earlier years.
No forecast is made for this condition: it averages 2.4 trial starts a year over the five years to September, under the 5 a year the method needs.
Recruitment duration, a model prediction: 2 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 Protoporphyria, Erythropoietic trials in? (8)
Shown in this order: phase order.
Show the trials by phase as a table
| Phase | Trials |
|---|---|
| Phase 1 | 1 |
| Phase 1/2 | 1 |
| Phase 2 | 5 |
| Phase 2/3 | 3 |
| Phase 3 | 7 |
| Not applicable | 5 |
| Observational | 6 |
| Expanded access | 1 |
How many Protoporphyria, Erythropoietic trials are recruiting? (8)
Shown in this order: the order a trial moves through, from not yet recruiting to unknown.
| Status | Trials |
|---|---|
| Recruiting | 3 |
| Enrolling by invitation | 1 |
| Active, not recruiting | 1 |
| Available | 1 |
| Completed | 19 |
Showing 5 of 8 statuses.
Status as registered on ClinicalTrials.gov. Each trial is counted once.
Which lead sponsors run the most Protoporphyria, Erythropoietic trials? (6)
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 |
|---|---|
| Clinuvel Pharmaceuticals Limited | 5 |
| Disc Medicine, Inc | 4 |
| Portal Therapeutics, Inc. | 3 |
| Tanabe Pharma America, Inc. | 2 |
| Clinuvel Europe Limited | 1 |
Showing 5 of 6 sponsors.
Which drugs are being tested for Protoporphyria, Erythropoietic? (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 |
|---|---|
| RO4917838 | 5 |
| Afamelanotide | 5 |
| cysteine hydrochloride | 2 |
| Dersimelagon | 1 |
| Cimetidine | 1 |
Showing 5 of 6 drugs.
Interventions as named in the registry (7)
Shown in this order: most trials first.
| Drug | Trials |
|---|---|
| PORT-77 | 2 |
| Oral Iron | 1 |
| Isoniazida | 1 |
| MT-7117 Low Dose | 1 |
| Exercise | 1 |
Showing 5 of 7 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 Protoporphyria, Erythropoietic 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 |
|---|---|---|
| Diverticulitis | 2 | 2 |
| Abdominal pain | 1 | 1 |
| Abnormal liver enzymes | 1 | 1 |
| Anaphylactic reaction | 1 | 1 |
| CRAMPY ABDOMINAL PAIN | 1 | 1 |
Showing 5 of 10 adverse events.
Which publications cover Protoporphyria, Erythropoietic? (4)
Shown in this order: major-topic papers first, then newest, then most linked to trials.
| Publication | Journal | Year | Major topic |
|---|---|---|---|
| Liver involvement in a large cohort of patients with erythropoietic protoporphyria or X-linked protoporphyria. | Hepatology communications | 2025 | Yes |
| Dersimelagon in Erythropoietic Protoporphyrias. | The New England journal of medicine | 2023 | Yes |
| Clinical, Biochemical, and Genetic Characterization of North American Patients With Erythropoietic Protoporphyria and X-linked Protoporphyria. | JAMA dermatology | 2017 | Yes |
| Afamelanotide for Erythropoietic Protoporphyria. | The New England journal of medicine | 2015 | Yes |
Prevalence (44)
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 |
|---|---|---|---|---|---|
| Autosomal erythropoietic protoporphyria | Point prevalence | 1-9 / 100 000 | United Kingdom | GB | Exact |
| Autosomal erythropoietic protoporphyria | Annual incidence | <1 / 1 000 000 | United Kingdom | GB | Exact |
| Autosomal erythropoietic protoporphyria | Point prevalence | 1-9 / 100 000 | United Kingdom | GB | Exact |
| Autosomal erythropoietic protoporphyria | Annual incidence | <1 / 1 000 000 | United Kingdom | GB | Exact |
| Autosomal erythropoietic protoporphyria | Annual incidence | <1 / 1 000 000 | Sweden | Not stated | Exact |
Showing 5 of 44 prevalence observations.
Rare-disease identity (2)
Shown in this order: exact, broader, narrower, other.
| Orphanet disease | ORPHA code | Relation | Source |
|---|---|---|---|
| Autosomal erythropoietic protoporphyria | 79278 | Exact | Orphanet Rare Disease Ontology (ORDO) |
| Autosomal erythropoietic protoporphyria | 79278 | 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.