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Erythropoietic Protoporphyria

1 published article · Updated continuously

Clinical Trial Landscape

Clinical Trials for Erythropoietic Protoporphyria

10 trials tracked for Erythropoietic Protoporphyria: 5 in phase 3 or 4 and 3 with published results. The most-cited published study has 280 citations.

10Trials tracked
5Phase 3 & 4
0Recruiting
3With published results
Phase distribution
Phase 3 5 Phase 2 3 Other / NA 2
  1. Phase 3 Phase III Confirmatory Study in Erythropoietic Protoporphyria (EPP) Completed · 280 cited
  2. Phase 3 Phase III Confirmatory Study in Erythropoietic Protoporphyria Completed · 279 cited
  3. Phase 3 Multicentre Phase III Erythropoietic Protoporphyria Study Completed
  4. Phase 3 Sorbent Therapy of the Cutaneous Porphyrias Completed
  5. Phase 3 A Safety Extension Study in Patients With Erythropoietic Protoporphyria (EPP) Completed
  6. Phase 2 Pharmacokinetics of Afamelanotide in Erythropoietic Protoporphyria Patients Completed
Show 4 more trials
  1. Phase 2 Study of Bitopertin to Evaluate the Safety, Tolerability, Efficacy, and PPIX Concentrations in Participants With EPP Completed
  2. Phase 2 Phase II Confirmatory Study in Erythropoietic Protoporphyria (EPP) Completed
  3. N/A Erythropoietic Protoporphyrias: Studies of the Natural History, Genotype-Phenotype Correlations, and Psychosocial Impact Completed
  4. N/A Oral Iron for Erythropoietic Protoporphyrias Completed

Showing the 10 most-cited and recently-updated of 10 trials. Browse the full registry →

Trial data sourced from ClinicalTrials.gov. Counts describe the research landscape and are not a treatment recommendation. Informational only — not medical advice.

What the trials found Updated — new results For clinicians

Erythropoietic Protoporphyria: what the trials found

Afamelanotide has been evaluated in multiple Phase 3 trials for the management of phototoxicity in Erythropoietic Protoporphyria. Evidence indicates that Afamelanotide is associated with significant increases in the duration of direct sunlight exposure on pain-free days (10:00 to 15:00) compared to placebo [2, 7]. Furthermore, some studies reported substantial gains in sun tolerance during the 10:00 to 18:00 window 3.

Clinical data from Afamelanotide trials also demonstrate a reduction in the cumulative number of days with phototoxic reactions 5. Patients treated with Afamelanotide showed significant improvements in Quality of Life scores (SF36 Physical Component) compared to those on placebo [3, 5]. Additionally, Colestipol has been evaluated for its effect on photosensitivity, showing a recorded sun tolerance of 65.8 minutes 6.

Recent results — preliminary, needs further review

  • DISC-1459 showed significant reductions in whole blood metal-free PPIX levels (ranging from -21.48% to -41.74%) and increased the total hours of pain-free sunlight exposure between 10:00 and 18:00 1.

For the clinician treating this condition

  • Afamelanotide is associated with increased duration of pain-free sun exposure and improved physical quality of life scores in patients with Erythropoietic Protoporphyria.
  • DISC-1459 has demonstrated a reduction in metal-free PPIX levels and an increase in hours of pain-free sunlight exposure.

AI synthesis of 4 cited trials, updated Jun 23, 2026. Informational only — not medical advice; trial data sourced from ClinicalTrials.gov. How we use AI.

HCP Mode — summaries include clinical detail, trial data, and statistical outcomes.
Patient Mode — summaries use plain language, avoiding clinical jargon.