20 completed, 3 recruiting, 2 unknown, 1 available. 10 Phase 3, 9 Phase 2, 5 Not applicable, 2 Phase 1.
Follow Protoporphyria, Erythropoietic: new trials by email Literature Review of Protoporphyria, Erythropoietic trials Research Report on Protoporphyria, Erythropoietic
Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.
| Trial | Phase | Status | Sponsor | Enrollment | Start | Updated |
|---|---|---|---|---|---|---|
| A Study to Investigate PORT-77 Oral Dosing Compared With Placebo in Participants With Erythropoietic Protoporphyria (EPP) or X-linked Protoporphyria (XLP) PORT-77, Placebo - Erythropoietic Protoporphyria (EPP), X-Linked Protoporphyria (XLP) | Phase 2 / Phase 3 | Recruiting | Portal Therapeutics, Inc. | 225 | 2026-09-01 | 2026-09-15 |
| Observational Study of Adults and Adolescents With Erythropoietic Protoporphyria (EPP) and X-linked Porphyria (XLP) Erythropoietic Protoporphyria (EPP), X-Linked Porphyria (XLP) | Recruiting | Portal Therapeutics, Inc. | 50 | 2026-04-30 | 2026-05-05 | |
| Effect of Bitopertin on the Liver and on Levels of Protoporphyrin IX in Bile, Blood, Liver, and Stool in Patients With Erythropoietic Protoporphyria/X-linked Protoporphyria and Increased Liver Stiffness and/or Liver Enzymes at Baseline bitopertin - Protoporphyria | Phase 1 | Withdrawn | Wake Forest University Health Sciences | 2026-01-01 | 2026-01-29 | |
| Study of Bitopertin in Participants With EPP or XLP (APOLLO) Placebo, DISC-1459 - Erythropoietic Protoporphyria (EPP), X-Linked Protoporphyria (XLP) | Phase 3 | Completed | Disc Medicine, Inc | 183 | 2025-04-04 | 2026-10-09 |
| GATEWAY: A Phase 2a Study of PORT-77 in Adults With Erythropoietic Protoporphyria PORT-77, Placebo - Erythropoietic Protoporphyria (EPP) | Phase 2 | Completed | Portal Therapeutics, Inc. | 28 | 2025-04-04 | 2026-07-29 |
| Pharmacokinetics of Afamelanotide in Erythropoietic Protoporphyria Patients Afamelanotide 16mg implant - Erythropoietic Protoporphyria | Phase 1 / Phase 2 | Completed | Clinuvel Europe Limited | 28 | 2024-03-07 | 2026-07-27 |
| INcreased Sun Exposure Without Pain In Research Participants With EPP or XLP Dersimelagon, Placebo - Erythropoietic Protoporphyria (EPP), X-Linked Protoporphyria (XLP) | Phase 3 | Completed | Tanabe Pharma America, Inc. | 165 | 2023-12-11 | 2026-06-04 |
| HELIOS: Open-Label, Long-Term Extension Study to Investigate the Safety, Tolerability, and Efficacy of DISC-1459 (Bitopertin) in Participants With EPP or XLP DISC-1459 - Erythropoietic Protoporphyria | Phase 2 / Phase 3 | Enrolling by invitation | Disc Medicine, Inc | 230 | 2023-08-31 | 2026-10-09 |
| Protection Against Visible Light by Dihydroxyacetone in Erythropoietic Protoporphyria Dihydroxyacetone, Placebo - Erythropoietic Protoporphyria | Not applicable | Unknown | Bispebjerg Hospital | 6 | 2023-02-23 | 2023-03-23 |
| Study of Bitopertin to Evaluate the Safety, Tolerability, Efficacy, and PPIX Concentrations in Participants With EPP DISC-1459, Placebo - Erythropoietic Protoporphyria | Phase 2 | Completed | Disc Medicine, Inc | 75 | 2022-10-31 | 2026-01-08 |
| Iron Therapy in Erythropoietic Protoporphyria Erythropoietic Protoporphyria, Iron-deficiency | Completed | Bispebjerg Hospital | 10 | 2022-10-17 | 2023-02-23 | |
| Effect of Oral Cimetidine in the Protoporphyrias Cimetidine, Placebo - Erythropoietic Protoporphyria, X-linked Protoporphyria | Phase 2 | Completed | Amy K. Dickey, M.D. | 26 | 2022-06-14 | 2026-09-04 |
| Study to Evaluate Efficacy, Safety, and Tolerability of MT-7117 in Subjects With Erythropoietic Protoporphyria MT-7117 low dose, MT-7117 high dose, Placebo - Erythropoietic Protoporphyria (EPP) | Phase 2 | Completed | Tanabe Pharma America, Inc. | 102 | 2018-07-05 | 2025-12-31 |
| Light Exposure Patterns and Symptoms Among Patients With Erythropoietic Protoporphyria Erythropoietic Protoporphyria | Completed | Bispebjerg Hospital | 14 | 2017-04-01 | 2018-10-02 | |
| Oral Iron for Erythropoietic Protoporphyrias Oral Iron - Erythropoietic Protoporphyria, EPP | Not applicable | Completed | Icahn School of Medicine at Mount Sinai | 16 | 2016-12-01 | 2020-09-11 |
| Erythropoietic Protoporphyrias: Studies of the Natural History, Genotype-Phenotype Correlations, and Psychosocial Impact Erythropoietic Protoporphyria, EPP | Completed | Icahn School of Medicine at Mount Sinai | 150 | 2012-07-01 | 2020-04-17 | |
| Phase III Confirmatory Study in Erythropoietic Protoporphyria Afamelanotide, Placebo - Erythropoietic Protoporphyria | Phase 3 | Completed | Clinuvel Pharmaceuticals Limited | 93 | 2012-05-01 | 2019-09-19 |
| Effect of Isoniazid on Protoporphyrin Levels in Erythropoietic Protoporphyria Isoniazid - Erythropoietic Protoporphyria (EPP), X Linked Erythropoietic Protoporphyria | Not applicable | Terminated | University of Utah | 11 | 2012-03-01 | 2017-01-16 |
| Mitoferrin-1 Expression in Erythropoietic Protoporphyria (Porphyria Rare Disease Clinical Research Consortium (RDCRC)) Erythropoietic Protoporphyria (EPP) | Completed | University of Alabama at Birmingham | 150 | 2011-11-01 | 2021-06-24 | |
| A Safety Extension Study in Patients With Erythropoietic Protoporphyria (EPP) Afamelanotide - Erythropoietic Protoporphyria | Phase 3 | Completed | Clinuvel Pharmaceuticals Limited | 16 | 2011-07-11 | 2020-11-19 |
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Porphyrias (11) · Protoporphyria, Erythropoietic, X-Linked Dominant (8) · Metabolism, Inborn Errors (2) · Rare Diseases (2) · Anemia, Iron-Deficiency (1) · Coproporphyria (1) · Photosensitivity Disorders (1) · Porphyria, Acute Intermittent (1)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (29) · Digestive System Diseases (29) · Genetic Diseases, Inborn (29) · Liver Diseases (29) · Metabolic Diseases (29) · Nutritional and Metabolic Diseases (29) · Porphyrias, Hepatic (29) · Skin Diseases (29) · Skin Diseases, Genetic (29) · Skin and Connective Tissue Diseases (29) · Porphyrias (18) · Disease Attributes (2) · Pathologic Processes (2) · Pathological Conditions, Signs and Symptoms (2) · Anemia (1)
afamelanotide (6) · (4-(3-fluoro-5-trifluoromethylpyridin-2-yl)piperazin-1-yl)(5-methanesulfonyl-2-(2,2,2-trifluoro-1-methylethoxy)phenyl)methanone (5) · Cysteine (2) · Cimetidine (1) · Colestipol (1) · Dihydroxyacetone (1) · Drug Implants (1) · Exercise (1) · Hot Temperature (1) · Iron (1) · Isoniazid (1)
Clinuvel Pharmaceuticals Limited (5) · Bispebjerg Hospital (4) · Disc Medicine, Inc (4) · Portal Therapeutics, Inc. (3) · Brigham and Women's Hospital (2) · Icahn School of Medicine at Mount Sinai (2) · Tanabe Pharma America, Inc. (2) · Amy K. Dickey, M.D. (1) · Clinuvel Europe Limited (1) · FDA Office of Orphan Products Development (1) · The American Porphyrias Expert Collaborative (1) · University of Alabama at Birmingham (1) · University of Utah (1) · Wake Forest University Health Sciences (1)
United States (18) · France (5) · Netherlands (5) · United Kingdom (5) · Belgium (4) · Denmark (4) · Italy (4) · Spain (4) · Australia (3) · Germany (3) · Ireland (3) · Canada (2) · Sweden (2) · Bulgaria (1) · Czechia (1)
Robert Desnick, MD (2) · Will Savage, MD PhD (2) · Will Savage, MD, PhD (2) · Alex Anstey, MBBS, FRCP (1) · Amy K Dickey, MD (1) · Brendan McGuire, MD (1) · Eric JG Sijbrands, MD, PhD (1) · Gillian M Murphy, MD PhD (1) · Hans C Wulf, MD, Sci (1) · Herbert Bonkovsky, MD (1) · Hetanshi Naik, PhD (1) · Jean-Charles Deybach, MD. PhD (1)
PMID 26132941, PMID 25016127, PMID 28614581, PMID 32681675, PMID 36406817, PMID 37043653, PMID 39887900, PMID 39969427, PMID 9521433
U54DK083909 (4), 2023 (1)