15 completed, 2 unknown, 1 enrolling by invitation. 7 Phase 3, 6 Phase 2, 4 Not applicable, 1 Phase 1.
| Phase | Trials | Status |
|---|---|---|
| Phase 3 | 7 | Completed 6, Enrolling by invitation 1 |
| Phase 2 | 6 | Completed 5, Enrolling by invitation 1 |
| Not applicable | 4 | Completed 2, Unknown 2 |
| Phase 1 | 1 | Completed 1 |
Follow Erythropoietic Protoporphyria: new trials by email Literature Review of Erythropoietic Protoporphyria trials Research Report on Erythropoietic Protoporphyria
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 |
|---|---|---|---|---|---|---|
| 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 |
| 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 |
| 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 |
| 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 |
| Sorbent Therapy of the Cutaneous Porphyrias Colestipol - Erythropoietic Protoporphyria | Phase 2 / Phase 3 | Completed | Brigham and Women's Hospital | 4 | 2011-05-01 | 2017-04-25 |
| Phase II Confirmatory Study in Erythropoietic Protoporphyria (EPP) Afamelanotide, Placebo - Erythropoietic Protoporphyria | Phase 2 | Completed | Clinuvel Pharmaceuticals Limited | 77 | 2010-04-01 | 2019-10-28 |
| Phase III Confirmatory Study in Erythropoietic Protoporphyria (EPP) Afamelanotide, Placebo - Erythropoietic Protoporphyria | Phase 3 | Completed | Clinuvel Pharmaceuticals Limited | 74 | 2009-09-01 | 2021-10-12 |
| Multicentre Phase III Erythropoietic Protoporphyria Study Afamelanotide, Placebo - Erythropoietic Protoporphyria | Phase 3 | Completed | Clinuvel Pharmaceuticals Limited | 100 | 2007-05-01 | 2019-10-10 |
| Does Exercise and Heat Increase the Lightsensibility in Patients With Erythropoietic Protoporphyria exercise, heat, red light illumination - Erythropoietic Protoporphyria | Not applicable | Unknown | Bispebjerg Hospital | 2003-01-01 | 2006-10-11 | |
| Study of Cysteine Hydrochloride for Erythropoietic Protoporphyria cysteine hydrochloride - Erythropoietic Protoporphyria | Not applicable | Completed | FDA Office of Orphan Products Development | 20 | 1996-10-01 | 2015-03-25 |
| Phase III Study of L-Cysteine in Patients With Erythropoietic Protoporphyria cysteine hydrochloride - Erythropoietic Protoporphyria | Phase 3 | Completed | Brigham and Women's Hospital | 50 | 1996-05-01 | 2015-03-25 |
Protoporphyria, Erythropoietic (18) · Porphyrias (6) · Protoporphyria, Erythropoietic, X-Linked Dominant (3) · Metabolism, Inborn Errors (2) · Rare Diseases (2) · Anemia, Iron-Deficiency (1) · Photosensitivity Disorders (1)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (18) · Digestive System Diseases (18) · Genetic Diseases, Inborn (18) · Liver Diseases (18) · Metabolic Diseases (18) · Nutritional and Metabolic Diseases (18) · Porphyrias, Hepatic (18) · Skin Diseases (18) · Skin Diseases, Genetic (18) · Skin and Connective Tissue Diseases (18) · Porphyrias (12) · 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 (2) · Cysteine (2) · Cimetidine (1) · Colestipol (1) · Dihydroxyacetone (1) · Drug Implants (1) · Exercise (1) · Hot Temperature (1) · Iron (1)
Clinuvel Pharmaceuticals Limited (5) · Bispebjerg Hospital (4) · Brigham and Women's Hospital (2) · Disc Medicine, Inc (2) · Icahn School of Medicine at Mount Sinai (2) · Amy K. Dickey, M.D. (1) · Clinuvel Europe Limited (1) · FDA Office of Orphan Products Development (1)
United States (8) · Denmark (4) · Netherlands (3) · Belgium (2) · France (2) · Germany (2) · Ireland (2) · United Kingdom (2) · Australia (1) · Finland (1) · Italy (1) · Spain (1) · Sweden (1)
Robert Desnick, MD (2) · Will Savage, MD PhD (2) · Alex Anstey, MBBS, FRCP (1) · Amy K Dickey, MD (1) · Eric JG Sijbrands, MD, PhD (1) · Gillian M Murphy, MD PhD (1) · Hans C Wulf, MD, Sci (1) · Jean-Charles Deybach, MD. PhD (1) · Jorge Frank, MD, PhD (1) · Karl Anderson, MD (1) · Lesley E Rhodes, MD PhD (1) · Manisha Balwani, MD (1) · Manisha Balwani, MD, MS (1) · Micheline M Mathews-Roth (1)
PMID 26132941, PMID 36406817, PMID 9521433
U54DK083909 (2)