11 completed, 1 enrolling by invitation, 1 recruiting. 13 Phase 3, 4 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Phase 3 | 13 | Completed 11, Enrolling by invitation 1, Recruiting 1 |
| Phase 2 | 4 | Completed 2, Enrolling by invitation 1, Recruiting 1 |
Follow Porphyrias: new trials by email Literature Review of Porphyrias trials Research Report on Porphyrias
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 |
| 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 |
| 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 |
| ENVISION: A Study to Evaluate the Efficacy and Safety of Givosiran (ALN-AS1) in Patients With Acute Hepatic Porphyrias (AHP) Givosiran, Placebo - Acute Hepatic Porphyria, Acute Intermittent Porphyria | Phase 3 | Completed | Alnylam Pharmaceuticals | 94 | 2017-11-16 | 2024-04-22 |
| 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 III Confirmatory Study in Erythropoietic Protoporphyria (EPP) Afamelanotide, Placebo - Erythropoietic Protoporphyria | Phase 3 | Completed | Clinuvel Pharmaceuticals Limited | 74 | 2009-09-01 | 2021-10-12 |
| Pilot Trial of Deferasirox in the Treatment of Porphyria Cutanea Tarda Deferasirox - Porphyria Cutanea Tarda | Phase 3 | Completed | University of Texas Southwestern Medical Center | 10 | 2008-01-01 | 2014-02-10 |
| Multicentre Phase III Erythropoietic Protoporphyria Study Afamelanotide, Placebo - Erythropoietic Protoporphyria | Phase 3 | Completed | Clinuvel Pharmaceuticals Limited | 100 | 2007-05-01 | 2019-10-10 |
| Porphozym in the Treatment of Acute Attacks in AIP recombinant human porphobilinogen deaminase (Porphozym) - Acute Intermittent Porphyria | Phase 2 / Phase 3 | Completed | Zymenex A/S | 36 | 2003-06-11 | 2018-03-09 |
| 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 (10) · Protoporphyria, Erythropoietic, X-Linked Dominant (3) · Porphyria, Acute Intermittent (2) · Coproporphyria, Hereditary (1) · Metabolism, Inborn Errors (1) · Porphyria Cutanea Tarda (1) · Porphyria, Variegate (1) · Rare Diseases (1)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (13) · Digestive System Diseases (13) · Genetic Diseases, Inborn (13) · Liver Diseases (13) · Metabolic Diseases (13) · Nutritional and Metabolic Diseases (13) · Porphyrias, Hepatic (13) · Skin Diseases (13) · Skin Diseases, Genetic (13) · Skin and Connective Tissue Diseases (13) · Disease Attributes (1) · Pathologic Processes (1) · Pathological Conditions, Signs and Symptoms (1)
afamelanotide (4) · (4-(3-fluoro-5-trifluoromethylpyridin-2-yl)piperazin-1-yl)(5-methanesulfonyl-2-(2,2,2-trifluoro-1-methylethoxy)phenyl)methanone (2) · Colestipol (1) · Cysteine (1) · Deferasirox (1) · givosiran (1)
Clinuvel Pharmaceuticals Limited (4) · Brigham and Women's Hospital (2) · Disc Medicine, Inc (2) · Alnylam Pharmaceuticals (1) · Portal Therapeutics, Inc. (1) · Tanabe Pharma America, Inc. (1) · University of Texas Southwestern Medical Center (1) · Zymenex A/S (1)
United States (9) · France (5) · Netherlands (5) · United Kingdom (5) · Australia (4) · Germany (4) · Italy (4) · Spain (4) · Ireland (3) · Sweden (3) · Belgium (2) · Bulgaria (2) · Canada (2) · Finland (2) · Japan (2)
Alex Anstey, MBBS, FRCP (1) · Amit Pandya, M.D. (1) · Christer Andersson, MD (1) · Eric JG Sijbrands, MD, PhD (1) · Gillian M Murphy, MD PhD (1) · Jean-Charles Deybach, MD. PhD (1) · Jorge Frank, MD, PhD (1) · Lesley E Rhodes, MD PhD (1) · Micheline Mary Mathews-Roth (1) · Pilar Bilbao (1) · Raili Kauppinen, MD, PhD (1)
PMID 26132941, PMID 22911183, PMID 32521132, PMID 36028858, PMID 37479139, PMID 38044204, PMID 41388232, PMID 9521433
2023 (1)