15 completed, 2 recruiting, 1 enrolling by invitation, 1 terminated. 20 Phase 2, 4 Phase 1, 4 Phase 3.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 20 | Completed 15, Recruiting 2, Enrolling by invitation 1, Terminated 1, Unknown 1 |
| Phase 1 | 4 | Completed 3, Recruiting 1 |
| Phase 3 | 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 |
| A Study to Investigate the Improvement of Photosensitivity in Terms of Skin Lesions Associated With CEP Following Administration of Oral ATL-001 (Ciclopirox Oral Solution) in Participants Aged >18 Years of Age With CEP ATL-001 (Ciclopirox oral solution) - Congenital Erythropoietic Porphyria (CEP) | Phase 1 / Phase 2 | Recruiting | Atlas Molecular Pharma | 6 | 2026-01-29 | 2026-03-24 |
| 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 |
| 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 |
| Study to Evaluate the Safety and Efficacy of Afamelanotide in Patients With Variegate Porphyria (VP) Afamelanotide 16 MG - Variegate Porphyria | Phase 2 | Completed | Clinuvel Pharmaceuticals Limited | 6 | 2023-03-28 | 2025-04-27 |
| 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 |
| 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 |
| Harvoni Treatment Porphyria Cutanea Tarda Harvoni - Porphyria Cutanea Tarda, Hepatitis C | Phase 2 | Completed | Wake Forest University Health Sciences | 23 | 2017-09-06 | 2023-07-03 |
| A Study to Evaluate Long-term Safety and Clinical Activity of Givosiran (ALN-AS1) in Patient With Acute Intermittent Porphyria (AIP) Givosiran - Acute Intermittent Porphyria | Phase 1 / Phase 2 | Completed | Alnylam Pharmaceuticals | 16 | 2016-10-01 | 2024-03-12 |
| Panhematin for Prevention of Acute Attacks of Porphyria Hemin for injection, Placebo - Acute Intermittent Porphyria, Hereditary Coproporphyria | Phase 2 | Terminated | The University of Texas Medical Branch, Galveston | 13 | 2015-10-30 | 2026-01-30 |
| Controlled Trial of Panhematin in Treatment of Acute Attacks of Porphyria Panhematin, Glucose - Acute Porphyrias | Phase 2 | Completed | The University of Texas Medical Branch, Galveston | 20 | 2014-04-28 | 2025-04-11 |
| 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 |
| Safety and Efficacy of Oral Deferasirox in Patients With Porphyria Cutanea Tarda Exjade - Porphyria Cutanea Tarda | Phase 2 | Unknown | Assistance Publique - Hôpitaux de Paris | 45 | 2011-01-01 | 2011-01-27 |
| 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 |
| Hydroxychloroquine and Phlebotomy for Treating Porphyria Cutanea Tarda Hydroxychloroquine, Phlebotomy - Porphyria Cutanea Tarda | Phase 2 | Completed | The University of Texas Medical Branch, Galveston | 48 | 2006-03-21 | 2023-02-17 |
| 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 |
| Studies in Porphyria III: Heme and Tin Mesoporphyrin in Acute Porphyrias heme arginate, tin mesoporphyrin - Porphyria | Phase 2 | Completed | National Center for Research Resources (NCRR) | 32 | 1997-09-01 | 2005-06-24 |
| Phase I/II Study of Heme Arginate and Tin Mesoporphyrin for Acute Porphyria heme arginate, tin mesoporphyrin - Porphyria | Phase 1 / Phase 2 | Completed | National Center for Research Resources (NCRR) | 59 | 1993-07-01 | 2005-06-24 |
Protoporphyria, Erythropoietic (9) · Porphyria, Acute Intermittent (5) · Porphyria Cutanea Tarda (3) · Rare Diseases (3) · Metabolism, Inborn Errors (2) · Porphyria, Variegate (2) · Protoporphyria, Erythropoietic, X-Linked Dominant (2) · Coproporphyria, Hereditary (1) · Hepatitis C (1) · Photosensitivity Disorders (1) · Porphyria, Erythropoietic (1)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (20) · Genetic Diseases, Inborn (20) · Metabolic Diseases (20) · Nutritional and Metabolic Diseases (20) · Skin Diseases (19) · Skin Diseases, Genetic (19) · Skin and Connective Tissue Diseases (19) · Digestive System Diseases (18) · Liver Diseases (18) · Porphyrias, Hepatic (18) · Disease Attributes (3) · Pathologic Processes (3) · Pathological Conditions, Signs and Symptoms (3) · Blood-Borne Infections (1)
afamelanotide (3) · (4-(3-fluoro-5-trifluoromethylpyridin-2-yl)piperazin-1-yl)(5-methanesulfonyl-2-(2,2,2-trifluoro-1-methylethoxy)phenyl)methanone (2) · Hemin (2) · heme arginate (2) · tin mesoporphyrin (2) · Ciclopirox (1) · Cimetidine (1) · Colestipol (1) · Deferasirox (1) · Drug Implants (1) · Glucose (1) · Hydroxychloroquine (1) · Injections (1) · Phlebotomy (1) · Sofosbuvir (1)
The University of Texas Medical Branch, Galveston (3) · Clinuvel Pharmaceuticals Limited (2) · Disc Medicine, Inc (2) · National Center for Research Resources (NCRR) (2) · Portal Therapeutics, Inc. (2) · Alnylam Pharmaceuticals (1) · Amy K. Dickey, M.D. (1) · Assistance Publique - Hôpitaux de Paris (1) · Atlas Molecular Pharma (1) · Brigham and Women's Hospital (1) · Clinuvel Europe Limited (1) · Tanabe Pharma America, Inc. (1) · Wake Forest University Health Sciences (1) · Zymenex A/S (1)
United States (16) · Netherlands (3) · Belgium (2) · France (2) · Sweden (2) · United Kingdom (2) · Australia (1) · Germany (1) · Ireland (1) · Italy (1) · Spain (1)
Karl E Anderson, MD (3) · Karl Elmo Anderson (2) · Will Savage, MD PhD (2) · Amy K Dickey, MD (1) · Christer Andersson, MD (1) · Deybach Jean-Charles, Professor (1) · Herbert L Bonkovsky, MD (1) · Karl Anderson, MD (1) · Robert Desnick, MD (1) · Sean Rudnick, MD (1)
PMID 20226990, PMID 22985607, PMID 36811718, PMID 37043653, PMID 39363243, PMID 39887900, PMID 42833135
2016 (1), U54DK083909 (1)