47 completed, 7 recruiting, 6 unknown, 4 terminated. 20 Phase 2, 13 Phase 3, 11 Phase 1, 8 Not applicable.
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 |
| 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 | |
| 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 |
| 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Protoporphyria and Increased Liver Stiffness and/or Liver Enzymes at Baseline bitopertin - Protoporphyria | Phase 1 | Withdrawn | Wake Forest University Health Sciences | 2026-01-01 | 2026-01-29 | |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 |
| Clinical Research on Acute Intermittent Porphyria and the Use of Carbohydrate-Rich Diet as a Treatment Carbohydrates - Porphyria, Acute Intermittent | Not applicable | Recruiting | Nordlandssykehuset HF | 50 | 2024-01-27 | 2026-03-27 |
| 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 |
| 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 |
| 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 | |
| Identification of Acute Intermittent Porphyria Modifying Genes Acute Intermittent Porphyria (AIP) | Recruiting | Icahn School of Medicine at Mount Sinai | 150 | 2022-09-23 | 2026-09-10 | |
| 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 |
| Evaluating the Prevalence of Acute Hepatic Porphyria in Postural Tachycardia Syndrome Autonomic Function Testing, Genetic Testing, Urine Testing - Postural Orthostatic Tachycardia Syndrome, Acute Hepatic Porphyria | Active not recruiting | Vanderbilt University Medical Center | 70 | 2022-01-19 | 2026-09-28 | |
| Prevalence of Acute Hepatic Porphyria Dosage of Delta-aminolevulinic acid and Porphobilinogen - Acute Hepatic Porphyria | Completed | Association pour la Recherche en Medecine Interne | 150 | 2021-08-23 | 2026-01-28 | |
| ELEVATE, a Registry of Patients With Acute Hepatic Porphyria (AHP) Acute Hepatic Porphyria | Recruiting | Alnylam Pharmaceuticals | 150 | 2021-04-26 | 2026-10-06 | |
| Colombia National Porphyria Registry Rare Diseases, Porphyrias | Unknown | Fundación Grupo de Investigación en Cuidados Intensivos y Obstetricia | 100 | 2021-04-01 | 2022-09-01 |
Showing 20 of 70. Sign in to see all of them.
Protoporphyria, Erythropoietic (29) · Porphyria, Acute Intermittent (21) · Coproporphyria, Hereditary (14) · Rare Diseases (12) · Metabolism, Inborn Errors (11) · Porphyria, Variegate (8) · Protoporphyria, Erythropoietic, X-Linked Dominant (8) · Porphyria Cutanea Tarda (6) · Coproporphyria (2) · Hepatitis C (2) · Porphyria, Erythropoietic (2) · Porphyrias, Hepatic (2) · Anemia, Iron-Deficiency (1) · Arrhythmias, Cardiac (1)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (70) · Genetic Diseases, Inborn (70) · Metabolic Diseases (70) · Nutritional and Metabolic Diseases (70) · Skin Diseases (64) · Skin Diseases, Genetic (64) · Skin and Connective Tissue Diseases (64) · Digestive System Diseases (62) · Liver Diseases (62) · Porphyrias, Hepatic (60) · Pathological Conditions, Signs and Symptoms (16) · Pathologic Processes (15) · Disease Attributes (12) · Blood-Borne Infections (2)
afamelanotide (7) · (4-(3-fluoro-5-trifluoromethylpyridin-2-yl)piperazin-1-yl)(5-methanesulfonyl-2-(2,2,2-trifluoro-1-methylethoxy)phenyl)methanone (5) · givosiran (5) · heme arginate (4) · tin mesoporphyrin (4) · Hemin (3) · Cysteine (2) · Deferasirox (2) · Exercise (2) · Carbohydrates (1) · Chlorpromazine (1) · Ciclopirox (1) · Cimetidine (1) · Colestipol (1) · Dihydroxyacetone (1)
National Center for Research Resources (NCRR) (9) · Alnylam Pharmaceuticals (8) · Clinuvel Pharmaceuticals Limited (6) · Bispebjerg Hospital (4) · Disc Medicine, Inc (4) · The University of Texas Medical Branch, Galveston (4) · Icahn School of Medicine at Mount Sinai (3) · Portal Therapeutics, Inc. (3) · Brigham and Women's Hospital (2) · Digna Biotech S.L. (2) · Nordlandssykehuset HF (2) · Tanabe Pharma America, Inc. (2) · University of Texas Southwestern Medical Center (2) · Wake Forest University Health Sciences (2) · Amy K. Dickey, M.D. (1)
United States (42) · France (10) · United Kingdom (10) · Spain (9) · Netherlands (8) · Sweden (8) · Italy (7) · Belgium (6) · Germany (6) · Australia (5) · Denmark (5) · Norway (4) · Poland (4) · Bulgaria (3) · Canada (3)
Karl Elmo Anderson (7) · Karl E Anderson, MD (3) · Jesus Prieto, MD (2) · Juan Ruiz, MD (2) · Karl E Anderson (2) · Rafael Enriquez de Salamanca, MD (2) · Robert Desnick, MD (2) · Will Savage, MD PhD (2) · Will Savage, MD, PhD (2) · Alex Anstey, MBBS, FRCP (1) · Amit Pandya, M.D. (1) · Amy K Dickey, MD (1)
PMID 42833135, PMID 26132941, PMID 27212246, PMID 39363243, PMID 10037110, PMID 16943900, PMID 17709712, PMID 20226990, PMID 22911183, PMID 22985607, PMID 25016127, PMID 25483980, PMID 28614581, PMID 30726693, PMID 32521132, PMID 32681675, PMID 32916704, PMID 36028858, PMID 36406817, PMID 36811718, PMID 37043653, PMID 37479139, PMID 38044204, PMID 39115302, PMID 39887900, PMID 39969427, PMID 41388232, PMID 42106273, PMID 8423877, PMID 9521433
U54DK083909 (6), 2016 (1), 2023 (1)