4 completed, 2 terminated, 1 active not recruiting, 1 approved for marketing. 2 Phase 2, 1 Phase 3.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 2 | Completed 1, Terminated 1 |
| Phase 3 | 1 | Completed 1 |
Follow Porphyria, Variegate: new trials by email Literature Review of Porphyria, Variegate trials Research Report on Porphyria, Variegate
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 |
|---|---|---|---|---|---|---|
| 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 |
| Evidence-based Assessment of Medication Sensitivity in Acute Hepatic Porphyria Web-based Survey - Porphyrias | Completed | University of South Florida | 60 | 2019-04-03 | 2019-10-16 | |
| INSIGHT-AHP: A Study to Characterize the Prevalence of Acute Hepatic Porphyria (AHP) in Patients With Clinical Presentation and History Consistent With AHP Acute Hepatic Porphyria, Hepatic Porphyrias | Terminated | Alnylam Pharmaceuticals | 212 | 2018-05-29 | 2019-01-25 | |
| 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 |
| 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 |
| Acute Porphyria Biomarkers for Disease Activity Hemin - Acute Intermittent Porphyria, Hereditary Coproporphyria | Active not recruiting | The University of Texas Medical Branch, Galveston | 50 | 2014-04-28 | 2026-05-12 | |
| Clinical Diagnosis of Acute Porphyria Hereditary Coproporphyria (HCP), Acute Intermittent Porphyria (AIP) | Completed | University of California, San Francisco | 148 | 2011-12-01 | 2021-09-28 | |
| Expanded Access Protocol of Givosiran for Patients With Acute Hepatic Porphyria Givosiran - Acute Hepatic Porphyria | Approved for marketing | Alnylam Pharmaceuticals | 2024-05-20 |
Coproporphyria, Hereditary (7) · Porphyria, Acute Intermittent (7) · Porphyrias (5) · Porphyrias, Hepatic (1)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (8) · Digestive System Diseases (8) · Genetic Diseases, Inborn (8) · Liver Diseases (8) · Metabolic Diseases (8) · Nutritional and Metabolic Diseases (8) · Skin Diseases (8) · Skin Diseases, Genetic (8) · Skin and Connective Tissue Diseases (8) · Porphyrias, Hepatic (7) · Porphyrias (3)
Hemin (2) · givosiran (2) · Injections (1) · afamelanotide (1)
Alnylam Pharmaceuticals (3) · The University of Texas Medical Branch, Galveston (2) · Clinuvel Pharmaceuticals Limited (1) · University of California, San Francisco (1) · University of South Florida (1)
United States (6) · Netherlands (2) · Australia (1) · Bulgaria (1) · Canada (1) · Denmark (1) · Finland (1) · France (1) · Germany (1) · Italy (1) · Japan (1) · Mexico (1) · Poland (1) · South Korea (1) · Spain (1)
Bruce Wang, M.D. (1) · Bruce Wang, MD (1) · D. Montgomery Bissell, MD (1) · Herbert L. Bonkovsky, M.D. (1) · James P. Kushner, M.D. (1) · Joseph R. Bloomer, M.D. (1) · Karl E Anderson (1) · Karl E Anderson, MD (1) · Karl E. Anderson, M.D. (1) · Quinn Dinh, MD (1) · Robert J. Desnick, Ph.D., M.D. (1)
PMID 32521132, PMID 36028858, PMID 37479139, PMID 38044204, PMID 41388232, PMID 42833135
U54DK083909 (1)