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TrialConditionsStatusPhaseUpdatedMore infoNCT ID
HELIOS: Open-Label, Long-Term Extension Study to Investigate the Safety, Tolerability, and Efficacy of DISC-1459 (Bitopertin) in Participants With EPP or XLPCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Digestive System Diseases, Nutritional and Metabolic Diseases, Skin and Connective Tissue Diseases, erythropoietic protoporphyria, hepatic porphyria, hereditary disease, hereditary skin disorder, liver disorder, metabolic disease, porphyria, skin disorderEnrolling by invitationPhase 2 / Phase 32026-10-09NCT05883748
ELEVATE, a Registry of Patients With Acute Hepatic Porphyria (AHP)Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Digestive System Diseases, Nutritional and Metabolic Diseases, Skin and Connective Tissue Diseases, hepatic porphyria, hereditary coproporphyria, hereditary disease, hereditary skin disorder, liver disorder, metabolic disease, porphyria, porphyria due to ALA dehydratase deficiency, skin disorderRecruitingNot stated2026-10-06NCT04883905
Evaluating the Prevalence of Acute Hepatic Porphyria in Postural Tachycardia SyndromeCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Digestive System Diseases, Nutritional and Metabolic Diseases, Skin and Connective Tissue Diseases, autonomic nervous system disorder, hepatic porphyria, hereditary coproporphyria, hereditary disease, hereditary skin disorder, liver disorder, metabolic disease, nervous system disorder, orthostatic intolerance, porphyria, porphyria due to ALA dehydratase deficiency, primary dysautonomia, skin disorderActive, not recruitingNot stated2026-09-28NCT05344599
A Study to Investigate PORT-77 Oral Dosing Compared With Placebo in Participants With Erythropoietic Protoporphyria (EPP) or X-linked Protoporphyria (XLP)Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Digestive System Diseases, Nutritional and Metabolic Diseases, Skin and Connective Tissue Diseases, X-linked erythropoietic protoporphyria, erythropoietic protoporphyria, hepatic porphyria, hereditary disease, hereditary skin disorder, liver disorder, metabolic disease, porphyria, skin disorderRecruitingPhase 2 / Phase 32026-09-15NCT07820085
Identification of Acute Intermittent Porphyria Modifying GenesCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Digestive System Diseases, Nutritional and Metabolic Diseases, Skin and Connective Tissue Diseases, acute intermittent porphyria, hepatic porphyria, hereditary disease, hereditary skin disorder, liver disorder, metabolic disease, porphyria, skin disorderRecruitingNot stated2026-09-10NCT05502133
Longitudinal Study of the PorphyriasCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Coproporphyria, Digestive System Diseases, Nutritional and Metabolic Diseases, Skin and Connective Tissue Diseases, acute intermittent porphyria, erythropoietic protoporphyria, hepatic porphyria, hereditary disease, hereditary skin disorder, liver disorder, metabolic disease, porphyria, skin disorderRecruitingNot stated2026-05-15NCT01561157
Acute Porphyria Biomarkers for Disease ActivityCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Digestive System Diseases, Nutritional and Metabolic Diseases, Skin and Connective Tissue Diseases, acute intermittent porphyria, hepatic porphyria, hereditary coproporphyria, hereditary disease, hereditary skin disorder, liver disorder, metabolic disease, porphyria, skin disorder, variegate porphyriaActive, not recruitingNot stated2026-05-12NCT02935400
Observational Study of Adults and Adolescents With Erythropoietic Protoporphyria (EPP) and X-linked Porphyria (XLP)Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Digestive System Diseases, Nutritional and Metabolic Diseases, Skin and Connective Tissue Diseases, erythropoietic protoporphyria, hepatic porphyria, hereditary disease, hereditary skin disorder, liver disorder, metabolic disease, porphyria, skin disorderRecruitingNot stated2026-05-05NCT07567131
Clinical Research on Acute Intermittent Porphyria and the Use of Carbohydrate-Rich Diet as a TreatmentCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Digestive System Diseases, Nutritional and Metabolic Diseases, Skin and Connective Tissue Diseases, acute intermittent porphyria, hepatic porphyria, hereditary disease, hereditary skin disorder, liver disorder, metabolic disease, porphyria, skin disorderRecruitingNot applicable2026-03-27NCT06273644
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 CEPCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Nutritional and Metabolic Diseases, Skin and Connective Tissue Diseases, cutaneous porphyria, hereditary disease, hereditary skin disorder, metabolic disease, porphyria, skin disorderRecruitingPhase 1 / Phase 22026-03-24NCT07024316