6 completed, 2 unknown, 1 withdrawn. 4 Phase 1, 1 Not applicable, 1 Phase 3.
| Phase | Trials | Status |
|---|---|---|
| Phase 1 | 4 | Completed 4 |
| Not applicable | 1 | Unknown 1 |
| Phase 3 | 1 | Completed 1 |
Follow Hereditary Tyrosinemia, Type I: new trials by email Literature Review of Hereditary Tyrosinemia, Type I trials Research Report on Hereditary Tyrosinemia, Type I
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 Non-interventional, Post-Marketing Study to Describe Outcome of Nitisinone Treatment in HT-1 Patients Nitisinone - Hereditary Tyrosinemia, Type I | Withdrawn | Swedish Orphan Biovitrum | 2025-09-01 | 2025-12-23 | ||
| Bio Equivalency 20 Mgm Orfadin and 20 Mgm of Nitisonine Nitisinone, Orfadin - Hereditary Tyrosinemia, Type I | Not applicable | Unknown | Sutphin Drugs | 4 | 2019-11-01 | 2019-10-03 |
| Hereditary Hepatorenal Tyrosinemia Natural History in Egypt and the Arab World (Multicenter Clinical Study) Hereditary Tyrosinemia, Type I | Unknown | Yassin Abdelghaffar Charity Center for Liver Disease and Research | 50 | 2019-04-05 | 2019-01-10 | |
| Bioequivalence Study of Two Oral Nitisinone Formulations to Treat Hereditary Tyrosinemia (HT-1) Nitisinone, Nitisinone Baked Tablet, Orfadin - Hereditary Tyrosinemia, Type I | Phase 1 | Completed | Cycle Pharmaceuticals Ltd. | 24 | 2016-03-01 | 2017-06-14 |
| Bioavailability Food-Effect Study of an Oral Nitisinone Formulation to Treat Hereditary Tyrosinemia (HT-1) Nitisinone - Hereditary Tyrosinemia, Type I | Phase 1 | Completed | Cycle Pharmaceuticals Ltd. | 20 | 2015-11-01 | 2017-03-15 |
| Bioequivalence Study of Two Nitisinone Formulations Compared to Orfadin Nitisinone, Nitisinone 10 mg Tablet High Compritol, Orfadin - Hereditary Tyrosinemia, Type I | Phase 1 | Completed | Cycle Pharmaceuticals Ltd. | 24 | 2015-10-01 | 2017-03-15 |
| Efficacy and Safety of Once Daily Dosing Compared to Twice Daily Dosing of Nitisinone in HT-1 Nitisinone - Hereditary Tyrosinemia, Type I | Phase 3 | Completed | Swedish Orphan Biovitrum | 18 | 2014-12-01 | 2015-11-11 |
| Long Term Safety Study of Orfadin Treatment in HT-1 Patients in Standard Clinical Care Nitisinone - Hereditary Tyrosinemia, Type I | Completed | Swedish Orphan Biovitrum | 315 | 2013-09-01 | 2024-09-19 | |
| Taste and Palatability of Orfadin Suspension Nitisinone - Hereditary Tyrosinemia, Type I | Phase 1 | Completed | Swedish Orphan Biovitrum | 18 | 2012-10-01 | 2014-11-06 |
Tyrosinemias (9) · Fanconi Anemia (1) · Liver Failure (1) · Rare Diseases (1)
Amino Acid Metabolism, Inborn Errors (9) · Brain Diseases (9) · Brain Diseases, Metabolic (9) · Brain Diseases, Metabolic, Inborn (9) · Central Nervous System Diseases (9) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (9) · Genetic Diseases, Inborn (9) · Metabolic Diseases (9) · Metabolism, Inborn Errors (9) · Nervous System Diseases (9) · Nutritional and Metabolic Diseases (9) · Anemia (1) · Anemia, Aplastic (1) · Anemia, Hypoplastic, Congenital (1) · Bone Marrow Diseases (1)
nitisinone (8)
Swedish Orphan Biovitrum (4) · Cycle Pharmaceuticals Ltd. (3) · Sutphin Drugs (1) · Yassin Abdelghaffar Charity Center for Liver Disease and Research (1)
France (3) · Germany (3) · South Africa (3) · Belgium (2) · Denmark (2) · Sweden (2) · United Kingdom (2) · Austria (1) · China (1) · Czechia (1) · Egypt (1) · Finland (1) · Hungary (1) · India (1) · Ireland (1)
Erik Brouwer, MD (1) · Ioannis Kottakis, MD, PhD (1) · Tawhida Y Abdelghaffar, MD (1) · Xiaoping Luo, MD, PhD (1)