14 completed, 2 unknown, 1 withdrawn. 8 Phase 1, 4 Phase 2, 3 Phase 3, 2 Not applicable.
| Phase | Trials | Status |
|---|---|---|
| Phase 1 | 8 | Completed 8 |
| Phase 2 | 4 | Completed 4 |
| Phase 3 | 3 | Completed 2, Unknown 1 |
| Not applicable | 2 | Completed 1, Unknown 1 |
Follow nitisinone: new trials by email Literature Review of nitisinone trials Research Report on nitisinone
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Study to Describe Outcome of Nitisinone Treatment in HT-1 Patients Nitisinone - Hereditary Tyrosinemia, Type I | Withdrawn | Swedish Orphan Biovitrum | 2025-09-01 | 2025-12-23 | ||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 |
| A Study to Investigate the Potential Influence of Nitisinone on the Metabolism and the Transport of Other Drugs in Healthy Volunteers Nitisinone in Arm A, Tolbutamide, Metoprolol - Drug Drug Interaction | Phase 1 | Completed | Swedish Orphan Biovitrum | 36 | 2017-03-28 | 2017-08-10 |
| 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 |
| Suitability of Nitisinone in Alkaptonuria 2 Nitisinone - Alkaptonuria | Phase 3 | Unknown | University of Liverpool | 140 | 2014-04-01 | 2018-05-31 |
| 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 | |
| Bioequivalence of Orfadin 20 mg Compared to Orfadin 10 mg Capsules. Nitisinone 20 mg, Nitisinone 10 mg - Healthy | Phase 1 | Completed | Swedish Orphan Biovitrum | 12 | 2013-05-01 | 2021-10-12 |
| Dose Response Study of Nitisinone in Alkaptonuria Nitisinone - Alkaptonuria | Phase 2 | Completed | University of Liverpool | 40 | 2013-05-01 | 2014-06-11 |
| Nitisinone for Type 1B Oculocutaneous Albinism Nitisinone - Albinism, Vision Loss | Phase 1 / Phase 2 | Completed | National Eye Institute (NEI) | 5 | 2013-04-16 | 2019-02-26 |
| Taste and Palatability of Orfadin Suspension Nitisinone - Hereditary Tyrosinemia, Type I | Phase 1 | Completed | Swedish Orphan Biovitrum | 18 | 2012-10-01 | 2014-11-06 |
| Bioequivalence of Orfadin Suspension Compared to Orfadin Capsules, and the Effect of Food on the Bioavailability of the Suspension Nitisinone, capsule, Nitisinone, suspension - Healthy Volunteers | Phase 1 | Completed | Swedish Orphan Biovitrum | 12 | 2012-08-01 | 2014-03-17 |
| Nitisinone (NTBC) In Different Age Groups Of Patients With Alkaptonuria Nitisinone - Alkaptonuria | Phase 2 / Phase 3 | Completed | University of California, San Diego | 8 | 2011-01-01 | 2021-04-22 |
| Long-Term Study of Nitisinone to Treat Alkaptonuria Nitisinone (NTBC) - Alkaptonuria | Phase 2 | Completed | National Human Genome Research Institute (NHGRI) | 40 | 2005-01-01 | 2021-08-26 |
| Prevention of Dichloroacetate Toxicity Nitisinone (NTBC), Dichloroacetate, Low-tyrosin diet - Acidosis, Lactic, Chronic Disease | Not applicable | Completed | University of Florida | 30 | 2001-09-01 | 2015-03-25 |
Tyrosinemias (8) · Alkaptonuria (4) · Acidosis, Lactic (1) · Albinism (1) · Arthritis (1) · Chronic Disease (1) · Corneal Injuries (1) · Fanconi Anemia (1) · Liver Failure (1) · Ochronosis (1) · Photophobia (1) · Rare Diseases (1) · Vision Disorders (1)
Swedish Orphan Biovitrum (7) · Cycle Pharmaceuticals Ltd. (3) · University of Liverpool (2) · National Eye Institute (NEI) (1) · National Human Genome Research Institute (NHGRI) (1) · Sutphin Drugs (1) · University of California, San Diego (1) · University of Florida (1)
Germany (4) · United Kingdom (4) · United States (4) · France (3) · Netherlands (3) · South Africa (3) · Belgium (2) · Denmark (2) · Sweden (2) · Austria (1) · China (1) · Czechia (1) · Finland (1) · Hungary (1) · India (1)
Erik Brouwer, MD (3) · L Ranganath (2) · Brian P Brooks, M.D. (1) · Ioannis Kottakis, MD, PhD (1) · William L Nyhan, MD, PhD (1) · Xiaoping Luo, MD, PhD (1)
PMID 25475116, PMID 25665838, PMID 30674731, PMID 31296884, PMID 32822600, PMID 36270742
2016 (1)