241. Hypertyrosinemia type I
[
11 clinical trials,
6 drugs（DrugBank:
1 drug),
1 target gene / 4 target pathways ]
Searched query = "Hypertyrosinemia type I", "Tyrosinemia type I", "Tyrosinemia I", "Hereditary tyrosinemia, Type I"
The queries were searched in Public_title, Scientific_title, and Condition of the data. Export date: 11/20/2019, 11/21/2019. Trials are sorted by Date_enrolment from most recent to oldest in the table.
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | NCT04113772 | November 1, 2019 | 14 October 2019 | Bio Equivalency 20 Mgm Orfadin and 20 Mgm of Nitisonine | Orfadin and Nitinosine Study | Hereditary Tyrosinemia, Type I | Drug: Nitisinone;Drug: Orfadin | Sutphin Drugs | Not recruiting | 18 Years | 50 Years | All | 4 | N/A | India | |
| 2 | NCT02750345 | March 2016 | 16 December 2017 | Bioequivalence Study of Two Oral Nitisinone Formulations to Treat Hereditary Tyrosinemia (HT-1) | A Three-Period Crossover Study to Determine the Bioequivalence of Two Oral Formulations Containing Nitisinone 10 mg Compared to Reference Formulation Orfadin In Healthy Subjects Under Fasting Conditions | Hereditary Tyrosinemia, Type I | Drug: Nitisinone;Drug: Nitisinone Baked Tablet;Drug: Orfadin | Cycle Pharmaceuticals Ltd. | Parexel | Not recruiting | 18 Years | 55 Years | All | 24 | Phase 1 | South Africa |
| 3 | NCT02750332 | November 2015 | 16 December 2017 | Bioavailability Food-Effect Study of an Oral Nitisinone Formulation to Treat Hereditary Tyrosinemia (HT-1) | A Single Center, Single-Dose, Open-Label, Randomized Study to Compare the Bioavailability of an Oral Test Formulation Containing Nitisinone 10 mg in at Least 16 Healthy Male and Female Subjects Under Fasting and Fed Conditions | Hereditary Tyrosinemia, Type I | Drug: Nitisinone | Cycle Pharmaceuticals Ltd. | Parexel | Not recruiting | 18 Years | 55 Years | All | 20 | Phase 1 | South Africa |
| 4 | NCT02750709 | October 2015 | 16 December 2017 | Bioequivalence Study of Two Nitisinone Formulations Compared to Orfadin | A Single Center, Single-Dose, Open-Label, Laboratory-Blind, Randomized, Three-Period Crossover Study to Determine the Bioequivalence of Two Oral Formulations Containing of Nitisinone 10 mg Compared to the Reference Formulation Orfadin 10 mg in at Least 18 Healthy Male and Female Subjects Under Fasting Conditions | Hereditary Tyrosinemia, Type I | Drug: Nitisinone;Drug: Nitisinone 10 mg Tablet High Compritol;Drug: Orfadin | Cycle Pharmaceuticals Ltd. | Parexel | Not recruiting | 18 Years | 55 Years | All | 24 | Phase 1 | South Africa |
| 5 | NCT02323529 | December 2014 | 23 November 2015 | Efficacy and Safety of Once Daily Dosing Compared to Twice Daily Dosing of Nitisinone in HT-1 | Open-label, Multicentre, Multiple-dose Trial to Evaluate Pharmacokinetics, Efficacy and Safety of Once Daily Dosing Compared to Twice Daily Dosing of Orfadin in Patients Diagnosed With Hereditary Tyrosinemia Type 1 | Hereditary Tyrosinemia, Type I | Drug: Nitisinone | Swedish Orphan Biovitrum | Not recruiting | N/A | N/A | Both | 18 | Phase 3 | Belgium;Denmark;France;Germany;Sweden | |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 6 | EUCTR2013-004132-29-SE | 15/10/2014 | 14 December 2015 | An open-label, non-randomized, sequential, multicenter study to evaluate the pharmacokinetics, efficacy and safety of once daily dosing compared to twice daily dosing of Orfadin in patients diagnosed with hereditary tyrosinemia type 1 | An open-label, non-randomized, sequential, multicenter study to evaluate the pharmacokinetics, efficacy and safety of once daily dosing compared to twice daily dosing of Orfadin in patients diagnosed with hereditary tyrosinemia type 1 | hereditary tyrosinemia type 1 MedDRA version: 17.1 Level: LLT Classification code 10069462 Term: Tyrosinemia type I System Organ Class: 100000004850 ;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03] | Trade Name: Orfadin Pharmaceutical Form: Capsule, hard INN or Proposed INN: NITISINONE CAS Number: 104206-65-7 Concentration unit: mg milligram(s) Concentration type: range Concentration number: 2-10 | Swedish Orphan Biovitrum AB (Publ) | Not Recruiting | Female: yes Male: yes | 20 | Belgium;Denmark;Sweden | ||||
| 7 | NCT02320084 | September 2013 | 11 November 2019 | Long Term Safety Study of Orfadin Treatment in HT-1 Patients in Standard Clinical Care | A Non-interventional Post Authorization Study (PASS) to Evaluate Long-term Safety of Orfadin Treatment in Hypertyrosinemia Type 1 (HT-1) Patients in Standard Care | Hereditary Tyrosinemia, Type I | Drug: Nitisinone | Swedish Orphan Biovitrum | Not recruiting | N/A | N/A | All | 315 | Phase 1 | Austria;Belgium;Czechia;Denmark;Finland;France;Germany;Hungary;Ireland;Italy;Netherlands;Norway;Poland;Portugal;Spain;Sweden;United Kingdom;Croatia;Czech Republic;Greece;Kosovo;Lithuania;Macedonia, The Former Yugoslav Republic of;Romania;Slovakia;Switzerland | |
| 8 | EUCTR2012-002286-36-DE | 19/11/2012 | 20 May 2013 | Taste and palatability of Orfadin suspension. | Taste and palatability of Orfadin suspension. An open, non-controlled 3-day study in pediatric patients with hereditary tyrosinemia type 1 treated with Orfadin. | Hereditary tyrosinemia type 1 (HT-1) MedDRA version: 14.1 Level: LLT Classification code 10069459 Term: Tyrosinaemia type I System Organ Class: 100000004850 ;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03] | Product Name: Orfadin oral suspension 4mg/ml Pharmaceutical Form: Oral suspension INN or Proposed INN: Nitisinone CAS Number: 104206-65-7 Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 4- | Swedish Orphan Biovitrum AB | Not Recruiting | Female: yes Male: yes | 18 | Germany;United Kingdom | ||||
| 9 | NCT01734889 | October 2012 | 19 October 2017 | Taste and Palatability of Orfadin Suspension | Taste and Palatability of Orfadin Suspension. An Open, Non-controlled 3 Day Study in Pediatric Patients With Hereditary Tyrosinemia Type 1 Treated With Orfadin. | Hereditary Tyrosinemia, Type I | Drug: Nitisinone | Swedish Orphan Biovitrum | Not recruiting | 1 Month | 17 Years | All | 18 | Phase 1 | France;Germany;United Kingdom | |
| 10 | NCT00004443 | October 1998 | 7 April 2015 | Study of NTBC for Tyrosinemia I | Tyrosinemia I | Drug: NTBC | University of Washington | Not recruiting | N/A | 17 Years | Both | 20 | N/A | United States;Canada | ||
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 11 | NCT00004333 | November 1994 | 19 February 2015 | Phase II Study of the Enzyme Inhibitor NTBC for Tyrosinemia Type I | Tyrosinemia, Type I | Drug: NTBC | National Center for Research Resources (NCRR) | University of Michigan | Not recruiting | N/A | N/A | Both | Phase 2 |