241. 高チロシン血症1型 Hypertyrosinemia type I Clinical trials / Disease details
臨床試験数 : 14 / 薬物数 : 7 - (DrugBank : 1) / 標的遺伝子数 : 1 - 標的パスウェイ数 : 5
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | NCT04113772 (ClinicalTrials.gov) | November 1, 2019 | 1/10/2019 | Bio Equivalency 20 Mgm Orfadin and 20 Mgm of Nitisonine | Orfadin and Nitinosine Study | Hereditary Tyrosinemia, Type I | Drug: Nitisinone;Drug: Orfadin | Sutphin Drugs | NULL | Not yet recruiting | 18 Years | 50 Years | All | 4 | N/A | India |
| 2 | NCT02750345 (ClinicalTrials.gov) | March 2016 | 21/4/2016 | 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 | Completed | 18 Years | 55 Years | All | 24 | Phase 1 | South Africa |
| 3 | NCT02750332 (ClinicalTrials.gov) | November 2015 | 21/4/2016 | 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 | Completed | 18 Years | 55 Years | All | 20 | Phase 1 | South Africa |
| 4 | NCT02750709 (ClinicalTrials.gov) | October 2015 | 20/4/2016 | 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 | Completed | 18 Years | 55 Years | All | 24 | Phase 1 | South Africa |
| 5 | EUCTR2013-004132-29-DK (EUCTR) | 16/12/2014 | 25/08/2014 | 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 INN or Proposed INN: NITISINONE | Swedish Orphan Biovitrum AB (Publ) | NULL | Not Recruiting | Female: yes Male: yes | 20 | Belgium;Denmark;Sweden | |||
| 6 | NCT02323529 (ClinicalTrials.gov) | December 2014 | 18/12/2014 | 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 | NULL | Completed | N/A | N/A | Both | 18 | Phase 3 | Belgium;Denmark;France;Germany;Sweden |
| 7 | EUCTR2013-004132-29-BE (EUCTR) | 06/11/2014 | 15/10/2014 | 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: 17.0;Level: LLT;Classification code 10069462;Term: Tyrosinemia type I;System Organ Class: 100000004850;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03] | Trade Name: Orfadin INN or Proposed INN: NITISINONE Trade Name: Orfadin INN or Proposed INN: NITISINONE Trade Name: Orfadin INN or Proposed INN: NITISINONE | Swedish Orphan Biovitrum AB (Publ) | NULL | Not Recruiting | Female: yes Male: yes | 20 | Phase 4 | Belgium;Denmark;Sweden | ||
| 8 | EUCTR2013-004132-29-SE (EUCTR) | 15/10/2014 | 18/08/2014 | 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 INN or Proposed INN: NITISINONE | Swedish Orphan Biovitrum AB (Publ) | NULL | Not Recruiting | Female: yes Male: yes | 20 | Belgium;Denmark;Sweden | |||
| 9 | NCT02320084 (ClinicalTrials.gov) | September 2013 | 23/10/2014 | 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 | NULL | Completed | N/A | N/A | All | 315 | 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 | |
| 10 | EUCTR2012-002286-36-DE (EUCTR) | 19/11/2012 | 03/08/2012 | 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 INN or Proposed INN: Nitisinone | Swedish Orphan Biovitrum AB | NULL | Not Recruiting | Female: yes Male: yes | 18 | Germany;United Kingdom | |||
| 11 | EUCTR2012-002286-36-GB (EUCTR) | 04/10/2012 | 10/08/2012 | 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: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03] | Product Name: Orfadin oral suspension 4mg/ml INN or Proposed INN: NITISINONE | Swedish Orphan Biovitrum AB | NULL | Not Recruiting | Female: yes Male: yes | 18 | Germany;United Kingdom | |||
| 12 | NCT01734889 (ClinicalTrials.gov) | October 2012 | 22/11/2012 | 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 | NULL | Completed | 1 Month | 17 Years | All | 18 | Phase 1 | France;Germany;United Kingdom |
| 13 | NCT00004443 (ClinicalTrials.gov) | October 1998 | 18/10/1999 | Study of NTBC for Tyrosinemia I | Tyrosinemia I | Drug: NTBC | University of Washington | NULL | Completed | N/A | 17 Years | Both | 20 | N/A | United States;Canada | |
| 14 | NCT00004333 (ClinicalTrials.gov) | November 1994 | 18/10/1999 | 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 | Completed | N/A | N/A | Both | Phase 2 | NULL |