241. Hypertyrosinemia type I
14 clinical trials, 7 drugs (DrugBank: 1 drug), 1 drug target gene, 5 drug target pathways
Searched query = "Hypertyrosinemia type I", "Tyrosinemia type I", "Tyrosinemia I", "Hereditary tyrosinemia, Type I", "Fumarylacetoacetate hydrolase deficiency", "FAH deficiency"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
| 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 | 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 | |||
| 2 | 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 |