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.

Search in Page e.g. "Phase 3", "Not recruiting", "Japan"
2 trials found
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1EUCTR2012-002286-36-DE
(EUCTR)
19/11/201203/08/2012Taste 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 ABNULLNot RecruitingFemale: yes
Male: yes
18Germany;United Kingdom
2EUCTR2012-002286-36-GB
(EUCTR)
04/10/201210/08/2012Taste 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 ABNULLNot RecruitingFemale: yes
Male: yes
18Germany;United Kingdom