240. Phenylketonuria
125 clinical trials,   95 drugs   (DrugBank: 11 drugs),   1 drug target gene,   5 drug target pathways
Searched query = "Phenylketonuria", "PKU", "Phenylalanine hydroxylase deficiency", "PAH deficiency", "Tetrahydrobiopterin deficiency", "BH4 deficiency", "BH4 reactive hyper pheemia"
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 |
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1 | EUCTR2009-018168-81-DK (EUCTR) | 16/04/2010 | 03/03/2010 | ENDURE: A Phase IV, prospective, open-label, uncontrolled, multi-centre cohort trial to assess the responsiveness of subjects with phenylketonuria (PKU) to treatment with Kuvan® 20 mg/kg/day for 28 days - ENDURE | ENDURE: A Phase IV, prospective, open-label, uncontrolled, multi-centre cohort trial to assess the responsiveness of subjects with phenylketonuria (PKU) to treatment with Kuvan® 20 mg/kg/day for 28 days - ENDURE | Subjects diagnosed with Phenylketonuria (PKU) (classic or mild PKU, or mild hyperphenylalaninaemia (HPA)). MedDRA version: 14.1;Level: LLT;Classification code 10034873;Term: Phenylketonuria (PKU);System Organ Class: 10010331 - Congenital, familial and genetic disorders | Trade Name: Kuvan INN or Proposed INN: Sapropterindihydrochloride Other descriptive name: SAPROPTERIN HYDROCHLORIDE | Merck Serono Norway | NULL | Not Recruiting | Female: yes Male: yes | 70 | Phase 4 | Denmark | ||
2 | EUCTR2009-018168-81-NO (EUCTR) | 10/03/2010 | ENDURE: A Phase IV, prospective, open-label, uncontrolled, multi-centre cohort trial to assess the responsiveness of subjects with phenylketonuria (PKU) to treatment with Kuvan® 20 mg/kg/day for 28 days - ENDURE | ENDURE: A Phase IV, prospective, open-label, uncontrolled, multi-centre cohort trial to assess the responsiveness of subjects with phenylketonuria (PKU) to treatment with Kuvan® 20 mg/kg/day for 28 days - ENDURE | Subjects diagnosed with Phenylketonuria (PKU) (classic or mild PKU, or mild hyperphenylalaninaemia (HPA)). MedDRA version: 12.1;Level: LLT;Classification code 10034873;Term: Phenylketonuria (PKU) | Trade Name: Kuvan INN or Proposed INN: Sapropterindihydrochloride Other descriptive name: SAPROPTERIN HYDROCHLORIDE | Merck Serono Norway | NULL | NA | Female: yes Male: yes | 150 | Phase 4 | Denmark;Norway |