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 | NCT04256655 (ClinicalTrials.gov) | December 1, 2020 | 28/1/2020 | Safety, Tolerability, Pharmacodynamics and Pharmacokinetics of CDX 6114 in PKU Patients | A Phase 1, Multi-Center, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety, Tolerability, Pharmacodynamics and Pharmacokinetics of CDX 6114 After Multiple Ascending Oral Dose Administration to Patients With Phenylketonuria (PKU). | Phenylketonurias | Drug: cohort 1 0.225g;Drug: Cohort 2 0.75g;Drug: Cohort 3 2.25 g | Nestlé | NULL | Withdrawn | 18 Years | 65 Years | All | 0 | Phase 1 | NULL |
2 | NCT04085666 (ClinicalTrials.gov) | June 1, 2019 | 3/8/2019 | Pharmacodynamics, Safety, Tolerability and Pharmacokinetics of CDX-6114 in Patients With Phenylketonuria (PKU) | A Phase 1, Multi-center, Randomized, Double-blind, Placebo-controlled, Cross-over Study to Evaluate the Pharmacodynamics, Safety, Tolerability and Pharmacokinetics of a Single Oral Dose of CDX-6114 in Patients With Phenylketonuria (PKU). | Phenylketonuria | Drug: CDX 6114;Other: Matching Placebo | Nestlé | NULL | Completed | 18 Years | 55 Years | All | 18 | Phase 1 | Australia;Germany |