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Cnsa-001    (DrugBank: -)

1 disease
IDDisease name (Link within this page)Number of trials
240Phenylketonuria1

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.
1 / 125 trial found
No.TrialIDDate_
enrollment		Date_
registration		Public_titleScientific_titleConditionInterventionPrimary_
sponsor		Secondary_
sponsor		Recruitment_
Status		Inclusion_
agemin		Inclusion_
agemax		Inclusion_
gender		Target_
size		PhaseCountries
1NCT03519711
(ClinicalTrials.gov)		June 24, 201811/4/2018A Study of CNSA-001 in Primary Tetrahydrobiopterin (BH4) Deficient Participants With HyperphenylalaninemiaA Phase 1/2, Open-Label, Randomized Parallel Arm, Intra-patient Dose Escalation Study to Evaluate the Safety, Pharmacokinetics and Preliminary Efficacy of CNSA-001(Sepiapterin) in Primary Tetrahydrobiopterin Deficient Patients With HyperphenylalaninemiaBH4 Deficiency;HyperphenylalaninemiaDrug: CNSA-001PTC TherapeuticsNULLActive, not recruiting12 MonthsN/AAll6Phase 1;Phase 2United States;Germany