DDrare: Database of Drug Development for Rare Diseases

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.

Drug = 5,6,7,8-tetrahydrobiopterin; 6R-BH4; Amino acids; BH4; BMN 165; BMN 165 (rAvPAL-PEG); BMN 307; BMN165 20mg/day; BMN165 40mg/day; Behavioral: Neuro-psychological Assessment; Behavioral: Restricted phenylalanine diet; Behavioral: phenylalanine restricted diet; CDX; CDX 6114; CNSA-001; Cohort 1 0.225g; Cohort 2 0.75g; Cohort 3 2.25 g; Corn; Corn/soy oil; Diagnostic Test: Whole body Phe oxidation testing; Diet; Dietary Supplement for PKU patients; Docosahexaenoic Acid; Docosahexaenoic acid; Free amino acids intake; Glycomacropeptide-based protein substitute; High oleic sunflower oil; Immunosuppression; KUVAN; Kuvan; Kuvan 100mg soluble tablet; KuvanTM Therapy; Kuvan®; Kuvan® (sapropterin dihydrochloride); Large Neutral Amino Acid Therapy; Low calorie protein substitute; Microalgal oil; Moxifloxacin; N.a.; NAP; Other: Diet; Other: Diet treatment; Other: Liver Evaluation; Other: Meal Challenge; Other: Normal (control) diet; Other: Phenylalanine (Phe)-restricted diet; Other: Routine; PEG; PEGVALIASE; PKU Explore; PKU Sphere; PKU Start; PKU sphere; Palynziq; Pegvaliase; Phe; Phenoptin; Phenylalanine; Phenylalanine restricted diet; Phenylalanine-free protein substitute in tablet form (XPhe minis); Phenylalanine-free protein substitute tablets; Phenylketonuria-type diet; Procedure: Hepatocyte Transplant; Procedure: Liver Biopsy; Protein intake; RAvPAL-PEG; RAvPAL-PEG 0.001 mg/kg; RAvPAL-PEG 0.003 mg/kg; RAvPAL-PEG 0.01 mg/kg; RAvPAL-PEG 0.03 mg/kg; RAvPAL-PEG 0.1 mg/kg; RTX; RTX-134; Radiation: Preparative Radiation Therapy; SAPROPTERIN; SAPROPTERIN DIHYDROCHLORIDE; SAPROPTERIN HYDROCHLORIDE; SYNB1618; Sapropterin; Sapropterin (Kuvan); Sapropterin Dihydrochloride; Sapropterin Dihydrocholoride; Sapropterin dihydrochloride; Sapropterin dihydrochloride, 6R-BH4, tetrahydrobiopterin; Sapropterin hydrochloride; Sapropterin, sapropterin dihydrochloride; Sapropterin, sapropterin hydrochloride; Sapropterindihydrochloride; Saproterin dihydrochloride; Sunflower oil; Synergy; T1401; Tetrahydrobiopterin; Tetrahydrobiopterin (BH4)

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	NCT00925054 (ClinicalTrials.gov)	September 2009	17/6/2009	Dose-Finding Study to Evaluate the Safety, Efficacy, & Tolerability of Multiple Doses of rAvPAL-PEG in Subjects With PKU	Phase 2, Open-Label Dose-Finding Study to Evaluate the Safety, Efficacy, and Tolerability of Multiple Subcutaneous (SC) Doses of rAvPAL-PEG in Subjects With PKU	Phenylketonuria	Drug: rAvPAL-PEG 0.001 mg/kg;Drug: rAvPAL-PEG 0.003 mg/kg;Drug: rAvPAL-PEG 0.01 mg/kg;Drug: rAvPAL-PEG 0.03 mg/kg;Drug: rAvPAL-PEG 0.1 mg/kg	BioMarin Pharmaceutical	NULL	Completed	16 Years	55 Years	All	40	Phase 2	United States

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agemin

Inclusion_
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NCT00925054
(ClinicalTrials.gov)

September 2009

17/6/2009

Dose-Finding Study to Evaluate the Safety, Efficacy, & Tolerability of Multiple Doses of rAvPAL-PEG in Subjects With PKU

Phase 2, Open-Label Dose-Finding Study to Evaluate the Safety, Efficacy, and Tolerability of Multiple Subcutaneous (SC) Doses of rAvPAL-PEG in Subjects With PKU

Phenylketonuria

Drug: rAvPAL-PEG 0.001 mg/kg;Drug: rAvPAL-PEG 0.003 mg/kg;Drug: rAvPAL-PEG 0.01 mg/kg;Drug: rAvPAL-PEG 0.03 mg/kg;Drug: rAvPAL-PEG 0.1 mg/kg

BioMarin Pharmaceutical

NULL

Completed

16 Years

55 Years

All

Phase 2

United States