DDrare: Database of Drug Development for Rare Diseases

240. フェニルケトン尿症
［臨床試験数：125，薬物数：95（DrugBank：11），標的遺伝子数：1，標的パスウェイ数：5］

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	NCT04224142 (ClinicalTrials.gov)	July 29, 2019	7/10/2019	Evaluation of PKU Sphere in Maternal PKU	An Observational Study to Evaluate Metabolic Control and Dietary Management in Women With PKU Taking PKU Sphere During Pre-conception and/or Pregnancy	Maternal Phenylketonuria	Dietary Supplement: PKU sphere	Vitaflo International, Ltd	University College London Hospitals	Recruiting	16 Years	N/A	Female	10		United Kingdom
2	NCT03939052 (ClinicalTrials.gov)	July 19, 2019	1/5/2019	Protein Requirements in Adults With Phenylketonuria (PKU)	Protein Requirements in Phenylketonuria (PKU) Patients Compared Using PKU Sphere™ \| Glycomacropeptide (GMP) and an L-amino Acid-based Product	Phenylketonuria	Dietary Supplement: Free amino acids intake	University of British Columbia	Vitaflo International, Ltd	Recruiting	19 Years	50 Years	All	6	N/A	Canada
3	NCT03771391 (ClinicalTrials.gov)	December 6, 2018	7/12/2018	A 16 Week Study Evaluating the Introduction of a GMP Based Protein Substitute in Participants With PKU	Multicentre Study Project to Evaluate the New Generation of Protein Supplements With Glycomacropeptide (GMP) in Patients With Phenylketonuria (PKU) Aged 10 Years and Older Over a 16 Week Period.	Phenylketonurias	Dietary Supplement: PKU Sphere	Vitaflo International, Ltd	Kreiskliniken Reutlingen;Universitätsmedizin der Johannes Gutenberg-Universität Mainz	Recruiting	10 Years	N/A	All	33	N/A	Germany
4	NCT03419819 (ClinicalTrials.gov)	May 1, 2018	3/1/2018	Evaluation of PKU Sphere	Evaluation of the Acceptability, Tolerance, and Satiety of PKU Sphere, a Glycomacropeptide (GMP) Based Medical Food in Patients With Phenylketonuria (PKU).	Phenylketonurias	Dietary Supplement: PKU Sphere	Vitaflo International, Ltd	Oregon Health and Science University	Completed	3 Years	N/A	All	28	N/A	United States

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

NCT04224142
(ClinicalTrials.gov)

July 29, 2019

7/10/2019

Evaluation of PKU Sphere in Maternal PKU

An Observational Study to Evaluate Metabolic Control and Dietary Management in Women With PKU Taking PKU Sphere During Pre-conception and/or Pregnancy

Maternal Phenylketonuria

Dietary Supplement: PKU sphere

Vitaflo International, Ltd

University College London Hospitals

Recruiting

16 Years

N/A

Female

United Kingdom

NCT03939052
(ClinicalTrials.gov)

July 19, 2019

1/5/2019

Protein Requirements in Adults With Phenylketonuria (PKU)

Protein Requirements in Phenylketonuria (PKU) Patients Compared Using PKU Sphere™ | Glycomacropeptide (GMP) and an L-amino Acid-based Product

Phenylketonuria

Dietary Supplement: Free amino acids intake

University of British Columbia

Vitaflo International, Ltd

Recruiting

19 Years

50 Years

All

N/A

Canada

NCT03771391
(ClinicalTrials.gov)

December 6, 2018

7/12/2018

A 16 Week Study Evaluating the Introduction of a GMP Based Protein Substitute in Participants With PKU

Multicentre Study Project to Evaluate the New Generation of Protein Supplements With Glycomacropeptide (GMP) in Patients With Phenylketonuria (PKU) Aged 10 Years and Older Over a 16 Week Period.

Phenylketonurias

Dietary Supplement: PKU Sphere

Vitaflo International, Ltd

Kreiskliniken Reutlingen;Universitätsmedizin der Johannes Gutenberg-Universität Mainz

Recruiting

10 Years

N/A

All

N/A

Germany

NCT03419819
(ClinicalTrials.gov)

May 1, 2018

3/1/2018

Evaluation of PKU Sphere

Evaluation of the Acceptability, Tolerance, and Satiety of PKU Sphere, a Glycomacropeptide (GMP) Based Medical Food in Patients With Phenylketonuria (PKU).

Phenylketonurias

Dietary Supplement: PKU Sphere

Vitaflo International, Ltd

Oregon Health and Science University

Completed

3 Years

N/A

All

N/A

United States