Phenylalanine ( DrugBank: Phenylalanine )

1 disease

告示番号	疾患名（ページ内リンク）	臨床試験数
240	フェニルケトン尿症	37

240. フェニルケトン尿症

臨床試験数 : 143 / 薬物数 : 90 - (DrugBank : 10) / 標的遺伝子数 : 1 - 標的パスウェイ数 : 5

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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	NCT05356377 (ClinicalTrials.gov)	May 25, 2022	27/4/2022	Preliminary Study of Brain Effects of Palynziq-Related Changes in Phenylalaninein Individuals With P ...	A Preliminary Study of the Neurological and Neuropsychological Effects of Palynziq-Related Changes i ...	Phenylketonurias	Drug: Pegvaliase-Pqpz	University of Missouri-Columbia	BioMarin Pharmaceutical	Enrolling by invitation	18 Years	55 Years	All	13		United States
2	NCT04375592 (ClinicalTrials.gov)	February 1, 2021	21/4/2020	Acceptability and Tolerance of a Ready-to-use Protein Substitute in Tablet Form for the Dietary Mana ...	XPhe Minis - Acceptability and Tolerance Market Research	Phenylketonurias;Hyperphenylalaninaemia;Tetrahydrobiopterin Deficiency	Dietary Supplement: Phenylalanine-free protein substitute in tablet form (XPhe minis)	metaX Institut fuer Diatetik GmbH	Birmingham Children's Hospital	Recruiting	7 Years	18 Years	All	10		United Kingdom
3	NCT04480567 (ClinicalTrials.gov)	September 24, 2020	8/7/2020	AAV Gene Therapy Study for Subjects With PKU	A Phase 1/2 Open-Label, Dose Escalation Study to Determine the Safety and Efficacy of BMN 307, an Ad ...	Phenylketonuria (PKU)	Drug: BMN 307	BioMarin Pharmaceutical	NULL	Active, not recruiting	15 Years	N/A	All	100	Phase 1/Phase 2	United States;United Kingdom
4	NCT04302194 (ClinicalTrials.gov)	May 1, 2020	7/3/2020	Early Dietary Treated Patients With Phenylketonuria Can Achieve Normal Growth and Mental Development ...	Early Dietary Treated Patients With Phenylketonuria Can Achieve Normal Growth and Mental Development ...	Phenylketonurias	Dietary Supplement: phenylalanine restricted diet	Assiut University	NULL	Not yet recruiting	1 Month	3 Years	All	50		NULL
5	EUCTR2019-001878-28-GB (EUCTR)	17/12/2019	21/11/2019	A Phase 1/2 study to evaluate the safety and efficacy of BMN 307 gene therapy in patients with pheny ...	A Phase 1/2 Open-Label, Dose Escalation Study to Determine the Safety and Efficacy of BMN 307, an Ad ...	Phenylketonuria MedDRA version: 20.0;Level: PT;Classification code 10034872;Term: Phenylketonuria;Sy ...	Product Code: BMN 307	BioMarin Pharmaceutical Inc.	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	100	Phase 1;Phase 2	United States;Taiwan;Spain;Turkey;Australia;Germany;Italy;United Kingdom
6	NCT03788343 (ClinicalTrials.gov)	August 19, 2019	17/12/2018	Phenylalanine and Its Impact on Cognition	PICO: Phenylalanineand Its Impact on Cognition - Impact of Phenylalanineon Cognitive, Cerebral and N ...	Phenylketonuria	Dietary Supplement: Phenylalanine;Drug: Placebo	University Hospital Inselspital, Berne	University of Zurich	Completed	18 Years	N/A	All	90	Phase 4	Switzerland
7	NCT03333720 (ClinicalTrials.gov)	July 15, 2019	18/9/2017	Compliance, Tolerance and Acceptability of a Tablet Protein Substitute for the Dietary Management of ...	Compliance, Tolerance and Acceptability of a Tablet Protein Substitute for the Dietary Management of ...	Phenylketonurias	Dietary Supplement: Phenylalanine-free protein substitute tablets	Nutricia UK Ltd	NULL	Withdrawn	8 Years	100 Years	All	0	N/A	NULL
8	NCT03168399 (ClinicalTrials.gov)	June 8, 2017	24/5/2017	Evaluation of PKU Explore	A Study to Evaluate the Acceptability of PKU Explore, a Renovated PhenylalanineFree Second Stage Pro ...	Phenylketonuria;Inborn Errors of Metabolism	Dietary Supplement: PKU Explore	Vitaflo International, Ltd	Birmingham Women's and Children's NHS Foundation Trust	Completed	6 Months	5 Years	All	22	N/A	United Kingdom
9	NCT03058848 (ClinicalTrials.gov)	March 6, 2017	16/2/2017	Evaluation of PKU Start	A Study to Evaluate the Acceptability of a New PhenylalanineFree Infant Formula for Use in the Dieta ...	Phenylketonuria;Inborn Errors of Metabolism	Dietary Supplement: PKU Start	Vitaflo International, Ltd	NULL	Completed	N/A	2 Years	All	10	N/A	United Kingdom
10	EUCTR2010-021343-41-NL (EUCTR)	21/03/2014	30/11/2011	Variations of blood phenylalanine and tyrosine in children with phenylketonuria under sapropterin	Effect of sapropterin on variations of blood phenylalanineand tyrosine over 24 hours and from day to ...	Phenylketonuria (PKU) is an autosomal recessive metabolic genetic disorder by a mutation in the gene ...	Trade Name: Kuvan	University Medical Center Groningen	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes		Phase 4	Netherlands

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