240. Phenylketonuria Clinical trials / Disease details


Clinical trials : 143 Drugs : 90 - (DrugBank : 10) / Drug target gene : 1 - Drug target pathways : 5

  
34 trials found
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1NCT04272736
(ClinicalTrials.gov)
December 1, 202229/8/2018PKU Low Calorie Drink StudyEvaluating the Compliance, Acceptability, Safety and Tolerance of a Lower Calorie Protein Substitute for the Dietary Management of Phenylketonuria in Children and Adults - a Pilot StudyPhenylketonurias;HyperphenylalaninaemiaDietary Supplement: Low calorie protein substituteNutricia UK LtdNULLNot yet recruiting3 YearsN/AAll30N/ANULL
2NCT05270837
(ClinicalTrials.gov)
June 17, 20221/2/2022Study to Evaluate the Safety and Efficacy of Pegvaliase in Adolescents (Ages 12-17) With PhenylketonuriaA Phase 3 Multi-Center Study to Evaluate the Safety and Efficacy of Subcutaneous Injections of Pegvaliase in Adolescent Subjects (Ages 12-17) With Phenylketonuria Featuring an Open-Label Randomized Two-Arm (Active vs Diet-Only Control) DesignPhenylketonuria (PKU)Drug: Pegvaliase;Other: Diet OnlyBioMarin PharmaceuticalNULLRecruiting12 Years17 YearsAll54Phase 3United States;Germany
3NCT04898829
(ClinicalTrials.gov)
May 18, 202211/5/2021Evaluation of PKU Explore FranceAn Exploratory Study to Evaluate the Acceptability of PKU Explore, a Food for Special Medical Purposes, for Use in the Dietary Management of Phenylketonuria in Infants From 6 Months to 3 Years of Age With Regard to Product-acceptability, Tolerance, Phe Levels and GrowthPhenylketonuriasDietary Supplement: PKU exploreVitaflo International, LtdNULLRecruiting6 Months3 YearsAll10N/AFrance
4NCT05096988
(ClinicalTrials.gov)
November 23, 202114/10/2021Evaluation of PKU Sphere LiquidA Study to Evaluate the Acceptability, Tolerance and Adherence of Children and Adults Consuming PKU Sphere Liquid, a Food for Special Medical Purposes (FSMP), for the Dietary Management of Phenylketonuria (PKU)PhenylketonuriasDietary Supplement: PKU sphere liquidVitaflo International, LtdNULLActive, not recruiting3 YearsN/AAll19N/AUnited Kingdom
5NCT05051657
(ClinicalTrials.gov)
June 14, 202110/9/2021Evaluation of PKU Express PlusA Study To Evaluate The Acceptability Of PKU Express Plus, A Food For Special Medical Purposes (FSMP), In Children And Adults With Phenylketonuria (PKU)Phenylketonurias;PKUDietary Supplement: PKU express plusVitaflo International, LtdNULLRecruiting3 YearsN/AAll15N/AUnited Kingdom
6NCT04375592
(ClinicalTrials.gov)
February 1, 202121/4/2020Acceptability and Tolerance of a Ready-to-use Protein Substitute in Tablet Form for the Dietary Management of PhenylketonuriaXPhe Minis - Acceptability and Tolerance Market ResearchPhenylketonurias;Hyperphenylalaninaemia;Tetrahydrobiopterin DeficiencyDietary Supplement: Phenylalanine-free protein substitute in tablet form (XPhe minis)metaX Institut fuer Diatetik GmbHBirmingham Children's HospitalRecruiting7 Years18 YearsAll10United Kingdom
7NCT05062226
(ClinicalTrials.gov)
September 16, 20206/9/2021GMP Case Studies of Tolerance, Safety and Acceptability in PKU and TYREvaluating the Tolerance, Safety and Acceptability of GMP-based Protein Substitutes for the Dietary Management of Phenylketonuria and Tyrosinaemia in Children and Adults: a Case Study SeriesPKU;TyrosinemiasDietary Supplement: PKU/TYR GMP Protein SubstituteNutricia UK LtdNULLRecruiting3 YearsN/AAll45N/AUnited Kingdom
8NCT04302194
(ClinicalTrials.gov)
May 1, 20207/3/2020Early 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.PhenylketonuriasDietary Supplement: phenylalanine restricted dietAssiut UniversityNULLNot yet recruiting1 Month3 YearsAll50NULL
9NCT04679467
(ClinicalTrials.gov)
January 15, 202026/11/2020Evaluation of PKU Sphere in ItalyAn Acceptability Study to Evaluate the Tolerance, Adherence and Metabolic Control of Patients With Phenylketonuria (PKU) Consuming PKU Sphere™, a Glycomacropeptide (GMP) Based Protein Substitute, When Introduced According to a Practical Guide to PKU Sphere.Phenylketonurias;PKUDietary Supplement: PKU sphereVitaflo International, LtdNULLCompleted3 YearsN/AAll13N/AItaly
10NCT04404530
(ClinicalTrials.gov)
October 8, 201921/5/2020Nutritional Impacts of Palynziq on Patients With Phenylketonuria (PKU)Palynziq and PKU: Treatment Impacts on Diet Quality, Neurological Health, Nutritional Status, and the MetabolomePhenylketonuriasDrug: PalynziqEmory UniversityBioMarin PharmaceuticalRecruiting16 YearsN/AAll45United States
11NCT03924180
(ClinicalTrials.gov)
September 19, 201922/4/2019Glytactin EfficiEncy in Non or Insufficiently Treated Adult PHENylketonuria PatientsGlytactin EfficiEncy in Non or Insufficiently Treated Adult PHENylketonuria PatientsAdult Phenylketonuria Non Treated PatientsDietary Supplement: Dietary Supplement for PKU patientsUniversity Hospital, ToursNULLCompleted18 YearsN/AAll13N/AFrance
12NCT03788343
(ClinicalTrials.gov)
August 19, 201917/12/2018Phenylalanine and Its Impact on CognitionPICO: Phenylalanine and Its Impact on Cognition - Impact of Phenylalanine on Cognitive, Cerebral and Neurometabolic Parameters in Adult Patients With PhenylketonuriaPhenylketonuriaDietary Supplement: Phenylalanine;Drug: PlaceboUniversity Hospital Inselspital, BerneUniversity of ZurichCompleted18 YearsN/AAll90Phase 4Switzerland
13NCT04224142
(ClinicalTrials.gov)
July 29, 20197/10/2019Evaluation of PKU Sphere in Maternal PKUAn Observational Study to Evaluate Metabolic Control and Dietary Management in Women With PKU Taking PKU Sphere During Pre-conception and/or PregnancyMaternal PhenylketonuriaDietary Supplement: PKU sphereVitaflo International, LtdUniversity College London HospitalsRecruiting16 YearsN/AFemale10United Kingdom
14NCT03939052
(ClinicalTrials.gov)
July 19, 20191/5/2019Protein Requirements in Adults With Phenylketonuria (PKU)Protein Requirements in Phenylketonuria (PKU) Patients Compared Using PKU Sphere™ | Glycomacropeptide (GMP) and an L-amino Acid-based ProductPhenylketonuriaDietary Supplement: Free amino acids intakeUniversity of British ColumbiaVitaflo International, LtdRecruiting19 Years50 YearsAll6N/ACanada
15NCT03333720
(ClinicalTrials.gov)
July 15, 201918/9/2017Compliance, Tolerance and Acceptability of a Tablet Protein Substitute for the Dietary Management of PhenylketonuriaCompliance, Tolerance and Acceptability of a Tablet Protein Substitute for the Dietary Management of PhenylketonuriaPhenylketonuriasDietary Supplement: Phenylalanine-free protein substitute tabletsNutricia UK LtdNULLWithdrawn8 Years100 YearsAll0N/ANULL
16NCT03820804
(ClinicalTrials.gov)
January 7, 201919/10/2018Nutritional Status in PhenylketonuriaTrends in Nutritional Status of Patients With PhenylketonuriaPhenylketonuriasOther: Diet;Drug: SapropterinUniversidade do PortoCentro Hospitalar do Porto;BioMarin PharmaceuticalCompleted3 YearsN/AAll94Portugal
17NCT03771391
(ClinicalTrials.gov)
December 6, 20187/12/2018A 16 Week Study Evaluating the Introduction of a GMP Based Protein Substitute in Participants With PKUMulticentre 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.PhenylketonuriasDietary Supplement: PKU SphereVitaflo International, LtdKreiskliniken Reutlingen;Johannes Gutenberg University MainzActive, not recruiting10 YearsN/AAll15N/AGermany
18NCT03419819
(ClinicalTrials.gov)
May 1, 20183/1/2018Evaluation of PKU SphereEvaluation of the Acceptability, Tolerance, and Satiety of PKU Sphere, a Glycomacropeptide (GMP) Based Medical Food in Patients With Phenylketonuria (PKU).PhenylketonuriasDietary Supplement: PKU SphereVitaflo International, LtdOregon Health and Science UniversityCompleted3 YearsN/AAll28N/AUnited States
19NCT02677870
(ClinicalTrials.gov)
January 22, 201826/1/2016The Effectiveness of Kuvan in Amish PKU PatientsThe Effectiveness of High-Dose Synthetic BH4 (Saproterin Dihydrochloride or Kuvan) in Amish PKU PatientsPhenylketonuriaDrug: saproterin dihydrochloride;Other: Diet treatmentUniversity Hospitals Cleveland Medical CenterBioMarin PharmaceuticalCompleted2 Years60 YearsAll7Phase 4United States
20NCT03168399
(ClinicalTrials.gov)
June 8, 201724/5/2017Evaluation of PKU ExploreA Study to Evaluate the Acceptability of PKU Explore, a Renovated Phenylalanine Free Second Stage Protein Substitute for Use in the Dietary Management of Phenylketonuria in Infants From 6 Months to 5 Years of Age With Regard to Product Tolerance and Adherence.Phenylketonuria;Inborn Errors of MetabolismDietary Supplement: PKU ExploreVitaflo International, LtdBirmingham Women's and Children's NHS Foundation TrustCompleted6 Months5 YearsAll22N/AUnited Kingdom
21NCT03167697
(ClinicalTrials.gov)
May 1, 201722/5/2017Evaluating the Efficacy of PKU Synergy in Patients Expressing Phenylketonuria or HyperphenylalaninemiaEvaluating the Efficacy of PKU Synergy in Patients Expressing Phenylketonuria or HyperphenylalaninemiaPhenylketonurias;Hyperphenylalaninaemia, Type IDietary Supplement: Synergy;Other: RoutineNutricia UK LtdNULLCompleted16 Years100 YearsAll14N/AUnited Kingdom
22NCT03058848
(ClinicalTrials.gov)
March 6, 201716/2/2017Evaluation of PKU StartA Study to Evaluate the Acceptability of a New Phenylalanine Free Infant Formula for Use in the Dietary Management of Phenylketonuria in Infants From Birth to 2 Year of Age With Regard to Product Tolerance and Adherence.Phenylketonuria;Inborn Errors of MetabolismDietary Supplement: PKU StartVitaflo International, LtdNULLCompletedN/A2 YearsAll10N/AUnited Kingdom
23NCT02915510
(ClinicalTrials.gov)
July 20168/8/2016GMP Drink for PKU StudyEvaluating the Tolerance, Safety and Acceptability of PKU GMPro, a Whey Protein Derived Feed for the Dietary Management of Phenylketonuria in Children and Adults - a Pilot TrialPhenylketonuriaDietary Supplement: Glycomacropeptide-based protein substituteNutricia UK LtdNULLCompleted3 YearsN/AAll40N/AUnited Kingdom
24NCT02440932
(ClinicalTrials.gov)
November 201430/4/2015Impact of Phenylketonuria-type Diet on Appetite, Appetite Hormones and Diet Induced ThermogenesisImpact of Phenylketonuria-type Diet on Appetite, Appetite Hormones and Diet Induced ThermogenesisPhenylketonuria (PKU)Dietary Supplement: Phenylketonuria-type diet;Other: Normal (control) dietUniversity of GlasgowNULLCompleted18 Years45 YearsBoth26N/AUnited Kingdom
25EUCTR2010-021343-41-NL
(EUCTR)
21/03/201430/11/2011Variations of blood phenylalanine and tyrosine in children with phenylketonuria under sapropterinEffect of sapropterin on variations of blood phenylalanine and tyrosine over 24 hours and from day to day in children with phenylketonuria Phenylketonuria (PKU) is an autosomal recessive metabolic genetic disorder by a mutation in the gene for the enzyme phenylalanine hydroxylase (PAH), rendering it nonfunctional. Left untreated, the disease will result in high concentrations of phenylalanine (Phe) in blood and tissues, likely resulting in severe mental retardation and behavioural problems. Treatment focusus on the restriction of dietary phenylalanine intake with supplementation of a synthetic phenylalaninefree amino acid mixture.;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]Trade Name: KuvanUniversity Medical Center GroningenNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
Phase 4Netherlands
26NCT01965691
(ClinicalTrials.gov)
October 201316/10/2013Protein Requirements in Children With Phenylketonuria (PKU)Application of Stable Isotopes to Determine Protein Requirements in Children With Phenylketonuria (PKU)PhenylketonuriaDietary Supplement: Protein intakeUniversity of British ColumbiaRare Disease Foundation, Vancouver, Canada;Saudi Arabian Cultural Bureau, OttawaCompleted5 Years18 YearsAll4N/ACanada
27NCT01376908
(ClinicalTrials.gov)
June 201117/6/2011Kuvan® in Phenylketonuria Patients Less Than 4 Years OldA Phase IIIb, Multicentre, Open-Label, Randomized, Controlled Study of the Efficacy, Safety, and Population Pharmacokinetics of Sapropterin Dihydrochloride (Kuvan®) in Phenylketonuria (PKU) Patients <4 Years Old.PhenylketonuriaDrug: Kuvan®;Other: Phenylalanine (Phe)-restricted dietBioMarin PharmaceuticalNULLCompletedN/A4 YearsAll56Phase 3Austria;Belgium;Czechia;Germany;Italy;Netherlands;Slovakia;Turkey;United Kingdom;Czech Republic;Portugal
28NCT01412437
(ClinicalTrials.gov)
April 20111/8/2011Neuroimaging and Neurocognitive Assessment and Response to Sapropterin Dihydrochloride Treatment in PhenylketonuriaMultimodal Neuroimaging and Neurocognitive Assessment of Biomarkers and Response to Sapropterin Dihydrochloride Treatment in PhenylketonuriaPKUDietary Supplement: diet;Drug: sapropterin dihydrochlorideChildren's Research InstituteGeorgetown UniversityWithdrawn18 Years40 YearsBoth0N/AUnited States
29NCT00841100
(ClinicalTrials.gov)
December 200810/2/2009Kuvan Therapy in Phenylketonuria (PKU): The Effect of Blood Phenylalanine Concentration on Kuvan ResponseKuvan Therapy in Phenylketonuria (PKU): The Effect of Blood Phenylalanine Concentration on Kuvan ResponsePhenylketonuriaDrug: Kuvan;Other: DietUniversity of MiamiNULLCompleted4 YearsN/AAll21Phase 2United States
30NCT00892554
(ClinicalTrials.gov)
June 20071/5/2009The Effect of Supplemental Docosahexaenoic Acid (DHA) on Neurocognitive Outcomes in Teen and Adult Women With Phenylketonuria(PKU)The Impact of Docosahexaenoic Acid on Neuropsychological Status in Females With PhenylketonuriaPhenylketonuriaDietary Supplement: Docosahexaenoic Acid;Dietary Supplement: Corn/soy oilEmory UniversityAtlanta Clinical and Translational Science InstituteCompleted12 Years50 YearsFemale33N/AUnited States
31EUCTR2005-003777-24-GB
(EUCTR)
17/08/200620/04/2006A Phase 3, Multicenter, Randomized, Double-Blind, Placebo-controlled Study to Evaluate the Safety and Efficacy of Phenoptin 20 mg/kg/day to Increase Phenylalanine Tolerance in Phenylketonuric Children on a Phenylalanine-restricted Diet - NAA Phase 3, Multicenter, Randomized, Double-Blind, Placebo-controlled Study to Evaluate the Safety and Efficacy of Phenoptin 20 mg/kg/day to Increase Phenylalanine Tolerance in Phenylketonuric Children on a Phenylalanine-restricted Diet - NA Phenylketonuria (PKU)
MedDRA version: 6.1;Classification code 10034872
Product Name: Phenoptin
Product Code: T1401
INN or Proposed INN: Sapropterin
Other descriptive name: sapropterin dihydrochloride
BioMarin Pharmaceutical Inc.NULLNot Recruiting Female: yes
Male: yes
300 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): noSpain;Germany;United Kingdom
32EUCTR2005-003777-24-DE
(EUCTR)
24/03/200614/12/2005A Phase 3, Multicenter, Randomized, Double-Blind, Placebo-controlled Study to Evaluate the Safety and Efficacy of Phenoptin 20 mg/kg/day to Increase Phenylalanine Tolerance in Phenylketonuric Children on a Phenylalanine-restricted Diet - NAA Phase 3, Multicenter, Randomized, Double-Blind, Placebo-controlled Study to Evaluate the Safety and Efficacy of Phenoptin 20 mg/kg/day to Increase Phenylalanine Tolerance in Phenylketonuric Children on a Phenylalanine-restricted Diet - NA Phenylketonuria (PKU)
MedDRA version: 6.1;Classification code 10034872
Product Name: Phenoptin
Product Code: T1401
INN or Proposed INN: Sapropterin
Other descriptive name: sapropterin dihydrochloride
BioMarin Pharmaceutical Inc.NULLNot RecruitingFemale: yes
Male: yes
300Phase 3Germany;Spain
33EUCTR2005-003777-24-ES
(EUCTR)
01/03/200619/01/2006Estudio fase 3, multicéntrico, aleatorizado, doble ciego y controlado con placebo para evaluar la seguridad y la eficacia de PhenoptinTM en dosis de 20 mg/kg/dia para aumentar la tolerancia a la fenilalanina en niños fenilcetonúricos que siguen dietas restrictivas en fenilalanina.Estudio fase 3, multicéntrico, aleatorizado, doble ciego y controlado con placebo para evaluar la seguridad y la eficacia de PhenoptinTM en dosis de 20 mg/kg/dia para aumentar la tolerancia a la fenilalanina en niños fenilcetonúricos que siguen dietas restrictivas en fenilalanina. Fenilcetonuria (Phenylketonuria-PKU)
MedDRA version: 6.1;Classification code 10034872
Product Name: Phenoptin
Product Code: T1401
INN or Proposed INN: Sapropterin
Other descriptive name: Sapropterin dihydrochloride
BioMarin Pharmaceutical Inc.NULLNot RecruitingFemale: yes
Male: yes
300Phase 3Spain;Germany;United Kingdom
34EUCTR2004-002365-21-DK
(EUCTR)
03/11/200409/07/2008Trial with BH4, a new and simple treatment of phenylketonuria, PKU - BH4 and PKUTrial with BH4, a new and simple treatment of phenylketonuria, PKU - BH4 and PKU PKU, phenylketonuria, is a rare, inherited metabolic disease that results in mental retardation if not a very strict low-protein diet is started within the first weeks of life. The conversion of phenylalanine to tyrosine is defect, phe accumulates and leads to brain damage. There are different degrees of severity, reflecting the spectrum of mutant genes. BH4, tetrahydrobiopterin, is co-enzym for the conversion of phe to tyrosine. BH4 can lower phe in some patients with milder forms of PKU.Product Name: tetrahydrobiopterinJohn F. Kennedy InstituteNULLNot RecruitingFemale: yes
Male: yes
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