240. フェニルケトン尿症 Phenylketonuria Clinical trials / Disease details


臨床試験数 : 138 薬物数 : 106 - (DrugBank : 11) / 標的遺伝子数 : 1 - 標的パスウェイ数 : 5

  
No.TrialIDDate_
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Public_titleScientific_titleConditionInterventionPrimary_
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agemin
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PhaseCountries
1NCT01465100
(ClinicalTrials.gov)
December 15, 202220/10/2011Liver Cell Transplant for PhenylketonuriaHepatocyte Transplantation for PhenylketonuriaPhenylketonuriaRadiation: Preparative Radiation Therapy;Procedure: Hepatocyte Transplant;Drug: Immunosuppression;Other: Liver Evaluation;Behavioral: Neuro-psychological Assessment;Diagnostic Test: Whole body Phe oxidation testing;Procedure: Liver BiopsyIra FoxNULLRecruiting14 Years55 YearsAll10Phase 1/Phase 2United States
2NCT05222178
(ClinicalTrials.gov)
March 202221/12/2021Safety and Efficacy of HMI-103, a Gene Editing Development Candidate in Adults With Classical PKU Due to PAH DeficiencyA Phase 1, Open-Label, Dose-Escalation Study to Evaluate the Safety and Efficacy of HMI-103 Administered Intravenously in Adult Participants With Classical PKU Due to PAH DeficiencyPhenylketonurias;PAH Deficiency;PhenylketonuriaDrug: HMI-103Homology Medicines, IncNULLNot yet recruiting18 Years55 YearsAll9Phase 1United States
3NCT05166161
(ClinicalTrials.gov)
February 14, 20228/12/2021A Long-Term Safety Study of PTC923 in Participants With PhenylketonuriaA Phase 3 Open-Label Extension Study of PTC923 in PhenylketonuriaPhenylketonuriaDrug: PTC923PTC TherapeuticsNULLRecruitingN/AN/AAll200Phase 3United States;Australia;Brazil;Canada;Denmark;France;Georgia;Germany;Italy;Mexico;Netherlands;Portugal;Spain;Turkey;United Kingdom
4EUCTR2021-000474-29-PT
(EUCTR)
02/02/202229/11/2021A Phase 3 Study of PTC923 in Subjects with PhenylketonuriaA Phase 3 Study of PTC923 in Subjects with Phenylketonuria Metabolic Disorders - Phenylketonuria
MedDRA version: 20.0;Level: LLT;Classification code 10034873;Term: Phenylketonuria (PKU);System Organ Class: 100000004850;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03]
Product Code: PTC923
INN or Proposed INN: not available
Other descriptive name: (S)-2-amino-6-(2-hydroxypropanoyl)-7,8-dihydropteridin-4(3H)-one
Product Code: PTC923
INN or Proposed INN: not available
Other descriptive name: (S)-2-amino-6-(2-hydroxypropanoyl)-7,8-dihydropteridin-4(3H)-one
PTC Therapeutics, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
178Phase 3United States;Portugal;Spain;Turkey;United Kingdom;Italy;France;Mexico;Canada;Brazil;Australia;Denmark;Georgia;Germany;Netherlands
5NCT05096988
(ClinicalTrials.gov)
November 23, 202114/10/2021A 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)A 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, LtdNULLRecruiting3 YearsN/AAll15N/AUnited Kingdom
6NCT05099640
(ClinicalTrials.gov)
September 30, 20216/10/2021A Study of PTC923 in Participants With PhenylketonuriaA Phase 3 Study of PTC923 in Subjects With PhenylketonuriaPhenylketonuriaDrug: PTC923;Drug: PlaceboPTC TherapeuticsNULLRecruitingN/AN/AAll178Phase 3United States;Australia;Brazil;Canada;Denmark;France;Georgia;Germany;Italy;Mexico;Netherlands;Portugal;Spain;Turkey;United Kingdom
7NCT04898829
(ClinicalTrials.gov)
September 3, 202111/5/2021Evaluation 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
8NCT05051657
(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
9EUCTR2021-001174-29-NL
(EUCTR)
31/05/202131/03/2021Effectiveness of methylphenidate for treating ADHD in adultswith PKUEffectiveness of methylphenidate in adults with phenylketonuria and attention-deficit/hyperactivity disorder: An N-of-1 series - MPH4PKU Phenylketonuria, ADHD;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]Trade Name: MedikinetAmsterdam UMCNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
5Phase 4Netherlands
10NCT04879277
(ClinicalTrials.gov)
May 26, 20214/5/2021Study of Low-grade Systemic Inflammation in Adult Patients With PhenylketonuriaEtude de l'INflammation systémique de Bas GRade Chez Les Patients Adultes Atteints de PHénylcétonuriePhenylketonuriaBiological: Blood samplesUniversity Hospital, ToursNULLCompleted18 YearsN/AAll40N/AFrance
11NCT04375592
(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
12NCT04256655
(ClinicalTrials.gov)
December 1, 202028/1/2020Safety, Tolerability, Pharmacodynamics and Pharmacokinetics of CDX 6114 in PKU PatientsA Phase 1, Multi-Center, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety, Tolerability, Pharmacodynamics and Pharmacokinetics of CDX 6114 After Multiple Ascending Oral Dose Administration to Patients With Phenylketonuria (PKU).PhenylketonuriasDrug: cohort 1 0.225g;Drug: Cohort 2 0.75g;Drug: Cohort 3 2.25 gNestléNULLWithdrawn18 Years65 YearsAll0Phase 1NULL
13NCT04480567
(ClinicalTrials.gov)
September 24, 20208/7/2020AAV Gene Therapy Study for Subjects With PKUA Phase 1/2 Open-Label, Dose Escalation Study to Determine the Safety and Efficacy of BMN 307, an Adeno-Associated Virus Vector-Mediated Gene Transfer of Human Phenylalanine Hydroxylase in Subjects With PhenylketonuriaPhenylketonuria (PKU)Drug: BMN 307BioMarin PharmaceuticalNULLActive, not recruiting15 YearsN/AAll100Phase 1/Phase 2United States;United Kingdom
14NCT05062226
(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
15NCT04272736
(ClinicalTrials.gov)
September 1, 202029/8/2018PKU 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
16NCT04534842
(ClinicalTrials.gov)
August 25, 202024/8/2020An Open-label Study of the Efficacy and Safety of SYNB1618 and SYNB1934 in Patients With Phenylketonuria (SynPheny-1)An Open-label Study of the Efficacy and Safety of SYNB1618 and SYNB1934 in Patients With Phenylketonuria (SynPheny-1)PhenylketonuriaDrug: SYNB1618;Drug: SYNB1934SynlogicNULLRecruiting18 YearsN/AAll24Phase 2United States
17NCT04227080
(ClinicalTrials.gov)
June 202010/1/2020BH4 Responsiveness in PAH Deficiency PKU PatientsTo Evaluate BH4 Responsiveness in PAH Deficiency PKU Patients Who Failed to Achieve 30% Blood Phe Reduction Within 24-hour BH4 Loading Test by Extending the Period of BH4 Response Test: A Pilot Study in TaiwanPku Phenylketonuria;PAH DeficiencyDrug: BH4Taipei Veterans General Hospital, TaiwanBioMarin PharmaceuticalNot yet recruitingN/AN/AAll40Phase 4NULL
18NCT04302194
(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
19NCT04110496
(ClinicalTrials.gov)
January 29, 202023/9/2019Safety and Tolerability of RTX-134 in Adults With PhenylketonuriaA Phase 1b Open-Label Single Dose Safety, Tolerability, and Pharmacokinetics Study of RTX-134 in Adults With PhenylketonuriaPhenylketonuriasDrug: RTX-134Rubius TherapeuticsNULLActive, not recruiting18 YearsN/AAll12Phase 1United States
20NCT04679467
(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, LtdNULLRecruiting3 YearsN/AAll20N/AItaly
21EUCTR2019-001878-28-GB
(EUCTR)
17/12/201921/11/2019A Phase 1/2 study to evaluate the safety and efficacy of BMN 307 gene therapy in patients with phenylketonuriaA Phase 1/2 Open-Label, Dose Escalation Study to Determine the Safety and Efficacy of BMN 307, an Adeno-Associated Virus Vector-Mediated Gene Transfer of Human Phenylalanine Hydroxylase in Subjects with Phenylketonuria and Plasma Phe Levels > 600 µmol/L - A Phase 1/2 study to evaluate the safety and efficacy of BMN 307 Phenylketonuria
MedDRA version: 20.0;Level: PT;Classification code 10034872;Term: Phenylketonuria;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Product Code: BMN 307BioMarin Pharmaceutical Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
100Phase 1;Phase 2United States;Taiwan;Spain;Turkey;Australia;Germany;Italy;United Kingdom
22NCT04404530
(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
23NCT03924180
(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, ToursINSERM CIC 1415Recruiting18 YearsN/AAll32N/AFrance
24NCT03788343
(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 ZurichRecruiting18 YearsN/AAll60Phase 4Switzerland
25NCT04014712
(ClinicalTrials.gov)
August 20193/7/2019O2 Transport and Utilization in Health and Lung DiseaseRole of Nitric Oxide Coupling in Muscle Dysfunction With COPDCOPD;Tetrahydrobiopterin Deficiency;Oxidative StressDrug: Tetrahydrobiopterin;Drug: Placebo oral tabletUniversity of Massachusetts, AmherstNULLNot yet recruiting18 Years85 YearsAll30Phase 1United States
26NCT04224142
(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
27NCT03939052
(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
28NCT03333720
(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
29EUCTR2018-000648-25-FR
(EUCTR)
05/07/201915/10/2018A Phase 3b, Open-Label, Single Arm, Multi-Centre Study to Assess the Safety and Efficacy of Pegvaliase (BMN 165) Treatment in adults With Phenylketonuria Not Controlled With Current ManagementA Phase 3b, Open-Label, Single Arm, Multi-Centre Study to Assess the Safety and Efficacy of Pegvaliase (BMN 165) Treatment in adults With Phenylketonuria Not Controlled With Current Management - OPTIC Study Phenylketonuria (PKU)
MedDRA version: 20.0;Level: LLT;Classification code 10034873;Term: Phenylketonuria (PKU);System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Product Name: Pegvaliase
INN or Proposed INN: PEGVALIASE
Product Name: Pegvaliase
INN or Proposed INN: PEGVALIASE
Product Name: Pegvaliase
INN or Proposed INN: PEGVALIASE
BioMarin Pharmaceutical Inc.NULLNot Recruiting Female: yes
Male: yes
76 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): noItaly;Germany;Netherlands;Russian Federation;Austria;Turkey;Spain;France
30EUCTR2018-000648-25-DE
(EUCTR)
28/06/201930/08/2018A Phase 3b, Open-Label, Single Arm, Multi-Centre Study to Assess the Safety and Efficacy of Pegvaliase (BMN 165) Treatment in adults With Phenylketonuria Not Controlled With Current ManagementA Phase 3b, Open-Label, Single Arm, Multi-Centre Study to Assess the Safety and Efficacy of Pegvaliase (BMN 165) Treatment in adults With Phenylketonuria Not Controlled With Current Management - OPTIC Study Phenylketonuria (PKU)
MedDRA version: 20.0;Level: LLT;Classification code 10034873;Term: Phenylketonuria (PKU);System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Product Name: Pegvaliase
INN or Proposed INN: PEGVALIASE
Product Name: Pegvaliase
INN or Proposed INN: PEGVALIASE
Product Name: Pegvaliase
INN or Proposed INN: PEGVALIASE
BioMarin Pharmaceutical Inc.NULLNot Recruiting Female: yes
Male: yes
85 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): noFrance;Spain;Turkey;Austria;Netherlands;Germany;Italy
31JPRN-JapicCTI-194642
13/6/201925/02/2019A Phase 3 Study to Assess the Safety and Efficacy of BMN 165 in Japanese Adults With PhenylketonuriaA Phase 3, Open-Label, Multi-Center Study to Assess the Safety and Efficacy of BMN 165 in Japanese Subjects 18 Years of Age and Older With Phenylketonuria PhenylketonuriaIntervention name : BMN 165
INN of the intervention : pegvaliase
Dosage And administration of the intervention : BMN 165 will be administered SC at dose levels of 2.5 to 60 mg. The minimum dose is a single weekly dose of 2.5 mg (for a total weekly dose of 2.5 mg). The maximum allowable daily dose is 40 mg/day (for a maximum weekly dose of 280 mg) after a minimum of 24 weeks on 20 mg/day during Part 1. Subjects may increase dose up to 60 mg/day in Part 2 (for a maximum weekly dose of 420 mg) after a minimum of 16 weeks on 40 mg/day.
Control intervention name : -
INN of the control intervention : -
Dosage And administration of the control intervention : -
BioMarin Pharmaceutical Inc./CMIC Co., Ltd.NULLcomplete1870BOTH10Phase 3Japan
32NCT04085666
(ClinicalTrials.gov)
June 1, 20193/8/2019Pharmacodynamics, Safety, Tolerability and Pharmacokinetics of CDX-6114 in Patients With Phenylketonuria (PKU)A Phase 1, Multi-center, Randomized, Double-blind, Placebo-controlled, Cross-over Study to Evaluate the Pharmacodynamics, Safety, Tolerability and Pharmacokinetics of a Single Oral Dose of CDX-6114 in Patients With Phenylketonuria (PKU).PhenylketonuriaDrug: CDX 6114;Other: Matching PlaceboNestléNULLCompleted18 Years55 YearsAll18Phase 1Australia;Germany
33NCT03792451
(ClinicalTrials.gov)
January 17, 201931/12/2018Nutrition Status of Adults Treated With PegvaliaseNutrition Status of Adults With Phenylketonuria Treated With PegvaliasePhenylketonuriasDrug: PegvaliaseBoston Children's HospitalBioMarin PharmaceuticalCompleted18 Years65 YearsAll18United States
34NCT03820804
(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
35NCT03771391
(ClinicalTrials.gov)
December 6, 20187/12/2018Multicentre 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.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.PhenylketonuriasDietary Supplement: PKU SphereVitaflo International, LtdKreiskliniken Reutlingen;Johannes Gutenberg University MainzActive, not recruiting10 YearsN/AAll15N/AGermany
36NCT03694353
(ClinicalTrials.gov)
September 13, 20184/9/2018Safety and Efficacy of Self Administered Injections of Pegvaliase (>40mg/Day Dose) in Adults With PKUAn Open-label Extension Study to Evaluate the Safety and Efficacy of Subcutaneous Injections of Pegvaliase (> 40 mg/Day Dose) in Adults With PhenylketonuriaPhenylketonuria (PKU)Drug: PegvaliaseBioMarin PharmaceuticalNULLCompleted18 Years70 YearsAll37Phase 3United States
37NCT03519711
(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 TherapeuticsNULLCompleted12 MonthsN/AAll6Phase 1/Phase 2United States;Germany
38NCT03419819
(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
39NCT03516487
(ClinicalTrials.gov)
April 17, 201811/4/2018Safety and Tolerability of SYNB1618 in Healthy Adult Volunteers and Adult Subjects With PhenylketonuriaA Phase 1/2a, First-in-human, Oral Single and Multiple Dose-escalation, Randomized, Double-blinded, Placebo-controlled Study of SYNB1618 in Healthy Adult Volunteers and Adult Subjects With Phenylketonuria to Evaluate Safety, Tolerability, Kinetics, and PharmacodynamicsPhenylketonuria;HealthyDrug: SYNB1618;Drug: PlaceboSynlogicNULLCompleted18 Years64 YearsAll70Phase 1/Phase 2United States
40NCT02677870
(ClinicalTrials.gov)
January 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 PharmaceuticalRecruiting2 Years60 YearsAll25Phase 4United States
41NCT03864029
(ClinicalTrials.gov)
October 10, 201722/2/2019Retrospective Observational Safety Effectiveness With Kuvan in hpAAn Observational Study Research to Collect the Effectiveness and Safety Data of KUVAN® Retrospectively in Chinese Subjects With Hyperphenylalaninemia (HPA) Caused by Tetrahydrobiopterin (BH4) DeficiencyTetrahydrobiopterin DeficiencyDrug: KUVANBioMarin PharmaceuticalQuintiles, Inc.CompletedN/AN/AAll26China
42NCT03168399
(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
43NCT03167697
(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
44NCT03058848
(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
45NCT02915510
(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
46NCT02440932
(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
47EUCTR2010-024311-13-NL
(EUCTR)
13/05/201427/01/2014A clinical study, carried out in different centers and using a placebo, to assess the effect of a medicinal product, containing sapropterin as the active substance, on mental abilities in young adults suffering from phenylketonuria, a genetic disease in which an enzyme defect leads to accumulation of a waste product (phenylketone) which can be detected in urine.A Multicenter, Double-Blind, Placebo Controlled, Randomized, 2-Arm Phase IIa Pilot Trial Assessing the Effect of Sapropterin on Cognitive Abilities in Young Adults with Phenylketonuria - SIGNAL (SapropterIn on coGnitive abilities in youNg Adults with phenyLketonuria) Phenylketonuria
MedDRA version: 16.1;Level: LLT;Classification code 10034873;Term: Phenylketonuria (PKU);System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Trade Name: Kuvan®
INN or Proposed INN: Sapropterin
Other descriptive name: SAPROPTERIN DIHYDROCHLORIDE
Merck KGaANULLNot RecruitingFemale: yes
Male: yes
30Phase 2France;Spain;Belgium;Germany;Netherlands;Italy;Switzerland
48EUCTR2010-021343-41-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
49NCT01977820
(ClinicalTrials.gov)
February 201431/10/2013Sapropterin on Cognitive Abilities in Young Adults With PhenylketonuriaA Multicenter, Double-Blind, Placebo-Controlled, Randomized, 2-Arm Phase IIa Pilot Trial Assessing the Effect of Sapropterin on Cognitive Abilities in Young Adults With PhenylketonuriaPhenylketonuriaDrug: Sapropterin;Drug: PlaceboBioMarin PharmaceuticalNULLTerminated18 Years29 YearsAll2Phase 2Italy;Switzerland;Belgium;Germany;Netherlands;Spain
50EUCTR2010-024311-13-DE
(EUCTR)
02/01/201423/07/2013A clinical study, carried out in different centers and using a placebo, to assess the effect of a medicinal product, containing sapropterin as the active substance, on mental abilities in young adults suffering from phenylketonuria, a genetic disease in which an enzyme defect leads to accumulation of a waste product (phenylketone) which can be detected in urine.A Multicenter, Double-Blind, Placebo Controlled, Randomized, 2-Arm Phase IIa Pilot Trial Assessing the Effect of Sapropterin on Cognitive Abilities in Young Adults with Phenylketonuria - SIGNAL (SapropterIn on coGnitive abilities in youNg Adults with phenyLketonuria) Phenylketonuria
MedDRA version: 16.0;Level: LLT;Classification code 10034873;Term: Phenylketonuria (PKU);System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Trade Name: Kuvan®
INN or Proposed INN: Sapropterin
Other descriptive name: SAPROPTERIN DIHYDROCHLORIDE
Merck KGaANULLNot RecruitingFemale: yes
Male: yes
30Phase 2France;Belgium;Spain;Netherlands;Germany;Italy;Switzerland
51EUCTR2010-024311-13-BE
(EUCTR)
23/12/201325/09/2013A clinical study, carried out in different centers and using a placebo, to assess the effect of a medicinal product, containing sapropterin as the active substance, on mental abilities in young adults suffering from phenylketonuria, a genetic disease in which an enzyme defect leads to accumulation of a waste product (phenylketone) which can be detected in urine.A Multicenter, Double-Blind, Placebo Controlled, Randomized, 2-Arm Phase IIa Pilot Trial Assessing the Effect of Sapropterin on Cognitive Abilities in Young Adults with Phenylketonuria - SIGNAL (SapropterIn on coGnitive abilities in youNg Adults with phenyLketonuria) Phenylketonuria
MedDRA version: 16.0;Level: LLT;Classification code 10034873;Term: Phenylketonuria (PKU);System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Trade Name: Kuvan®
INN or Proposed INN: Sapropterin
Other descriptive name: SAPROPTERIN DIHYDROCHLORIDE
Merck KGaANULLNot RecruitingFemale: yes
Male: yes
30Phase 2France;Spain;Belgium;Netherlands;Germany;Italy;Switzerland
52EUCTR2010-024311-13-ES
(EUCTR)
21/11/201301/10/2013A clinical study, carried out in different centers and using a placebo, to assess the effect of a medicinal product, containing sapropterin as the active substance, on mental abilities in young adults suffering from phenylketonuria, a genetic disease in which an enzyme defect leads to accumulation of a waste product (phenylketone) which can be detected in urine.A Multicenter, Double-Blind, Placebo Controlled, Randomized, 2-Arm Phase IIa Pilot Trial Assessing the Effect of Sapropterin on Cognitive Abilities in Young Adults with Phenylketonuria - SIGNAL (SapropterIn on coGnitive abilities in youNg Adults with phenyLketonuria) Phenylketonuria
MedDRA version: 16.0;Level: LLT;Classification code 10034873;Term: Phenylketonuria (PKU);System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Trade Name: Kuvan®
INN or Proposed INN: Sapropterin
Other descriptive name: SAPROPTERIN DIHYDROCHLORIDE
Merck KGaANULLNot RecruitingFemale: yes
Male: yes
30Phase 2France;Belgium;Spain;Netherlands;Germany;Italy;Switzerland
53EUCTR2009-015844-41-ES
(EUCTR)
31/10/201316/01/2014A Study of the Long-term Effects on Mental Abilities of 4 to 5 Year-old Children with a Disease Caused by an Enzyme Defect (Phenylketonuria) that Have Been Treated with Kuvan® (a Medicinal Product aimed at Restoring the Defect) for 7 Years.A Phase IV Open-Label, Single-Cohort Study of the Long-Term Neurocognitive Outcomes in 4 to 5 Year-Old Children with Phenylketonuria Treated with Sapropterin Dihydrochloride (Kuvan®) for 7 Years. - KOGNITO (Kuvan®'s effect on the cOGNITion of children with phenylketOnuria) Phenylketonuria
MedDRA version: 16.1;Level: LLT;Classification code 10034873;Term: Phenylketonuria (PKU);System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Trade Name: Kuvan
INN or Proposed INN: SAPROPTERIN
Other descriptive name: tetrahydrobiopterin
Merck KGaANULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
30Phase 4Spain;Germany;United Kingdom
54NCT01965691
(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
55NCT01965912
(ClinicalTrials.gov)
October 201316/10/2013Kuvan®'s Effect on the Cognition of Children With PhenylketonuriaA Phase 4 Open-Label, Single-Cohort Study of the Long-Term Neurocognitive Outcomes in 4 to 5 Year-Old Children With Phenylketonuria Treated With Sapropterin Dihydrochloride (Kuvan®) for 7 YearsPhenylketonuriaDrug: Kuvan®BioMarin PharmaceuticalNULLActive, not recruiting4 Years5 YearsAll34Phase 4Germany;Italy;Spain;United Kingdom
56EUCTR2010-024311-13-IT
(EUCTR)
21/09/201326/07/2013A clinical study, carried out in different centers and using a placebo, to assess the effect of a medicinal product, containing sapropterin as the active substance, on mental abilities in young adults suffering from phenylketonuria, a genetic disease in which an enzyme defect leads to accumulation of a waste product (phenylketone) which can be detected in urine.A Multicenter, Double-Blind, Placebo Controlled, Randomized, 2-Arm Phase IIa Pilot Trial Assessing the Effect of Sapropterin on Cognitive Abilities in Young Adults with Phenylketonuria - SIGNAL (SapropterIn on coGnitive abilities in youNg Adults with phenyLketonuria) Phenylketonuria
MedDRA version: 14.1;Level: LLT;Classification code 10034873;Term: Phenylketonuria (PKU);System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Trade Name: Kuvan®
INN or Proposed INN: Sapropterin
Other descriptive name: SAPROPTERIN DIHYDROCHLORIDE
Merck KGaANULLNot RecruitingFemale: yes
Male: yes
30Phase 2France;Belgium;Spain;Netherlands;Germany;Switzerland;Italy
57EUCTR2009-015844-41-DE
(EUCTR)
10/09/201313/06/2013A Study of the Long-term Effects on Mental Abilities of 4 to 5 Year-old Children with a Disease Caused by an Enzyme Defect (Phenylketonuria) that Have Been Treated with Kuvan® (a Medicinal Product aimed at Restoring the Defect) for 7 Years.A Phase IV Open-Label, Single-Cohort Study of the Long-Term Neurocognitive Outcomes in 4 to 5 Year-Old Children with Phenylketonuria Treated with Sapropterin Dihydrochloride (Kuvan®) for 7 Years. - KOGNITO (Kuvan®’s effect on the cOGNITion of children with phenylketOnuria) Phenylketonuria
MedDRA version: 20.0;Level: LLT;Classification code 10034873;Term: Phenylketonuria (PKU);System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Trade Name: Kuvan
INN or Proposed INN: SAPROPTERIN
Other descriptive name: tetrahydrobiopterin
BioMarin International Ltd.NULLAuthorised-recruitment may be ongoing or finished Female: yes
Male: yes
60 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): yesSpain;Germany;Italy;United Kingdom
58EUCTR2009-015844-41-IT
(EUCTR)
02/09/201325/06/2013A Study of the Long-term Effects on Mental Abilities of 4 to 5 Year-old Children with a Disease Caused by an Enzyme Defect (Phenylketonuria) that Have Been Treated with Kuvan® (a Medicinal Product aimed at Restoring the Defect) for 7 Years.A Phase IV Open-Label, Single-Cohort Study of the Long-Term Neurocognitive Outcomes in 4 to 5 Year-Old Children with Phenylketonuria Treated with Sapropterin Dihydrochloride (Kuvan®) for 7 Years. - KOGNITO (Kuvan®’s effect on the cOGNITion of children with phenylketOnuria) Phenylketonuria
MedDRA version: 14.1;Level: LLT;Classification code 10034873;Term: Phenylketonuria (PKU);System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Trade Name: Kuvan
INN or Proposed INN: SAPROPTERIN
Other descriptive name: tetrahydrobiopterin
Merck KGaANULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
30Phase 4Spain;Germany;United Kingdom;Italy
59NCT01889862
(ClinicalTrials.gov)
July 29, 201318/6/2013Phase 3 Study to Evaluate the Efficacy & Safety of Self-Administered Injections of BMN165 by Adults With PKUA Four-Part, Phase 3, Randomized, Double-Blind, Placebo- Controlled, Four-Arm, Discontinuation Study to Evaluate the Efficacy and Safety of Subcutaneous Injections of BMN 165 Self-Administered by Adults With Phenylketonuria (PKU)Phenylketonuria (PKU)Drug: BMN165 20mg/day;Drug: BMN165 40mg/day;Drug: PlaceboBioMarin PharmaceuticalNULLCompleted18 Years70 YearsAll215Phase 3United States
60EUCTR2009-015844-41-GB
(EUCTR)
25/07/201313/06/2013A Study of the Long-term Effects on Mental Abilities of 4 to 5 Year-old Children with a Disease Caused by an Enzyme Defect (Phenylketonuria) that Have Been Treated with Kuvan® (a Medicinal Product aimed at Restoring the Defect) for 7 Years.A Phase IV Open-Label, Single-Cohort Study of the Long-Term Neurocognitive Outcomes in 4 to 5 Year-Old Children with Phenylketonuria Treated with Sapropterin Dihydrochloride (Kuvan®) for 7 Years. - KOGNITO (Kuvan®’s effect on the cOGNITion of children with phenylketOnuria) Phenylketonuria
MedDRA version: 19.0;Level: LLT;Classification code 10034873;Term: Phenylketonuria (PKU);System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Trade Name: Kuvan
Product Name: Kuvan
INN or Proposed INN: SAPROPTERIN
Other descriptive name: tetrahydrobiopterin
BioMarin International Ltd.NULLNot RecruitingFemale: yes
Male: yes
30Phase 4Spain;Germany;Italy;United Kingdom
61NCT01819727
(ClinicalTrials.gov)
May 201318/3/2013An Open-Label Phase 3 Study of BMN 165 for Adults With PKU Not Previously Treated w/ BMN 165A Phase 3, Open-Label, Randomized, Multi-Center Study to Assess the Safety & Tolerability of an Induction, Titration, and Maintenance Dose Regimen of BMN 165 Self Administered by Adults With PKU Not Previously Treated With BMN 165PhenylketonuriaDrug: BMN 165BioMarin PharmaceuticalNULLCompleted18 Years70 YearsAll261Phase 3United States
62NCT01806051
(ClinicalTrials.gov)
March 20134/3/2013A Pilot Study on Diurnal VariationA Pilot Study on the Diurnal Variation in PKU Patients With KuvanPhenylketonuria (PKU)Drug: KuvanDr. Linda RandolphBioMarin PharmaceuticalTerminated4 YearsN/AAll6N/AUnited States
63NCT01732471
(ClinicalTrials.gov)
November 201219/11/2012Phase 3 Open-label Study to Evaluate the Response and Safety of Kuvan® in Subjects With PhenylketonuriaA Phase III Non-comparative Open-label Clinical Study to Evaluate the Response to and Safety of Kuvan (Sapropterin Dihydrochloride) After 6 Weeks of Treatment in Patients of 4 to 18 Years of Age With Phenylketonuria Who Have Elevated Blood Phenylalanine LevelsPhenylketonuriaDrug: Kuvan®Merck KGaANULLCompleted4 Years18 YearsAll90Phase 3Germany
64NCT01560286
(ClinicalTrials.gov)
May 20125/3/2012A Study to Evaluate Subcutaneously Administered rAvPAL-PEG in Patients With Phenylketonuria for 24 WeeksA Phase II, Multi-center, Open-label, Dose-finding Study to Evaluate Safety, Efficacy and Tolerability of Subcutaneously (SC) Administered rAvPAL-PEG in Patients With PKU for 24 WeeksPhenylketonuriaBiological: BMN 165 (rAvPAL-PEG)BioMarin PharmaceuticalNULLCompleted16 Years70 YearsAll24Phase 2United States
65NCT01617070
(ClinicalTrials.gov)
May 20128/6/2012Effects of Kuvan on Melatonin SecretionPilot Study to Evaluate Melatonin Secretion as a Marker of Decreased Serotonin in Individuals With PKU: Evaluation of the CNS Effects of TetrahydrobiopterinPhenylketonuria (PKU)Drug: Kuvan;Dietary Supplement: Large Neutral Amino Acid TherapyUniversity of Southern CaliforniaBioMarin PharmaceuticalCompleted18 YearsN/AAll10Phase 4United States
66NCT01541397
(ClinicalTrials.gov)
June 201111/7/2011Bone Mineral Density in Adults With Hyperphenylalaninemia on Kuvan TherapyBone Mineral Density in Adults With Hyperphenylalaninemia on Kuvan TherapyHyperphenylalaninemia;PhenylketonuriaDrug: SapropterinThe University of Texas Health Science Center, HoustonBioMarin PharmaceuticalTerminated18 Years50 YearsAll6N/AUnited States
67NCT01395394
(ClinicalTrials.gov)
June 201123/6/2011Phenylketonuria, Oxidative Stress, and BH4The Ability of Kuvan® (Sapropterin Dihydrochloride) to Prevent Meal-induced Lipid Peroxidation and Endothelial Dysfunction in Patients With Phenylketonuria: a Pilot StudyPhenylketonuriaDrug: Kuvan;Other: Meal ChallengeEmory UniversityNULLTerminated10 Years45 YearsAll12Phase 2United States
68NCT01376908
(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
69EUCTR2009-015768-33-DE
(EUCTR)
20/05/201104/11/2010Safety Paediatric efficAcy phaRmacokinetic with Kuvan®A 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 - SPARK (Safety Paediatric efficAcy phaRmacokinetic with Kuvan®) Phenylketonuria
MedDRA version: 19.0;Level: LLT;Classification code 10034873;Term: Phenylketonuria (PKU);System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Trade Name: Kuvan
Product Name: Sapropterin Dihydrocholoride
Product Code: NAP
INN or Proposed INN: SAPROPTERIN
Other descriptive name: tetrahydrobiopterin
BioMarin International LtdNULLNot RecruitingFemale: yes
Male: yes
56Phase 3Portugal;Czech Republic;Slovakia;Belgium;Turkey;Austria;Netherlands;Germany;Italy;United Kingdom
70EUCTR2009-015768-33-PT
(EUCTR)
13/05/201116/12/2010Safety Paediatric efficAcy phaRmacokinetic with Kuvan®A 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 - SPARK (Safety Paediatric efficAcy phaRmacokinetic with Kuvan®) Phenylketonuria
MedDRA version: 14.1;Level: LLT;Classification code 10034873;Term: Phenylketonuria (PKU);System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Trade Name: Kuvan
Product Name: Sapropterin Dihydrocholoride
Product Code: NAP
INN or Proposed INN: SAPROPTERIN
Other descriptive name: tetrahydrobiopterin
Merck Serono S.A. - GenevaNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
50Phase 3Austria;Italy;Belgium;Netherlands;Turkey;United Kingdom;Slovakia;Germany;Czech Republic;Portugal
71EUCTR2009-015768-33-NL
(EUCTR)
03/05/201114/02/2011Safety Paediatric efficAcy phaRmacokinetic with Kuvan®A 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 - SPARK (Safety Paediatric efficAcy phaRmacokinetic with Kuvan®) Phenylketonuria
MedDRA version: 14.1;Level: LLT;Classification code 10034873;Term: Phenylketonuria (PKU);System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Trade Name: Kuvan
Product Name: Sapropterin Dihydrochloride
Product Code: NAP
INN or Proposed INN: SAPROPTERIN
Other descriptive name: tetrahydrobiopterin
Merck Serono S.A. - GenevaNULLNot RecruitingFemale: yes
Male: yes
50Phase 3Portugal;Czech Republic;Slovakia;Belgium;Turkey;Austria;Germany;Netherlands;Italy;United Kingdom
72NCT01412437
(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
73EUCTR2009-015768-33-IT
(EUCTR)
15/03/201128/12/2011SPARK (Safety Paediatric efficAcy phaRmacokinetic with Kuvan)A Phase IIIb, Multicenter, Open-Label, Randomized, Controlled Study of the Efficacy, Safety, and Population Pharmacokinetics of Sapropterin Dihydrochloride (Kuvan) in Phenylketonuria (PKU) Patients <4 Years Old - SPARK Phenylketonuria
MedDRA version: 14.1;Level: SOC;Classification code 10010331;Term: Congenital, familial and genetic disorders;System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 14.1;Level: PT;Classification code 10034872;Term: Phenylketonuria;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Trade Name: KUVAN
INN or Proposed INN: SAPROPTERIN
MERCK SERONO SANULLNot RecruitingFemale: yes
Male: yes
50Phase 3Portugal;Czech Republic;Slovakia;Belgium;Turkey;Austria;Netherlands;Germany;United Kingdom;Italy
74NCT01212744
(ClinicalTrials.gov)
March 201129/9/2010Safety, Tolerability, and Efficacy Study of rAvPAL-PEG Administered Daily in Subjects With Phenylketonuria (PKU)A Phase 2, Open-Label Study to Evaluate the Safety, Tolerability, and Efficacy of Subcutaneous Dose Levels of rAvPAL-PEG Administered Daily in Subjects With PhenylketonuriaPhenylketonuriaDrug: rAvPAL-PEGBioMarin PharmaceuticalNULLCompleted16 Years70 YearsAll16Phase 2United States
75EUCTR2009-015768-33-BE
(EUCTR)
16/02/201119/01/2011Safety Paediatric efficAcy phaRmacokinetic with Kuvan®A 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 - SPARK (Safety Paediatric efficAcy phaRmacokinetic with Kuvan®) Phenylketonuria
MedDRA version: 18.1;Level: LLT;Classification code 10034873;Term: Phenylketonuria (PKU);System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Trade Name: Kuvan
Product Name: Sapropterin Dihydrocholoride
Product Code: NAP
INN or Proposed INN: SAPROPTERIN
Other descriptive name: tetrahydrobiopterin
BioMarin International LtdNULLNot RecruitingFemale: yes
Male: yes
50Phase 3Portugal;Czech Republic;Slovakia;Belgium;Turkey;Austria;Netherlands;Germany;Italy;United Kingdom
76EUCTR2009-015768-33-SK
(EUCTR)
10/02/201103/11/2010Safety Paediatric efficAcy phaRmacokinetic with Kuvan®A 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 - SPARK (Safety Paediatric efficAcy phaRmacokinetic with Kuvan®) Phenylketonuria
MedDRA version: 18.1;Level: LLT;Classification code 10034873;Term: Phenylketonuria (PKU);System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Trade Name: Kuvan
Product Name: Sapropterin Dihydrocholoride
Product Code: NAP
INN or Proposed INN: SAPROPTERIN
Other descriptive name: tetrahydrobiopterin
BioMarin International LtdNULLNot RecruitingFemale: yes
Male: yes
50Phase 3Portugal;Czech Republic;Slovakia;Belgium;Turkey;Austria;Netherlands;Germany;Italy;United Kingdom
77EUCTR2009-015768-33-AT
(EUCTR)
05/01/201125/11/2010Safety Paediatric efficAcy phaRmacokinetic with Kuvan®A 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 - SPARK (Safety Paediatric efficAcy phaRmacokinetic with Kuvan®) Phenylketonuria
MedDRA version: 18.1;Level: LLT;Classification code 10034873;Term: Phenylketonuria (PKU);System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Trade Name: Kuvan
Product Name: Sapropterin Dihydrocholoride
Product Code: NAP
INN or Proposed INN: SAPROPTERIN
Other descriptive name: tetrahydrobiopterin
BioMarin International LtdNULLNot RecruitingFemale: yes
Male: yes
54Phase 3Portugal;Czech Republic;Slovakia;Belgium;Turkey;Austria;Netherlands;Germany;Italy;United Kingdom
78EUCTR2009-015768-33-GB
(EUCTR)
04/01/201111/10/2010Safety Paediatric efficAcy phaRmacokinetic with Kuvan®A 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 - SPARK (Safety Paediatric efficAcy phaRmacokinetic with Kuvan®) Phenylketonuria
MedDRA version: 19.0;Level: LLT;Classification code 10034873;Term: Phenylketonuria (PKU);System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Trade Name: Kuvan
Product Name: Sapropterin Dihydrocholoride
Product Code: NAP
INN or Proposed INN: SAPROPTERIN
Other descriptive name: tetrahydrobiopterin
BioMarin International Ltd.NULLNot Recruiting Female: yes
Male: yes
56 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): noPortugal;Czech Republic;Slovakia;Belgium;Turkey;Austria;Netherlands;Germany;Italy;United Kingdom
79NCT01274026
(ClinicalTrials.gov)
January 201110/1/2011Evaluation of Behavior, Executive Function, Neurotransmitter Function and Genomic Expression Kuvan NonrespondersEvaluation of Behavior, Executive Function, Neurotransmitter Function and Genomic Expression in PKU Nonresponders to Kuvan® (Sapropterin Dihydrochloride)Phenylketonuria;Behavior and Behavior Mechanisms;PAH Gene ExpressionDrug: sapropterin dihydrochlorideTulane University School of MedicineBioMarin PharmaceuticalCompleted2 YearsN/AAll21United States
80EUCTR2009-015768-33-CZ
(EUCTR)
15/12/201010/12/2010Safety Paediatric efficAcy phaRmacokinetic with Kuvan®A 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 - SPARK (Safety Paediatric efficAcy phaRmacokinetic with Kuvan®) Phenylketonuria
MedDRA version: 19.0;Level: LLT;Classification code 10034873;Term: Phenylketonuria (PKU);System Organ Class: 100000004850
Trade Name: Kuvan
Product Name: Sapropterin Dihydrocholoride
Product Code: NAP
INN or Proposed INN: SAPROPTERIN
Other descriptive name: tetrahydrobiopterin
BioMarin International LtdNULLNot RecruitingFemale: yes
Male: yes
56Phase 3Portugal;Czech Republic;Slovakia;Belgium;Turkey;Austria;Netherlands;Germany;Italy;United Kingdom
81EUCTR2010-019767-11-AT
(EUCTR)
08/09/201010/08/2010Ö-PKU 1 – Evaluation of a Test for the identication of BH4 responsive PKU patients - Ö-PKU1Ö-PKU 1 – Evaluation of a Test for the identication of BH4 responsive PKU patients - Ö-PKU1 Hyperphenylalaninaemia (HPA) in adult and paediatric patients of 4 years of age and over with phenylketonuria (PKU).Trade Name: KuvanGraz Medical UniversityNULLNot RecruitingFemale: yes
Male: yes
Austria
82NCT01114737
(ClinicalTrials.gov)
August 201027/4/2010Safety and Therapeutic Effects of Sapropterin Dihydrochloride on Neuropsychiatric Symptoms in Phenylketonuria (PKU) PatientsA Double-blind, Placebo-controlled, Randomized Study to Evaluate the Safety and Therapeutic Effects of Sapropterin Dihydrochloride on Neuropsychiatric Symptoms in Subjects With PhenylketonuriaPhenylketonuriaDrug: Sapropterin dihydrochloride;Drug: PlaceboBioMarin PharmaceuticalNULLCompleted8 Years65 YearsAll206Phase 3United States;Canada
83NCT01082328
(ClinicalTrials.gov)
May 20105/3/2010Response to Kuvan® in Subjects With Phenylketonuria (PKU) in a 4 Weeks Testing PeriodENDURE: A Phase IV, Prospective, Open-label, Uncontrolled, Multi-centre Cohort Trial to Assess the Responsiveness of Subjects With Phenylketonuria (PKU) to Treatment With Kuvan® 20 mg/kg/Day for 28 DaysPhenylketonuriaDrug: Kuvan®Merck KGaAMerck Serono Norway;Smerud Medical Research International ASCompleted4 YearsN/AAll59Phase 4Norway
84EUCTR2009-018168-81-DK
(EUCTR)
16/04/201003/03/2010ENDURE: A Phase IV, prospective, open-label, uncontrolled, multi-centre cohort trial to assess the responsiveness of subjects with phenylketonuria (PKU) to treatment with Kuvan® 20 mg/kg/day for 28 days - ENDUREENDURE: A Phase IV, prospective, open-label, uncontrolled, multi-centre cohort trial to assess the responsiveness of subjects with phenylketonuria (PKU) to treatment with Kuvan® 20 mg/kg/day for 28 days - ENDURE Subjects diagnosed with Phenylketonuria (PKU) (classic or mild PKU, or mild hyperphenylalaninaemia (HPA)).
MedDRA version: 14.1;Level: LLT;Classification code 10034873;Term: Phenylketonuria (PKU);System Organ Class: 10010331 - Congenital, familial and genetic disorders
Trade Name: Kuvan
INN or Proposed INN: Sapropterindihydrochloride
Other descriptive name: SAPROPTERIN HYDROCHLORIDE
Merck Serono NorwayNULLNot RecruitingFemale: yes
Male: yes
70Phase 4Denmark
85NCT00986973
(ClinicalTrials.gov)
March 201028/9/2009Fluorodeoxyglucose Positron Emission Tomography (FDG PET) Findings in Patients With Phenylketonuria Before and After KUVAN TherapyA Pilot Study of FDG PET Findings in Patients With Phenylketonuria Before and After BH4 SupplementationPhenylketonuriaDrug: SapropterinChildren's Hospital of PhiladelphiaNULLCompleted18 Years50 YearsAll6N/AUnited States
86NCT00924703
(ClinicalTrials.gov)
October 8, 200918/6/2009Long-Term Extension of Previous rAvPAL-PEG Protocols in Subjects With PKU (PAL-003)Long-term Extension of a Phase 2, Open-Label Dose-Finding Study to Evaluate the Safety, Efficacy, and Tolerability of Multiple Subcutaneous Doses of rAvPAL-PEG in Subjects With PKUPhenylketonuriaDrug: rAvPAL-PEGBioMarin PharmaceuticalNULLCompleted16 Years55 YearsAll68Phase 2United States
87NCT00925054
(ClinicalTrials.gov)
September 200917/6/2009Dose-Finding Study to Evaluate the Safety, Efficacy, & Tolerability of Multiple Doses of rAvPAL-PEG in Subjects With PKUPhase 2, Open-Label Dose-Finding Study to Evaluate the Safety, Efficacy, and Tolerability of Multiple Subcutaneous (SC) Doses of rAvPAL-PEG in Subjects With PKUPhenylketonuriaDrug: 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/kgBioMarin PharmaceuticalNULLCompleted16 Years55 YearsAll40Phase 2United States
88NCT00964236
(ClinicalTrials.gov)
August 200921/8/2009The Effects of Kuvan on Functional Brain Connectivity in Individuals With Phenylketonuria (PKU)The Effects of Kuvan on Functional Brain Connectivity in Individuals With Phenylketonuria (PKU)PhenylketonuriaDrug: SapropterinUniversity of Missouri-ColumbiaBioMarin PharmaceuticalCompleted6 YearsN/AAll20United States
89NCT00909012
(ClinicalTrials.gov)
May 200922/4/2009Quantitative Requirements of Docosahexaenoic Acid for Neural Function in Children With PhenylketonuriaQuantitative Requirements of Docosahexaenoic Acid for Neural Function in Children With PhenylketonuriaPhenylketonuriaDietary Supplement: high oleic sunflower oil;Dietary Supplement: microalgal oilLudwig-Maximilians - University of MunichEuropean UnionCompleted5 Years13 YearsAll114N/AGermany;Italy;Spain;United Kingdom
90NCT00838435
(ClinicalTrials.gov)
February 20095/2/2009Effect of Kuvan on Neurocognitive Function, Blood Phenylalanine Level, Safety, and Pharmacokinetics in Children With PKUA Phase 3b Open-Label Study to Evaluate the Effect of Kuvan® on Neurocognitive Function, Maintenance of Blood Phenylalanine Concentrations, Safety, and Population Pharmacokinetics in Young Children With PhenylketonuriaPhenylketonuriaDrug: sapropterin dihydrochlorideBioMarin PharmaceuticalNULLCompletedN/A6 YearsAll95Phase 3United States;Canada
91NCT00827762
(ClinicalTrials.gov)
January 200921/1/2009Behavioral Effects of Kuvan in Children With Mild PhenylketonuriaBehavioral Effects of Kuvan in Children With Mild PhenylketonuriaPhenylketonuriaDrug: KuvanWashington University School of MedicineBioMarin Pharmaceutical;University of Missouri-Columbia;Northwestern University;Oregon Health and Science UniversityTerminated6 Years18 YearsBoth2N/AUnited States
92NCT00841100
(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
93NCT00688844
(ClinicalTrials.gov)
October 200829/5/2008Nutritional and Neurotransmitter Changes in PKU Subjects on BH4Baseline Evaluation and Long-term Follow-up of Nutritional Status and Neurotransmitter Concentrations in Phenylketonuria Patients Initiating Treatment With Sapropterin Dihydrochloride (KuvanTM), a Tetrahydrobiopterin Analog.PhenylketonuriaDrug: KuvanTM TherapyEmory UniversityBioMarin Pharmaceutical;Atlanta Clinical and Translational Science InstituteCompleted4 YearsN/AAll58N/AUnited States
94NCT00789568
(ClinicalTrials.gov)
October 200810/11/2008A Phase 1 Study to Evaluate Effects of Sapropterin Dihydrochloride on QTc Intervals in Healthy Adult SubjectsA Phase 1, Randomized, Placebo- and Active-controlled Crossover Study to Evaluate the Effects of Sapropterin Dihydrochloride Oral Administration on QTc Intervals in Healthy Adult SubjectsPhenylketonuriaDrug: sapropterin dihydrochloride;Drug: Moxifloxacin;Drug: Moxifloxacin placeboBioMarin PharmaceuticalNULLCompleted18 Years50 YearsAll56Phase 1United States
95NCT00778206
(ClinicalTrials.gov)
September 200821/10/2008PKUDOS: Phenylketonuria (PKU) Demographic, Outcomes, and Safety RegistryPKUDOS: Phenylketonuria (PKU) Demographic, Outcomes, and Safety RegistryPhenylketonuria;HyperphenylalaninaemiaDrug: KuvanBioMarin PharmaceuticalNULLCompletedN/AN/AAll1890United States;Puerto Rico
96NCT00730080
(ClinicalTrials.gov)
July 20081/8/2008Sapropterin in Individuals With PhenylketonuriaEffects of Sapropterin on Brain and Cognition in Individuals With PhenylketonuriaPhenylketonuriaDrug: Sapropterin (Kuvan)Washington University School of MedicineBioMarin Pharmaceutical;University of Missouri-ColumbiaCompleted6 Years50 YearsAll45United States
97NCT00634660
(ClinicalTrials.gov)
May 20086/3/2008Safety and Tolerability Study of rAvPAL-PEG to Treat PhenylketonuriaA Phase I, Open-Label, Dose-Escalation Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of Single, Subcutaneous Doses of rAvPAL-PEG in Subjects With PhenylketonuriaPhenylketonuriaDrug: rAvPAL-PEGBioMarin PharmaceuticalNULLCompleted16 Years50 YearsAll25Phase 1United States
98NCT00892554
(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
99NCT00432822
(ClinicalTrials.gov)
February 20077/2/2007Long-Term Tetrahydrobiopterin Treatment in PKU Patients of 0-18 Years - Study on Phenylalanine Tolerance and SafetyDouble-Blind, Placebo Controlled, Multicentre Study With an Open Label Extension to Evaluate the Efficacy and Safety of Tetrahydrobiopterin (BH4) in Children and Adolescents With Hyperphenylalaninemia Caused by Phenylalanine Hydroxylase DeficiencyPhenylalanine Hydroxylase DeficienciesDrug: tetrahydrobiopterin (BH4)Orphanetics Pharma Entwicklungs GmbHNULLTerminatedN/A18 YearsBoth50Phase 2/Phase 3NULL
100NCT00484991
(ClinicalTrials.gov)
February 20078/6/2007Sapropterin Expanded Access ProgramSapropterin Expanded Access ProgramPhenylketonuriaDrug: Sapropterin dihydrochlorideBioMarin PharmaceuticalNULLApproved for marketing9 YearsN/ABothN/AUnited States
101EUCTR2006-000839-10-ES
(EUCTR)
06/10/200613/07/2006A Phase 3b, Multicenter, Open-Label Extension Study of Phenoptin in Subjects with Phenylketonuria Who Participated in Protocols PKU-004 or PKU-006Estudio de extensión fase 3b, multicéntrico y abierto de PhenoptinTM en sujetos fenilcetonúricos que participaron en los estudios PKU-004 o PKU-006A Phase 3b, Multicenter, Open-Label Extension Study of Phenoptin in Subjects with Phenylketonuria Who Participated in Protocols PKU-004 or PKU-006Estudio de extensión fase 3b, multicéntrico y abierto de PhenoptinTM en sujetos fenilcetonúricos que participaron en los estudios PKU-004 o PKU-006 Fenilcetonuria (PKU)
MedDRA version: 8.1;Level: LLT;Classification code 10034872;Term: Phenylketonuria
Product Name: Phenoptin
Product Code: T1401
INN or Proposed INN: Sapropterin
Other descriptive name: sapropterin dihydrochloride
BioMarin Pharmaceutical Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
128Phase 3United Kingdom;Germany;Ireland;Spain;Italy
102EUCTR2006-000839-10-IT
(EUCTR)
07/09/200602/08/2007A Phase 3b, Multicenter, Open?Label Extension Study of Phenoptin in Subjects with Phenylketonuria Who Participated in Studies PKU?004 or PKU?006 - NDA Phase 3b, Multicenter, Open?Label Extension Study of Phenoptin in Subjects with Phenylketonuria Who Participated in Studies PKU?004 or PKU?006 - ND phenylketonuria
Level: PTClassification code 10034872
Trade Name: Phenoptin
INN or Proposed INN: Sapropterin
BIOMARINNULLNot RecruitingFemale: yes
Male: yes
128Phase 3United Kingdom;Germany;Spain;Ireland;Italy
103EUCTR2006-000839-10-IE
(EUCTR)
02/09/200612/06/2006A Phase 3b, Multicenter, Open-Label Extension Study of Phenoptin in Subjects with Phenylketonuria Who Participated in Protocols PKU-004 or PKU-006 - NAA Phase 3b, Multicenter, Open-Label Extension Study of Phenoptin in Subjects with Phenylketonuria Who Participated in Protocols PKU-004 or PKU-006 - NA Phenylketonuria (PKU)
MedDRA version: 8.1;Level: LLT;Classification code 10034872;Term: Phenylketonuria
Product Name: Phenoptin
Product Code: T1401
INN or Proposed INN: Sapropterin
Other descriptive name: sapropterin dihydrochloride
BioMarin Pharmaceutical Inc.NULLNot RecruitingFemale: yes
Male: yes
128Phase 3United Kingdom;Germany;Spain;Ireland;Italy
104EUCTR2005-003777-24-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
105EUCTR2006-000839-10-GB
(EUCTR)
14/08/200620/09/2006A Phase 3b, Multicenter, Open-Label Extension Study of Phenoptin in Subjects with Phenylketonuria Who Participated in Protocols PKU-004 or PKU-006 - NAA Phase 3b, Multicenter, Open-Label Extension Study of Phenoptin in Subjects with Phenylketonuria Who Participated in Protocols PKU-004 or PKU-006 - NA Phenylketonuria (PKU)
MedDRA version: 8.1;Level: LLT;Classification code 10034872;Term: Phenylketonuria
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
128 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): noSpain;Ireland;Germany;Italy;United Kingdom
106EUCTR2006-000839-10-DE
(EUCTR)
09/08/200626/05/2006A Phase 3b, Multicenter, Open-Label Extension Study of Phenoptin in Subjects with Phenylketonuria Who Participated in Protocols PKU-004 or PKU-006 - NAA Phase 3b, Multicenter, Open-Label Extension Study of Phenoptin in Subjects with Phenylketonuria Who Participated in Protocols PKU-004 or PKU-006 - 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
128Phase 3United Kingdom;Germany;Spain;Ireland;Italy
107NCT00355264
(ClinicalTrials.gov)
August 200619/7/2006Safety and Efficacy Study of Phenoptin in Subjects With Hyperphenylalaninemia Due to BH4 DeficiencyPhase 2, Multicenter, Open Label Study of Phenoptin in Subjects With Hyperphenylalaninemia Due to Primary BH4 DeficiencyTetrahydrobiopterin Deficiencies;Hyperphenylalaninemia, Non-PhenylketonuricDrug: PhenoptinBioMarin PharmaceuticalNULLCompletedN/AN/AAll12Phase 2United States;Germany
108EUCTR2005-003778-13-DE
(EUCTR)
13/07/200614/06/2006A Phase 2, Multicenter, Open-label Study to Evaluate the Safety and Efficacy of Phenoptin in Subjects with Hyperphenylalaninemia Due to Primary BH4 DeficiencyA Phase 2, Multicenter, Open-label Study to Evaluate the Safety and Efficacy of Phenoptin in Subjects with Hyperphenylalaninemia Due to Primary BH4 Deficiency Phenylketonuria (PKU)
MedDRA version: 8.1;Level: LLT;Classification code 10034872;Term: Phenylketonuria
Product Name: Phenoptin
Product Code: T1401
INN or Proposed INN: Sapropterin
Other descriptive name: saproterin dihydrochloride
BioMarin Pharmaceutical IncNULLNot RecruitingFemale: yes
Male: yes
15Phase 2Germany
109NCT00332189
(ClinicalTrials.gov)
July 200630/5/2006Study of Phenoptin in Subjects With Phenylketonuria Who Participated in Protocols PKU-004 or PKU-006A Phase 3b, Multicenter, Open-Label Extension Study of Phenoptin in Subjects With Phenylketonuria Who Participated in Protocols PKU-004 or PKU-006PhenylketonuriaDrug: sapropterin dihydrochlorideBioMarin PharmaceuticalNULLCompleted4 YearsN/AAll111Phase 3United States
110EUCTR2006-000648-15-AT
(EUCTR)
08/06/200605/04/2006A double-blind, placebo-controlled, multicentre study with an open-label extension to evaluate the efficacy and safety of tetrahydrobiopterin (BH4) in children and adolescents with hyperphenylalaninemia caused by phenylalanine hydroxylase deficiencyA double-blind, placebo-controlled, multicentre study with an open-label extension to evaluate the efficacy and safety of tetrahydrobiopterin (BH4) in children and adolescents with hyperphenylalaninemia caused by phenylalanine hydroxylase deficiency Hyperphenylalaninemia due to phenylalanine hydroxylase deficiency. Phenotypes: classic phenylketonuria (PKU), mild PKU (MPK) or mild hyperphenylalaninemia (HPA).
MedDRA version: 81;Level: LLT;Classification code 10034873
Product Name: tetrahydrobiopterin
Product Code: BH4
INN or Proposed INN: Sapropterin
Other descriptive name: n.a.
ORPHANETICS Pharma Entwicklungs- GmbHNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
75Austria
111EUCTR2005-003777-24-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
112EUCTR2005-003777-24-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.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
300Germany;Spain
113NCT00225615
(ClinicalTrials.gov)
November 200522/9/2005A Phase 3, Multicenter, Open-Label Extension Study of Phenoptin in Subjects With PKU Who Have Elevated Phenylalanine LevelsA Phase 3, Multicenter, Open-Label Extension Study of Phenoptin in Subjects With Phenylketonuria Who Have Elevated Phenylalanine LevelsPhenylketonuriasDrug: sapropterin dihydrochlorideBioMarin PharmaceuticalNULLCompleted8 YearsN/ABoth100Phase 3United States
114EUCTR2004-004513-41-DE
(EUCTR)
27/10/200512/05/2006A Phase 3, Multicenter, Open-Label Extension Study of Phenoptin in Subjects with Phenylketonuria Who Have Elevated Phenylalanine LevelsA Phase 3, Multicenter, Open-Label Extension Study of Phenoptin in Subjects with Phenylketonuria Who Have Elevated Phenylalanine Levels Phenylketonuria (PKU)
MedDRA version: 6.1;Classification code 10034872
Product Name: Phenoptin
Product Code: T1401
INN or Proposed INN: Sapropterin, sapropterin dihydrochloride
BioMarin Pharmaceutical Inc.NULLNot RecruitingFemale: yes
Male: yes
100Phase 3Ireland;Germany;Italy;United Kingdom
115EUCTR2004-004513-41-GB
(EUCTR)
25/10/200502/06/2005A Phase 3, Multicenter, Open-Label Extension Study of Phenoptin in Subjects with Phenylketonuria Who Have Elevated Phenylalanine LevelsA Phase 3, Multicenter, Open-Label Extension Study of Phenoptin in Subjects with Phenylketonuria Who Have Elevated Phenylalanine Levels Phenylketonuria (PKU)
MedDRA version: 6.1;Classification code 10034872
Product Name: Phenoptin
Product Code: T1401
INN or Proposed INN: Sapropterin, sapropterin dihydrochloride
BioMarin Pharmaceutical Inc.NULLNot Recruiting Female: yes
Male: yes
100 Human pharmacology (Phase 1): Therapeutic exploratory (Phase 2): Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4):Ireland;Germany;Italy;United Kingdom
116EUCTR2004-004513-41-IT
(EUCTR)
23/09/200511/11/2005A Phase 3, Multicenter, Open-Label Extension Study of Phenoptin in Subjects with Phenylketonuria Who Have Elevated Phenylalanine LevelsA Phase 3, Multicenter, Open-Label Extension Study of Phenoptin in Subjects with Phenylketonuria Who Have Elevated Phenylalanine Levels Treatment of phenylketonuria
MedDRA version: 6.1;Level: SOC;Classification code 10027433
Product Name: PhenoptinBIOMARINNULLNot RecruitingFemale: yes
Male: yes
50Phase 3Germany;United Kingdom;Ireland;Italy
117EUCTR2004-004513-41-IE
(EUCTR)
19/08/200510/06/2005A Phase 3, Multicenter, Open-Label Extension Study of Phenoptin in Subjects with Phenylketonuria Who Have Elevated Phenylalanine LevelsA Phase 3, Multicenter, Open-Label Extension Study of Phenoptin in Subjects with Phenylketonuria Who Have Elevated Phenylalanine Levels Phenylketonuria (PKU)
MedDRA version: 6.1;Classification code 10034872
Product Name: Phenoptin
Product Code: T1401
INN or Proposed INN: Sapropterin, sapropterin hydrochloride
BioMarin Pharmaceutical Inc.NULLNot Recruiting Female: yes
Male: yes
100 Human pharmacology (Phase 1): Therapeutic exploratory (Phase 2): Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4):Ireland;Germany;Italy;United Kingdom
118EUCTR2004-004512-23-GB
(EUCTR)
15/08/200523/02/2005A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Phenoptin in Subjects with Phenylketonuria Who Have Elevated Phenylalanine Levels - NAA Phase 3, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Phenoptin in Subjects with Phenylketonuria Who Have Elevated Phenylalanine Levels - NA Phenylketonuria (PKU)
MedDRA version: 6.1;Classification code 10034872
Product Name: Phenoptin
Product Code: T1401
INN or Proposed INN: Sapropterin, sapropterin hydrochloride
BioMarin Pharmaceutical Inc.NULLNot Recruiting Female: yes
Male: yes
100 Human pharmacology (Phase 1): Therapeutic exploratory (Phase 2): Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4):Ireland;Germany;Italy;United Kingdom
119EUCTR2004-004512-23-IT
(EUCTR)
09/06/200522/06/2005A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Phenoptin in Subjects with Phenylketonuria Who Have Elevated Phenylalanine LevelsA Phase 3, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Phenoptin in Subjects with Phenylketonuria Who Have Elevated Phenylalanine Levels Treatment of phenylketonuria
MedDRA version: 6.1;Level: SOC;Classification code 10027433
Product Name: PhenoptinBIOMARINNULLNot RecruitingFemale: yes
Male: yes
100Phase 3Germany;United Kingdom;Ireland;Italy
120EUCTR2004-004512-23-IE
(EUCTR)
22/05/200515/02/2005A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Phenoptin in Subjects with Phenylketonuria Who Have Elevated Phenylalanine Levels - NAA Phase 3, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Phenoptin in Subjects with Phenylketonuria Who Have Elevated Phenylalanine Levels - NA Phenylketonuria (PKU)
MedDRA version: 6.1;Classification code 10034872
Product Name: Phenoptin
Product Code: T1401
INN or Proposed INN: Sapropterin, sapropterin hydrochloride
BioMarin Pharmaceutical Inc.NULLNot Recruiting Female: yes
Male: yes
100 Human pharmacology (Phase 1): Therapeutic exploratory (Phase 2): Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4):Ireland;Germany;Italy;United Kingdom
121NCT00244218
(ClinicalTrials.gov)
April 200525/10/2005Response to Phenylketonuria to Tetrahydrobiopterin (BH4)Response to Phenylketonuria to Tetrahydrobiopterin (BH4)PhenylketonuriaDrug: tetrahydrobiopterin (BH4)The University of Texas Medical Branch, GalvestonNULLTerminated10 YearsN/AAll57Phase 1United States
122NCT00260000
(ClinicalTrials.gov)
April 200530/11/2005Study of BH4, a New and Simple Treatment of Mild PKUStudy of the Response of Tetrahydrobiopterin on S-Phenylalanine in Patients With PKU Housing the Y414C MutationPhenylketonuriaDrug: 5,6,7,8-tetrahydrobiopterinThe Kennedy Institute-National Eye ClinicSygekassernes HelsefondCompleted8 YearsN/ABoth15Phase 2Denmark
123EUCTR2004-002071-16-GB
(EUCTR)
18/03/200522/02/2005A Phase 2, Multicenter, Open-Label Study to Evaluate the Response to and Safety of an 8-Day Course of Phenoptin Treatment in Subjects with Phenylketonuria Who Have Elevated Phenylalanine Levels - NAA Phase 2, Multicenter, Open-Label Study to Evaluate the Response to and Safety of an 8-Day Course of Phenoptin Treatment in Subjects with Phenylketonuria Who Have Elevated Phenylalanine Levels - NA Phenylketonuria (PKU)
MedDRA version: 6.1;Classification code 10034872
Product Name: Phenoptin
Product Code: T1401
INN or Proposed INN: Sapropterin, sapropterin hydrochloride
BioMarin Pharmaceutical Inc.NULLNot Recruiting Female: yes
Male: yes
700 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): noIreland;Germany;Italy;United Kingdom
124EUCTR2004-002071-16-IT
(EUCTR)
17/03/200527/04/2005A Phase 2, Multicenter, Open-Label Study to Evaluate the Response to and Safety of an 8-Day Course of Phenoptin Treatment in Subjects with Phenylketonuria Who Have Elevated Phenylalanine LevelsA Phase 2, Multicenter, Open-Label Study to Evaluate the Response to and Safety of an 8-Day Course of Phenoptin Treatment in Subjects with Phenylketonuria Who Have Elevated Phenylalanine Levels Treatment of Phenylketonuria
MedDRA version: 6.1;Level: SOC;Classification code 10027433
Product Name: Phenoptin
Product Code: NA
INN or Proposed INN: sapropterin hydrochloride
Other descriptive name: NA
BIOMARINNULLNot RecruitingFemale: yes
Male: yes
Phase 2United Kingdom;Germany;Italy
125NCT00104247
(ClinicalTrials.gov)
March 200524/2/2005Study to Evaluate the Safety and Efficacy of Phenoptin™ in Subjects With Phenylketonuria Who Have Elevated Phenylalanine LevelsA Phase 3, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Phenoptin™ in Subjects With Phenylketonuria Who Have Elevated Phenylalanine LevelsPhenylketonuriasDrug: sapropterin dihydrochloride, 6R-BH4, tetrahydrobiopterinBioMarin PharmaceuticalNULLCompleted8 YearsN/AAll89Phase 3United States
126EUCTR2004-002071-16-DE
(EUCTR)
28/01/200514/10/2004A Phase 2, Multicenter, Open-Label Study to Evaluate the Response to and Safety of an 8-Day Course of Phenoptin Treatment in Subjects with Phenylketonuria Who Have Elevated Phenylalanine Levels - NAA Phase 2, Multicenter, Open-Label Study to Evaluate the Response to and Safety of an 8-Day Course of Phenoptin Treatment in Subjects with Phenylketonuria Who Have Elevated Phenylalanine Levels - NA Phenylketonuria (PKU)
MedDRA version: 6.1;Classification code 10034872
Product Name: Phenoptin
Product Code: T1401
INN or Proposed INN: Sapropterin, sapropterin hydrochloride
BioMarin Pharmaceutical Inc.NULLNot RecruitingFemale: yes
Male: yes
700Phase 2United Kingdom;Germany;Italy
127NCT00104260
(ClinicalTrials.gov)
December 200424/2/2005Study to Evaluate the Response to and Safety of an 8-Day Course of Phenoptin™ Treatment in Subjects With PhenylketonuriaA Phase 2, Multicenter, Open-Label Study to Evaluate the Response to and Safety of an 8-Day Course of Phenoptin™ Treatment in Subjects With Phenylketonuria Who Have Elevated Phenylalanine LevelsPhenylketonuriasDrug: sapropterin dihydrochlorideBioMarin PharmaceuticalNULLCompleted8 YearsN/ABoth700Phase 2United States
128EUCTR2004-002365-21-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
Denmark
129NCT00065299
(ClinicalTrials.gov)
May 198421/7/2003Low Phenylalanine Diet for Mothers With Phenylketonuria (PKU)Effects of Maternal Phenylketonuria (PKU) on Pregnancy OutcomePhenylketonuriaBehavioral: Restricted phenylalanine dietEunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)NULLCompletedN/AN/AFemale572N/AUnited States
130NCT00006142
(ClinicalTrials.gov)
December 19833/8/2000Study of a Phenylalanine Restricted Diet During Pregnancy to Prevent Symptoms in Offspring of Patients With PhenylketonuriaPhenylketonuriaBehavioral: phenylalanine restricted dietNational Center for Research Resources (NCRR)University of TexasCompletedN/AN/AFemaleN/ANULL
131EUCTR2004-002071-16-IE
(EUCTR)
13/10/2004A Phase 2, Multicenter, Open-Label Study to Evaluate the Response to and Safety of an 8-Day Course of Phenoptin Treatment in Subjects with Phenylketonuria Who Have Elevated Phenylalanine Levels - NAA Phase 2, Multicenter, Open-Label Study to Evaluate the Response to and Safety of an 8-Day Course of Phenoptin Treatment in Subjects with Phenylketonuria Who Have Elevated Phenylalanine Levels - NA Phenylketonuria (PKU)
MedDRA version: 6.1;Classification code 10034872
Product Name: Phenoptin
Product Code: T1401
INN or Proposed INN: Sapropterin, sapropterin hydrochloride
BioMarin Pharmaceutical Inc.NULLNot Recruiting Female: yes
Male: yes
400 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): noIreland;Germany;Italy;United Kingdom
132EUCTR2021-000474-29-DE
(EUCTR)
13/09/2021A Phase 3 Study of PTC923 in Subjects with PhenylketonuriaA Phase 3 Study of PTC923 in Subjects with Phenylketonuria Metabolic Disorders - Phenylketonuria
MedDRA version: 20.0;Level: LLT;Classification code 10034873;Term: Phenylketonuria (PKU);System Organ Class: 100000004850;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03]
Product Code: PTC923
INN or Proposed INN: not available
Other descriptive name: (S)-2-amino-6-(2-hydroxypropanoyl)-7,8-dihydropteridin-4(3H)-one
Product Code: PTC923
INN or Proposed INN: not available
Other descriptive name: (S)-2-amino-6-(2-hydroxypropanoyl)-7,8-dihydropteridin-4(3H)-one
PTC Therapeutics, Inc.NULLNAFemale: yes
Male: yes
178Phase 3United States;Portugal;Spain;Turkey;United Kingdom;Italy;France;Mexico;Canada;Brazil;Australia;Denmark;Georgia;Germany;Netherlands
133EUCTR2009-018168-81-NO
(EUCTR)
10/03/2010ENDURE: A Phase IV, prospective, open-label, uncontrolled, multi-centre cohort trial to assess the responsiveness of subjects with phenylketonuria (PKU) to treatment with Kuvan® 20 mg/kg/day for 28 days - ENDUREENDURE: A Phase IV, prospective, open-label, uncontrolled, multi-centre cohort trial to assess the responsiveness of subjects with phenylketonuria (PKU) to treatment with Kuvan® 20 mg/kg/day for 28 days - ENDURE Subjects diagnosed with Phenylketonuria (PKU) (classic or mild PKU, or mild hyperphenylalaninaemia (HPA)).
MedDRA version: 12.1;Level: LLT;Classification code 10034873;Term: Phenylketonuria (PKU)
Trade Name: Kuvan
INN or Proposed INN: Sapropterindihydrochloride
Other descriptive name: SAPROPTERIN HYDROCHLORIDE
Merck Serono NorwayNULLNAFemale: yes
Male: yes
150Phase 4Denmark;Norway
134EUCTR2015-001650-15-Outside-EU/EEA
(EUCTR)
26/06/2015Phase 3 Open-label Study to Evaluate the Response and Safety of Kuvan® in Subjects With PhenylketonuriaA Phase III Non-comparative Open-label Clinical Study to Evaluate the Response to and Safety of Kuvan (Sapropterin Dihydrochloride) After 6 Weeks of Treatment in Patients of 4 to 18 Years of Age With Phenylketonuria Who Have Elevated Blood Phenylalanine Levels Phenylketonuria;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]Trade Name: Kuvan 100mg soluble tablet
Product Name: Sapropterin Dihydrocholoride
Merck Serono Middle East FZ-LLCNULLNAFemale: yes
Male: yes
90Phase 3Ukraine;Russian Federation
135EUCTR2004-004512-23-DE
(EUCTR)
17/03/2005A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Phenoptin in Subjects with Phenylketonuria Who Have Elevated Phenylalanine Levels - NAA Phase 3, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Phenoptin in Subjects with Phenylketonuria Who Have Elevated Phenylalanine Levels - NA Phenylketonuria (PKU)
MedDRA version: 6.1;Classification code 10034872
Product Name: Phenoptin
Product Code: T1401
INN or Proposed INN: Sapropterin, sapropterin hydrochloride
BioMarin Pharmaceutical Inc.NULLNot RecruitingFemale: yes
Male: yes
100Phase 3United Kingdom;Germany;Ireland;Italy
136EUCTR2009-012978-12-NO
(EUCTR)
02/06/2009A Phase IV, prospective, open-label, uncontrolled, single-centre cohort trial to be conducted in Norway to assess the responsiveness of subjects with phenylketonuria (PKU) to treatment with Kuvan® 20 mg/kg/day for 7 days. - Response to Kuvan® in subjects with PKU from a single centre in NorwayA Phase IV, prospective, open-label, uncontrolled, single-centre cohort trial to be conducted in Norway to assess the responsiveness of subjects with phenylketonuria (PKU) to treatment with Kuvan® 20 mg/kg/day for 7 days. - Response to Kuvan® in subjects with PKU from a single centre in Norway Hyperphenylalaninemia (HPA) in adults and children >=16 years of age with phenylketonuri (PKU) (classic or mild PKU, or mild hyperphenylalaninemia (HPA)).
MedDRA version: 9.1;Level: LLT;Classification code 10034872;Term: Phenylketonuria
MedDRA version: 9.1;Classification code 10034873;Term: Phenylketonuria (PKU)
MedDRA version: 9.1;Level: PT;Classification code 10034871;Term: Phenylalaninemia
Trade Name: Kuvan® (sapropterin dihydrochloride)
INN or Proposed INN: sapropterin
Merck Serono, an affiliate of E. Merck ABNULLNot RecruitingFemale: yes
Male: yes
Phase 4Norway
137EUCTR2021-003777-63-NL
(EUCTR)
24/11/2021BH4 (Kuvan©) treatment in Phenylketonuria - comparing different practices of dosing regimensTetrahydrobiopterin (BH4) treatment in Phenylketonuria - comparing different practices of dosing regimen - BH4 dose Phenylketonuria (PKU; OMIM 261600);Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]Trade Name: Kuvan® (sapropterin dihydrochloride)
Product Name: Kuvan® (sapropterin dihydrochloride)
Product Code: A16AX07
University Medical Center GroningenNULLNAFemale: yes
Male: yes
28Phase 4Netherlands
138EUCTR2018-000648-25-NL
(EUCTR)
13/11/2018A Phase 3b, Open-Label, Single Arm, Multi-Centre Study to Assess the Safety and Efficacy of Pegvaliase (BMN 165) Treatment in adults With Phenylketonuria Not Controlled With Current ManagementA Phase 3b, Open-Label, Single Arm, Multi-Centre Study to Assess the Safety and Efficacy of Pegvaliase (BMN 165) Treatment in adults With Phenylketonuria Not Controlled With Current Management - OPTIC Study Phenylketonuria (PKU)
MedDRA version: 20.0;Level: LLT;Classification code 10034873;Term: Phenylketonuria (PKU);System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Product Name: Pegvaliase
INN or Proposed INN: PEGVALIASE
Product Name: Pegvaliase
INN or Proposed INN: PEGVALIASE
Product Name: Pegvaliase
INN or Proposed INN: PEGVALIASE
BioMarin Pharmaceutical Inc.NULLNot Recruiting Female: yes
Male: yes
85 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): noItaly;Netherlands;Germany;Austria;Turkey;Spain;France