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 240. フェニルケトン尿症 [臨床試験数:78,薬物数:53(DrugBank:8),標的遺伝子数:0,標的パスウェイ数:0] 

Searched query = "Phenylketonuria", "Phenylalanine hydroxylase deficiency", "PAH deficiency", "Tetrahydrobiopterin deficiency", "BH4 deficiency", "BH4 reactive hyper pheemia"
The queries were searched in Public_title, Scientific_title, and Condition of the data. Export date: 11/20/2019, 11/21/2019. Trials are sorted by Date_enrolment from most recent to oldest in the table.

Search in Page    e.g. "Phase 3", "Not recruiting", "Japan"    Synonyms (DrugBank) were also searched for.
No.TrialIDDate_
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1NCT04110496September 28, 201914 October 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 TherapeuticsRecruiting18 YearsN/AAll12Phase 1United States
2NCT03924180September 19, 201930 September 2019Glytactin EfficiEncy in Non Treated Adult PHENylketonuria PatientsGlytactin EfficiEncy in Non Treated Adult PHENylketonuria PatientsAdult Phenylketonuria Non Treated PatientsDietary Supplement: Dietary Supplement for PKU patientsUniversity Hospital, ToursINSERM 1415-TOURSRecruiting18 Years75 YearsAll32N/AFrance
3NCT01465100September 201926 August 2019Liver 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 FoxRecruiting14 Years55 YearsAll10Phase 1/Phase 2United States
4NCT03788343August 19, 201916 September 2019Phenylalanine 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
5NCT04014712August 201922 July 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, AmherstNot recruiting18 Years85 YearsAll30Phase 1United States
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6NCT03939052July 19, 20192 September 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
7NCT03333720July 15, 201925 March 2019Compliance, 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 LtdNot recruiting8 Years100 YearsAll0N/A
8NCT04085666June 1, 201930 September 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 PlaceboNestléRecruiting18 Years55 YearsAll20Phase 1Australia;Germany
9JPRN-JapicCTI-19464217/4/201916 July 2019A Phase 3 Study to Assess the Safety and Efficacy of BMN 165 in Japanese Subjects 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 PhenylketonuriaPhenylketonuriaIntervention 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) during Part 1. Subjects may increase dose up to 60 mg/day in Part 2 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 Pharmaceuticals Inc./CMIC Co., Ltd.Recruiting1870BOTH10Phase 3Japan
10NCT03792451January 17, 201926 August 2019Nutrition Status of Adults Treated With PegvaliaseNutrition Status of Adults With Phenylketonuria Treated With PegvaliasePhenylketonuriasDrug: PegvaliaseBoston Children’s HospitalBioMarin PharmaceuticalRecruiting18 Years65 YearsAll20Phase 1United States
No.TrialIDDate_
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11NCT03820804January 7, 201930 September 2019Nutritional Status in PhenylketonuriaTrends in Nutritional Status of Patients With PhenylketonuriaPhenylketonuriasOther: Diet;Drug: SapropterinUniversidade do PortoCentro Hospitalar do Porto;BioMarin PharmaceuticalNot recruiting3 YearsN/AAll94Phase 3Portugal
12NCT03771391December 6, 201818 December 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;Universitätsmedizin der Johannes Gutenberg-Universität MainzRecruiting10 YearsN/AAll33N/AGermany
13NCT03694353September 13, 201825 March 2019Study to Evaluate the Safety 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 PharmaceuticalNot recruiting18 Years70 YearsAll37Phase 3United States
14NCT03519711June 24, 201826 August 2019A Study of CNSA-001 in Primary Tetrahydrobiopterin Deficient Patients With HyperphenylalaninemiaA Phase 1/2, Open-Label, Randomized Parallel Arm, Intra-patient Dose Escalation Study to Evaluate Safety, PK and Preliminary Efficacy of CNSA-001 in Primary Tetrahydrobiopterin Deficient Patients With HyperphenylalaninemiaBH4 Deficiency;Hyperphenylalaninemia, Non-PhenylketonuricDrug: CNSA-001Censa Pharmaceuticals, Inc.Recruiting12 YearsN/AAll6Phase 1/Phase 2United States;Germany
15NCT03419819May 1, 201817 June 2019Evaluation 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 UniversityNot recruiting3 YearsN/AAll28N/AUnited States
No.TrialIDDate_
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16NCT03516487April 2, 201826 August 2019Safety 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: PlaceboSynlogicNot recruiting18 Years64 YearsAll70Phase 1/Phase 2United States
17NCT02677870January 201816 December 2017The 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
18EUCTR2018-000648-25-NL19 November 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 StudyPhenylketonuria (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
Pharmaceutical Form: Solution for injection
INN or Proposed INN: PEGVALIASE
CAS Number: 1585984-95-7
Current Sponsor code: pegvaliase, rAvPAL-PEG, PEG-PAL, PAL-PEG
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 2.5-
Product Name: Pegvaliase
Pharmaceutical Form: Solution for injection
INN or Proposed INN: PEGVALIASE
CAS Number: 1585984-95-7
Current Sponsor code: pegvaliase, rAvPAL-PEG, PEG-PAL, PAL-PEG
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 10-
Product Name: Pegvaliase
Pharmaceutical Form: Solution for injection
INN or Proposed INN: PEGVALIASE
CAS Number: 1585984-95-7
Current Sponsor code: pegvaliase, rAvPAL-PEG, PEG-PAL, PAL-PEG
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 20-
BioMarin Pharmaceutical Inc.Not AvailableFemale: yes
Male: yes
85Phase 3France;Spain;Turkey;Austria;Germany;Netherlands;Italy
19NCT03864029October 10, 201718 March 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.Not recruitingN/AN/AAll26N/AChina
20NCT03168399June 8, 201711 June 2018Evaluation 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 TrustNot recruiting6 Months5 YearsAll22N/AUnited Kingdom
No.TrialIDDate_
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21NCT03167697May 1, 201725 March 2019Evaluating 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 LtdNot recruiting16 Years100 YearsAll14N/AUnited Kingdom
22NCT03058848March 6, 201716 December 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, LtdNot recruitingN/A2 YearsAll10N/AUnited Kingdom
23NCT02915510July 201614 October 2019GMP 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 LtdNot recruiting3 YearsN/AAll40N/AUnited Kingdom
24NCT02440932November 201430 May 2016Impact 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 GlasgowNot recruiting18 Years45 YearsBoth26N/AUnited Kingdom
25EUCTR2010-021343-41-NL21/03/201431 October 2016Variations 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 phenylketonuriaPhenylketonuria (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: Kuvan
Pharmaceutical Form: Soluble tablet
University Medical Center GroningenAuthorisedFemale: yes
Male: yes
Phase 4Netherlands
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26EUCTR2010-024311-13-DE02/01/201426 October 2015A 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®
Pharmaceutical Form: Coated tablet
INN or Proposed INN: Sapropterin
CAS Number: 62989-33-7
Other descriptive name: SAPROPTERIN DIHYDROCHLORIDE
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 100-
Pharmaceutical form of the placebo: Coated tablet
Route of administration of the placebo: Oral use
Merck KGaANot RecruitingFemale: yes
Male: yes
30Phase 2aFrance;Belgium;Spain;Netherlands;Germany;Italy;Switzerland
27NCT01965691October 201313 June 2016Protein 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;Royal Embassy of Saudi Arabia-Cultural Bureau OttawaNot recruiting5 Years18 YearsBoth4N/ACanada
28NCT01965912October 20135 February 2018Kuvan®'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 PharmaceuticalNot recruiting4 Years5 YearsAll34Phase 4Germany;Italy;Spain;United Kingdom
29EUCTR2009-015844-41-DE10/09/201323 May 2016A 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
Pharmaceutical Form: Soluble tablet
INN or Proposed INN: SAPROPTERIN
CAS Number: 62989-33-7
Other descriptive name: tetrahydrobiopterin
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 100-
BioMarin International Ltd.AuthorisedFemale: yes
Male: yes
60Phase 4Spain;Germany;United Kingdom
30NCT01889862July 29, 201326 August 2019Phase 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 PharmaceuticalNot recruiting18 Years70 YearsAll215Phase 3United States
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31NCT01819727May 201316 December 2017An 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 PharmaceuticalNot recruiting18 Years70 YearsAll261Phase 3United States
32NCT01806051March 201319 October 2017A Pilot Study on Diurnal VariationA Pilot Study on the Diurnal Variation in PKU Patients With KuvanPhenylketonuria (PKU)Drug: KuvanDr. Linda RandolphBioMarin PharmaceuticalNot recruiting4 YearsN/AAll6N/AUnited States
33NCT01732471November 201219 October 2017Phase 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 KGaANot recruiting4 Years18 YearsAll90Phase 3Germany
34NCT01560286May 201226 August 2019A 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 PharmaceuticalNot recruiting16 Years70 YearsAll24Phase 2United States
35NCT01617070May 201219 October 2017Effects 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 PharmaceuticalNot recruiting18 YearsN/AAll10Phase 4United States
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36NCT01395394June 201119 October 2017Phenylketonuria, 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 UniversityNot recruiting10 Years45 YearsAll12Phase 2United States
37NCT01541397June 201119 October 2017Bone 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 PharmaceuticalNot recruiting18 Years50 YearsAll6N/AUnited States
38NCT01412437April 201119 February 2015Neuroimaging 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 UniversityNot recruiting18 Years40 YearsBoth0N/AUnited States
39NCT01212744March 201116 December 2017Safety, 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 PharmaceuticalNot recruiting16 Years70 YearsAll16Phase 2United States
40EUCTR2009-015768-33-GB04/01/201130 April 2019Safety 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
Pharmaceutical Form: Soluble tablet
INN or Proposed INN: SAPROPTERIN
CAS Number: 62989337
Other descriptive name: tetrahydrobiopterin
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 100-
BioMarin International Ltd.Not Recruiting Female: yes
Male: yes
56Phase 3Portugal;Czech Republic;Slovakia;Belgium;Turkey;Austria;Netherlands;Germany;Italy;United Kingdom
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41NCT01274026January 201124 September 2018Evaluation 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 PharmaceuticalNot recruiting2 YearsN/AAll21Phase 4United States
42EUCTR2010-019767-11-AT08/09/20104 August 2015Ö-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-PKU1Hyperphenylalaninaemia (HPA) in adult and paediatric patients of 4 years of age and over with phenylketonuria (PKU).Trade Name: Kuvan
Pharmaceutical Form: Soluble tablet
Graz Medical UniversityNot RecruitingFemale: yes
Male: yes
Austria
43NCT01114737August 201019 October 2017Safety 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 PharmaceuticalNot recruiting8 Years65 YearsAll206Phase 3United States;Canada
44NCT01082328May 201019 October 2017Response 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 ASNot recruiting4 YearsN/AAll59Phase 4Norway
45EUCTR2009-018168-81-DK16/04/20106 May 2013ENDURE: 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 - ENDURESubjects 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
CAS Number: 69056-38-8
Other descriptive name: SAPROPTERIN HYDROCHLORIDE
Concentration unit: mg/kg milligram(s)/kilogram
Concentration number: 100 -
Merck Serono NorwayNot RecruitingFemale: yes
Male: yes
70Phase 4Denmark
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46NCT00986973March 201019 October 2017Fluorodeoxyglucose 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 PhiladelphiaNot recruiting18 Years50 YearsAll6N/AUnited States
47NCT00924703October 8, 200915 April 2019Long-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 PharmaceuticalNot recruiting16 Years55 YearsAll68Phase 2United States
48NCT00925054September 200916 December 2017Dose-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-PEGBioMarin PharmaceuticalNot recruiting16 Years55 YearsAll40Phase 2United States
49NCT00964236August 200918 January 2018The 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 PharmaceuticalNot recruiting6 YearsN/AAll20N/AUnited States
50NCT00909012May 200911 June 2018Quantitative 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 UnionNot recruiting5 Years13 YearsAll114N/AGermany;Italy;Spain;United Kingdom
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
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PhaseCountries
51NCT00838435February 20094 February 2019Effect 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 PharmaceuticalNot recruitingN/A6 YearsAll230Phase 3United States;Canada
52NCT00827762January 200919 February 2015Behavioral 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 UniversityNot recruiting6 Years18 YearsBoth2N/AUnited States
53EUCTR2009-012978-12-NO2 October 2017A 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 NorwayHyperphenylalaninemia (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 Level: LLT Classification code 10034873 Term: Phenylketonuria (PKU)
MedDRA version: 9.1 Level: PT Classification code 10034872 Term: Phenylketonuria
MedDRA version: 9.1 Level: LLT Classification code 10034871 Term: Phenylalaninemia
Trade Name: Kuvan® (sapropterin dihydrochloride)
Pharmaceutical Form: Soluble tablet
INN or Proposed INN: sapropterin
Concentration unit: mg/kg milligram(s)/kilogram
Concentration type: equal
Concentration number: 20-
Merck Serono, an affiliate of E. Merck ABNot RecruitingFemale: yes
Male: yes
Phase 4Norway
54NCT00841100December 200819 February 2015Kuvan 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: KuvanUniversity of MiamiRecruiting4 YearsN/ABoth20N/AUnited States
55NCT00688844October 200819 October 2017Nutritional 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 InstituteNot recruiting4 YearsN/AAll58N/AUnited States
No.TrialIDDate_
enrollement
Last_Refreshed_
on
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PhaseCountries
56NCT00789568October 200819 February 2015A 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: MoxifloxacinBioMarin PharmaceuticalNot recruiting18 Years50 YearsBoth56Phase 1United States
57NCT00778206September 20084 February 2019PKUDOS: Phenylketonuria (PKU) Demographic, Outcomes, and Safety RegistryPKUDOS: Phenylketonuria (PKU) Demographic, Outcomes, and Safety RegistryPhenylketonuria;HyperphenylalaninaemiaDrug: KuvanBioMarin PharmaceuticalRecruitingN/AN/AAll3500Phase 1/Phase 2United States;Puerto Rico
58NCT00730080July 200825 June 2018Sapropterin 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-ColumbiaNot recruiting6 Years50 YearsAll45Phase 1United States
59NCT00634660May 200816 December 2017Safety 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 PharmaceuticalNot recruiting16 Years50 YearsAll25Phase 1United States
60NCT00892554June 200721 December 2015The 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 InstituteNot recruiting12 Years50 YearsFemale33N/AUnited States
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
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sponsor
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Status
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agemin
Inclusion_
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PhaseCountries
61NCT00484991February 200719 February 2015Sapropterin Expanded Access ProgramSapropterin Expanded Access ProgramPhenylketonuriaDrug: Sapropterin dihydrochlorideBioMarin PharmaceuticalNot recruiting9 YearsN/ABothN/AUnited States
62EUCTR2006-000839-10-DE09/08/200619 March 2012A 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 - NAPhenylketonuria (PKU)
MedDRA version: 6.1 Classification code 10034872
Product Name: Phenoptin
Product Code: T1401
Pharmaceutical Form: Tablet
INN or Proposed INN: Sapropterin
CAS Number: 69056-38-8
Current Sponsor code: T1401
Other descriptive name: sapropterin dihydrochloride
Concentration unit: mg milligram(s)
Concentration type: not less then
Concentration number: 100-
BioMarin Pharmaceutical Inc.Not RecruitingFemale: yes
Male: yes
128Phase 3bUnited Kingdom;Germany;Spain;Ireland;Italy
63NCT00355264August 200619 February 2015Safety 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 PharmaceuticalNot recruitingN/AN/ABoth12Phase 2United States
64EUCTR2005-003778-13-DE13/07/200619 March 2012A 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 DeficiencyPhenylketonuria (PKU)
MedDRA version: 8.1 Level: LLT Classification code 10034872 Term: Phenylketonuria
Product Name: Phenoptin
Product Code: T1401
Pharmaceutical Form: Tablet
INN or Proposed INN: Sapropterin
CAS Number: 69056-38-8
Current Sponsor code: T1401
Other descriptive name: saproterin dihydrochloride
Concentration unit: mg milligram(s)
Concentration type: not less then
Concentration number: 100-
BioMarin Pharmaceutical IncNot RecruitingFemale: yes
Male: yes
15Phase 2Germany
65NCT00332189July 200619 October 2017Study 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 PharmaceuticalNot recruiting4 YearsN/AAll111Phase 3United States
No.TrialIDDate_
enrollement
Last_Refreshed_
on
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sponsor
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PhaseCountries
66EUCTR2006-000648-15-AT08/06/200610 June 2013A 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 deficiencyHyperphenylalaninemia 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
Pharmaceutical Form: Oral solution
INN or Proposed INN: Sapropterin
CAS Number: 69056-38-8
Current Sponsor code: BH4
Other descriptive name: n.a.
Concentration unit: mg/kg milligram(s)/kilogram
Concentration type: equal
Concentration number: 10-10
Pharmaceutical form of the placebo: Oral solution
Route of administration of the placebo: Oral use
ORPHANETICS Pharma Entwicklungs- GmbHAuthorisedFemale: yes
Male: yes
75Austria
67EUCTR2005-003777-24-DE24/03/200619 March 2012A 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 - NAPhenylketonuria (PKU)
MedDRA version: 6.1 Classification code 10034872
Product Name: Phenoptin
Product Code: T1401
Pharmaceutical Form: Tablet
INN or Proposed INN: Sapropterin
CAS Number: 69056-38-8
Current Sponsor code: T1401
Other descriptive name: sapropterin dihydrochloride
Concentration unit: mg milligram(s)
Concentration type: not less then
Concentration number: 100-
Pharmaceutical form of the placebo: Tablet
Route of administration of the placebo: Oral use
BioMarin Pharmaceutical Inc.Not RecruitingFemale: yes
Male: yes
300Phase 3Germany;Spain
68NCT00225615November 200519 February 2015A 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 PharmaceuticalNot recruiting8 YearsN/ABoth100Phase 3United States
69EUCTR2004-004513-41-GB25/10/200519 March 2012A 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 LevelsPhenylketonuria (PKU)
MedDRA version: 6.1 Classification code 10034872
Product Name: Phenoptin
Product Code: T1401
Pharmaceutical Form: Tablet
INN or Proposed INN: Sapropterin, sapropterin dihydrochloride
CAS Number: 69056-38-8
Concentration unit: mg milligram(s)
Concentration type: not less then
Concentration number: 100-
BioMarin Pharmaceutical Inc.AuthorisedFemale: yes
Male: yes
100Phase 3Germany;United Kingdom;Ireland;Italy
70EUCTR2004-004512-23-IE22/05/20051 May 2012A 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 - NAPhenylketonuria (PKU)
MedDRA version: 6.1 Classification code 10034872
Product Name: Phenoptin
Product Code: T1401
Pharmaceutical Form: Tablet
INN or Proposed INN: Sapropterin, sapropterin hydrochloride
CAS Number: 69056-38-8
Concentration unit: mg milligram(s)
Concentration type: not less then
Concentration number: 100-
Pharmaceutical form of the placebo: Tablet
Route of administration of the placebo: Oral use
BioMarin Pharmaceutical Inc.AuthorisedFemale: yes
Male: yes
100Phase 3Germany;United Kingdom;Ireland;Italy
No.TrialIDDate_
enrollement
Last_Refreshed_
on
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agemin
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PhaseCountries
71NCT00244218April 200519 February 2015Response to Phenylketonuria to Tetrahydrobiopterin (BH4)Response to Phenylketonuria to Tetrahydrobiopterin (BH4)PhenylketonuriaDrug: tetrahydrobiopterin (BH4)FDA Office of Orphan Products DevelopmentRecruiting10 YearsN/ABoth36Phase 1United States
72NCT00260000April 200519 February 2015Study 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 HelsefondNot recruiting8 YearsN/ABoth15Phase 2Denmark
73NCT00104247March 200519 October 2017Study 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 PharmaceuticalNot recruiting8 YearsN/AAll89Phase 3United States
74NCT00104260December 200419 February 2015Study 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 PharmaceuticalNot recruiting8 YearsN/ABoth700Phase 2United States
75EUCTR2004-002365-21-DK03/11/20043 April 2012Trial 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 PKUPKU, 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: tetrahydrobiopterin
Pharmaceutical Form: Tablet
John F. Kennedy InstituteNot RecruitingFemale: yes
Male: yes
Denmark
No.TrialIDDate_
enrollement
Last_Refreshed_
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agemin
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size
PhaseCountries
76EUCTR2004-002071-16-IE2 October 2017A 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 - NAPhenylketonuria (PKU)
MedDRA version: 6.1 Classification code 10034872
Product Name: Phenoptin
Product Code: T1401
Pharmaceutical Form: Tablet
INN or Proposed INN: Sapropterin, sapropterin hydrochloride
CAS Number: 69056-38-8
Concentration unit: mg milligram(s)
Concentration type: not less then
Concentration number: 100 mg-
BioMarin Pharmaceutical Inc.Not AvailableFemale: yes
Male: yes
400Phase 2Ireland;Germany;Italy;United Kingdom
77NCT00065299May 198419 February 2015Low 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)Not recruitingN/AN/AFemale572N/AUnited States
78NCT00006142December 198319 February 2015Study 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 TexasNot recruitingN/AN/AFemaleN/A

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