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

Searched query = "Phenylketonuria", "PKU", "Phenylalanine hydroxylase deficiency", "PAH deficiency", "Tetrahydrobiopterin deficiency", "BH4 deficiency", "BH4 reactive hyper pheemia"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.

Search in Page e.g. "Phase 3", "Not recruiting", "Japan"
43 trials found
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1NCT02677870
(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
2NCT03864029
(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
3EUCTR2010-024311-13-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 2aFrance;Spain;Belgium;Germany;Netherlands;Italy;Switzerland
4EUCTR2010-021343-41-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
5EUCTR2010-024311-13-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 2aFrance;Belgium;Spain;Netherlands;Germany;Italy;Switzerland
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
6EUCTR2010-024311-13-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 2aFrance;Spain;Belgium;Netherlands;Germany;Italy;Switzerland
7EUCTR2010-024311-13-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 2aFrance;Belgium;Spain;Netherlands;Germany;Italy;Switzerland
8EUCTR2009-015844-41-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
9NCT01965912
(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
10EUCTR2010-024311-13-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 2aFrance;Belgium;Spain;Netherlands;Germany;Switzerland;Italy
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
11EUCTR2009-015844-41-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]
BioMarin International Ltd.NULLAuthorised-recruitment may be ongoing or finished Female: yes
Male: yes
60Phase 4Spain;Germany;Italy;United Kingdom
12EUCTR2009-015844-41-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
13EUCTR2009-015844-41-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
14NCT01806051
(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
15NCT01732471
(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
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
16NCT01617070
(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
17NCT01541397
(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
18NCT01376908
(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
19NCT01395394
(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
20EUCTR2009-015768-33-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
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
21EUCTR2009-015768-33-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 3bPortugal;Czech Republic;Germany;Slovakia;United Kingdom;Turkey;Netherlands;Belgium;Italy;Austria
22EUCTR2009-015768-33-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
23EUCTR2009-015768-33-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
24EUCTR2009-015768-33-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
25EUCTR2009-015768-33-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
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
26EUCTR2009-015768-33-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
27EUCTR2009-015768-33-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]
BioMarin International Ltd.NULLNot Recruiting Female: yes
Male: yes
56Phase 3Portugal;Czech Republic;Slovakia;Belgium;Turkey;Austria;Netherlands;Germany;Italy;United Kingdom
28NCT01274026
(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
29EUCTR2009-015768-33-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
30EUCTR2010-019767-11-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
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
31NCT01082328
(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
32EUCTR2009-018168-81-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
33NCT00986973
(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
34NCT00964236
(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
35NCT00838435
(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
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
36NCT00827762
(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
37NCT00841100
(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
38NCT00688844
(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
39NCT00778206
(ClinicalTrials.gov)
September 200821/10/2008PKUDOS: Phenylketonuria (PKU) Demographic, Outcomes, and Safety RegistryPKUDOS: Phenylketonuria (PKU) Demographic, Outcomes, and Safety RegistryPhenylketonuria;HyperphenylalaninaemiaDrug: KuvanBioMarin PharmaceuticalNULLActive, not recruitingN/AN/AAll1927United States;Puerto Rico
40NCT00730080
(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
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
41EUCTR2009-018168-81-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
42EUCTR2009-012978-12-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
43EUCTR2015-001650-15-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