240. Phenylketonuria
125 clinical trials,   95 drugs   (DrugBank: 11 drugs),   1 drug target gene,   5 drug target pathways

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"
29 trials found
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1NCT03864029
(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
2EUCTR2010-024311-13-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
3EUCTR2010-024311-13-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
4EUCTR2010-024311-13-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
5EUCTR2010-024311-13-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
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
6EUCTR2009-015844-41-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
7NCT01965912
(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
8EUCTR2010-024311-13-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
9EUCTR2009-015844-41-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
10EUCTR2009-015844-41-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
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-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
12NCT01732471
(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
13NCT01376908
(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
14NCT01395394
(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
15EUCTR2009-015768-33-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
16EUCTR2009-015768-33-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
17EUCTR2009-015768-33-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
18EUCTR2009-015768-33-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
19EUCTR2009-015768-33-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
20EUCTR2009-015768-33-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
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-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
22NCT01274026
(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
23EUCTR2009-015768-33-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
24NCT01082328
(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
25EUCTR2009-018168-81-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
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
26NCT00838435
(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
27EUCTR2009-012978-12-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
28EUCTR2015-001650-15-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
29EUCTR2009-018168-81-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