Phenylalanine    (DrugBank: Phenylalanine)

1 disease
IDDisease name (Link within this page)Number of trials
240Phenylketonuria38

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.
38 / 125 trials found
No.TrialIDDate_
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1NCT04375592
(ClinicalTrials.gov)
October 202021/4/2020Acceptability and Tolerance of a Ready-to-use Protein Substitute in Tablet Form for the Dietary Management of PhenylketonuriaXPhe Minis - Acceptability and Tolerance Market ResearchPhenylketonurias;Hyperphenylalaninaemia;Tetrahydrobiopterin DeficiencyDietary Supplement: Phenylalanine-free protein substitute in tablet form (XPhe minis)metaX Institut fuer Diatetik GmbHBirmingham Children's HospitalNot yet recruiting7 Years18 YearsAll10NULL
2NCT04480567
(ClinicalTrials.gov)
September 24, 20208/7/2020AAV Gene Therapy Study for Subjects With PKUA Phase 1/2 Open-Label, Dose Escalation Study to Determine the Safety and Efficacy of BMN 307, an Adeno-Associated Virus Vector-Mediated Gene Transfer of Human Phenylalanine Hydroxylase in Subjects With PhenylketonuriaPhenylketonuria (PKU)Drug: BMN 307BioMarin PharmaceuticalNULLRecruiting15 YearsN/AAll100Phase 1;Phase 2United States;United Kingdom
3NCT04302194
(ClinicalTrials.gov)
May 1, 20207/3/2020Early Dietary Treated Patients With Phenylketonuria Can Achieve Normal Growth and Mental Development.Early Dietary Treated Patients With Phenylketonuria Can Achieve Normal Growth and Mental Development.PhenylketonuriasDietary Supplement: phenylalanine restricted dietAssiut UniversityNULLNot yet recruiting1 Month3 YearsAll50NULL
4EUCTR2019-001878-28-GB
(EUCTR)
17/12/201921/11/2019A Phase 1/2 study to evaluate the safety and efficacy of BMN 307 gene therapy in patients with phenylketonuriaA Phase 1/2 Open-Label, Dose Escalation Study to Determine the Safety and Efficacy of BMN 307, an Adeno-Associated Virus Vector-Mediated Gene Transfer of Human Phenylalanine Hydroxylase in Subjects with Phenylketonuria and Plasma Phe Levels > 600 µmol/L - A Phase 1/2 study to evaluate the safety and efficacy of BMN 307 Phenylketonuria
MedDRA version: 20.0;Level: PT;Classification code 10034872;Term: Phenylketonuria;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Product Code: BMN 307BioMarin Pharmaceutical Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
100Phase 1;Phase 2United States;Taiwan;Spain;Turkey;Australia;Germany;Italy;United Kingdom
5NCT03788343
(ClinicalTrials.gov)
August 19, 201917/12/2018Phenylalanine and Its Impact on CognitionPICO: Phenylalanine and Its Impact on Cognition - Impact of Phenylalanine on Cognitive, Cerebral and Neurometabolic Parameters in Adult Patients With PhenylketonuriaPhenylketonuriaDietary Supplement: Phenylalanine;Drug: PlaceboUniversity Hospital Inselspital, BerneUniversity of ZurichRecruiting18 YearsN/AAll60Phase 4Switzerland
No.TrialIDDate_
enrollment
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Public_titleScientific_titleConditionInterventionPrimary_
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agemin
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PhaseCountries
6NCT03333720
(ClinicalTrials.gov)
July 15, 201918/9/2017Compliance, Tolerance and Acceptability of a Tablet Protein Substitute for the Dietary Management of PhenylketonuriaCompliance, Tolerance and Acceptability of a Tablet Protein Substitute for the Dietary Management of PhenylketonuriaPhenylketonuriasDietary Supplement: Phenylalanine-free protein substitute tabletsNutricia UK LtdNULLWithdrawn8 Years100 YearsAll0N/ANULL
7NCT03168399
(ClinicalTrials.gov)
June 8, 201724/5/2017Evaluation of PKU ExploreA Study to Evaluate the Acceptability of PKU Explore, a Renovated Phenylalanine Free Second Stage Protein Substitute for Use in the Dietary Management of Phenylketonuria in Infants From 6 Months to 5 Years of Age With Regard to Product Tolerance and Adherence.Phenylketonuria;Inborn Errors of MetabolismDietary Supplement: PKU ExploreVitaflo International, LtdBirmingham Women's and Children's NHS Foundation TrustCompleted6 Months5 YearsAll22N/AUnited Kingdom
8NCT03058848
(ClinicalTrials.gov)
March 6, 201716/2/2017Evaluation of PKU StartA Study to Evaluate the Acceptability of a New Phenylalanine Free Infant Formula for Use in the Dietary Management of Phenylketonuria in Infants From Birth to 2 Year of Age With Regard to Product Tolerance and Adherence.Phenylketonuria;Inborn Errors of MetabolismDietary Supplement: PKU StartVitaflo International, LtdNULLCompletedN/A2 YearsAll10N/AUnited Kingdom
9EUCTR2010-021343-41-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
10NCT01732471
(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
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agemin
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agemax
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PhaseCountries
11NCT01376908
(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
12NCT00838435
(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
13NCT00841100
(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
14NCT00432822
(ClinicalTrials.gov)
February 20077/2/2007Long-Term Tetrahydrobiopterin Treatment in PKU Patients of 0-18 Years - Study on Phenylalanine Tolerance and SafetyDouble-Blind, Placebo Controlled, Multicentre Study With an Open Label Extension to Evaluate the Efficacy and Safety of Tetrahydrobiopterin (BH4) in Children and Adolescents With Hyperphenylalaninemia Caused by Phenylalanine Hydroxylase DeficiencyPhenylalanine Hydroxylase DeficienciesDrug: tetrahydrobiopterin (BH4)Orphanetics Pharma Entwicklungs GmbHNULLTerminatedN/A18 YearsBoth50Phase 2;Phase 3NULL
15EUCTR2005-003777-24-GB
(EUCTR)
17/08/200620/04/2006A Phase 3, Multicenter, Randomized, Double-Blind, Placebo-controlled Study to Evaluate the Safety and Efficacy of Phenoptin 20 mg/kg/day to Increase Phenylalanine Tolerance in Phenylketonuric Children on a Phenylalanine-restricted Diet - NAA Phase 3, Multicenter, Randomized, Double-Blind, Placebo-controlled Study to Evaluate the Safety and Efficacy of Phenoptin 20 mg/kg/day to Increase Phenylalanine Tolerance in Phenylketonuric Children on a Phenylalanine-restricted Diet - NA Phenylketonuria (PKU)
MedDRA version: 6.1;Classification code 10034872
BioMarin Pharmaceutical Inc.NULLNot Recruiting Female: yes
Male: yes
300Phase 3Spain;Germany;United Kingdom
No.TrialIDDate_
enrollment
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Public_titleScientific_titleConditionInterventionPrimary_
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PhaseCountries
16EUCTR2006-000648-15-AT
(EUCTR)
08/06/200605/04/2006A double-blind, placebo-controlled, multicentre study with an open-label extension to evaluate the efficacy and safety of tetrahydrobiopterin (BH4) in children and adolescents with hyperphenylalaninemia caused by phenylalanine hydroxylase deficiencyA double-blind, placebo-controlled, multicentre study with an open-label extension to evaluate the efficacy and safety of tetrahydrobiopterin (BH4) in children and adolescents with hyperphenylalaninemia caused by phenylalanine hydroxylase deficiency Hyperphenylalaninemia due to phenylalanine hydroxylase deficiency. Phenotypes: classic phenylketonuria (PKU), mild PKU (MPK) or mild hyperphenylalaninemia (HPA).
MedDRA version: 81;Level: LLT;Classification code 10034873
Product Name: tetrahydrobiopterin
Product Code: BH4
INN or Proposed INN: Sapropterin
Other descriptive name: n.a.
ORPHANETICS Pharma Entwicklungs- GmbHNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
75Austria
17EUCTR2005-003777-24-DE
(EUCTR)
24/03/200614/12/2005A Phase 3, Multicenter, Randomized, Double-Blind, Placebo-controlled Study to Evaluate the Safety and Efficacy of Phenoptin 20 mg/kg/day to Increase Phenylalanine Tolerance in Phenylketonuric Children on a Phenylalanine-restricted Diet - NAA Phase 3, Multicenter, Randomized, Double-Blind, Placebo-controlled Study to Evaluate the Safety and Efficacy of Phenoptin 20 mg/kg/day to Increase Phenylalanine Tolerance in Phenylketonuric Children on a Phenylalanine-restricted Diet - NA Phenylketonuria (PKU)
MedDRA version: 6.1;Classification code 10034872
Product Name: Phenoptin
Product Code: T1401
INN or Proposed INN: Sapropterin
Other descriptive name: sapropterin dihydrochloride
BioMarin Pharmaceutical Inc.NULLNot RecruitingFemale: yes
Male: yes
300Phase 3Germany;Spain
18NCT00225615
(ClinicalTrials.gov)
November 200522/9/2005A Phase 3, Multicenter, Open-Label Extension Study of Phenoptin in Subjects With PKU Who Have Elevated Phenylalanine LevelsA Phase 3, Multicenter, Open-Label Extension Study of Phenoptin in Subjects With Phenylketonuria Who Have Elevated Phenylalanine LevelsPhenylketonuriasDrug: sapropterin dihydrochlorideBioMarin PharmaceuticalNULLCompleted8 YearsN/ABoth100Phase 3United States
19EUCTR2004-004513-41-DE
(EUCTR)
27/10/200512/05/2006A Phase 3, Multicenter, Open-Label Extension Study of Phenoptin in Subjects with Phenylketonuria Who Have Elevated Phenylalanine LevelsA Phase 3, Multicenter, Open-Label Extension Study of Phenoptin in Subjects with Phenylketonuria Who Have Elevated Phenylalanine Levels Phenylketonuria (PKU)
MedDRA version: 6.1;Classification code 10034872
Product Name: Phenoptin
Product Code: T1401
INN or Proposed INN: Sapropterin, sapropterin dihydrochloride
BioMarin Pharmaceutical Inc.NULLNot RecruitingFemale: yes
Male: yes
100Phase 3Ireland;Germany;Italy;United Kingdom
20EUCTR2004-004513-41-GB
(EUCTR)
25/10/200502/06/2005A Phase 3, Multicenter, Open-Label Extension Study of Phenoptin in Subjects with Phenylketonuria Who Have Elevated Phenylalanine LevelsA Phase 3, Multicenter, Open-Label Extension Study of Phenoptin in Subjects with Phenylketonuria Who Have Elevated Phenylalanine Levels Phenylketonuria (PKU)
MedDRA version: 6.1;Classification code 10034872
BioMarin Pharmaceutical Inc.NULLNot Recruiting Female: yes
Male: yes
100Phase 3Ireland;Germany;Italy;United Kingdom
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
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agemin
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agemax
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PhaseCountries
21EUCTR2004-004513-41-IT
(EUCTR)
23/09/200511/11/2005A Phase 3, Multicenter, Open-Label Extension Study of Phenoptin in Subjects with Phenylketonuria Who Have Elevated Phenylalanine LevelsA Phase 3, Multicenter, Open-Label Extension Study of Phenoptin in Subjects with Phenylketonuria Who Have Elevated Phenylalanine Levels Treatment of phenylketonuria
MedDRA version: 6.1;Level: SOC;Classification code 10027433
Product Name: PhenoptinBIOMARINNULLNot RecruitingFemale: yes
Male: yes
50Phase 3Germany;United Kingdom;Ireland;Italy
22EUCTR2004-004513-41-IE
(EUCTR)
19/08/200510/06/2005A Phase 3, Multicenter, Open-Label Extension Study of Phenoptin in Subjects with Phenylketonuria Who Have Elevated Phenylalanine LevelsA Phase 3, Multicenter, Open-Label Extension Study of Phenoptin in Subjects with Phenylketonuria Who Have Elevated Phenylalanine Levels Phenylketonuria (PKU)
MedDRA version: 6.1;Classification code 10034872
BioMarin Pharmaceutical Inc.NULLNot Recruiting Female: yes
Male: yes
100Phase 3Ireland;Germany;Italy;United Kingdom
23EUCTR2004-004512-23-GB
(EUCTR)
15/08/200523/02/2005A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Phenoptin in Subjects with Phenylketonuria Who Have Elevated Phenylalanine Levels - NAA Phase 3, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Phenoptin in Subjects with Phenylketonuria Who Have Elevated Phenylalanine Levels - NA Phenylketonuria (PKU)
MedDRA version: 6.1;Classification code 10034872
BioMarin Pharmaceutical Inc.NULLNot Recruiting Female: yes
Male: yes
100Phase 3Ireland;Germany;Italy;United Kingdom
24EUCTR2004-004512-23-IT
(EUCTR)
09/06/200522/06/2005A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Phenoptin in Subjects with Phenylketonuria Who Have Elevated Phenylalanine LevelsA Phase 3, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Phenoptin in Subjects with Phenylketonuria Who Have Elevated Phenylalanine Levels Treatment of phenylketonuria
MedDRA version: 6.1;Level: SOC;Classification code 10027433
Product Name: PhenoptinBIOMARINNULLNot RecruitingFemale: yes
Male: yes
100Phase 3Germany;United Kingdom;Ireland;Italy
25EUCTR2004-004512-23-IE
(EUCTR)
22/05/200515/02/2005A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Phenoptin in Subjects with Phenylketonuria Who Have Elevated Phenylalanine Levels - NAA Phase 3, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Phenoptin in Subjects with Phenylketonuria Who Have Elevated Phenylalanine Levels - NA Phenylketonuria (PKU)
MedDRA version: 6.1;Classification code 10034872
BioMarin Pharmaceutical Inc.NULLNot Recruiting Female: yes
Male: yes
100Phase 3Ireland;Germany;Italy;United Kingdom
No.TrialIDDate_
enrollment
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Public_titleScientific_titleConditionInterventionPrimary_
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agemin
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PhaseCountries
26NCT00260000
(ClinicalTrials.gov)
April 200530/11/2005Study of BH4, a New and Simple Treatment of Mild PKUStudy of the Response of Tetrahydrobiopterin on S-Phenylalanine in Patients With PKU Housing the Y414C MutationPhenylketonuriaDrug: 5,6,7,8-tetrahydrobiopterinThe Kennedy Institute-National Eye ClinicSygekassernes HelsefondCompleted8 YearsN/ABoth15Phase 2Denmark
27EUCTR2004-002071-16-GB
(EUCTR)
18/03/200522/02/2005A Phase 2, Multicenter, Open-Label Study to Evaluate the Response to and Safety of an 8-Day Course of Phenoptin Treatment in Subjects with Phenylketonuria Who Have Elevated Phenylalanine Levels - NAA Phase 2, Multicenter, Open-Label Study to Evaluate the Response to and Safety of an 8-Day Course of Phenoptin Treatment in Subjects with Phenylketonuria Who Have Elevated Phenylalanine Levels - NA Phenylketonuria (PKU)
MedDRA version: 6.1;Classification code 10034872
BioMarin Pharmaceutical Inc.NULLNot Recruiting Female: yes
Male: yes
700Phase 2Ireland;Germany;Italy;United Kingdom
28EUCTR2004-002071-16-IT
(EUCTR)
17/03/200527/04/2005A Phase 2, Multicenter, Open-Label Study to Evaluate the Response to and Safety of an 8-Day Course of Phenoptin Treatment in Subjects with Phenylketonuria Who Have Elevated Phenylalanine LevelsA Phase 2, Multicenter, Open-Label Study to Evaluate the Response to and Safety of an 8-Day Course of Phenoptin Treatment in Subjects with Phenylketonuria Who Have Elevated Phenylalanine Levels Treatment of Phenylketonuria
MedDRA version: 6.1;Level: SOC;Classification code 10027433
Product Name: Phenoptin
Product Code: NA
INN or Proposed INN: sapropterin hydrochloride
Other descriptive name: NA
BIOMARINNULLNot RecruitingFemale: yes
Male: yes
Phase 2United Kingdom;Germany;Italy
29NCT00104247
(ClinicalTrials.gov)
March 200524/2/2005Study to Evaluate the Safety and Efficacy of Phenoptin™ in Subjects With Phenylketonuria Who Have Elevated Phenylalanine LevelsA Phase 3, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Phenoptin™ in Subjects With Phenylketonuria Who Have Elevated Phenylalanine LevelsPhenylketonuriasDrug: sapropterin dihydrochloride, 6R-BH4, tetrahydrobiopterinBioMarin PharmaceuticalNULLCompleted8 YearsN/AAll89Phase 3United States
30EUCTR2004-002071-16-DE
(EUCTR)
28/01/200514/10/2004A Phase 2, Multicenter, Open-Label Study to Evaluate the Response to and Safety of an 8-Day Course of Phenoptin Treatment in Subjects with Phenylketonuria Who Have Elevated Phenylalanine Levels - NAA Phase 2, Multicenter, Open-Label Study to Evaluate the Response to and Safety of an 8-Day Course of Phenoptin Treatment in Subjects with Phenylketonuria Who Have Elevated Phenylalanine Levels - NA Phenylketonuria (PKU)
MedDRA version: 6.1;Classification code 10034872
Product Name: Phenoptin
Product Code: T1401
INN or Proposed INN: Sapropterin, sapropterin hydrochloride
BioMarin Pharmaceutical Inc.NULLNot RecruitingFemale: yes
Male: yes
700Phase 2United Kingdom;Germany;Italy
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
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Inclusion_
agemin
Inclusion_
agemax
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gender
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size
PhaseCountries
31NCT00104260
(ClinicalTrials.gov)
December 200424/2/2005Study to Evaluate the Response to and Safety of an 8-Day Course of Phenoptin™ Treatment in Subjects With PhenylketonuriaA Phase 2, Multicenter, Open-Label Study to Evaluate the Response to and Safety of an 8-Day Course of Phenoptin™ Treatment in Subjects With Phenylketonuria Who Have Elevated Phenylalanine LevelsPhenylketonuriasDrug: sapropterin dihydrochlorideBioMarin PharmaceuticalNULLCompleted8 YearsN/ABoth700Phase 2United States
32EUCTR2004-002365-21-DK
(EUCTR)
03/11/200409/07/2008Trial with BH4, a new and simple treatment of phenylketonuria, PKU - BH4 and PKUTrial with BH4, a new and simple treatment of phenylketonuria, PKU - BH4 and PKU PKU, phenylketonuria, is a rare, inherited metabolic disease that results in mental retardation if not a very strict low-protein diet is started within the first weeks of life. The conversion of phenylalanine to tyrosine is defect, phe accumulates and leads to brain damage. There are different degrees of severity, reflecting the spectrum of mutant genes. BH4, tetrahydrobiopterin, is co-enzym for the conversion of phe to tyrosine. BH4 can lower phe in some patients with milder forms of PKU.Product Name: tetrahydrobiopterinJohn F. Kennedy InstituteNULLNot RecruitingFemale: yes
Male: yes
Denmark
33NCT00065299
(ClinicalTrials.gov)
May 198421/7/2003Low Phenylalanine Diet for Mothers With Phenylketonuria (PKU)Effects of Maternal Phenylketonuria (PKU) on Pregnancy OutcomePhenylketonuriaBehavioral: Restricted phenylalanine dietEunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)NULLCompletedN/AN/AFemale572N/AUnited States
34NCT00006142
(ClinicalTrials.gov)
December 19833/8/2000Study of a Phenylalanine Restricted Diet During Pregnancy to Prevent Symptoms in Offspring of Patients With PhenylketonuriaPhenylketonuriaBehavioral: phenylalanine restricted dietNational Center for Research Resources (NCRR)University of TexasCompletedN/AN/AFemaleN/ANULL
35EUCTR2015-001650-15-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
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
36EUCTR2009-012978-12-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
37EUCTR2004-002071-16-IE
(EUCTR)
13/10/2004A Phase 2, Multicenter, Open-Label Study to Evaluate the Response to and Safety of an 8-Day Course of Phenoptin Treatment in Subjects with Phenylketonuria Who Have Elevated Phenylalanine Levels - NAA Phase 2, Multicenter, Open-Label Study to Evaluate the Response to and Safety of an 8-Day Course of Phenoptin Treatment in Subjects with Phenylketonuria Who Have Elevated Phenylalanine Levels - NA Phenylketonuria (PKU)
MedDRA version: 6.1;Classification code 10034872
BioMarin Pharmaceutical Inc.NULLNot Recruiting Female: yes
Male: yes
400Phase 2Ireland;Germany;Italy;United Kingdom
38EUCTR2004-004512-23-DE
(EUCTR)
17/03/2005A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Phenoptin in Subjects with Phenylketonuria Who Have Elevated Phenylalanine Levels - NAA Phase 3, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Phenoptin in Subjects with Phenylketonuria Who Have Elevated Phenylalanine Levels - NA Phenylketonuria (PKU)
MedDRA version: 6.1;Classification code 10034872
Product Name: Phenoptin
Product Code: T1401
INN or Proposed INN: Sapropterin, sapropterin hydrochloride
BioMarin Pharmaceutical Inc.NULLNot RecruitingFemale: yes
Male: yes
100Phase 3United Kingdom;Germany;Ireland;Italy