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Clinical trials for Phenylketonuria

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    The EU Clinical Trials Register currently displays   44334   clinical trials with a EudraCT protocol, of which   7366   are clinical trials conducted with subjects less than 18 years old.   The register also displays information on   18700   older paediatric trials (in scope of Article 45 of the Paediatric Regulation (EC) No 1901/2006).

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    24 result(s) found for: Phenylketonuria. Displaying page 1 of 2.
    1  2  Next»
    EudraCT Number: 2009-012978-12 Sponsor Protocol Number: ME026 Start Date*: Information not available in EudraCT
    Sponsor Name:Merck Serono, an affiliate of E. Merck AB
    Full Title: A 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® ...
    Medical condition: Hyperphenylalaninemia (HPA) in adults and children >=16 years of age with phenylketonuri (PKU) (classic or mild PKU, or mild hyperphenylalaninemia (HPA)).
    Disease: Version SOC Term Classification Code Term Level
    9.1 10034872 Phenylketonuria LLT
    9.1 10034873 Phenylketonuria (PKU) LLT
    9.1 10034872 Phenylketonuria PT
    9.1 10034871 Phenylalaninemia LLT
    Population Age: Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: NO (Prematurely Ended)
    Trial results: (No results available)
    EudraCT Number: 2010-024311-13 Sponsor Protocol Number: EMR700773-004 Start Date*: 2014-01-02
    Sponsor Name:Merck KGaA
    Full Title: 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
    Medical condition: Phenylketonuria
    Disease: Version SOC Term Classification Code Term Level
    16.0 100000004850 10034873 Phenylketonuria (PKU) LLT
    Population Age: Adults Gender: Male, Female
    Trial protocol: DE (Prematurely Ended) IT (Prematurely Ended) BE (Completed) ES (Completed) NL (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2006-000839-10 Sponsor Protocol Number: PKU-008 Start Date*: 2006-08-09
    Sponsor Name:BioMarin Pharmaceutical Inc.
    Full Title: A Phase 3b, Multicenter, Open-Label Extension Study of Phenoptin in Subjects with Phenylketonuria Who Participated in Protocols PKU-004 or PKU-006
    Medical condition: Phenylketonuria (PKU)
    Disease: Version SOC Term Classification Code Term Level
    6.1 10034872
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: DE (Completed) IE (Completed) ES (Ongoing) GB (Completed) IT (Completed)
    Trial results: (No results available)
    EudraCT Number: 2004-004512-23 Sponsor Protocol Number: PKU-003 Start Date*: 2005-05-22
    Sponsor Name:BioMarin Pharmaceutical Inc.
    Full Title: A 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
    Medical condition: Phenylketonuria (PKU)
    Disease: Version SOC Term Classification Code Term Level
    6.1 10034872
    Population Age: Children, Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: IE (Completed) GB (Completed) DE (Completed) IT (Completed)
    Trial results: View results
    EudraCT Number: 2004-002071-16 Sponsor Protocol Number: PKU-001 Start Date*: Information not available in EudraCT
    Sponsor Name:BioMarin Pharmaceutical Inc.
    Full Title: A 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
    Medical condition: Phenylketonuria (PKU)
    Disease: Version SOC Term Classification Code Term Level
    6.1 10034872
    Population Age: Children, Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: IE (Completed) DE (Completed) GB (Completed) IT (Completed)
    Trial results: View results
    EudraCT Number: 2005-003777-24 Sponsor Protocol Number: PKU-006 Start Date*: 2006-03-24
    Sponsor Name:BioMarin Pharmaceutical Inc.
    Full Title: A 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 Child...
    Medical condition: Phenylketonuria (PKU)
    Disease: Version SOC Term Classification Code Term Level
    6.1 10034872
    Population Age: Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: DE (Completed) ES (Completed) GB (Completed)
    Trial results: (No results available)
    EudraCT Number: 2021-005058-27 Sponsor Protocol Number: 165-306 Start Date*: Information not available in EudraCT
    Sponsor Name:BioMarin Pharmaceutical Inc.
    Full Title: A Phase 3 Multi-Center Study to Evaluate the Safety and Efficacy of Subcutaneous Injections of Pegvaliase in Adolescent Subjects (Ages 12-17) with Phenylketonuria Featuring an Open-Label Randomized...
    Medical condition: Phenylketonuria (PKU)
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10034873 Phenylketonuria (PKU) LLT
    Population Age: Adolescents, Under 18 Gender: Male, Female
    Trial protocol: DE (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2004-004513-41 Sponsor Protocol Number: PKU-004 Start Date*: 2005-10-25
    Sponsor Name:BioMarin Pharmaceutical Inc.
    Full Title: A Phase 3, Multicenter, Open-Label Extension Study of Phenoptin in Subjects with Phenylketonuria Who Have Elevated Phenylalanine Levels
    Medical condition: Phenylketonuria (PKU)
    Disease: Version SOC Term Classification Code Term Level
    6.1 10034872
    Population Age: Children, Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: GB (Completed) IE (Completed) IT (Completed) DE (Completed)
    Trial results: View results
    EudraCT Number: 2009-015768-33 Sponsor Protocol Number: EMR700773-003/BMN162-503 Start Date*: 2011-01-04
    Sponsor Name:BioMarin International Ltd.
    Full Title: A Phase IIIb, Multicentre, Open-Label, Randomized, Controlled Study of the Efficacy, Safety, and Population Pharmacokinetics of Sapropterin Dihydrochloride (Kuvan®) in Phenylketonuria (PKU) Patient...
    Medical condition: Phenylketonuria
    Disease: Version SOC Term Classification Code Term Level
    19.0 100000004850 10034873 Phenylketonuria (PKU) LLT
    Population Age: Infants and toddlers, Children, Under 18 Gender: Male, Female
    Trial protocol: GB (Completed) SK (Completed) DE (Completed) AT (Completed) CZ (Completed) PT (Completed) BE (Completed) NL (Completed) IT (Completed)
    Trial results: View results
    EudraCT Number: 2009-018168-81 Sponsor Protocol Number: EMR-700773-503 Start Date*: 2010-04-16
    Sponsor Name:Merck Serono Norway
    Full Title: ENDURE: 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...
    Medical condition: Subjects diagnosed with Phenylketonuria (PKU) (classic or mild PKU, or mild hyperphenylalaninaemia (HPA)).
    Disease: Version SOC Term Classification Code Term Level
    14.1 10010331 - Congenital, familial and genetic disorders 10034873 Phenylketonuria (PKU) LLT
    Population Age: Children, Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: DK (Completed) NO (Ongoing)
    Trial results: View results
    EudraCT Number: 2005-003778-13 Sponsor Protocol Number: PKU-007 Start Date*: 2006-07-13
    Sponsor Name:BioMarin Pharmaceutical Inc
    Full Title: A Phase 2, Multicenter, Open-label Study to Evaluate the Safety and Efficacy of Phenoptin in Subjects with Hyperphenylalaninemia Due to Primary BH4 Deficiency
    Medical condition: Phenylketonuria (PKU)
    Disease: Version SOC Term Classification Code Term Level
    8.1 10034872 Phenylketonuria LLT
    Population Age: Children, Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: DE (Completed)
    Trial results: View results
    EudraCT Number: 2021-000497-28 Sponsor Protocol Number: PTC923-MD-004-PKU Start Date*: Information not available in EudraCT
    Sponsor Name:PTC Therapeutics, Inc.
    Full Title: Phase 3 Open-Label Study of PTC923 (Sepiapterin) in Phenylketonuria
    Medical condition: Metabolic Disorders - Phenylketonuria
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004850 10034873 Phenylketonuria (PKU) LLT
    Population Age: Newborns, Infants and toddlers, Children, Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: DE (Trial now transitioned) ES (Ongoing) PT (Trial now transitioned) DK (Trial now transitioned) NL (Trial now transitioned) IT (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2015-001650-15 Sponsor Protocol Number: EMR700773-510 Start Date*: 2015-06-26
    Sponsor Name:Merck Serono Middle East FZ-LLC
    Full Title: A 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 Wi...
    Medical condition: Phenylketonuria
    Disease:
    Population Age: Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2021-000474-29 Sponsor Protocol Number: PTC923-MD-003-PKU Start Date*: Information not available in EudraCT
    Sponsor Name:PTC Therapeutics, Inc.
    Full Title: A Phase 3 Study of PTC923 in Subjects with Phenylketonuria
    Medical condition: Metabolic Disorders - Phenylketonuria
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004850 10034873 Phenylketonuria (PKU) LLT
    Population Age: Infants and toddlers, Children, Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: DE (Completed) ES (Ongoing) PT (Completed) DK (Completed) NL (Completed) IT (Completed)
    Trial results: View results
    EudraCT Number: 2009-015844-41 Sponsor Protocol Number: BMN162-502 Start Date*: 2013-07-25
    Sponsor Name:BioMarin International Ltd.
    Full Title: 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.
    Medical condition: Phenylketonuria
    Disease: Version SOC Term Classification Code Term Level
    19.0 100000004850 10034873 Phenylketonuria (PKU) LLT
    Population Age: Children, Under 18 Gender: Male, Female
    Trial protocol: GB (Completed) DE (Completed) IT (Completed) ES (Completed)
    Trial results: View results
    EudraCT Number: 2006-000648-15 Sponsor Protocol Number: BH4/III/05/001 Start Date*: 2006-06-08
    Sponsor Name:ORPHANETICS Pharma Entwicklungs- GmbH
    Full Title: A 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 hyperphenylalani...
    Medical condition: Hyperphenylalaninemia due to phenylalanine hydroxylase deficiency. Phenotypes: classic phenylketonuria (PKU), mild PKU (MPK) or mild hyperphenylalaninemia (HPA).
    Disease: Version SOC Term Classification Code Term Level
    81 10034873 LLT
    Population Age: Newborns, Infants and toddlers, Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: AT (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2008-005394-35 Sponsor Protocol Number: EC81/00474 Start Date*: 2009-11-10
    Sponsor Name:Intituto Aragonés de Ciencias de la Salud
    Full Title: Tratamiento de la hiperfenilalaninemia con dihidrocloruro de sapropterina (tetrahidrobiopterina, 6R-BH4) y su influencia en el patrón de aminoácidos y ácidos grasos desde la infancia hasta la edad ...
    Medical condition: La indicación terapéutica de la sapropterina es el diagnóstico y tratamiento de la hiperfenilalaninemia, causada por deficiencia de tetrahidrobiopterina o por una disminución de la afinidad de la e...
    Disease: Version SOC Term Classification Code Term Level
    9 10034871 Phenylalaninemia LLT
    Population Age: Infants and toddlers, Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: ES (Completed)
    Trial results: (No results available)
    EudraCT Number: 2019-001878-28 Sponsor Protocol Number: 307-201 Start Date*: 2019-12-17
    Sponsor Name:BioMarin Pharmaceutical Inc.
    Full Title: A 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...
    Medical condition: Phenylketonuria
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10034872 Phenylketonuria PT
    Population Age: Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: GB (GB - no longer in EU/EEA)
    Trial results: (No results available)
    EudraCT Number: 2021-003777-63 Sponsor Protocol Number: 202100482 Start Date*: Information not available in EudraCT
    Sponsor Name:University Medical Center Groningen
    Full Title: Tetrahydrobiopterin (BH4) treatment in Phenylketonuria - comparing different practices of dosing regimen
    Medical condition: Phenylketonuria (PKU; OMIM 261600)
    Disease:
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: NL (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2010-021343-41 Sponsor Protocol Number: ME040 Start Date*: 2014-03-21
    Sponsor Name:University Medical Center Groningen
    Full Title: Effect of sapropterin on variations of blood phenylalanine and tyrosine over 24 hours and from day to day in children with phenylketonuria
    Medical condition: 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:
    Population Age: Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: NL (Ongoing)
    Trial results: (No results available)
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