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Clinical trials for Hyperphenylalaninemia

The European Union Clinical Trials Register   allows you to search for protocol and results information on:
  • interventional clinical trials that were approved in the European Union (EU)/European Economic Area (EEA) under the Clinical Trials Directive 2001/20/EC
  • clinical trials conducted outside the EU/EEA that are linked to European paediatric-medicine development

    • EU/EEA interventional clinical trials approved under or transitioned to the Clinical Trial Regulation 536/2014 are publicly accessible through the
    Clinical Trials Information System (CTIS).

    The EU Clinical Trials Register currently displays   44364   clinical trials with a EudraCT protocol, of which   7388   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).

    Phase 1 trials conducted solely on adults and that are not part of an agreed paediatric investigation plan (PIP) are not publicly available (see Frequently Asked Questions ).  
     
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    9 result(s) found for: Hyperphenylalaninemia. Displaying page 1 of 1.
    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: 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: 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: 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: 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: 2021-000474-29 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: 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: 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: 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)
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    The status and protocol content of GB trials is no longer updated since 1 January 2021. For the UK, as of 31 January 2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI. Legal notice
    As of 31 January 2023, all EU/EEA initial clinical trial applications must be submitted through CTIS . Updated EudraCT trials information and information on PIP/Art 46 trials conducted exclusively in third countries continues to be submitted through EudraCT and published on this website.

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