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Clinical trials for Pediatric end-stage liver disease

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   44346   clinical trials with a EudraCT protocol, of which   7374   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 ).  
     
    Examples: Cancer AND drug name. Pneumonia AND sponsor name.
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    6 result(s) found for: Pediatric end-stage liver disease. Displaying page 1 of 1.
    EudraCT Number: 2011-004287-30 Sponsor Protocol Number: LAL-CL06 Start Date*: 2014-06-19
    Sponsor Name:Alexion Pharmaceuticals inc
    Full Title: A Multicenter, Open-Label Study of Sebelipase Alfa in Patients with Lysosomal Acid Lipase Deficiency
    Medical condition: Lysosomal Acid Lipase Deficiency (LALD)
    Disease: Version SOC Term Classification Code Term Level
    19.1 100000004850 10024579 Lysosomal storage disorders HLT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: DK (Completed) ES (Completed) GB (Completed) IT (Prematurely Ended) DE (Completed) BE (Completed) HR (Completed) NL (Completed)
    Trial results: View results
    EudraCT Number: 2017-002325-38 Sponsor Protocol Number: A4250-008 Start Date*: Information not available in EudraCT
    Sponsor Name:Albireo AB
    Full Title: An Open-label Extension Study to Evaluate Long-term Efficacy and Safety of A4250 in Children with Progressive Familial Intrahepatic Cholestasis Types 1 and 2 (PEDFIC 2)
    Medical condition: Progressive Familial Intrahepatic Cholestasis Types 1 and 2
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10076033 Progressive familial intrahepatic cholestasis PT
    Population Age: Infants and toddlers, Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: DE (Completed) FR (Completed) SE (Completed) GB (GB - no longer in EU/EEA) NL (Completed) BE (Completed) ES (Prematurely Ended) IT (Completed) PL (Completed)
    Trial results: (No results available)
    EudraCT Number: 2019-003807-37 Sponsor Protocol Number: A4250-011 Start Date*: 2020-07-29
    Sponsor Name:Albireo AB
    Full Title: A Double-Blind, Randomized, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Odevixibat (A4250) in Children with Biliary Atresia Who Have Undergone a Kasai Hepatoportoenterostomy (BOLD)
    Medical condition: Biliary Atresia
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004850 10004653 Biliary atresia LLT
    Population Age: Infants and toddlers, Under 18 Gender: Male, Female
    Trial protocol: HU (Trial now transitioned) GB (GB - no longer in EU/EEA) FR (Trial now transitioned) NL (Trial now transitioned) PL (Trial now transitioned) DE (Ongoing) BE (Trial now transitioned) IT (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2020-000974-22 Sponsor Protocol Number: MRX-701 Start Date*: 2021-01-29
    Sponsor Name:Mirum Pharmaceuticals Inc.
    Full Title: Randomized, Double-blind, Placebo-Controlled Phase 2 Study to Evaluate the Efficacy and Safety of Maralixibat in the Treatment of Subjects with Biliary Atresia after Hepatoportoenterostomy
    Medical condition: Biliary atresia (BA) is a rare, inflammatory condition of the biliary tree that presents in the first weeks of life and leads to bile duct obstruction and consequent liver injury, fibrosis and cirr...
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004850 10004653 Biliary atresia LLT
    Population Age: Newborns, Infants and toddlers, Under 18 Gender: Male, Female
    Trial protocol: DE (Completed) FR (Completed) PL (Completed) GB (GB - no longer in EU/EEA) IT (Completed)
    Trial results: View results
    EudraCT Number: 2018-003099-10 Sponsor Protocol Number: DCR-PHXC-301 Start Date*: 2019-06-12
    Sponsor Name:Dicerna Pharmaceuticals Inc
    Full Title: An Open-Label Roll-Over Study to Evaluate the Long-Term Safety and Efficacy of DCR PHXC Solution for Injection (subcutaneous use) in Patients with Primary Hyperoxaluria
    Medical condition: Primary Hyperoxaluria
    Disease: Version SOC Term Classification Code Term Level
    20.1 10038359 - Renal and urinary disorders 10020703 Hyperoxaluria PT
    Population Age: Children, Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: FR (Trial now transitioned) GB (GB - no longer in EU/EEA) DE (Trial now transitioned) NL (Trial now transitioned) ES (Ongoing) PL (Completed) IT (Trial now transitioned) RO (Completed) NO (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2018-003098-91 Sponsor Protocol Number: DCR-PHXC-201 Start Date*: 2019-03-05
    Sponsor Name:Dicerna Pharmaceuticals Inc
    Full Title: A Phase 2 Placebo-Controlled, Double-Blind, Multicenter Study to Evaluate the Efficacy, Safety, and Tolerability of DCR-PHXC Solution for Injection (subcutaneous use) in Patients with Primary Hyper...
    Medical condition: Primary Hyperoxaluria
    Disease: Version SOC Term Classification Code Term Level
    20.1 10038359 - Renal and urinary disorders 10020703 Hyperoxaluria PT
    Population Age: Children, Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: FR (Completed) GB (GB - no longer in EU/EEA) DE (Completed) NL (Completed) ES (Ongoing) PL (Completed) IT (Completed) RO (Completed)
    Trial results: View results
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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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