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Clinical trials for 2013-000051-40

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
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    • 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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    2 result(s) found for: 2013-000051-40. Displaying page 1 of 1.
    EudraCT Number: 2013-000051-40 Sponsor Protocol Number: LTS13632 Start Date*: 2013-12-16
    Sponsor Name:Genzyme Corporation
    Full Title: A Long-Term Study to Assess the Ongoing Safety and Efficacy of Olipudase Alfa in Patients With Acid Sphingomyelinase Deficiency
    Medical condition: Patients with acid sphingomyelinase deficiency (Niemann-Pick Type B disease)
    Disease: Version SOC Term Classification Code Term Level
    20.1 100000004850 10041515 Sphingomyelin lipidosis LLT
    Population Age: Infants and toddlers, Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: GB (GB - no longer in EU/EEA) IT (Completed) DE (Completed) BE (Completed)
    Trial results: View results
    EudraCT Number: 2010-023953-12 Sponsor Protocol Number: DFI12712 Start Date*: 2013-12-16
    Sponsor Name:Genzyme Corporation
    Full Title: A Phase 2, Multi-Center, Randomized, Open-Label, Repeat Dose, Dose-Comparison Study to Evaluate the Safety, Efficacy, and Pharmacokinetics of Recombinant Human Acid Sphingomyelinase in Patients Wit...
    Medical condition: Patients with acid sphingomyelinase deficiency (Niemann-Pick Type B disease)
    Disease: Version SOC Term Classification Code Term Level
    17.1 100000004850 10041515 Sphingomyelin lipidosis LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: GB (Completed) IT (Completed) FR (Ongoing)
    Trial results: (No results available)
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    Query did not match any studies.
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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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