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Clinical trials for Sly syndrome

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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    4 result(s) found for: Sly syndrome. Displaying page 1 of 1.
    EudraCT Number: 2015-000104-26 Sponsor Protocol Number: UX003-CL203 Start Date*: 2015-07-23
    Sponsor Name:Ultragenyx Pharmaceutical Inc.
    Full Title: An Open-label Study of UX003 rhGUS Enzyme Replacement Therapy in MPS 7 Patients Less than 5 years old
    Medical condition: Mucopolysaccharidosis type 7 (MPS 7, Sly syndrome)
    Disease:
    Population Age: Preterm newborn infants, Newborns, Infants and toddlers, Children, Under 18 Gender: Male, Female
    Trial protocol: Outside EU/EEA ES (Prematurely Ended) PT (Completed)
    Trial results: View results
    EudraCT Number: 2015-001875-32 Sponsor Protocol Number: UX003-CL202 Start Date*: 2015-11-12
    Sponsor Name:Ultragenyx Pharmaceutical Inc.
    Full Title: A Long-Term Open-Label Treatment and Extension Study of UX003 rhGUS Enzyme Replacement Therapy in Subjects with MPS 7
    Medical condition: Mucopolysaccharidosis type 7 (MPS 7, Sly syndrome)
    Disease:
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: Outside EU/EEA PT (Completed)
    Trial results: View results
    EudraCT Number: 2013-001152-35 Sponsor Protocol Number: UX003-CL201 Start Date*: 2013-08-02
    Sponsor Name:Ultragenyx Pharmaceutical Inc.
    Full Title: An Open-Label Phase 1/2 Study to Assess the Safety, Efficacy and Dose of UX003 rhGUS Enzyme Replacement Therapy in Patients with MPS 7
    Medical condition: Mucopolysaccharidosis type 7 (MPS 7, Sly syndrome)
    Disease:
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: GB (Completed) ES (Completed)
    Trial results: View results
    EudraCT Number: 2014-005638-71 Sponsor Protocol Number: UX003-CL301 Start Date*: 2014-12-22
    Sponsor Name:Ultragenyx Pharmaceutical Inc.
    Full Title: A Randomized, Placebo-Controlled, Blind-Start, Single-Crossover Phase 3 Study to Assess the Efficacy and Safety of UX003 rhGUS Enzyme Replacement Therapy in Patients with MPS 7
    Medical condition: Mucopolysaccharidosis type 7 (MPS 7, Sly syndrome)
    Disease:
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: Outside EU/EEA
    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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