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Clinical trials for Lipid-storage diseases

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
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    The EU Clinical Trials Register currently displays   44336   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).

    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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    5 result(s) found for: Lipid-storage diseases. Displaying page 1 of 1.
    EudraCT Number: 2015-004846-25 Sponsor Protocol Number: Pfizer-Prevenar13-2015 Start Date*: 2016-07-15
    Sponsor Name:Maastricht University
    Full Title: Immunization against oxLDL in patients with lysosomal lipid diseases and associated metabolic disorders
    Medical condition: Familial hypercholesterolemia Niemann-Pick disease type B (NPB) Niemann-Pick disease type C (NPC) Partial lipodystrophy in children and adults
    Disease:
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: BE (Completed)
    Trial results: (No results available)
    EudraCT Number: 2011-001205-27 Sponsor Protocol Number: ABR35884 Start Date*: 2011-08-04
    Sponsor Name:
    Full Title: The effect of fibrate therapy in two patients with neutral lipid storage disease with myopathy (NLSDM).
    Medical condition: Neutral lipid storage disease with myopathy
    Disease: Version SOC Term Classification Code Term Level
    13.1 10007541 - Cardiac disorders 10007636 Cardiomyopathy PT
    13.1 10027433 - Metabolism and nutrition disorders 10013317 Lipid metabolism disorders HLGT
    13.1 10028395 - Musculoskeletal and connective tissue disorders 10028641 Myopathy PT
    Population Age: Gender:
    Trial protocol: NL (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2021-002320-20 Sponsor Protocol Number: EFC16158 Start Date*: 2022-04-11
    Sponsor Name:sanofi-aventis recherche et développement
    Full Title: A randomized, open-label, parallel-group, 18-month Phase 3 study to evaluate the effect of venglustat compared with usual standard of care on left ventricular mass index in participants with Fabry ...
    Medical condition: Fabry’s disease
    Disease: Version SOC Term Classification Code Term Level
    24.1 10010331 - Congenital, familial and genetic disorders 10016016 Fabry's disease PT
    Population Age: Adults Gender: Male, Female
    Trial protocol: NO (Trial now transitioned) CZ (Trial now transitioned) IT (Trial now transitioned) DK (Trial now transitioned) ES (Ongoing) GR (Trial now transitioned) AT (Trial now transitioned) PL (Trial now transitioned) NL (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2011-001513-13 Sponsor Protocol Number: LAL-CL04 Start Date*: 2011-10-24
    Sponsor Name:Synageva Biopharma Corp.
    Full Title: AN OPEN LABEL MULTICENTER EXTENSION STUDY TO EVALUATE THE LONG-TERM SAFETY, TOLERABILITY, AND EFFICACY OF SBC-102 IN ADULT SUBJECTS WITH LIVER DYSFUNCTION DUE TO LYSOSOMAL ACID LIPASE DEFICIENCY WH...
    Medical condition: Lysosomal Acid Lipase (LAL) Deficiency is a rare autosomal recessive lipid storage disorder that is caused by deficient activity or absence, of the lysosomal enzyme, LAL. It is an extremely rare di...
    Disease: Version SOC Term Classification Code Term Level
    14.1 10027433 - Metabolism and nutrition disorders 10027433 Metabolism and nutrition disorders SOC
    14.1 10027433 - Metabolism and nutrition disorders 10021605 Inborn errors of metabolism HLGT
    14.1 10010331 - Congenital, familial and genetic disorders 10024579 Lysosomal storage disorders HLT
    Population Age: Adults Gender: Male, Female
    Trial protocol: GB (Completed) CZ (Completed)
    Trial results: View results
    EudraCT Number: 2010-024068-16 Sponsor Protocol Number: LAL-CL01 Start Date*: 2011-03-08
    Sponsor Name:Synageva Biopharma Corp.
    Full Title: An open label multicenter study to evaluate the safety, tolerability and pharmacokinetics of SBC-102 in adult patients with liver dysfunction due to lysosomal acid lipase deficiency.
    Medical condition: Lysosomal Acid Lipase (LAL) Deficiency is a rare autosomal recessive lipid storage disorder that is caused by deficient activity or absence, of the lysosomal enzyme, LAL. It is an extremely rare di...
    Disease: Version SOC Term Classification Code Term Level
    14.0 10027433 - Metabolism and nutrition disorders 10027433 Metabolism and nutrition disorders SOC
    14.0 10027433 - Metabolism and nutrition disorders 10021605 Inborn errors of metabolism HLGT
    14.0 10010331 - Congenital, familial and genetic disorders 10024579 Lysosomal storage disorders HLT
    Population Age: Adults Gender: Male, Female
    Trial protocol: GB (Completed) CZ (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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