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Clinical trials for Lipid storage 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   44335   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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    17 result(s) found for: Lipid storage disease. 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: 2021-000903-19 Sponsor Protocol Number: UX053-CL101 Start Date*: 2022-08-24
    Sponsor Name:Ultragenyx Pharmaceutical Inc.
    Full Title: A Phase 1/2 First-in-human, 2-part Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of Single Ascending Doses (Part 1: Open-label) and Repeat Doses (Part 2: Randomized, Double-blind...
    Medical condition: Glycogen Storage Disease Type III (GSD III)
    Disease: Version SOC Term Classification Code Term Level
    20.1 10010331 - Congenital, familial and genetic disorders 10053250 Glycogen storage disease type III PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: FR (Completed) IT (Prematurely Ended) ES (Ongoing)
    Trial results: View results
    EudraCT Number: 2017-003014-22 Sponsor Protocol Number: Thalamus_V3 Start Date*: 2018-06-25
    Sponsor Name:Medical University of Vienna
    Full Title: The role of leptin in regulating hepatic lipid metabolism in humans
    Medical condition: lipodystrophy patients, patients after liver transplantation and healthy controls
    Disease:
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: AT (Completed)
    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: 2011-002750-31 Sponsor Protocol Number: LAL-CL02 Start Date*: 2013-05-29
    Sponsor Name:Alexion Pharmaceuticals, Inc.
    Full Title: A multicenter, randomized, placebo-controlled study of SBC-102 in patients with lysosomal acid lipase deficiency
    Medical condition: Lysosomal Acid Lipase Deficiency
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004915 10024579 Lysosomal storage disorders HLT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: DE (Completed) GB (Completed) IT (Completed) ES (Completed) CZ (Completed) PL (Completed) GR (Completed) HR (Completed) FR (Completed)
    Trial results: View results
    EudraCT Number: 2014-000533-22 Sponsor Protocol Number: LAL-CL08 Start Date*: 2014-05-28
    Sponsor Name:Alexion Pharmaceuticals Inc
    Full Title: A Phase 2, Open Label, Multicenter Study to Evaluate the Safety, Tolerability, Efficacy, and Pharmacokinetics of Sebelipase Alfa in Infants with Rapidly Progressive Lysosomal Acid Lipase Deficiency
    Medical condition: Lysosomal Acid Lipase Deficiency (LALD)
    Disease: Version SOC Term Classification Code Term Level
    18.1 100000004850 10024579 Lysosomal storage disorders HLT
    Population Age: Newborns, Infants and toddlers, Under 18 Gender: Male, Female
    Trial protocol: GB (Completed) IT (Completed) FI (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
    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: 2017-001508-31 Sponsor Protocol Number: R1500-CL-1643 Start Date*: 2017-11-20
    Sponsor Name:Regeneron Pharmaceuticals, Inc.
    Full Title: A Randomized, Double-Blind, Placebo-Controlled Study of the Safety and Efficacy of Varying Doses and Dose Regimens of Evinacumab in Patients with Persistent Hypercholesterolemia Despite Maximally T...
    Medical condition: Persistent Hypercholesterolemia Despite Maximally Tolerated Lipid Modifying Therapy
    Disease: Version SOC Term Classification Code Term Level
    21.0 100000004861 10020604 Hypercholesterolemia LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: CZ (Completed) NL (Completed) NO (Completed) SE (Completed) DK (Completed) PL (Completed) AT (Completed) ES (Completed) GB (GB - no longer in EU/EEA)
    Trial results: View results
    EudraCT Number: 2021-006766-21 Sponsor Protocol Number: MINI-SPIOMET Start Date*: 2022-09-02
    Sponsor Name:Hospital de Girona Dr. Josep Trueta
    Full Title: Towards a treatment for accelerated maturation in girls testing spiomet in a randomised placebo controlled, multicentre study.
    Medical condition: Girls with advanced puberty and accelerated bone maturation. Polycystic Ovary Syndrome (PCOS)
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004860 10014054 Early puberty LLT
    Population Age: Children, Under 18 Gender: Female
    Trial protocol: ES (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2005-001651-37 Sponsor Protocol Number: OGT 918-011 Start Date*: 2005-10-27
    Sponsor Name:Actelion Pharmaceuticals Ltd
    Full Title: A phase IV, open-label, non comparative, multi-center study to evaluate the long term efficacy and safety and tolerability of oral miglustat as a maintenance after a switch from Enzyme Replacement ...
    Medical condition: Gaucher disease is the most common of the glycosphingolipid storage diseases and has autosomal recessive inheritance. For a more precise description, please refer to page 16 of the protocol: 1 BA...
    Disease:
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: GB (Completed) ES (Completed) IT (Completed) DE (Completed) CZ (Completed) HU (Completed)
    Trial results: View results
    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: 2012-005590-32 Sponsor Protocol Number: APCP-113 Start Date*: 2013-02-20
    Sponsor Name:Leiden University Medical Center
    Full Title: A double blind, placebo controlled Phase 2 study comparing the effects of ARA 290 on neuropathic symptoms of patients with type 2 diabetes
    Medical condition: Diabetes type 2: neuropathic symptoms
    Disease:
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: NL (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2011-000942-39 Sponsor Protocol Number: NL34476.068.11 Start Date*: 2011-06-20
    Sponsor Name:CTMM, the Center for Translational Molecular Medicine
    Full Title: Effects of thyroid hormone treatment on mitochondrial function, ectopic fat accumulation, insulin sensitivity and brown adipose tissue in type 2 diabetes mellitus.
    Medical condition: This trial includes overweight patients with both hypothyroidism and type 2 diabetes.
    Disease: Version SOC Term Classification Code Term Level
    13.1 10014698 - Endocrine disorders 10021114 Hypothyroidism PT
    13.1 10027433 - Metabolism and nutrition disorders 10045242 Type II diabetes mellitus LLT
    Population Age: Adults Gender: Male, Female
    Trial protocol: NL (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2020-001488-10 Sponsor Protocol Number: APHP200080 Start Date*: 2020-10-05
    Sponsor Name:Assistance Publique Hopitaux de Paris
    Full Title: Multisite open-label randomized phase II clinical trial in newly diagnosed glioblastoma treated by concurrent TemoRadiation and adjuvant temozolomide +/- ultrasound-induced blood brain barrier opening
    Medical condition: Newly diagnosed Glioblastoma (IDH wild-type) patients at initial radiological diagnosis eligible for tumor resection , and for the standard of care including concurrent temoradiation and adjuvant T...
    Disease:
    Population Age: Gender: Male, Female
    Trial protocol: FR (Trial now transitioned) BE (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2014-003721-18 Sponsor Protocol Number: CV181365 Start Date*: 2015-09-14
    Sponsor Name:AstraZeneca AB
    Full Title: A 52-week International, Multicenter, Randomized, Double-Blind, Active-Controlled, Parallel Group, Phase 3bTrial with a Blinded 104-week Long -term Extension Period to Evaluate the Efficacy and Saf...
    Medical condition: Inadequately controlled Diabetes Mellitus Type 2.
    Disease: Version SOC Term Classification Code Term Level
    20.0 10027433 - Metabolism and nutrition disorders 10067585 Type 2 diabetes mellitus PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: CZ (Completed) DE (Completed) HU (Completed) SE (Completed) PL (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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