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Clinical trials for Lysosomal

The European Union Clinical Trials Register   allows you to search for protocol and results information on:
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    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).

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    71 result(s) found for: Lysosomal. Displaying page 1 of 4.
    1  2  3  4  Next»
    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: 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: 2005-001629-27 Sponsor Protocol Number: AGLU02403 Start Date*: 2005-08-03
    Sponsor Name:Genzyme Europe BV
    Full Title: A Long-term Continuation Study of Patients with Infantile-Onset Pompe Disease who were previously enrolled in Protocol AGLU01602
    Medical condition: Pompe disease is a rare metabolic muscle disease inherited in an autosomal recessive fashion. Pompe disease is caused by a deficiency of GAA, which is needed for the degradation of lysosomal glyco...
    Disease:
    Population Age: Infants and toddlers, Children, Under 18 Gender: Male, Female
    Trial protocol: DE (Completed) IT (Completed)
    Trial results: View results
    EudraCT Number: 2011-002880-42 Sponsor Protocol Number: LAL-CL05 Start Date*: 2011-12-05
    Sponsor Name:Synageva Biopharma Corp.
    Full Title: An Open Label Multicenter Extension Study to Evaluate the Long-Term Efficacy and Safety of SBC 102 in Children with Lysosomal Acid Lipase Deficiency Who Previously Received Treatment with SBC-102
    Medical condition: Growth failure in children due to lysosomal acid lipase deficiency (Wolman disease).
    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: Preterm newborn infants, Newborns, Infants and toddlers, Children, Under 18 Gender: Male, Female
    Trial protocol: GB (Prematurely Ended) FR (Ongoing)
    Trial results: (No results available)
    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: 2011-000032-28 Sponsor Protocol Number: LAL-CL03 Start Date*: 2011-04-18
    Sponsor Name:Synageva Biopharma Corp.
    Full Title: An Open Label, Multicenter, Dose Escalation Study to Evaluate the Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of SBC-102 in Children with Growth Failure Due to Lysosomal ...
    Medical condition: Growth failure in children due to lysosomal acid lipase deficiency (Wolman disease).
    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: Preterm newborn infants, Newborns, Infants and toddlers, Under 18 Gender: Male, Female
    Trial protocol: GB (Completed) FR (Completed) DE (Prematurely Ended) IT (Completed) IE (Completed)
    Trial results: View results
    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: 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: 2013-003321-28 Sponsor Protocol Number: LTS13769 Start Date*: 2014-05-12
    Sponsor Name:Genzyme Corporation
    Full Title: An Open-Label, Multicenter, Multinational Extension Study Of The Long-Term Safety And Pharmacokinetics Of Repeated Biweekly Infusions Of Avalglucosidase Alfa (NeoGAA, GZ402666) In Patients With Pom...
    Medical condition: Pompe disease (acid alpha-glucosidase deficiency)
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10036143 Pompe's disease LLT
    20.1 10010331 - Congenital, familial and genetic disorders 10053185 Glycogen storage disease type II PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: BE (Completed) NL (Completed) DK (Completed) DE (Completed) FR (Ongoing)
    Trial results: View results
    EudraCT Number: 2008-002302-18 Sponsor Protocol Number: POM-CL-201 Start Date*: 2009-02-16
    Sponsor Name:Amicus Therapeutics, Inc.
    Full Title: An open-label, multicenter, study to evaluate the safety, tolerability, pharmacodynamics, and pharmacokinetics of three dosing regimens of oral AT2220 in patients with Pompe disease
    Medical condition: Pompe Disease
    Disease: Version SOC Term Classification Code Term Level
    9.1 10036143 Pompe's disease LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: DE (Completed) GB (Prematurely Ended)
    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: 2014-002158-38 Sponsor Protocol Number: 701-201 Start Date*: 2015-03-23
    Sponsor Name:BioMarin Pharmaceutical Inc
    Full Title: A Study of Respiratory Muscle Strength, including Effort-Independent Measures, in Subjects with Late-Onset Pompe Disease
    Medical condition: Pompe disease
    Disease: Version SOC Term Classification Code Term Level
    18.0 100000004850 10036143 Pompe's disease LLT
    Population Age: Adults Gender: Male, Female
    Trial protocol: FR (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2008-008293-31 Sponsor Protocol Number: Pompe phys 03 Start Date*: 2009-02-10
    Sponsor Name:Erasmus MC Sophia
    Full Title: Early introduction in the Netherlands of alglucosidase alfa manufactured in a 4000 liter bioreactor
    Medical condition: Glycogen Storage Disease Type II (Pompe's Disease)
    Disease: Version SOC Term Classification Code Term Level
    9.1 10036143 Pompe's disease LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: NL (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2015-000512-18 Sponsor Protocol Number: AGLU09411/EFC12720 Start Date*: 2015-03-02
    Sponsor Name:Genzyme Corporation Inc
    Full Title: A Phase 4 Open Label, Prospective Study in Patients With Pompe Disease to Evaluate The Efficacy and Safety of Alglucosidase Alfa Produced at the 4000L Scale
    Medical condition: Pompe disease (acid alpha-glucosidase deficiency)
    Disease: Version SOC Term Classification Code Term Level
    17.1 100000004850 10036143 Pompe's disease LLT
    Population Age: Infants and toddlers, Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2010-024647-32 Sponsor Protocol Number: MIGLU-3 Start Date*: 2011-03-18
    Sponsor Name:UNIVERSITA` DEGLI STUDI DI NAPOLI FEDERICO II
    Full Title: Combined enzyme enhancement therapy (EET) and enzyme replacement therapy (ERT) in patients with Pompe disease
    Medical condition: Patients with Pompe Disease (Type II Glycogenosis) on therapy with Enzyme Replacement Therapy (ERT) for at least 6 months
    Disease: Version SOC Term Classification Code Term Level
    13.1 10010331 - Congenital, familial and genetic disorders 10036143 Pompe's disease LLT
    Population Age: Infants and toddlers, Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: IT (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2019-002251-42 Sponsor Protocol Number: GZ-2016-11512. Start Date*: Information not available in EudraCT
    Sponsor Name:
    Full Title: An open-label, single-center study on the safety and efficacy of avalglucosidase alpha in late-onset Pompe patients ≥5 years
    Medical condition: Late onset Pompe disease
    Disease: Version SOC Term Classification Code Term Level
    24.0 100000004850 10036143 Pompe's disease LLT
    Population Age: Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: NL (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2004-002168-59 Sponsor Protocol Number: AGLU017-02 Start Date*: 2004-10-14
    Sponsor Name:GENZYME
    Full Title: An Open-Label, Multicenter, Multinational, Study of the Safety, Efficacy, Pharmacokinetics, and Pharmacodynamics of Recombinant Human Acid alpha- Glucosidase (rhGAA) Treatment in Patients > 6 and <...
    Medical condition: Treatment for Type II Glycogenosis
    Disease: Version SOC Term Classification Code Term Level
    6.1 10053185 PT
    Population Age: Infants and toddlers, Children, Under 18 Gender: Male, Female
    Trial protocol: IT (Completed)
    Trial results: View results
    EudraCT Number: 2019-003595-38 Sponsor Protocol Number: AT845-01 Start Date*: 2020-11-20
    Sponsor Name:Audentes Therapeutics, Inc.
    Full Title: FORTIS: A Phase 1/2, Open-Label, Ascending-Dose Clinical Study to Evaluate the Safety and Preliminary Efficacy of AT845, an AAV8-Delivered Gene Transfer Therapy in Patients with Late Onset Pompe Di...
    Medical condition: Late Onset Pompe Disease
    Disease: Version SOC Term Classification Code Term Level
    20.1 10010331 - Congenital, familial and genetic disorders 10053185 Glycogen storage disease type II PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: GB (GB - no longer in EU/EEA)
    Trial results: (No results available)
    EudraCT Number: 2021-004047-25 Sponsor Protocol Number: ALGMYL08718 Start Date*: 2021-07-27
    Sponsor Name:Sanofi(China) Investment Co Ltd, Shanghai Branch
    Full Title: A Single Arm, Prospective, Open-label, Multi-center Study to Evaluate Efficacy and Safety in Chinese Patients with Infantile-Onset Pompe Disease with One Year Alglucosidase Alfa Treatment
    Medical condition: Infantile-Onset Pompe Disease
    Disease: Version SOC Term Classification Code Term Level
    20.1 10010331 - Congenital, familial and genetic disorders 10053185 Glycogen storage disease type II PT
    Population Age: Newborns, Infants and toddlers, Under 18 Gender: Male, Female
    Trial protocol: Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2021-005552-11 Sponsor Protocol Number: LTS12869 Start Date*: 2022-03-07
    Sponsor Name:Genzyme Corporation
    Full Title: A Long-term Study to Evaluate Growth and Development Outcomes in Patients With Infantile-Onset Pompe Disease Who Are Receiving Alglucosidase Alfa.
    Medical condition: Pompe disease
    Disease: Version SOC Term Classification Code Term Level
    20.1 10010331 - Congenital, familial and genetic disorders 10053185 Glycogen storage disease type II PT
    Population Age: Newborns, Infants and toddlers, Under 18 Gender: Male, Female
    Trial protocol: Outside EU/EEA
    Trial results: View results
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