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Clinical trials for Wolman disease

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    The EU Clinical Trials Register currently displays   44346   clinical trials with a EudraCT protocol, of which   7374   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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    5 result(s) found for: Wolman disease. Displaying page 1 of 1.
    EudraCT Number: 2011-004730-34 Sponsor Protocol Number: 361.11 Start Date*: 2011-07-20
    Sponsor Name:OSPEDALE PEDIATRICO BAMBINO GESU' DI ROMA
    Full Title: A phase II multicentre, randomized, controlled open-label study on the use of anti-thymocyte globulin and rituximab for immunomodulation of graft-versus-host disease in allogeneic matched transpla...
    Medical condition: graft-versus-host disease
    Disease: Version SOC Term Classification Code Term Level
    14.1 10021428 - Immune system disorders 10021428 Immune system disorders SOC
    Population Age: Infants and toddlers, Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: IT (Prematurely Ended)
    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: 2011-002880-42 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: 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: 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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