(Q63812993)

English

Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of Sebelipase Alfa in Children With Growth Failure Due to Lysosomal Acid Lipase Deficiency

clinical trial

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clinical trial

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title

An Open Label, Multicenter, Dose Escalation Study to Evaluate the Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of SBC-102 (Sebelipase Alfa) in Children With Growth Failure Due to Lysosomal Acid Lipase Deficiency (English)

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Cryptogenic cirrhosis in a patient with familial hypocholesterolemia due to a new truncated form of apolipoprotein B

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start time

4 May 2011

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end time

3 January 2018

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number of participants

9

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clinical trial phase

phase II clinical trial

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phase III clinical trial

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maximum age

24 month

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study type

interventional study

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Identifiers

ClinicalTrials.gov ID

NCT01371825

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(Q63812993)

Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of Sebelipase Alfa in Children With Growth Failure Due to Lysosomal Acid Lipase Deficiency

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