CompletedPhase 2

Extension Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of SBC-102 (Sebelipase Alfa) in Adult Subjects With Lysosomal Acid Lipase Deficiency

United States8 participantsStarted 2011-12-12

Plain-language summary

This was an extension study to Study LAL-CL01 (NCT01307098). The primary objective of the study was to evaluate the long-term safety and tolerability of sebelipase alfa in participants with liver dysfunction due to lysosomal acid lipase (LAL) deficiency.

Who can participate

Age range18 Years

SexALL

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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion Criteria: * Participant received all 4 scheduled doses of sebelipase alfa in Study LAL-CL01 with no life-threatening or unmanageable study drug toxicity. Exclusion Criteria: * Clinically significant concurrent disease, serious inter-current illness, concomitant medications or other extenuating circumstances * Clinically significant abnormal values on laboratory screening tests, other than LFTs or lipid panel tests

What they're measuring

Number Of Participants Reporting TEAEs And IARs

Timeframe: From after first dose administration post-Baseline through EOS during study LAL-CL04

Trial details

NCT IDNCT01488097

SponsorAlexion Pharmaceuticals, Inc.

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2017-06-21

Results submitted2016-01-14

View on ClinicalTrials.gov More Cholesterol Ester Storage Disease (CESD) trials