CompletedPhase 1/2

Safety, Tolerability and Pharmacokinetics of SBC-102 (Sebelipase Alfa) in Adult Participants With Lysosomal Acid Lipase Deficiency

United States, Czechia, France9 participantsStarted 2011-04-25

Plain-language summary

This was the first clinical study of SBC-102 (sebelipase alfa) for the treatment of Lysosomal Acid Lipase (LAL) Deficiency. It was an open-label dose escalation study in adult participants with liver dysfunction due to LAL Deficiency and was designed to examine 3 doses of sebelipase alfa. The targeted number for this study was 9 evaluable participants.

Who can participate

Age range18 Years – 65 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: * Male or female participants ≥ 18 and ≤ 65 years of age * Documented decreased LAL activity * Evidence of liver involvement 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 liver function or lipid panel tests * Aspartate aminotransferase and/or alanine aminotransferase persistently elevated \> 3x upper limit of normal at screening * Previous hemopoietic bone marrow or liver transplant * Current history of alcohol abuse

What they're measuring

Number Of Participants Reporting TEAEs And Infusion-Related Reactions (IRRs)

Timeframe: Screening up to Day 52

Trial details

NCT IDNCT01307098

SponsorAlexion Pharmaceuticals, Inc.

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2012-01-06

Results submitted2018-10-30

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