NCT02193867 - Clinical Study In Infants With Rapidly Progressive Lysosomal Acid Lipase Deficiency | Biotech Hunter

Enrollment

Participants

Timeline

Start Date

June 6, 2014

Primary Completion Date

October 30, 2018

Study Completion Date

October 30, 2018

Conditions

Lysosomal Acid Lipase Deficiency

Interventions

DRUG

Sebelipase Alfa

Sebelipase alfa is a recombinant human lysosomal acid lipase. The investigational medicinal product is an enzyme replacement therapy intended for treatment of participants with LAL-D. Dosing occurred once weekly for up to 3 years.

Trial Locations (5)

85016

Phoenix

Unknown

Kuopio

Naples

Birmingham

Manchester