CL06: OPEN-LABEL STUDY OF SEBELIPASE ALFA IN PATIENTS WITH LALD
Research type
Research Study
Full title
A MULTICENTER, OPEN-LABEL STUDY OF SEBELIPASE ALFA IN PATIENTS WITH LYSOSOMAL ACID LIPASE DEFICIENCY
IRAS ID
147611
Contact name
Saikat Santra
Contact email
Sponsor organisation
Synageva BioPharma Corp
Eudract number
2011-004287-30
Duration of Study in the UK
1 years, 5 months, 31 days
Research summary
Lysosomal Acid Lipase (LAL) Deficiency is a rare disorder caused by the body not producing enough of the enzyme LAL which plays an important role in breaking down fatty material. This causes fatty material to build up in various tissues of the body such as the liver and small intestine.
LAL deficiency in infants is an extremely rare form of LAL deficiency which leads to malabsorption (inability to absorb nutrients from food), growth failure, and liver failure. Affected babies usually die in the first 6 months of life and there are currently no safe or effective treatments. Milder forms of LAL deficiency affect older children and adults.
The purpose of this study is to evaluate the safety and efficacy of sebelipase alfa (a replacement LAL enzyme) in a broad population of patients with LAL Deficiency who have been excluded from other studies. The study is funded by Synageva BioPharma Corp.
Approximately 20 patients will take part in the study worldwide with approximately 2 taking part in the UK.
All participants will have an evaluation (screening) period and if eligible will receive sebelipase alfa by intravenous infusion every other week for up to 96 weeks, and a follow-up phone call 4 weeks after the last dose of sebelipase alfa. The dose may be increased for participants who are not responding well to treatment after a minimum time on the previous dose.Initially the Participant will receive intravenous sebelipase alfa at the study centre. If the participant has received the same dose for the 24 weeks prior to the week 48 visit, they may be allowed to receive the infusion at home.
Study procedures include medical history, physical examination, ECG, height, weight, MRI, liver biopsy, blood and urine samples, questionnaires, vital signs, adverse event and concomitant medications recording and sebelipase alfa dosing.
REC name
Yorkshire & The Humber - Sheffield Research Ethics Committee
REC reference
15/YH/0006
Date of REC Opinion
19 Jan 2015
REC opinion
Favourable Opinion