ARISE (Acid Lipase Replacement Investigating Safety and Efficacy)

• Research type

  Research Study

• Full title

  A Multicenter, Randomized, Placebo-Controlled Study of SBC-102 in Patients with Lysosomal Acid Lipase Deficiency

• IRAS ID

  115042

• Contact name

  Patrick Deegan

• Sponsor organisation

  Synageva Biopharma Corporation

• Eudract number

  2011-002750-31

• ISRCTN Number

  n.a

• Clinicaltrials.gov Identifier

  n.a

• Research summary

  We are asking potential participants to take part in this study because they have a genetic disease called late onset Lysosomal Acid Lipase Deficiency (LALD) or cholesterol ester storage disease (CESD). This disease leads to a build-up of fat (lipids) in the liver and other parts of the body. This is due to lack of the enzyme called Lysosomal acid lipase, a substance that normally breaks down fat. The build-up causes a number of problems, including swelling and damage (scarring) of the liver and high cholesterol in the blood. High cholesterol in the blood may increase the risk of heart disease and other problems due to blocking of these arteries. This study will evaluate the safety and effectiveness of an experimental drug called SBC-102 in patients with LALD. SBC-102 replaces the enzyme that would normally be produced by the body. SBC-102 is produced in and purified from egg white from special hens. About 50 patients at hospitals and clinics all over the world will take part in this study. Potential participants would take part in the study for up to 160 weeks (37 months). It is expected that they will receive treatment in the study for at least 78 weeks (18 months). Depending on the time they enter the study and how they respond to the study treatment, they may continue to be treated for up to 150 weeks (35 months).

• REC name

  East of England - Cambridge South Research Ethics Committee

• REC reference

  12/EE/0492

• Date of REC Opinion

  14 Jan 2013

• REC opinion

  Further Information Favourable Opinion