Extension to LAL-CL01 in patients with liver dysfunction due to LAL de

• Research type

  Research Study

• Full title

  AN OPEN LABEL MULTICENTER EXTENSION STUDY TO EVALUATE THE LONG-TERM SAFETY, TOLERABILITY, AND EFFICACY OF SBC-102 IN ADULT SUBJECTS WITH LIVER DYSFUNCTION DUE TO LYSOSOMAL ACID LIPASE DEFICIENCY WHO PREVIOUSLY RECEIVED TREATMENT IN STUDY LAL-CL01

• IRAS ID

  82101

• Contact name

  Patrick Deegan

• Eudract number

  2011-001513-13

• Research summary

  LAL (Lysosomal Acid Lipase) deficiency is a rare genetic enzyme deficiency disease and is associated with a shortened life expectancy and significant ill health. Currently there are no approved therapies for this disease, other than supportive therapies that attempt to lessen some of the effects of the disease.SBC-102 is a novel therapy for this deficiency, which is hoped to work through replacement of the LAL enzyme. There are currently two SBC-102 studies underway; one in infants and one in adults, looking at the safety and efficacy of SBC-102 over a relatively short period of time. This study is an extension to the adult study, and will be looking at the more long-term safety and efficacy of SBC-102 in adults.Up to 9 patients who have taken part in the previous adult study will be considered for participation in this study. This extension will follow on directly after the first study to avoid patients being without access to SBC-102. This study will continue until marketing authorisation is granted or until the sponsor decides to discontinue the persuit of a marketing authorisation.

• REC name

  North West - Liverpool Central Research Ethics Committee

• REC reference

  11/NW/0619

• Date of REC Opinion

  24 Oct 2011

• REC opinion

  Further Information Favourable Opinion