SBC-102 in patients with liver dysfunction due to LAL deficiency

• Research type

  Research Study

• Full title

  An open label multicenter study to evaluate the safety, tolerability and pharmacokinetics of SBC-102 in adult patients with liver dysfunction due to lysosomal acid lipase deficiency.

• IRAS ID

  69684

• Contact name

  Stephen Waldek

• Sponsor organisation

  Synageva Biopharma Corp.

• Eudract number

  2010-024068-16

• ISRCTN Number

  N/A

• 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. This study will be the first administration of SBC-102 in humans and will investigate the safety, tolerability and pharmacokinetics of SBC-102 in patients with liver dysfunction due to LAL deficiency. A total of 9 patients will be enrolled to populate 3 cohorts of 3 patients. Each cohort will be given a different dose of SBC-102, administered intravenously once a week for four weeks. The dose will be escalated from 0.35 mg/kg in the first cohort up to 3 mg/kg in the third cohort, based on the recommendations of the safety committee who will be responsible for monitoring the safety of the patients throughout the study. As this will be the first time SBC-102 is administered to humans, the safety will be followed very closely through physical examinations, ECGs, clinical laboratory assessments, vital signs and the reporting of adverse events. Pharmacokinetic and pharmacodinamic assessments will also be performed to understand how the drug behaves in the human body. Further clinical laboratory assessments will be used to determine the efficacy of SBC-102 and to further characterise this rare deficiency.

• REC name

  North West - Liverpool Central Research Ethics Committee

• REC reference

  11/H1005/5

• Date of REC Opinion

  21 Feb 2011

• REC opinion

  Further Information Favourable Opinion