Long term administration of SBC-102 in children with LAL deficiency

• Research type

  Research Study

• Full title

  An Open Label Multicenter Extension Study to Evaluate the Long-Term Efficacy and Safety of SBC 102 in Children with Lysosomal Acid Lipase Deficiency Who Previously Received Treatment with SBC-102

• IRAS ID

  86113

• Contact name

  James Edmond Wraith

• Sponsor organisation

  Synageva Biopharma Corp.

• Eudract number

  2011-002880-42

• 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. In the early onset form of the disease which affects less than 2 people in a million, the life expectancy is usually less than 12 months. 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 infant study, and will be looking at the more long-term safety and efficacy of SBC-102. Up to 10 patients who have either taken part in the previous infant study or who have had access to SBC-102 through other means, 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 pursuit of a marketing authorisation.

• REC name

  North West - Greater Manchester Central Research Ethics Committee

• REC reference

  11/NW/0684

• Date of REC Opinion

  5 Dec 2011

• REC opinion

  Further Information Favourable Opinion