SBC-102 in children with growth failure due to LAL deficiency

• Research type

  Research Study

• Full title

  An open label, multicentre, dose escalation study to evaluate the safety, tolerability, efficacy, pharmacokinetics, and pharmacodynamics of SBC-102 in children with growth failure due to lysosomal acid lipase deficiency

• IRAS ID

  72479

• Contact name

  James Edmond Wraith

• Sponsor organisation

  Synageva Biopharma Corp.

• Eudract number

  2011-000032-28

• ISRCTN Number

  N/A

• Research summary

  (Lysosomal Acid Lipase) deficiency is a rare genetic enzyme deficiency disease which presents as either a Cholesteryl Ester Storage Disease or Wolman Disease. This study involves patients with Wolman Disease, which is characterised by malabsorption, growth failure and liver failure, and is usually fatal within the first year of life. Currently there are no approved therapies for this disease other than supportive therapies that attempt to lessen some of the effects of this rapidly fatal 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 children. It will investigate the safety, tolerability, efficacy, pharmacokinetics and pharmacodynamics of SBC-102 in children with growth failure due to LAL deficiency. Given the short life-expectancy of a patient suffering with Wolman disease LAL deficiency, this study will enroll patients of up to around 2 years of age. Consent will be sought from a parent or legal guardian. A total of 8 patients will be enrolled to populate 2 cohorts of 4 patients. The administration of SBC-102 will be staggered within each cohort and between the 2 cohorts, with dosing starting with the first patient in the 1st cohort at the lowest dose of SBC-102. Dosing of the further 3 patients in the 1st cohort and initiation of dosing in the 2nd cohort will be based on the recommendations of the safety committee who will be responsible for monitoring the safety of the patients throughout the study. Patients may receive an increase or decrease of dose within the set dose range, depending on how well they respond to the treatment, and how well it is tolerated. Patients in this study will receive treatment for 16 weeks, and may have the opportunity to take part in an extension study.

• REC name

  North West - Greater Manchester Central Research Ethics Committee

• REC reference

  11/NW/0016

• Date of REC Opinion

  18 Apr 2011

• REC opinion

  Further Information Favourable Opinion