Safety and Tolerability of SB-318 Gene Transfer in Subjects with MPS I

• Research type

  Research Study

• Full title

  A Phase I, Multicenter, Open-Label, Single-Dose, Dose-Ranging Study to Assess the Safety and Tolerability of SB-318, a rAAV2/6-based Gene Transfer in Subjects with Mucopolysaccharidosis I (MPS I)

• IRAS ID

  246598

• Contact name

  Derralynn Hughes

• Contact email

  derralynnhughes@nhs.net

• Sponsor organisation

  Sangamo Therapeutics, Inc.

• Eudract number

  2018-000206-28

• Clinicaltrials.gov Identifier

  NCT02702115

• Clinicaltrials.gov Identifier

  16821, BB-IND

• Duration of Study in the UK

  4 years, 1 months, 19 days

• Research summary

  This study will test to see if doctors can use a process called “gene therapy” to treat a lysosomal storage disease called Mucopolysaccharidosis type I (MPS I). The deficiency of these enzymes causes the change in a particular gene called alpha-L-iduronidase (IDUA).IDUA is an enzyme that is needed to break down a particular sugar called glycosaminoglycans (GAGs)

• REC name

  Scotland A: Adults with Incapacity only

• REC reference

  18/SS/0074

• Date of REC Opinion

  14 Aug 2018

• REC opinion

  Further Information Favourable Opinion