ELIKIDS

• Research type

  Research Study

• Full title

  Open label, two cohort (with and without imiglucerase), multicenter study to evaluate pharmacokinetics, safety, and efficacy of eliglustat in pediatric patients with Gaucher disease type 1 and type 3

• IRAS ID

  232727

• Contact name

  Anupam Chakrapani

• Contact email

  Anupam.Chakrapani@gosh.nhs.uk

• Sponsor organisation

  Sanofi

• Eudract number

  2016-000301-37

• Clinicaltrials.gov Identifier

  NCT03485677

• Clinicaltrials.gov Identifier

  N/A, N/A

• Duration of Study in the UK

  4 years, 8 months, 16 days

• Research summary

  The purpose of this study is to evaluate the possible risks and efficacy (improvement of disease) with an oral study drug named eliglustat in pediatric patients aged 6 to 17 years of age with Gaucher Type 1 or 3 disease.

  Based on the patient's disease symptoms, they will receive either eliglustat alone (cohort 1) or eliglustat in combination with the enzyme replacement therapy named imiglucerase (cohort 2).

• REC name

  South West - Central Bristol Research Ethics Committee

• REC reference

  18/SW/0218

• Date of REC Opinion

  9 Jan 2019

• REC opinion

  Further Information Favourable Opinion