BCX7353-304

• Research type

  Research Study

• Full title

  A PHASE 3 STUDY TO EVALUATE THE SAFETY AND PHARMACOKINETICS OF BEROTRALSTAT PROPHYLAXIS IN CHILDREN WITH HEREDITARY ANGIOEDEMA WHO ARE 2 TO < 12 YEARS OF AGE

• IRAS ID

  1005476

• Contact name

  Meredith Lilley

• Contact email

  MLilley@biocryst.com

• Sponsor organisation

  BioCryst Pharmaceuticals, Inc.

• Eudract number

  2021-005932-50

• Clinicaltrials.gov Identifier

  NCT05453968

• Research summary

  This is a single-arm, open-label study designed to evaluate the pharmacokinetics (PK) of berotralstat in. About 20 children aged 2 to less than 12 years old with hereditary angioedema (HAE) will take part in this study. The main purpose of this study is to examine how the body processes the study drug, how safe the study drug is, and how well the study drug works compared to standard of care (SOC) therapy. During the SOC period, each participant will only receive SOC therapy and will not receive any study drug. Researchers will use the SOC period as a control period before the participant starts taking the study drug.
  The study is divided into 3 time periods:
  • SOC treatment period: 12-week period including a screening period
  • Berotralstat treatment period: up to 144 weeks total
  o Treatment period 1: first 12 weeks on berotralstat
  o Treatment period 2: weeks 12-48, dose adjustment as necessary
  The study will take approximately 5 years to complete
  o Treatment period 3: weeks 48-144, dose adjustment as necessary
  • Follow-up period: 3 weeks (+/- 3 days) after last dose

• REC name

  East Midlands - Nottingham 2 Research Ethics Committee

• REC reference

  22/EM/0135

• Date of REC Opinion

  26 Jul 2022

• REC opinion

  Further Information Favourable Opinion