Long Term treatment with BCX9930 in PNH

• Research type

  Research Study

• Full title

  A Phase 2, Open-Label Study to Evaluate the Long-term Safety of Oral BCX9930 in Subjects with Paroxysmal Nocturnal Hemoglobinuria (PNH)

• IRAS ID

  286350

• Contact name

  F Dyer

• Contact email

  operations@ams-europe.com

• Sponsor organisation

  BioCryst Pharmaceuticals Inc

• Eudract number

  2020-000501-93

• Clinicaltrials.gov Identifier

  NCT04702568

• Duration of Study in the UK

  1 years, 0 months, 30 days

• Research summary

  The protocol is to allow patients who have participated in a prior BCX9930 study in PNH to have continued access to BCX9930.
  Participants must have demonstrated that they were achieving a clinical benefit on their previous trial.
  This protocol will also allow for a limited number of dose increases, if their treating investigator feels that they are not achieving an optimal clinical response.
  The current protocol is for 48 weeks treatment, with the plan to extend this as further non-clinical and clinical data become available.
  The only current protocol available in the UK is BCX9930-101 IRAS 261835

• REC name

  North East - Newcastle & North Tyneside 2 Research Ethics Committee

• REC reference

  20/NE/0192

• Date of REC Opinion

  29 Sep 2020

• REC opinion

  Further Information Favourable Opinion