BCX7353 long term safety study

• Research type

  Research Study

• Full title

  An open-label study to evaluate the long-term safety of daily oral BCX7353 in subjects with Type I and II hereditary angioedema

• IRAS ID

  236603

• Contact name

  Melanie Cornpropst

• Contact email

  mcornpropst@biocryst.com

• Sponsor organisation

  BioCryst Pharmaceuticals Inc.

• Eudract number

  2017-003281-27

• Duration of Study in the UK

  2 years, 0 months, 0 days

• Research summary

  Research Summary

  Hereditary angioedema (HAE) is an autosomal dominant disorder characterized by periodic episodes of swelling of the skin, pharynx, larynx, gastrointestinal tract, genitals, and extremities. The frequency of these attacks varies between subjects, with attacks occurring rarely in some patients to as frequently as every few days in others. HAE attacks, which may or may not be precipitated by a stimulus (such as stress, trauma, or estrogen) are typically slow in onset, peaking in intensity within 24 hours, and gradually subside over the subsequent 5 days.
  This study for patients who participated in a previous efficiacy studies to allow long term access to BCX7353 and to determine the long term safety profile of BCX7353, together with an assessment of efficacy over time.
  Patients will complete diaries to document the attacks they have, and how they treat them. Patients will be allowed to treat their attacks as per normal clinical practice, without restriction.

  Summary of Results

  A long-term safety study to evaluate the safety of BCX7353 (berotralstat) in patients with type I and type II Hereditary Angioedema (HAE).
  387 patients were treated with oral BCX7353 daily for up to 5 years.
  Common adverse effects included nasopharyngitis, headache, diarrhoea, abdominal pain, and nausea.
  Full results will be published online and can be found using the following link: https://eur03.safelinks.protection.outlook.com/?url=https%3A%2F%2Fu2790089.ct.sendgrid.net%2Fls%2Fclick%3Fupn%3DXv3JSvJ-2B3M71ppf7N9agba4yu73OCS9U-2BkKS40W1kfatiYvtk1-2B2gi1K6vKImual9G6VG4-2FeFhNKGHEnJKTVSkxeezaAtY-2FW0FQUhDGjcGoUaVlIiLgKQPsXXCiPJfRKUFsx_E1aO2-2BZlVOSJJV-2FajQqskegTd6IRomHYTi-2Fbt8SH3YJ5Q916LzrCzFOei8-2B7ee2XoDgjRZSY6DI3cWiotlzEkBzBvVJSHdrzcfz2ImDTNL2D1gWxZXVvjp-2BHUElR5d-2FJyXCzDjkmDgGsDM8U8telHtZRMdZe4mEFWS2Gyfw1NQbPV6JCs6FraS0Q3fdK18Q-2BokJ8tfNJZThdoQK8Cs4QsA-3D-3D&data=05%7C01%7Capprovals%40hra.nhs.uk%7C26d8ed281a5c4e44e49008daf8a171ba%7C8e1f0acad87d4f20939e36243d574267%7C0%7C0%7C638095669523343331%7CUnknown%7CTWFpbGZsb3d8eyJWIjoiMC4wLjAwMDAiLCJQIjoiV2luMzIiLCJBTiI6Ik1haWwiLCJXVCI6Mn0%3D%7C3000%7C%7C%7C&sdata=mDpvm2Yfkxh67DbyvukZBIMskjVaHRI2qN3BhbTkOb0%3D&reserved=0

• REC name

  South Central - Hampshire B Research Ethics Committee

• REC reference

  17/SC/0638

• Date of REC Opinion

  27 Dec 2017

• REC opinion

  Further Information Favourable Opinion