9930 Phase I study - volunteers and patients

• Research type

  Research Study

• Full title

  A Phase 1 dose-ranging study to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of single and multiple doses of BCX9930 in healthy subjects and in subjects with paroxysmal nocturnal hemoglobinuria (PNH)

• IRAS ID

  261835

• Contact name

  Diane Gesty-Palmer

• Contact email

  dgesty-palmer@biocryst.com

• Sponsor organisation

  BioCryst Pharmaceuticals Inc

• Eudract number

  2019-001637-14

• Duration of Study in the UK

  2 years, 0 months, 0 days

• Research summary

  Biocryst Pharmaceuticals Inc. (the sponsor) is developing a new test medicine to treat Paroxysmal Nocturnal Hemoglobinuria (PNH).
  
  PNH is a rare blood condition where blood cells are prone to be attacked by part of the body’s immune system. The disorder affects red blood cells, which carry oxygen; white blood cells, which protect the body from infection; and platelets, which are involved in blood clotting. People with PNH have sudden, recurring episodes of symptoms which may be activated by stresses on the body, such as infections or physical exertion. During these episodes, red blood cells are destroyed earlier than should be (haemolysis).

  This 3 part study will involve 96 healthy volunteers and 16 patients. Part 1 and 2 will look at the safety and tolerability after single and multiple doses of BCX9930 (the test medicine) in healthy volunteers in the UK. Part 3 of this study will look at the safety and tolerability after multiple doses of BCX9930 in patients with PNH at multiple sites globally, including in the UK.

  Part 1 will involve 6 groups of 8 volunteers. Volunteers will attend the clinic for 4 days where they will receive one dose of BCX9930 or placebo following an overnight fast. One group of volunteers will return to receive a second dose of either BCX9930 or placebo following breakfast. This will be the first time BCX9930 is given to humans.

  Part 2 will involve 4 groups of 12 volunteers. Volunteers will attend the clinic and be dosed up to 3 times a day for up to 14 days with either BCX9930 or placebo.

  Part 3 will involve 4 groups of 4 patients with PNH. The patients will receive BCX9930 alone or with other medicines used to treat PNH for up to 1 year.

  Each group may include up to 2 more volunteers or patients.

• REC name

  Wales REC 2

• REC reference

  19/WA/0158

• Date of REC Opinion

  7 Jun 2019

• REC opinion

  Further Information Favourable Opinion