Baxter 281101: Phase 1 Study of Safety and Pharmacokinetics in TTP

• Research type

  Research Study

• Full title

  A PHASE 1 PROSPECTIVE, UNCONTROLLED, OPEN-LABEL, MULTICENTER, DOSE-ESCALATION STUDY EVALUATING THE SAFETY AND PHARMACOKINETICS IN HEREDITARY TTP (THROMBOTIC THROMBOCYTOPENIC PURPURA)

• IRAS ID

  119414

• Contact name

  Marie Scully

• Sponsor organisation

  Baxter Innovations GmbH

• Eudract number

  2012-003221-19

• ISRCTN Number

  n/a

• Research summary

  Hereditary Thrombotic Thrombocytopenic Purpura (hTTP) is an extremely rare genetic disorder of the blood coagulation system, characterised by the formation of small clots (thrombi) within the circulation, resulting in the consumption of platelets and therefore a low platelet count (thrombocytopenia). This is due to the lack of activity by ADAMTS13, an enzyme, which splits large plasma glycoproteins involved in blood clot formation, called von Willebrand factor (VWF) multimers. As a result, VWF is not cut into smaller fragments by the ADAMTS13 enzyme and abnormal blood clots are formed throughout the body, which can block blood flow through small vessels, causing damage to multiple organs mainly the brain, kidneys, heart and other organs. The exact incidence of hTTP is not known but is estimated to be less than 1/1,000,000 patients per year. The clinical presentation of hTTP is quite variable although symptoms develop soon after birth in approximately 50% of patients and in others, symptoms do not occur until the second or third decade of life.Currently the available treatment for hTTP is plasma products, containing the missing ADAMTS13 enzyme, The Sponsor, Baxter, has developed a new recombinant ADAMTS13 enzyme (BAX930), which is manufactured using genetically engineered hamster cells intended for treatment of patients with hTTP. This manufacturing process greatly decreases the risk of transmission of diseases since it is manufactured without materials of human or animal origin. The only protein other than BAX930 in the study drug may be traces from hamster protein.This is a phase 1, uncontrolled, open-label dose-escalation study to evaluate the safety and pharmacokinetics of BAX930 in patients with hTTP. Approximately 14 patients with hTTP will participate in this study which will be conducted at approximately 10 to 14 sites in USA, Europe and Japan. Participants will be in the study for approximately 8 weeks.

• REC name

  London - Hampstead Research Ethics Committee

• REC reference

  13/LO/0016

• Date of REC Opinion

  5 Feb 2013

• REC opinion

  Favourable Opinion