NCT02216084

Phase 1 Dose Escalation, Single Dose Study to Assess Safety and Pharmacokinetics of BAX930 in Hereditary Thrombotic Thrombocytopenic Purpura (TTP)

BAX930 (rADAMTS13): A Phase 1 Prospective, Uncontrolled, Open-Label, Multicenter, Dose-Escalation Study Evaluating the Safety and Pharmacokinetics in Hereditary Thrombotic Thrombocytopenic Purpura (TTP)

• Status: Completed
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 16 (actual)
• Sponsor: Baxalta now part of Shire · Industry
• Sex: All
• Age: 12 Years – 65 Years
• Healthy volunteers: Not accepted

Summary

The purpose of this Phase 1, prospective, uncontrolled, open-label, multicenter, dose-escalation study is to evaluate the safety, including immunogenicity, and pharmacokinetics of BAX930 (rADAMTS13) in a total of 14 evaluable subjects diagnosed with severe hereditary thrombotic thrombocytopenic purpura (TTP) (plasma ADAMTS13 activity \<6%) who are assigned to one of three dose cohorts.

Conditions

• Hereditary Thrombotic Thrombocytopenic Purpura (TTP)

Interventions

Type	Name	Description
DRUG	Recombinant ADAMTS13	rADAMTS13 (a disintegrin and metalloproteinase with a thrombospondin type 1 motif, member 13) is a lyophilized formulation for intravenous injection. The lyophilized rADAMTS13 is reconstituted with sterile water for injection. Subjects will receive an intravenous injection with rADAMTS13 at a dose of either 5 U/kg bodyweight (Cohort 1), or 20 U/kg bodyweight (Cohort 2), or 40 U/kg bodyweight (Cohort 3).

Timeline

• Start date: 2014-09-30
• Primary completion: 2016-02-22
• Completion: 2016-02-22
• First posted: 2014-08-13
• Last updated: 2021-05-05

Locations

11 sites across 7 countries: United States, Austria, Germany, Japan, Poland, Switzerland, United Kingdom