Clinical Trial: Phase IIa Trial of ALXN1007 for the Treatment of Non-criteria Manifestations of Antiphospholipid Syndrome
Study Status: Completed
Recruit Status: Completed
Study Type: Interventional
Official Title: An Open-Label Proof of Concept Phase IIa Trial of ALXN1007 for the Treatment of Non-criteria Manifestations of Antiphospholipid Syndrome
Brief Summary: The primary purpose of this study is to evaluate the safety and tolerability of intravenous (IV) ALXN1007 in persistently antiphospholipid (aPL)-positive patients with at least 1 of the following non-criteria manifestations of APS: aPL-nephropathy, skin ulcers and/or thrombocytopenia.
Detailed Summary: Enrollment discontinued early due to recruitment difficulties. Initially enrolled patients will complete the study.
Sponsor: Alexion Pharmaceuticals
Current Primary Outcome: To evaluate the safety and tolerability of intravenous (IV) ALXN1007 in persistently antiphospholipid (aPL)-positive patients with at least 1 of the following non-criteria manifestations of APS: aPL-nephropathy, skin ulcers and/or thrombocytopenia [ Time Frame: Treatment Period (24 weeks) and Follow-up Period (12 weeks). ]
Original Primary Outcome: Same as current
Current Secondary Outcome:
- To evaluate the pharmacokinetic (PK) parameters of IV ALXN1007. [ Time Frame: Treatment Period (24 weeks) and Follow-up Period (12 weeks). ]Estimates of PK parameters will include, at a minimum, time to maximum observed plasma concentration (tmax), maximum observed plasma concentration (Cmax), and total body clearance (CLR).
- To evaluate the pharmacodynamic (PD) effects of IV ALXN1007. [ Time Frame: Treatment Period (24 weeks) and Follow-up Period (12 weeks). ]The PD endpoints will include changes from baseline in several complement protein levels and assessment of effect on terminal complement activity through Week 24.
Original Secondary Outcome: Same as current
Information By: Alexion Pharmaceuticals
Dates:
Date Received: April 29, 2014
Date Started: April 2014
Date Completion:
Last Updated: September 2, 2016
Last Verified: September 2016