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). ]

Safety will be assessed based on the incidence and severity of treatment emergent adverse events and serious adverse events. Incidence of clinical laboratory abnormalities and changes from baseline through study completion in vital signs, physical examinations, ECGs and laboratory tests will also be evaluated


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