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Trial record 1 of 1 for:    Phase IIa Trial of ALXN1007 for the Treatment of Non-criteria Manifestations of Antiphospholipid Syndrome
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Phase IIa Trial of ALXN1007 for the Treatment of Non-criteria Manifestations of Antiphospholipid Syndrome

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT02128269
Recruitment Status : Terminated (Screening discontinued early due to slow patient enrollment)
First Posted : May 1, 2014
Results First Posted : July 13, 2017
Last Update Posted : July 13, 2017
Sponsor:
Information provided by (Responsible Party):
Alexion Pharmaceuticals

Tracking Information
First Submitted Date  ICMJE April 29, 2014
First Posted Date  ICMJE May 1, 2014
Results First Submitted Date  ICMJE June 14, 2017
Results First Posted Date  ICMJE July 13, 2017
Last Update Posted Date July 13, 2017
Study Start Date  ICMJE April 2014
Actual Primary Completion Date June 2016   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: June 14, 2017)
Safety and Tolerability of Intravenous (IV) ALXN1007 as Measured by Percentage of Patients Reporting Adverse Events [ Time Frame: Treatment Period (24 weeks) ]
Original Primary Outcome Measures  ICMJE
 (submitted: April 30, 2014)
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
Change History Complete list of historical versions of study NCT02128269 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE Not Provided
Original Secondary Outcome Measures  ICMJE
 (submitted: April 30, 2014)
  • 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.
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures
 (submitted: April 30, 2014)
To assess the effect of ALXN1007 on aPL-nephropathy, skin ulcers and thrombocytopenia [ Time Frame: Treatment Period (24 weeks) and Follow-up Period (12 weeks). ]
Urine protein to creatinine ratio for patients with aPL-nephropathy. Manual measurement of ulcer area and objective analysis of digital photographs for patients with skin ulcers. Platelet count for patients with thrombocytopenia.
 
Descriptive Information
Brief Title  ICMJE Phase IIa Trial of ALXN1007 for the Treatment of Non-criteria Manifestations of Antiphospholipid Syndrome
Official Title  ICMJE 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 was 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 Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Antiphospholipid (aPL)-Positive
Intervention  ICMJE Biological: Study Drug- ALXN1007
10 mg/kg IV q 2 weeks x 12 doses
Study Arms  ICMJE Experimental: ALXN1007- Open label study
ALXN1007
Intervention: Biological: Study Drug- ALXN1007
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Terminated
Actual Enrollment  ICMJE
 (submitted: October 20, 2015)
9
Original Estimated Enrollment  ICMJE
 (submitted: April 30, 2014)
20
Actual Study Completion Date  ICMJE June 2016
Actual Primary Completion Date June 2016   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

Patients with a persistent and clinically significant aPL profile

Patients with at least 1 of the following non-criteria manifestations of APS:

  1. aPL-nephropathy (diagnosed by kidney biopsy within 12 months of Screening) confirmed based on the updated APS Classification Criteria recommendations, and urine protein to creatinine ratio > 1.0 at the time of the Screening visit and/or
  2. Skin ulcers (non-infected livedoid vasculitis-like skin ulcer and/or large skin ulceration resembling pyoderma gangrenosum) for at least 4 weeks prior to the Screening visit, diagnosed by physical examination, and/or
  3. Persistent active thrombocytopenia (diagnosed by platelet counts <100 x 103/μL and ≥20 x 103/μL [SI: <100 x 109/L and ≥ 20 x 109/L]) and confirmed at the time of screening (at least 4 weeks after previous test) based on the updated APS Classification Criteria recommendations Patients and spouse/partner who is of childbearing potential must be using highly effective contraception consisting of 2 forms of birth control (at least 1 of which must be a barrier method) starting at Screening and continuing through the entire study.

Patients with aPL-nephropathy must be receiving or agree to initiate an ACE inhibitor or angiotensin receptor blocker at least 4 weeks prior to initiation of ALXN1007 treatment; unless patient is documented to be intolerant. Patients receiving oral corticosteroids must be on a stable dose of ≤ 10 mg/day of prednisone or equivalent dose of another corticosteroid preparation for at least 4 weeks prior to the first dose of ALXN1007. Patients receiving immunosuppressive medications (including but not limited to methotrexate, hydroxychloroquine, azathioprine, cyclosporine and mycophenolate mofetil) must be on a stable dose for at least 4 weeks prior to the first dose of ALXN1007. Patients receiving oral anticoagulants or antiplatelet agents (including but not limited to aspirin) must be on stable doses for at least 4 weeks prior to first dose of ALXN1007.

Patients must be willing and able to give written informed consent and to comply with all study visits and procedures.

Exclusion Criteria:

Patients meeting the ACR classification criteria for systemic lupus erythematosus, systemic sclerosis or other systemic autoimmune diseases other than Primary APS.

Patients experiencing an acute thrombosis or a Major Adverse Vascular Event (MAVE) within 12 weeks prior to first administration of ALXN1007.

Patients with skin ulcers from causes other than aPL or who are positive for DVT or venous insufficiency at Screening. Patients with renal function status requiring chronic dialysis (defined as dialysis on a regular basis as renal replacement therapy). Patients with unresolved meningococcal disease or with known active bacterial, viral, fungal, mycobacterial or other infection. Patients that have received IVIg treatment within 4 weeks prior to first dose of ALXN1007. Patients that have received a course of rituximab (RITUXAN®) therapy within 12 months prior to first dose of ALXN1007 or have evidence of persistent depletion of the targeted lymphocyte population. Women who are pregnant or nursing.

Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years to 75 Years   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Brazil,   France,   Italy,   Japan,   United Kingdom,   United States
Removed Location Countries Canada,   Colombia,   Korea, Republic of,   Serbia,   Spain,   Taiwan
 
Administrative Information
NCT Number  ICMJE NCT02128269
Other Study ID Numbers  ICMJE ALXN1007-APS-201
2013-003588-73 ( EudraCT Number )
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Alexion Pharmaceuticals
Study Sponsor  ICMJE Alexion Pharmaceuticals
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: Bert Yao, M.D., Ph.D. Alexion Medical Monitor
PRS Account Alexion Pharmaceuticals
Verification Date June 2017

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP