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

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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

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.

Condition or disease Intervention/treatment Phase
Antiphospholipid (aPL)-Positive Biological: Study Drug- ALXN1007 Phase 2

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 9 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: An Open-Label Proof of Concept Phase IIa Trial of ALXN1007 for the Treatment of Non-criteria Manifestations of Antiphospholipid Syndrome
Study Start Date : April 2014
Actual Primary Completion Date : June 2016
Actual Study Completion Date : June 2016

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
Experimental: ALXN1007- Open label study
ALXN1007
Biological: Study Drug- ALXN1007
10 mg/kg IV q 2 weeks x 12 doses




Primary Outcome Measures :
  1. Safety and Tolerability of Intravenous (IV) ALXN1007 as Measured by Percentage of Patients Reporting Adverse Events [ Time Frame: Treatment Period (24 weeks) ]


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Ages Eligible for Study:   18 Years to 75 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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.


Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02128269


Locations
United States, New York
Hospital for Special Surgery
New York, New York, United States, 10021
United States, Texas
University of Texas Medical Branch
Galveston, Texas, United States, 77555
United States, Virginia
O & O Alpan, LLC
Fairfax, Virginia, United States, 22030
Brazil
Hospital das Clínicas da Faculdade de Medicina da USP
São Paulo, Sao Paulo, Brazil, 05403-000
France
Hôpital Cochin
Paris, France 75679, France, 75679
Hopital Claude Huriez - CHU Lille
Lille, Nord, France, 59037
Italy
Azienda Ospedaliera di Padova
Padova, Italy, 35128
Japan
Hokkaido University Hospital
Sapporo-shi, Hokkaido 060-8648, Japan, 060-8648
United Kingdom
University College London Hospitals
London, Greater London, United Kingdom, NW12PG
Sponsors and Collaborators
Alexion Pharmaceuticals
Investigators
Study Director: Bert Yao, M.D., Ph.D. Alexion Medical Monitor

Responsible Party: Alexion Pharmaceuticals
ClinicalTrials.gov Identifier: NCT02128269     History of Changes
Other Study ID Numbers: ALXN1007-APS-201
2013-003588-73 ( EudraCT Number )
First Posted: May 1, 2014    Key Record Dates
Results First Posted: July 13, 2017
Last Update Posted: July 13, 2017
Last Verified: June 2017

Keywords provided by Alexion Pharmaceuticals:
Antiphospholipid (aPL)-positive
Antiphospholid syndrome (APS)

Additional relevant MeSH terms:
Antiphospholipid Syndrome
Autoimmune Diseases
Immune System Diseases