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

NCT ID: NCT02128269

Last Updated: 2017-07-13

Basic Information

• Recruitment Status: TERMINATED
• Clinical Phase: PHASE2
• Total Enrollment: 9 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2014-04-30
• Study Completion Date: 2016-06-30

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.

Conditions

Antiphospholipid (aPL)-Positive

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

ALXN1007- Open label study

ALXN1007

Group Type: EXPERIMENTAL

Study Drug- ALXN1007

Intervention Type: BIOLOGICAL

10 mg/kg IV q 2 weeks x 12 doses

Interventions

Study Drug- ALXN1007

10 mg/kg IV q 2 weeks x 12 doses

Intervention Type: BIOLOGICAL

Eligibility 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.

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 75 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Alexion Pharmaceuticals, Inc.

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Bert Yao, M.D., Ph.D.

Role: STUDY_DIRECTOR

Alexion Medical Monitor

Locations

Hospital for Special Surgery

New York, New York, United States

University of Texas Medical Branch

Galveston, Texas, United States

O & O Alpan, LLC

Fairfax, Virginia, United States

Hospital das Clínicas da Faculdade de Medicina da USP

São Paulo, São Paulo, Brazil

Hôpital Cochin

Paris, France 75679, France

Hopital Claude Huriez - CHU Lille

Lille, Nord, France

Azienda Ospedaliera di Padova

Padua, , Italy

Hokkaido University Hospital

Sapporo, Hokkaido 060-8648, Japan

University College London Hospitals

London, Greater London, United Kingdom

Countries

United States; Brazil; France; Italy; Japan; United Kingdom

Other Identifiers

2013-003588-73

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

ALXN1007-APS-201

Identifier Type: -

Identifier Source: org_study_id