A Phase III, Multicenter, Randomized, Double-Blind Placebo-Controlled Study to Assess the Efficacy and Safety of Tocilizumab in Subjects with Giant Cell Arteritis

IRB Number: 13044

Institutional Review Board, Hospital for Special Surgery

April 02, 2014

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Principal Investigator

Robert F. Spiera, MD


Jessica K. Gordon, MD
Lindsay Lally, MD
Uzunma Udeh
Nina Paddu
Daniele Lerner
Jason Zheng, PharmD
Aisha Ali, PharmD


This multicenter, randomized, double-blind, placebo-controlled study will evaluate the efficacy and safety of RoActemra/Actemra (tocilizumab) in patients with giant cell arteritis. Patients will be randomized to receive either RoActemra/Actemra 162 mg subcutaneously weekly or every 2 weeks or placebo for 52 weeks, with tapering oral daily doses of prednisone. After Week 52, patients in remission will stop study treatment and enter long-term follow-up, whereas patients with disease activity or flares will receive open-label RoActemra/Actemra 162 mg subcutaneously weekly for a maximum period of 104 weeks at the discretion of the investigator. Anticipated time on study is 39 months. Six patients will be recruited from this site.

Inclusion/Exclusion Criteria

Inclusion Criteria:
• Diagnosis of giant cell arteritis classified according to the following criteria:
o Age >/= 50 years
o History of ESR >/= 50 mm/hour
o and at least one of the following:
 Unequivocal cranial symptoms of giant cell arteritis
 Symptoms of polymyalgia rheumatica
o and at least one of the following
 Temporal artery biopsy revealing features of giant cell arteritis
 Evidence of large-vessel vasculitis
• New onset active disease (diagnosis within 6 weeks of baseline) or refractory active disease (diagnosis > 6 weeks before baseline and previous treatment with >/= 40 mg/day prednisone (or equivalent) for at least 2 consecutive weeks at any time); active disease defined as presence of clinical signs and symptoms and ESR >/= 30 mm/hour or CRP >/= 1 mg/dl within 6 weeks of baseline
Exclusion Criteria:
• Recent or incoming major surgery
• Organ transplantation recipient (except corneas within 3 months prior to baseline visit)
• Major ischemic event, unrelated to giant cell arteritis, within 12 weeks of screening
• Prior treatment with any of the following:
o Investigational agent within 12 weeks (or 5 half-lives of the investigational drug, whichever is longer) of screening visit
o Cell-depleting agents (e.g. anti CD 20)
o Tocilizumab
o Tofacitinib
o Alkylating agents including CYC within 6 months of baseline
o HCQ, CsA, AZA, or MMF within 4 weeks of baseline
o Tumor necrosis factor inhibitors within 2-8 weeks of baseline
o Anakinra within 1 week of baseline
o Corticosteroids for conditions other than GCA
o IV corticosteroids within 6 weeks of baseline
• History of severe allergic reactions to monoclonal antibodies
• Evidence of serious uncontrolled concomitant disease (e.g. cardiovascular, respiratory, renal, endocrine)
• Current liver disease that could interfere with the trial as determined by the investigator
• History of diverticulitis, inflammatory bowel disease, or other symptomatic GI tract condition that might predispose to bowel perforation
• Infections:
o Active current or history of recurrent bacterial, viral fungal, mycobacterial, or other infection
o Prior episode of major infection
o Active TB requiring treatment within the previous 3 years
o Untreated latent TB infection (LTBI)
• Primary or secondary immunodeficiency
• Malignancy (except for basal and squamous cell carcinoma of the skin or carcinoma in situ of the cervix uteri that has been excised and cured)
• Inadequate hematologic, renal or liver function
• Positive for hepatitis B or hepatitis C infection

Contact Information

Uzunma Udeh