Research

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

The safety of study participants is our top priority. The trial is approved and periodically reviewed by an Institutional Review Board (IRB), which includes doctors, administrators, ethicists, and members of the general public. The safety of clinical trials is reviewed by the U.S. Food and Drug Administration.

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For further information, see Understanding Clinical Trials.

Principal Investigator

Robert F. Spiera, MD

Co-Investigators

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

Summary

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
 udehu@hss.edu
212-774-2123



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