Research

Phase II/III, Multicenter, Randomized, Double-blind Placebo-Controlled Study to Assess the Efficacy and Safety of Tocilizumab vs. Placebo in Patients with Systemic Sclerosis

IRB Number: 12136

Institutional Review Board, Hospital for Special Surgery

July 29, 2013

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.

Before enrolling in a clinical trial, the investigator will explain the purpose of the trial, its expected benefits, any possible risks or side effects, and what your role will be. This is the time to ask questions! If you want to join the trial, you must sign the informed consent documents. You can leave a clinical trial at any time without penalty.

For further information, see Understanding Clinical Trials.

Principal Investigator

Robert F. Spiera, MD

Co-Investigators

Jessica K. Gordon, MD
Uzunma Udeh, BA
Nina Paddu, BA
Jason Zheng, Pharm.D

Summary

The purpose of this study is to evaluate the safety and effectiveness of an experimental drug called Tocilizumab. You are being invited to take part in a clinical research study of Tocilizumab (TCZ) for the treatment of systemic sclerosis (SSc).  We hope to learn how safe and effective TCZ treatment is in patients with SSc compared with treatment with placebo.

Inclusion/Exclusion Criteria

INCLUSION CRITERIA
• Patients must meet the following criteria for study entry:
o Ability and willingness to give written informed consent and comply with the requirements of the study protocol
o Diagnosis of SSc, as defined using the ACR criteria (1980; see Appendix 1)
o Disease duration of = 60 months (defined as time from the first non Raynaudphenomenon manifestation)
o Age = 18 years at baseline
o = 15 and = 40 mRSS units at the screening visit
o Uninvolved skin at one of the following locations:
# Front of the middle region of the thighs
# Lower part of the abdomen below the navel except for the 2-inch area directly around the navel
# Outer area of the upper arms (if a caregiver is giving the patient injections)
o Active disease defined as at least one A criterion and one B criterion each:
# Criteria A at screening
• Increase = 3 in mRSS units at screening compared with the last visit within previous 1–6 months
• Involvement of one new body area with = 2 mRSS units at screening compared with the last visit within the previous 1–6 months
• Involvement of two new body areas with = 1 mRSS units at screening compared with the last visit within the previous 1–6 months
• Other documentation of worsening skin thickening at screening compared with the last visit within the previous 1–6 months consistent with the progression of skin thickening described in the above criteria using mRSS
• Presence of 1 or more TFRs at screening
# Criteria B at screening
• High-sensitivity CRP = 1 mg/dL
• ESR = 28 mm/hr
• Platelet count (= 330 Χ 103/΅L)
o Treatment with oral corticosteroids (= 10 mg/day of prednisone or equivalent) is permitted if the patient is on a stable dose regimen for = 2 weeks prior to and including at baseline
o Treatment with NSAIDs is permitted if the patient is on a stable dose regimen for = 2 weeks prior to and including at baseline.
o ACE inhibitors, calcium-channel blockers, protein-pump inhibitors, and/or oral vasodilators are permitted if the patient is on a stable dose for = 4 weeks prior to and including at baseline.
o If female of childbearing potential, the patient must have a negative pregnancy test at screening and the baseline visit.

EXCLUSION CRITERIA
• Patients who meet any of the following criteria will be excluded from study entry:
o Major surgery (including joint surgery) within 8 weeks prior to screening or planned major surgery within 6 months following randomization
o Rheumatic autoimmune disease other than SSc, including but not limited to, RA, systemic lupus erythematosis, mixed connective tissue disorder, polymyositis, dermatomyositis, eosinophilic fasciitis, primary Sjφgren syndrome, and eosinophilic myalgia syndrome, as determined by the
o Principal Investigator
o Skin thickening (scleroderma) limited to areas distal to the elbows or knees at screening

Contact Information

Uzunma Udeh, BA
udehu@hss.edu
212-774-2123



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