Clinical Trial: Study of Tocilizumab to Treat Polymyalgia Rheumatica

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

Official Title: Phase IIa of Tocilizumab In the Treatment of Polymyalgia Rheumatica

Brief Summary: This is a fifteen-month open label, Phase IIa clinical trial is being conducted to assess the tolerability, safety and efficacy of a medication called Tocilizumab (Actemra®) in patients with polymyalgia rheumatica (PMR).

Detailed Summary:

This fifteen-month open label, Phase IIa clinical trial is being conducted to assess the tolerability, safety and efficacy of a medication called Tocilizumab (Actemra®) in patients with polymyalgia rheumatica (PMR). The typical symptoms of PMR are muscle pain and stiffness in the shoulder, neck or hip region. Steroids have traditionally been used to treat this condition with great success, although long courses of steroids, up to 2 years in many cases, are often required. This can result in many unwanted side effects including diabetes, high blood pressure, heart disease, cataracts, weak bones with fractures, weak muscles, skin bruising, difficulty sleeping and mood disturbances. In this trial, the steroid dosage will be decreased much more quickly than what is done in routine clinical practice; there is an expectation that steroid therapy will be withdrawn within six months.

Tocilizumab is a medication already on the market that has been FDA approved in the US and Japan for the treatment of rheumatoid arthritis, and in Japan it is also approved for certain types of juvenile idiopathic arthritis (which is like rheumatoid arthritis in children) and Castleman's disease (which is a rare disease that causes enlarged lymph nodes). It is not FDA approved to treat polymyalgia rheumatica at this time. In this study, it will be given as an intravenous infusion once a month for a treatment period of one year. Experiments done on the blood of patients with PMR show one particular cytokine or small molecule that circulates throughout the body, interleukin-6, to be elevated in this disease. Tocilizumab is a medication that is designed to specifically block this cytokine. The co-primary endpoints for this study include efficacy, as well as evaluations of safety and tolerability.

  • Efficacy will be defi
    Sponsor: Hospital for Special Surgery, New York

    Current Primary Outcome:

    • Proportion of Patients in Disease Remission at Six Months from Trial Entry [ Time Frame: Six months ]

      The co-primary endpoints for this study include efficacy, as well as evaluations of safety and tolerability.

      • Efficacy will be defined by the proportion of patients in Disease Remission (DR) off corticosteroids, without relapse or recurrence, at six months from trial entry
      • Safety and tolerability of Tocilizumab will be evaluated during the fifteen-month study period by the monitoring of adverse events and immunogenicity surveillance
    • Number of Participants with Adverse Events as a Measure of Safety and Tolerability [ Time Frame: 15 months ]


    Original Primary Outcome: Same as current

    Current Secondary Outcome:

    • Proportion of patients able to achieve Disease Remission (DR) off corticosteroids, without Disease Relapse or Recurrence [ Time Frame: 12 and 15 months from trial entry ]
    • Proportion of patients who develop Disease Relapses [ Time Frame: 6, 12 and 15 months from trial entry ]
    • The proportion of patients who develop Disease Recurrences [ Time Frame: 6, 12 and 15 months from trial entry ]
    • The cumulative dose of prednisone [ Time Frame: 6, 12 and 15 months from trial entry ]


    Original Secondary Outcome: Same as current

    Information By: Hospital for Special Surgery, New York

    Dates:
    Date Received: July 11, 2011
    Date Started: April 2011
    Date Completion:
    Last Updated: March 15, 2017
    Last Verified: March 2017