Study of Tocilizumab to Treat Polymyalgia Rheumatica
|ClinicalTrials.gov Identifier: NCT01396317|
Recruitment Status : Completed
First Posted : July 18, 2011
Results First Posted : January 17, 2018
Last Update Posted : January 17, 2018
|Condition or disease||Intervention/treatment||Phase|
|Polymyalgia Rheumatica (PMR)||Drug: Tocilizumab||Phase 2|
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much more quickly than what is done in routine clinical practice; there is an expectation that steroid therapy will be withdrawn within six months.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 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
Disease Remission (DR) will be defined as the disappearance of signs and symptoms of polymyalgia rheumatica (aching and stiffness of the shoulder, hip girdle, or both) with normalization of erythrocyte sedimentation rate (ESR<30 mm/hr) and c-reactive protein (CRP ≤1.0 mg/dl), unless elevation of ESR and/or CRP are attributable to causes other than PMR (i.e., infection).
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||10 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||Phase IIa of Tocilizumab In the Treatment of Polymyalgia Rheumatica|
|Study Start Date :||April 2011|
|Primary Completion Date :||December 2015|
|Study Completion Date :||January 2016|
This is a single-arm study. All subjects will receive the active study treatment for 12 months, and will then be evaluated for 3 months of long-term follow-up.
Tocilizumab is a humanized anti-interleukin-6 receptor antibody that has been FDA approved for the treatment of rheumatoid arthritis (RA). This molecule binds to the IL-6 binding site of human IL-6 receptor, and competitively inhibits IL-6 signaling.
Other Name: Actemra
- 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:
• 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
Relapse was defined as the reappearance of signs and symptoms of PMR, accompanied by an increasing erythrocyte sedimentation rate and/or C-reactive protein level attributable to disease activity. Recurrence was similarly defined as the return of PMR symptoms in conjunction with elevations in levels of inflammation markers, occurring 1 month after discontinuation of glucocorticoid therapy.
- Number of Participants With Adverse Events as a Measure of Safety and Tolerability [ Time Frame: 15 months ]
The co-primary endpoints for this study include evaluations of safety and tolerability:
• Safety and tolerability of Tocilizumab will be evaluated during the fifteen-month study period by the monitoring of adverse events and immunogenicity surveillance
- 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 ]Relapse was defined as the reappearance of signs and symptoms of PMR, accompanied by an increasing erythrocyte sedimentation rate and/or C-reactive\ protein level attributable to disease activity. Recurrence was similarly defined as the return of PMR symptoms in conjunction with elevations in levels of inflammation markers, occurring 1 month after discontinuation of GC therapy.
- The Cumulative Dose of Prednisone [ Time Frame: 6, 12 and 15 months from trial entry ]
- Total Number of Relapses/Recurrences [ Time Frame: 12 months ]
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01396317
|United States, New York|
|Hospital for Special Surgery|
|New York, New York, United States, 100214898|
|Principal Investigator:||Robert F Spiera, MD||Hospital for Special Surgery, New York|