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Study of Tocilizumab to Treat Polymyalgia Rheumatica

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ClinicalTrials.gov Identifier: NCT01396317
Recruitment Status : Completed
First Posted : July 18, 2011
Results First Posted : January 17, 2018
Last Update Posted : January 17, 2018
Sponsor:
Collaborator:
Genentech, Inc.
Information provided by (Responsible Party):
Hospital for Special Surgery, New York

Tracking Information
First Submitted Date  ICMJE July 11, 2011
First Posted Date  ICMJE July 18, 2011
Results First Submitted Date  ICMJE April 21, 2017
Results First Posted Date  ICMJE January 17, 2018
Last Update Posted Date January 17, 2018
Study Start Date  ICMJE April 2011
Actual Primary Completion Date December 2015   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: January 15, 2018)
  • 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
Original Primary Outcome Measures  ICMJE
 (submitted: July 15, 2011)
  • 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 ]
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: January 15, 2018)
  • 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 ]
Original Secondary Outcome Measures  ICMJE
 (submitted: July 15, 2011)
  • 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 ]
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Study of Tocilizumab to Treat Polymyalgia Rheumatica
Official Title  ICMJE 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 Description

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  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Polymyalgia Rheumatica (PMR)
Intervention  ICMJE Drug: Tocilizumab
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
Study Arms  ICMJE Experimental: Tocilizumab
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.
Intervention: Drug: Tocilizumab
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: July 15, 2011)
10
Original Estimated Enrollment  ICMJE Same as current
Actual Study Completion Date  ICMJE January 2016
Actual Primary Completion Date December 2015   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Disease- Specific Inclusion Criteria:

Patients must meet the following inclusion criteria to be eligible for study entry:

Diagnosed with polymyalgia rheumatica and enrolled within one month of diagnosis.

Disease Specific Exclusion Criteria:

Patients will be excluded from the study based on the following criteria:

  • Symptoms or diagnosis of temporal arteritis, including headache, jaw claudication, hyperesthesia of scalp, abnormal palpated temporal artery, visual disturbances, temporal artery biopsy positivity
  • Concurrent rheumatoid arthritis
  • Presence of rheumatoid factor and CCP
  • Other inflammatory arthritis or other connective tissue diseases, such as but not limited to systemic lupus erythematosus, systemic sclerosis, vasculitis, polymyositis, dermatomyositis, mixed connective tissue disease
  • Treatment of polymyalgia rheumatica with more than 20mg of daily prednisone or its equivalent at the time of screening
  • Treatment with more than 30mg of daily prednisone or its equivalent since diagnosis. Treatment with 30mg of daily prednisone or its equivalent since diagnosis for more than 2 weeks.
  • More than 4 weeks of corticosteroid therapy prior to enrollment
  • History of bowel perforation within the past five years.
  • Active diverticulitis.
  • Pre-existing or recent onset demyelinating disorders
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 50 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT01396317
Other Study ID Numbers  ICMJE 11081
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Hospital for Special Surgery, New York
Study Sponsor  ICMJE Hospital for Special Surgery, New York
Collaborators  ICMJE Genentech, Inc.
Investigators  ICMJE
Principal Investigator: Robert F Spiera, MD Hospital for Special Surgery, New York
PRS Account Hospital for Special Surgery, New York
Verification Date January 2018

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP