ClinicalTrials.gov
ClinicalTrials.gov Menu

Study of Tocilizumab to Treat Polymyalgia Rheumatica

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
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

Study Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Polymyalgia Rheumatica (PMR)
Intervention Drug: Tocilizumab
Enrollment 10

Recruitment Details  
Pre-assignment Details  
Arm/Group Title Tocilizumab + Corticosteroid Taper Control (Corticosteroid Taper Alone)
Hide Arm/Group Description In a single-center open-label study, subjects with newly diagnosed PMR (Healey criteria) and prior treatment with <1 month of corticosteroids (CS) were treated with TCZ 8mg/kg IV monthly for 12 months plus a rapid CS taper. Subjects were followed for 15 months. Those with concurrent GCA or those treated with >30mg prednisone were excluded. A cohort of consecutively evaluated patients with newly diagnosed PMR who declined participation in the trial or failed to meet inclusion criteria served as a control group. These patients were treated contemporaneously by a single rheumatologist with expertise in PMR and received CS alone, tapered at the treating physician’s discretion.
Period Title: Overall Study
Started 10 10
Completed 9 10
Not Completed 1 0
Reason Not Completed
Adverse Event             1             0
Arm/Group Title Tocilizumab + Corticosteroid Taper Control (Corticosteroid Taper Alone) Total
Hide Arm/Group Description

All subjects will receive the open-label active study treatment for 12 months, and will then be evaluated for 3 months of long-term follow-up.

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.

A cohort of consecutively evaluated patients with newly diagnosed PMR who declined participation in the trial, or failed to meet inclusion criteria, served as a comparator group.These patients were treated contemporaneously with the comparator group by a single rheumatologist with expertise in PMR and received glucocorticoids alone, tapered at the treating physician’s discretion, as is the standard of care in PMR. Total of all reporting groups
Overall Number of Baseline Participants 10 10 20
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 10 participants 10 participants 20 participants
68  (8.5) 72  (10.7) 70  (9.6)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 10 participants 10 participants 20 participants
Female
5
  50.0%
5
  50.0%
10
  50.0%
Male
5
  50.0%
5
  50.0%
10
  50.0%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 10 participants 10 participants 20 participants
10 10 20
1.Primary Outcome
Title Proportion of Patients in Disease Remission at Six Months From Trial Entry
Hide Description

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.

Time Frame Six months
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Tocilizumab + Corticosteroid Taper Control (Corticosteroid Taper Alone)
Hide Arm/Group Description:
In a single-center open-label study, subjects with newly diagnosed PMR (Healey criteria) and prior treatment with <1 month of corticosteroids (CS) were treated with TCZ 8mg/kg IV monthly for 12 months plus a rapid CS taper. Subjects were followed for 15 months. Those with concurrent GCA or those treated with >30mg prednisone were excluded.
A cohort of consecutively evaluated patients with newly diagnosed PMR who declined participation in the trial or failed to meet inclusion criteria served as a control group. These patients were treated contemporaneously by a single rheumatologist with expertise in PMR and received CS alone, tapered at the treating physician’s discretion.
Overall Number of Participants Analyzed 9 10
Measure Type: Count of Participants
Unit of Measure: Participants
9
 100.0%
0
   0.0%
2.Primary Outcome
Title Number of Participants With Adverse Events as a Measure of Safety and Tolerability
Hide Description

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

Time Frame 15 months
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Tocilizumab
Hide Arm/Group Description:

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: 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.

Overall Number of Participants Analyzed 10
Measure Type: Number
Unit of Measure: Number of Adverse Events
Adverse Events 23
Serious Adverse Events 1
3.Secondary Outcome
Title Proportion of Patients Able to Achieve Disease Remission (DR) Off Corticosteroids, Without Disease Relapse or Recurrence
Hide Description [Not Specified]
Time Frame 12 and 15 months from trial entry
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Tocilizumab + Corticosteroid Taper Control (Corticosteroid Taper Alone)
Hide Arm/Group Description:
In a single-center open-label study, subjects with newly diagnosed PMR (Healey criteria) and prior treatment with <1 month of corticosteroids (CS) were treated with TCZ 8mg/kg IV monthly for 12 months plus a rapid CS taper. Subjects were followed for 15 months. Those with concurrent GCA or those treated with >30mg prednisone were excluded.
A cohort of consecutively evaluated patients with newly diagnosed PMR who declined participation in the trial or failed to meet inclusion criteria served as a control group. These patients were treated contemporaneously by a single rheumatologist with expertise in PMR and received CS alone, tapered at the treating physician’s discretion.
Overall Number of Participants Analyzed 9 0
Measure Type: Count of Participants
Unit of Measure: Participants
9
 100.0%
0
4.Secondary Outcome
Title Proportion of Patients Who Develop Disease Relapses
Hide Description 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.
Time Frame 6, 12 and 15 months from trial entry
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Tocilizumab + Corticosteroid Taper Control (Corticosteroid Taper Alone)
Hide Arm/Group Description:

All subjects will receive the open-label active study treatment for 12 months, and will then be evaluated for 3 months of long-term follow-up.

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.

A cohort of consecutively evaluated patients with newly diagnosed PMR who declined participation in the trial, or failed to meet inclusion criteria, served as a comparator group.These patients were treated contemporaneously with the comparator group by a single rheumatologist with expertise in PMR and received glucocorticoids alone, tapered at the treating physician’s discretion, as is the standard of care in PMR.
Overall Number of Participants Analyzed 9 10
Measure Type: Count of Participants
Unit of Measure: Participants
0
   0.0%
10
 100.0%
5.Secondary Outcome
Title The Cumulative Dose of Prednisone
Hide Description [Not Specified]
Time Frame 6, 12 and 15 months from trial entry
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Tocilizumab + Corticosteroid Taper Control (Corticosteroid Taper Alone)
Hide Arm/Group Description:

All subjects will receive the open-label active study treatment for 12 months, and will then be evaluated for 3 months of long-term follow-up.

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.

A cohort of consecutively evaluated patients with newly diagnosed PMR who declined participation in the trial, or failed to meet inclusion criteria, served as a comparator group.These patients were treated contemporaneously with the comparator group by a single rheumatologist with expertise in PMR and received glucocorticoids alone, tapered at the treating physician’s discretion, as is the standard of care in PMR.
Overall Number of Participants Analyzed 9 10
Mean (Standard Deviation)
Unit of Measure: milligrams
1,085.3  (301.3) 2562.0  (1355.9)
6.Secondary Outcome
Title Total Number of Relapses/Recurrences
Hide Description [Not Specified]
Time Frame 12 months
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Tocilizumab + Corticosteroid Taper Control (Corticosteroid Taper Alone)
Hide Arm/Group Description:

All subjects will receive the open-label active study treatment for 12 months, and will then be evaluated for 3 months of long-term follow-up.

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.

A cohort of consecutively evaluated patients with newly diagnosed PMR who declined participation in the trial, or failed to meet inclusion criteria, served as a comparator group.These patients were treated contemporaneously with the comparator group by a single rheumatologist with expertise in PMR and received glucocorticoids alone, tapered at the treating physician’s discretion, as is the standard of care in PMR.
Overall Number of Participants Analyzed 9 10
Measure Type: Number
Unit of Measure: Incidents
0 7
Time Frame 4 years
Adverse Event Reporting Description Serious and Other (Not Including Serious) Adverse Events were not monitored/assessed for the control group (corticosteroid taper alone).
 
Arm/Group Title Tocilizumab + Corticosteroid Taper
Hide Arm/Group Description In a single-center open-label study, subjects with newly diagnosed PMR (Healey criteria) and prior treatment with <1 month of corticosteroids (CS) were treated with TCZ 8mg/kg IV monthly for 12 months plus a rapid CS taper. Subjects were followed for 15 months. Those with concurrent GCA or those treated with >30mg prednisone were excluded.
All-Cause Mortality
Tocilizumab + Corticosteroid Taper
Affected / at Risk (%)
Total   --/--    
Show Serious Adverse Events Hide Serious Adverse Events
Tocilizumab + Corticosteroid Taper
Affected / at Risk (%) # Events
Total   1/10 (10.00%)    
Injury, poisoning and procedural complications   
Non-displaced fracture of sternum with small anterior hematoma  [1]  1/10 (10.00%)  1
Indicates events were collected by systematic assessment
[1]
Car accident: Patient was seated on the passenger side of the vehicle, and was hit from the rear passenger’s side and the vehicle hit the center divider. Patient was diagnosed with a non-displaced fracture of the sternum with small anterior hematoma.
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Tocilizumab + Corticosteroid Taper
Affected / at Risk (%) # Events
Total   7/10 (70.00%)    
Blood and lymphatic system disorders   
Neutropenia   1/10 (10.00%)  7
Nose bleeds   1/10 (10.00%)  1
Elevated Cholesterol   1/10 (10.00%)  2
Anemia   1/10 (10.00%)  1
General disorders   
Light-Headedness   1/10 (10.00%)  1
Infections and infestations   
Viral Infection   1/10 (10.00%)  1
Sinus Infection   1/10 (10.00%)  1
Oral Herpes   1/10 (10.00%)  1
Injury, poisoning and procedural complications   
Infusion Reaction  [1]  1/10 (10.00%)  1
Musculoskeletal and connective tissue disorders   
Left Rotator cuff tendinosis   1/10 (10.00%)  1
Lower Back Pain   1/10 (10.00%)  1
Tricompartmental Osteoarthritis of Right Knee   1/10 (10.00%)  1
Respiratory, thoracic and mediastinal disorders   
Upper Respiratory Infection   2/10 (20.00%)  3
Surgical and medical procedures   
Trigger Finger Surgery   1/10 (10.00%)  1
Indicates events were collected by systematic assessment
[1]
After only receiving about 30cc of study drug during this infusion, the subject began to experience flushing and subsequently, chills. Subject was treated appropriately for the infusion reaction, and was then withdrawn from the trial.
Certain Agreements
All Principal Investigators ARE employed by the organization sponsoring the study.
Results Point of Contact
Name/Title: Dr. Robert Spiera
Organization: Hospital for Special Surgery
Phone: 212-774-2048
Responsible Party: Hospital for Special Surgery, New York
ClinicalTrials.gov Identifier: NCT01396317     History of Changes
Other Study ID Numbers: 11081
First Submitted: July 11, 2011
First Posted: July 18, 2011
Results First Submitted: April 21, 2017
Results First Posted: January 17, 2018
Last Update Posted: January 17, 2018