Trial Outcomes & Findings for Study of Tocilizumab to Treat Polymyalgia Rheumatica (NCT NCT01396317)

Last Updated: 2018-01-17

Results Overview

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.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

10 participants

Primary outcome timeframe

Six months

Results posted on

2018-01-17

Participant Flow

Participant milestones

Participant milestones
Measure	Tocilizumab + Corticosteroid Taper 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.	Control (Corticosteroid Taper Alone) 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 Study STARTED	10	10
Overall Study COMPLETED	9	10
Overall Study NOT COMPLETED	1	0

Reasons for withdrawal

Reasons for withdrawal
Measure	Tocilizumab + Corticosteroid Taper 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.	Control (Corticosteroid Taper Alone) 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 Study Adverse Event	1	0

Baseline Characteristics

Study of Tocilizumab to Treat Polymyalgia Rheumatica

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Tocilizumab + Corticosteroid Taper n=10 Participants 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.	Control (Corticosteroid Taper Alone) n=10 Participants 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 n=20 Participants Total of all reporting groups
Age, Continuous	68 years STANDARD_DEVIATION 8.5 • n=5 Participants	72 years STANDARD_DEVIATION 10.7 • n=7 Participants	70 years STANDARD_DEVIATION 9.6 • n=5 Participants
Sex: Female, Male Female	5 Participants n=5 Participants	5 Participants n=7 Participants	10 Participants n=5 Participants
Sex: Female, Male Male	5 Participants n=5 Participants	5 Participants n=7 Participants	10 Participants n=5 Participants
Region of Enrollment United States	10 participants n=5 Participants	10 participants n=7 Participants	20 participants n=5 Participants

PRIMARY outcome

Timeframe: Six months

Outcome measures

Outcome measures
Measure	Tocilizumab + Corticosteroid Taper n=9 Participants 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.	Control (Corticosteroid Taper Alone) n=10 Participants 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.
Proportion of Patients in Disease Remission at Six Months From Trial Entry	9 Participants	0 Participants

PRIMARY outcome

Timeframe: 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

Outcome measures

Outcome measures
Measure	Tocilizumab + Corticosteroid Taper n=10 Participants 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.	Control (Corticosteroid Taper Alone) 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.
Number of Participants With Adverse Events as a Measure of Safety and Tolerability Adverse Events	23 Number of Adverse Events	—
Number of Participants With Adverse Events as a Measure of Safety and Tolerability Serious Adverse Events	1 Number of Adverse Events	—

SECONDARY outcome

Timeframe: 12 and 15 months from trial entry

Outcome measures

Outcome measures
Measure	Tocilizumab + Corticosteroid Taper n=9 Participants 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.	Control (Corticosteroid Taper Alone) 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.
Proportion of Patients Able to Achieve Disease Remission (DR) Off Corticosteroids, Without Disease Relapse or Recurrence	9 Participants	0 Participants

SECONDARY outcome

Timeframe: 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.

Outcome measures

Outcome measures
Measure	Tocilizumab + Corticosteroid Taper n=9 Participants 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.	Control (Corticosteroid Taper Alone) n=10 Participants 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.
Proportion of Patients Who Develop Disease Relapses	0 Participants	10 Participants

SECONDARY outcome

Timeframe: 6, 12 and 15 months from trial entry

Outcome measures

Outcome measures
Measure	Tocilizumab + Corticosteroid Taper n=9 Participants 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.	Control (Corticosteroid Taper Alone) n=10 Participants 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.
The Cumulative Dose of Prednisone	1,085.3 milligrams Standard Deviation 301.3	2562.0 milligrams Standard Deviation 1355.9

SECONDARY outcome

Timeframe: 12 months

Outcome measures

Outcome measures
Measure	Tocilizumab + Corticosteroid Taper n=9 Participants 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.	Control (Corticosteroid Taper Alone) n=10 Participants 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.
Total Number of Relapses/Recurrences	0 Incidents	7 Incidents

Adverse Events

Tocilizumab + Corticosteroid Taper

Serious events: 1 serious events

Other events: 7 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Tocilizumab + Corticosteroid Taper n=10 participants at risk 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.
Injury, poisoning and procedural complications Non-displaced fracture of sternum with small anterior hematoma	10.0% 1/10 • Number of events 1 • 4 years Serious and Other (Not Including Serious) Adverse Events were not monitored/assessed for the control group (corticosteroid taper alone).

Other adverse events

Other adverse events
Measure	Tocilizumab + Corticosteroid Taper n=10 participants at risk 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.
Blood and lymphatic system disorders Neutropenia	10.0% 1/10 • Number of events 7 • 4 years Serious and Other (Not Including Serious) Adverse Events were not monitored/assessed for the control group (corticosteroid taper alone).
Infections and infestations Viral Infection	10.0% 1/10 • Number of events 1 • 4 years Serious and Other (Not Including Serious) Adverse Events were not monitored/assessed for the control group (corticosteroid taper alone).
Injury, poisoning and procedural complications Infusion Reaction	10.0% 1/10 • Number of events 1 • 4 years Serious and Other (Not Including Serious) Adverse Events were not monitored/assessed for the control group (corticosteroid taper alone).
General disorders Light-Headedness	10.0% 1/10 • Number of events 1 • 4 years Serious and Other (Not Including Serious) Adverse Events were not monitored/assessed for the control group (corticosteroid taper alone).
Blood and lymphatic system disorders Nose bleeds	10.0% 1/10 • Number of events 1 • 4 years Serious and Other (Not Including Serious) Adverse Events were not monitored/assessed for the control group (corticosteroid taper alone).
Blood and lymphatic system disorders Elevated Cholesterol	10.0% 1/10 • Number of events 2 • 4 years Serious and Other (Not Including Serious) Adverse Events were not monitored/assessed for the control group (corticosteroid taper alone).
Musculoskeletal and connective tissue disorders Left Rotator cuff tendinosis	10.0% 1/10 • Number of events 1 • 4 years Serious and Other (Not Including Serious) Adverse Events were not monitored/assessed for the control group (corticosteroid taper alone).
Blood and lymphatic system disorders Anemia	10.0% 1/10 • Number of events 1 • 4 years Serious and Other (Not Including Serious) Adverse Events were not monitored/assessed for the control group (corticosteroid taper alone).
Musculoskeletal and connective tissue disorders Lower Back Pain	10.0% 1/10 • Number of events 1 • 4 years Serious and Other (Not Including Serious) Adverse Events were not monitored/assessed for the control group (corticosteroid taper alone).
Respiratory, thoracic and mediastinal disorders Upper Respiratory Infection	20.0% 2/10 • Number of events 3 • 4 years Serious and Other (Not Including Serious) Adverse Events were not monitored/assessed for the control group (corticosteroid taper alone).
Infections and infestations Sinus Infection	10.0% 1/10 • Number of events 1 • 4 years Serious and Other (Not Including Serious) Adverse Events were not monitored/assessed for the control group (corticosteroid taper alone).
Surgical and medical procedures Trigger Finger Surgery	10.0% 1/10 • Number of events 1 • 4 years Serious and Other (Not Including Serious) Adverse Events were not monitored/assessed for the control group (corticosteroid taper alone).
Musculoskeletal and connective tissue disorders Tricompartmental Osteoarthritis of Right Knee	10.0% 1/10 • Number of events 1 • 4 years Serious and Other (Not Including Serious) Adverse Events were not monitored/assessed for the control group (corticosteroid taper alone).
Infections and infestations Oral Herpes	10.0% 1/10 • Number of events 1 • 4 years Serious and Other (Not Including Serious) Adverse Events were not monitored/assessed for the control group (corticosteroid taper alone).

Additional Information

Dr. Robert Spiera

Hospital for Special Surgery

Phone: 212-774-2048

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place