Polymyalgia Rheumatica (PMR)Phase 2NCT01396317
Study of Tocilizumab to Treat Polymyalgia Rheumatica
A Phase 2 study testing Tocilizumab in people with Polymyalgia Rheumatica (PMR), run by Hospital for Special Surgery, New York at 1 site in United States.
Trial 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). Read more about this study
- Who is it for?
- Ages 50 years and older
- Conditions the study is for
- Polymyalgia Rheumatica (PMR)
- What is being tested?
- Tocilizumab
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 10 participants
- Where
- 1 siteUnited States
- Study dates
- Apr 2011to Jan 2016
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 50 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
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
Trial locations and contacts
1 site in 1 country.
United States1 site
Hospital for Special Surgery
New York, New York, 100214898
About this study
Official title: Phase IIa of Tocilizumab In the Treatment of Polymyalgia Rheumatica
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).
Read the 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).
- Conditions
- Polymyalgia Rheumatica (PMR)
- Being tested
- Tocilizumab
- Keywords
- PMR, POLYMYALGIA RHEUMATICA, Tocilizumab, Hospital for Special Surgery, HSS, Robert Spiera
- Medical subject headings
- Polymyalgia Rheumatica
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
TocilizumabDrug
Also called Actemra
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.
| Study group | Role | What they receive |
|---|---|---|
| Tocilizumab | Experimental | 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. Receives: Drug: Tocilizumab |
What is being measured
The primary outcome is the main question the study is designed to answer.
Proportion of Patients in Disease Remission at Six Months From Trial Entry
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.
Measured: Six months
Number of Participants With Adverse Events as a Measure of Safety and Tolerability
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
Measured: 15 months
Secondary outcomes (4)
Proportion of Patients Able to Achieve Disease Remission (DR) Off Corticosteroids, Without Disease Relapse or Recurrence
Measured: 12 and 15 months from trial entry
Proportion of Patients Who Develop Disease Relapses
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.
Measured: 6, 12 and 15 months from trial entry
The Cumulative Dose of Prednisone
Measured: 6, 12 and 15 months from trial entry
Total Number of Relapses/Recurrences
Measured: 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Jul 2011 (estimated)
- Study startApr 2011
- Primary completionDec 2015
- Study completionJan 2016
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Polymyalgia Rheumatica (PMR)Studies of the same condition, open and under way.
- More trials run by Hospital for Special Surgery, New YorkThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 10 participants (actual)
Sponsor and investigators
- Lead sponsor
- Hospital for Special Surgery, New York
- Sponsor type
- University, hospital or other
- Collaborators
- Genentech, Inc.
- Responsible party
- Sponsor
- Investigators
- Robert F Spiera, MD, Principal investigator, Hospital for Special Surgery, New York
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01396317
- Sponsor's protocol ID
- 11081
- Status last verified
- Jan 2018
- First submitted
- 11 Jul 2011
- Last updated
- 17 Jan 2018
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 17 Jan 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase IIa of Tocilizumab In the Treatment of Polymyalgia Rheumatica
- ClinicalTrials.gov ID
- NCT01396317
- Acronym / sponsor ID
- 11081
- Conditions
- Polymyalgia Rheumatica (PMR)
- Keywords
- PMR, POLYMYALGIA RHEUMATICA, Tocilizumab, Hospital for Special Surgery, HSS, Robert Spiera
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 10 participants (actual)
- Lead sponsor
- Hospital for Special Surgery, New YorkUniversity, hospital or other
- Collaborators
- Genentech, Inc.
- Study start
- Apr 2011
- Primary completion
- Dec 2015 (actual)
- Study completion
- Jan 2016 (actual)
- First posted
- 18 Jul 2011 (estimated)
- Results
- Posted 17 Jan 2018
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Tocilizumab | Experimental | Drug: TocilizumabThis 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. |
Who may qualify
- Ages
- 50 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| TocilizumabAlso: ActemraTocilizumab 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. | Drug | Tocilizumab |
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: • 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
4 secondary outcomes
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 Cumulative Dose of Prednisone
Time frame: 6, 12 and 15 months from trial entry
Total Number of Relapses/Recurrences
Time frame: 12 months
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 17 Jan 2018.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Robert F Spiera, MD principal investigator
Hospital for Special Surgery, New York
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Tocilizumab in people with Polymyalgia Rheumatica (PMR), run by Hospital for Special Surgery, New York at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 17 Jan 2018. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 17 Jan 2018). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Proportion of Patients in Disease Remission at Six Months From Trial Entry (time frame: Six months). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 17 Jan 2018.