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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT03600818




Registration number
NCT03600818
Ethics application status
Date submitted
17/07/2018
Date registered
26/07/2018

Titles & IDs
Public title
Evaluation of the Efficacy and Safety of Sarilumab in Patients With Polymyalgia Rheumatica
Scientific title
A Randomized, Double-blind, Placebo-controlled Study to Evaluate the Efficacy and Safety of Sarilumab in Patients With Polymyalgia Rheumatica
Secondary ID [1] 0 0
2017-002989-42
Secondary ID [2] 0 0
EFC15160
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Polymyalgia Rheumatica 0 0
Condition category
Condition code
Musculoskeletal 0 0 0 0
Other muscular and skeletal disorders
Inflammatory and Immune System 0 0 0 0
Other inflammatory or immune system disorders
Cardiovascular 0 0 0 0
Diseases of the vasculature and circulation including the lymphatic system
Neurological 0 0 0 0
Other neurological disorders
Inflammatory and Immune System 0 0 0 0
Autoimmune diseases

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Sarilumab SAR153191 (REGN88)
Treatment: Drugs - Sarilumab-matching placebo
Treatment: Drugs - Prednisone
Treatment: Drugs - Prednisone-matching placebo
Treatment: Drugs - Prednisone

Placebo comparator: Placebo+52 Week Taper - Participants received sarilumab-matching placebo as subcutaneous (SC) injection every 2 weeks (q2w) up to 52 weeks along with the combination of prednisone and/or prednisone-matching placebo according to the protocol-defined schedule. Participants received prednisone/prednisone-matching placebo tapering oral daily doses for 52 weeks.

Experimental: Sarilumab 200mg q2w+14 Week Taper - Participants received sarilumab 200 milligrams (mg) as SC injection q2w up to 52 weeks along with the combination of prednisone and/or prednisone-matching placebo according to the protocol-defined schedule. Participants received prednisone tapering oral daily doses during the first 14 weeks and prednisone-matching placebo from Week 14 up to Week 52.


Treatment: Drugs: Sarilumab SAR153191 (REGN88)
Pharmaceutical form:solution for injection Route of administration: subcutaneous

Treatment: Drugs: Sarilumab-matching placebo
Pharmaceutical form:solution for injection Route of administration: subcutaneous

Treatment: Drugs: Prednisone
Pharmaceutical form:over-encapsulated tablets Route of administration: oral administration

Treatment: Drugs: Prednisone-matching placebo
Pharmaceutical form:over-encapsulated tablets Route of administration: oral administration

Treatment: Drugs: Prednisone
Pharmaceutical form:tablets Route of administration: oral administration

Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Percentage of Participants Achieving Sustained Remission at Week 52
Timepoint [1] 0 0
At Week 52
Secondary outcome [1] 0 0
Total Cumulative Corticosteroid Dose
Timepoint [1] 0 0
Up to Week 52
Secondary outcome [2] 0 0
Number of Participants Who Achieved Disease Remission up to Week 12
Timepoint [2] 0 0
Up to Week 12
Secondary outcome [3] 0 0
Number of Participants With Absence of Disease Flare From Week 12 Through Week 52
Timepoint [3] 0 0
From Week 12 Through Week 52
Secondary outcome [4] 0 0
Number of Participants With Sustained Reduction of CRP From Week 12 Through Week 52
Timepoint [4] 0 0
From Week 12 through Week 52
Secondary outcome [5] 0 0
Number of Participants With Successful Adherence to the Prednisone Taper From Week 12 Through Week 52
Timepoint [5] 0 0
From Week 12 through Week 52
Secondary outcome [6] 0 0
Time to First Polymyalgia Rheumatica Flare After Clinical Remission up to Week 52
Timepoint [6] 0 0
Up to Week 52
Secondary outcome [7] 0 0
Composite Glucocorticoid Toxicity Index (C-GTI): Cumulative Worsening Score (CWS) and Aggregate Improvement Score (AIS) at Week 52
Timepoint [7] 0 0
At Week 52
Secondary outcome [8] 0 0
Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Treatment-emergent Serious Adverse Events (TESAEs)
Timepoint [8] 0 0
From first dose (i.e. Day 1) up to 60 days after last dose date of study drug (i.e. up to Week 60)
Secondary outcome [9] 0 0
Number of Participants With Potentially Clinically Significant Vital Signs Abnormalities During TEAE Period
Timepoint [9] 0 0
From first dose (i.e., Day 1) up to 60 days after last dose date of study drug (i.e., up to Week 60)
Secondary outcome [10] 0 0
Number of Participants With Potentially Clinically Significant Abnormalities - Hematological Parameter
Timepoint [10] 0 0
From first dose (i.e., Day 1) up to 60 days after last dose date of study drug (i.e., up to Week 60)
Secondary outcome [11] 0 0
Number of Participants With Potentially Clinically Significant Abnormalities - Metabolic Parameters
Timepoint [11] 0 0
From first dose (i.e., Day 1) up to 60 days after last dose date of study drug (i.e., up to Week 60)
Secondary outcome [12] 0 0
Number of Participants With Potentially Clinically Significant Abnormalities - Renal Function
Timepoint [12] 0 0
From first dose (i.e., Day 1) up to 60 days after last dose date of study drug (i.e., up to Week 60)
Secondary outcome [13] 0 0
Number of Participants With Potentially Clinically Significant Abnormalities - Liver Function
Timepoint [13] 0 0
From first dose (i.e., Day 1) up to 60 days after last dose date of study drug (i.e., up to Week 60)
Secondary outcome [14] 0 0
Number of Participants With Treatment-emergent Antidrug Antibodies (ADA) Response
Timepoint [14] 0 0
From first dose (i.e., Day 1) up to 60 days after last dose date of study drug (i.e., up to Week 60)
Secondary outcome [15] 0 0
Pharmacokinetics (PK): Serum Trough Concentration (Ctrough) of Sarilumab
Timepoint [15] 0 0
Pre-dose on Week 0 (Baseline), Week 2, 4, 12, 16, 24, and 52
Secondary outcome [16] 0 0
Pharmacokinetics: Serum Drug Concentration of Sarilumab Post-dose at Week 24
Timepoint [16] 0 0
Post-dose at Week 24

Eligibility
Key inclusion criteria
Inclusion criteria :

* Diagnosis of PMR according to European League Against Rheumatism/American College of Rheumatology classification criteria.
* Participants must be on prednisone of at least 7.5 milligrams per day (mg/day) (or equivalent) and not exceeding 20 mg/day at screening and during the screening period.
* Participant was willing and able to take prednisone of 15 mg/day at randomization.
* Participants had a history of being treated for at least 8 weeks with prednisone (greater than or equal to [>=]10 mg/day or equivalent).
* Participants must have had at least one episode of unequivocal PMR flare while attempting to taper prednisone at a dose that was >= 7.5 mg/day (or equivalent) within the past 12 Weeks prior to screening:

* Unequivocal symptoms of PMR flare included shoulder and/or hip girdle pain associated with inflammatory stiffness.
* Participants had erythrocyte sedimentation rate >=30 millimeters per hour (mm/hr) and/or C-reactive protein >=10 milligrams per liter (mg/L) associated with PMR disease activity within 12 weeks prior to screening.
Minimum age
50 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
Exclusion criteria:

* Diagnosis of giant cell arteritis (e.g., persistent or recurrent localized headache, temporal artery or scalp tenderness, jaw claudication, extremity claudication, blurry or loss of vision, symptoms of stroke).
* Diagnosis of active fibromyalgia.
* Concurrent rheumatoid arthritis or other inflammatory arthritis or other connective tissue diseases, such as but not limited to systemic lupus erythematosus, systemic sclerosis, vasculitis, myositis, mixed connective tissue disease, and ankylosing spondylitis.
* Concurrent diagnosis of rhabdomyolysis or neuropathic muscular diseases.
* Inadequately treated hypothyroidism.
* Organ transplant recipient.
* Therapeutic failure including inadequate response or intolerance, or contraindication, to biological interleukin-6 antagonist.
* Any prior (within the defined period below) or concurrent use of immunosuppressive therapies but not limited to any of the following:

* Janus kinase inhibitor within 4 weeks of Baseline.
* Alkylating agents including cyclophosphamide within 6 months of Baseline.
* Cell-depletion agents (e.g., anti CD20) without evidence of recovery of B cells to Baseline level.
* Tumor necrosis factor inhibitors within 2-8 weeks (etanercept within 2 weeks, infliximab, certolizumab, golimumab, or adalimumab within 8 weeks), or after at least 5 half-lives have elapsed, whichever was longer.
* Abatacept within 8 weeks of Baseline.
* Anakinra within 1 week of Baseline.
* Cyclosporine, azathioprine or mycophenolate mofetil or leflunomide within 4 weeks of Baseline.
* Unstable methotrexate (MTX) dose and/or MTX dose greater than (>) 15 mg/week within 3 months of Baseline
* Concurrent use of systemic CS for conditions other than PMR.
* Pregnant or breastfeeding woman.
* Participants with active or untreated latent tuberculosis.
* Participants with history of invasive opportunistic infections.
* Participants with fever associated with infection or chronic, persistent or recurring infections required active treatment.
* Participants with uncontrolled diabetes mellitus.
* Participants with non-healed or healing skin ulcers.
* Participants who received any live, attenuated vaccine within 3 months of Baseline.
* Participants who were positive for hepatitis B, hepatitis C and/or human immunodeficiency virus.
* Participants with a history of active or recurrent herpes zoster.
* Participants with a history of or prior articular or prosthetic joint infection.
* Prior or current history of malignancy.
* Participants who have had surgery within 4 weeks of screening or planned surgery during study.
* Participants with a history of inflammatory bowel disease or severe diverticulitis or previous gastrointestinal perforation.

The above information was not intended to contain all considerations relevant to a participant's potential participation in a clinical trial.

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Stopped early
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
Investigational Site Number 0360003 - Camberwell
Recruitment hospital [2] 0 0
Investigational Site Number 0360001 - Kogarah
Recruitment hospital [3] 0 0
Investigational Site Number 0360002 - Maroochydore
Recruitment hospital [4] 0 0
Investigational Site Number 0360004 - Woodville South
Recruitment postcode(s) [1] 0 0
3124 - Camberwell
Recruitment postcode(s) [2] 0 0
2217 - Kogarah
Recruitment postcode(s) [3] 0 0
4558 - Maroochydore
Recruitment postcode(s) [4] 0 0
5011 - Woodville South
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
California
Country [2] 0 0
United States of America
State/province [2] 0 0
Colorado
Country [3] 0 0
United States of America
State/province [3] 0 0
Connecticut
Country [4] 0 0
United States of America
State/province [4] 0 0
Florida
Country [5] 0 0
United States of America
State/province [5] 0 0
Iowa
Country [6] 0 0
United States of America
State/province [6] 0 0
Massachusetts
Country [7] 0 0
United States of America
State/province [7] 0 0
New York
Country [8] 0 0
United States of America
State/province [8] 0 0
Texas
Country [9] 0 0
United States of America
State/province [9] 0 0
Washington
Country [10] 0 0
Argentina
State/province [10] 0 0
Buenos Aires
Country [11] 0 0
Argentina
State/province [11] 0 0
Caba
Country [12] 0 0
Argentina
State/province [12] 0 0
San Miguel de Tucuman
Country [13] 0 0
Belgium
State/province [13] 0 0
Gent
Country [14] 0 0
Belgium
State/province [14] 0 0
Leuven
Country [15] 0 0
Canada
State/province [15] 0 0
Hamilton
Country [16] 0 0
Canada
State/province [16] 0 0
Montreal
Country [17] 0 0
Canada
State/province [17] 0 0
Rimouski
Country [18] 0 0
Canada
State/province [18] 0 0
Sherbrooke
Country [19] 0 0
Canada
State/province [19] 0 0
Trois-Rivières
Country [20] 0 0
Estonia
State/province [20] 0 0
Tallinn
Country [21] 0 0
France
State/province [21] 0 0
Brest Cedex
Country [22] 0 0
France
State/province [22] 0 0
Caen Cedex 9
Country [23] 0 0
France
State/province [23] 0 0
Le Kremlin Bicetre
Country [24] 0 0
France
State/province [24] 0 0
Lille Cedex
Country [25] 0 0
France
State/province [25] 0 0
Montivilliers
Country [26] 0 0
France
State/province [26] 0 0
Montpellier
Country [27] 0 0
France
State/province [27] 0 0
Paris
Country [28] 0 0
France
State/province [28] 0 0
Pierre Benite Cedex
Country [29] 0 0
France
State/province [29] 0 0
Toulouse Cedex 09
Country [30] 0 0
Germany
State/province [30] 0 0
Bad Abbach
Country [31] 0 0
Germany
State/province [31] 0 0
Berlin
Country [32] 0 0
Germany
State/province [32] 0 0
Dresden
Country [33] 0 0
Germany
State/province [33] 0 0
Kirchheim Unter Teck
Country [34] 0 0
Germany
State/province [34] 0 0
München
Country [35] 0 0
Germany
State/province [35] 0 0
Tübingen
Country [36] 0 0
Hungary
State/province [36] 0 0
Debrecen
Country [37] 0 0
Israel
State/province [37] 0 0
Haifa
Country [38] 0 0
Israel
State/province [38] 0 0
Kfar Saba
Country [39] 0 0
Israel
State/province [39] 0 0
Petah-Tikva
Country [40] 0 0
Israel
State/province [40] 0 0
Tel Hashomer
Country [41] 0 0
Italy
State/province [41] 0 0
Milano
Country [42] 0 0
Italy
State/province [42] 0 0
Pisa
Country [43] 0 0
Italy
State/province [43] 0 0
Reggio Emilia
Country [44] 0 0
Italy
State/province [44] 0 0
Rozzano
Country [45] 0 0
Italy
State/province [45] 0 0
Verona
Country [46] 0 0
Japan
State/province [46] 0 0
Fuchu-Shi
Country [47] 0 0
Japan
State/province [47] 0 0
Kamakura-Shi
Country [48] 0 0
Japan
State/province [48] 0 0
Kawachinagano-Shi
Country [49] 0 0
Japan
State/province [49] 0 0
Takasaki-Shi
Country [50] 0 0
Netherlands
State/province [50] 0 0
Alkmaar
Country [51] 0 0
Netherlands
State/province [51] 0 0
Almelo
Country [52] 0 0
Netherlands
State/province [52] 0 0
Den Haag
Country [53] 0 0
Netherlands
State/province [53] 0 0
Leeuwarden
Country [54] 0 0
Netherlands
State/province [54] 0 0
Nijmegen
Country [55] 0 0
Netherlands
State/province [55] 0 0
Rotterdam
Country [56] 0 0
Russian Federation
State/province [56] 0 0
Moscow
Country [57] 0 0
Russian Federation
State/province [57] 0 0
Saint-Petersburg
Country [58] 0 0
Spain
State/province [58] 0 0
A Coruña / Santiago De Compostela
Country [59] 0 0
Spain
State/province [59] 0 0
Badalona
Country [60] 0 0
Spain
State/province [60] 0 0
Getafe
Country [61] 0 0
Spain
State/province [61] 0 0
Granada
Country [62] 0 0
Spain
State/province [62] 0 0
Madrid
Country [63] 0 0
Spain
State/province [63] 0 0
Santander
Country [64] 0 0
Spain
State/province [64] 0 0
Valencia
Country [65] 0 0
Switzerland
State/province [65] 0 0
Bern
Country [66] 0 0
Switzerland
State/province [66] 0 0
St. Gallen
Country [67] 0 0
United Kingdom
State/province [67] 0 0
Gateshead
Country [68] 0 0
United Kingdom
State/province [68] 0 0
Leeds
Country [69] 0 0
United Kingdom
State/province [69] 0 0
Manchester
Country [70] 0 0
United Kingdom
State/province [70] 0 0
Newport
Country [71] 0 0
United Kingdom
State/province [71] 0 0
Plymouth
Country [72] 0 0
United Kingdom
State/province [72] 0 0
Southend

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Sanofi
Address
Country
Other collaborator category [1] 0 0
Commercial sector/industry
Name [1] 0 0
Regeneron Pharmaceuticals
Address [1] 0 0
Country [1] 0 0

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Clinical Sciences & Operations
Address 0 0
Sanofi
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
Yes
What data in particular will be shared?
Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org
When will data be available (start and end dates)?
Available to whom?
Available for what types of analyses?
How or where can data be obtained?
IPD available at link: https://vivli.org


What supporting documents are/will be available?

Results publications and other study-related documents

No documents have been uploaded by study researchers.