Please note that the copy function is not enabled for this field.
If you wish to
modify
existing outcomes, please copy and paste the current outcome text into the Update field.
LOGIN
CREATE ACCOUNT
LOGIN
CREATE ACCOUNT
MY TRIALS
REGISTER TRIAL
FAQs
HINTS AND TIPS
DEFINITIONS
Trial Review
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 endorsed by the ANZCTR. Before participating in a study, talk to your health care provider and refer to this
information for consumers
Download to PDF
Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/ct2/show/NCT03827018
Registration number
NCT03827018
Ethics application status
Date submitted
25/01/2019
Date registered
1/02/2019
Date last updated
23/10/2023
Titles & IDs
Public title
KPL-301 for Subjects With Giant Cell Arteritis
Query!
Scientific title
A Phase 2, Randomized, Double-blind Placebo-controlled Study to Test the Efficacy and Safety of KPL-301 in Giant Cell Arteritis
Query!
Secondary ID [1]
0
0
KPL-301-C001
Query!
Universal Trial Number (UTN)
Query!
Trial acronym
Query!
Linked study record
Query!
Health condition
Health condition(s) or problem(s) studied:
Giant Cell Arteritis
0
0
Query!
Condition category
Condition code
Musculoskeletal
0
0
0
0
Query!
Other muscular and skeletal disorders
Query!
Inflammatory and Immune System
0
0
0
0
Query!
Other inflammatory or immune system disorders
Query!
Cardiovascular
0
0
0
0
Query!
Diseases of the vasculature and circulation including the lymphatic system
Query!
Neurological
0
0
0
0
Query!
Other neurological disorders
Query!
Inflammatory and Immune System
0
0
0
0
Query!
Autoimmune diseases
Query!
Intervention/exposure
Study type
Interventional
Query!
Description of intervention(s) / exposure
Combination Product - mavrilimumab
Combination Product - placebo
Treatment: Drugs - prednisone
Active Comparator: mavrilimumab - Subjects randomized to mavrilimumab will receive 150 mg every other week by subcutaneous injection co-administered with a 26-week corticosteroid taper.
Placebo Comparator: placebo - Subjects randomized to placebo will receive placebo every other week by subcutaneous injection co-administered with a 26-week corticosteroid taper.
Combination Product: mavrilimumab
1 mL of 150 mg in a pre-filled syringe
Combination Product: placebo
1 mL of placebo in a pre-filled syringe
Treatment: Drugs: prednisone
Prednisone tablets for oral administration containing either 1 mg, 2.5 mg, 5 mg, 10 mg, 20 mg or 50 mg of prednisone United States Pharmacopeia (USP)
Query!
Intervention code [1]
0
0
Combination Product
Query!
Intervention code [2]
0
0
Treatment: Drugs
Query!
Comparator / control treatment
Query!
Control group
Query!
Outcomes
Primary outcome [1]
0
0
Time to Flare by Week 26
Query!
Assessment method [1]
0
0
Time to flare by Week 26 was defined as time from randomization to the date of first flare occurring within the 26-week period, as assessed by independent adjudication. Kaplan-Meier method used to estimate the survival functions for each treatment arm.
Flare/relapse was defined as a C-reactive protein (CRP) of 1 mg/dL or greater and/or erythrocyte sedimentation rate (ESR) of 30 mm/h or greater AND at least one of the following signs or symptoms attributed to GCA: Cranial symptoms (new-onset localized headache; scalp or temporal artery tenderness; ischemic-related vision loss; unexplained mouth or jaw pain upon mastication; transient ischemic attack or stroke related to GCA); Extracranial symptoms (claudication of the extremities; symptoms of polymyalgia rheumatica); New or worsening angiographic abnormalities detected via MRI, CT/CTA, or PET-CT of the aorta or other great vessels or via ultrasound of the temporal arteries.
Query!
Timepoint [1]
0
0
Week 26
Query!
Secondary outcome [1]
0
0
Sustained Remission Rate at Week 26
Query!
Assessment method [1]
0
0
The sustained remission rate at Week 26 is defined as the percentage of participants with sustained remission, as assessed by independent adjudication, at Week 26, derived from the time to flare curve. Kaplan-Meier Survival Estimates with standard error and 95% CI for each arm. Participants who completed the treatment period without a flare by Week 26 were considered to have sustained remission.
Query!
Timepoint [1]
0
0
Week 26
Query!
Secondary outcome [2]
0
0
Time to Elevated Erythrocyte Sedimentation Rate (ESR) by Week 26
Query!
Assessment method [2]
0
0
Elevated ESR is defined as first occurrence of ESR value = 30 mm/hr. Participants with elevated ESR within 3 days of first dose are excluded from the analysis. Kaplan-Meier method used to estimate the survival functions for each treatment arm.
Query!
Timepoint [2]
0
0
Week 26
Query!
Secondary outcome [3]
0
0
Time to Elevated C-Reactive Protein (CRP) by Week 26
Query!
Assessment method [3]
0
0
Elevated CRP is defined as first occurrence of CRP value = 1.0 mg/dL. Participants with elevated CRP within 3 days of first dose are excluded from the analysis. Kaplan-Meier method used to estimate the survival functions for each treatment arm.
Query!
Timepoint [3]
0
0
Week 26
Query!
Secondary outcome [4]
0
0
Time to Signs/Symptoms of Giant Cell Arteritis (GCA) or New or Worsening Vasculitis on Imaging by Week 26
Query!
Assessment method [4]
0
0
Kaplan-Meier method used to estimate the survival functions for each treatment arm.
Query!
Timepoint [4]
0
0
Week 26
Query!
Secondary outcome [5]
0
0
Cumulative Corticosteroid Dose at Week 26
Query!
Assessment method [5]
0
0
Query!
Timepoint [5]
0
0
Week 26
Query!
Secondary outcome [6]
0
0
Percentage of Participants Who Completed the 26-Week Corticosteroid Taper and Who Had a Normal ESR
Query!
Assessment method [6]
0
0
Participants were considered to have completed the corticosteroid taper if by week 26 receiving 1 mg/day for those who start with 60 mg/day, or 0 mg/day for those who start with doses < 60 mg/day. 95% CI calculated using Clopper-Pearson confidence intervals.
Query!
Timepoint [6]
0
0
Week 26
Query!
Secondary outcome [7]
0
0
Percentage of Participants Who Completed the 26-Week Corticosteroid Taper and Who Had a Normal CRP Level
Query!
Assessment method [7]
0
0
Participants were considered to have completed the corticosteroid taper if by week 26 receiving 1 mg/day for those who start with 60 mg/day, or 0 mg/day for those who start with doses < 60 mg/day. 95% CI calculated using Clopper-Pearson confidence intervals.
Query!
Timepoint [7]
0
0
Week 26
Query!
Secondary outcome [8]
0
0
Percentage of Participants Who Completed the 26-week Corticosteroid Taper and Who Had No Signs or Symptoms of GCA Nor New or Worsening Vasculitis by Imaging by Week 26
Query!
Assessment method [8]
0
0
Participants were considered to have completed the corticosteroid taper if by week 26 receiving 1 mg/day for those who start with 60 mg/day, or 0 mg/day for those who start with doses < 60 mg/day. 95% CI calculated using Clopper-Pearson confidence intervals.
Query!
Timepoint [8]
0
0
Week 26
Query!
Secondary outcome [9]
0
0
Cumulative Corticosteroid Dose at the End of the Washout Safety Follow-up Period
Query!
Assessment method [9]
0
0
Query!
Timepoint [9]
0
0
Final Safety Follow-up visit (Week 38)
Query!
Eligibility
Key inclusion criteria
Selected
1. Subjects with new-onset or relapsing/refractory GCA.
2. Westergren erythrocyte sedimentation rate > 30 mm/hour or c-reactive protein = 1 mg/
dL.
3. Remission of GCA at or before Day 0.
4. Female subjects must be postmenopausal or permanently sterile following documented
hysterectomy, bilateral salpingectomy, bilateral oophorectomy, or tubal ligation or
having a male partner with vasectomy as affirmed by the subject, or nonpregnant,
nonlactating, and if sexually active having agreed to use a highly effective method of
contraception.
5. Male subjects must have documented vasectomy or if sexually active must agree to use a
highly effective method of contraception with their partners of childbearing
potential.
Selected
Query!
Minimum age
50
Years
Query!
Query!
Maximum age
85
Years
Query!
Query!
Sex
Both males and females
Query!
Can healthy volunteers participate?
No
Query!
Key exclusion criteria
1. Transplanted organs (except corneal transplant performed more than 3 months prior to
randomization).
2. Concurrent enrollment in another interventional clinical study.
3. Treatment with non-biologic investigational drug therapy within 4 weeks or 5
half-lives of the study agent, whichever was longer, prior to screening.
4. Cell-depleting biological therapies within 12 months prior to Day 0, or
noncell-depleting biological therapies within 8 weeks (or 5 half-lives, whichever is
longer) prior to screening.
5. Treatment with alkylating agents within 12 weeks prior to screening.
6. Intramuscular, Intra-articular or IV corticosteroids within 4 weeks prior to
screening.
7. Receipt of live (attenuated) vaccine within the 4 weeks before Day 0.
8. Treatment with hydroxychloroquine, cyclosporine A, azathioprine, cyclophosphamide, or
mycophenolate mofetil (MMF) within 4 weeks of screening.
9. Female subjects who are pregnant, intending to become pregnant, or are breastfeeding.
10. Known history of allergy or reaction to any component of the mavrilimumab or placebo
formulation or to any other biologic therapy or prednisone or any of its excipients.
11. Positive (or 2 indeterminate) QuantiFERON test results.
12. Clinically significant active infection or infection requiring hospitalization or IV
antibiotics within 12 weeks before screening or opportunistic infection within 6
months before screening.
13. Chronic active hepatitis B infection.
14. Subjects at a high risk of infection, a history of an infected joint prosthesis still
in situ, leg ulcers, indwelling urinary catheter, or persistent or recurrent chest
infections.
15. History of cancer within the last 10 years, except for basal and squamous cell
carcinoma of the skin or in situ carcinoma of the cervix treated and considered cured.
16. Evidence of clinically-uncontrolled respiratory disease.
17. History of chronic respiratory tract infections.
Query!
Study design
Purpose of the study
Treatment
Query!
Allocation to intervention
Randomised controlled trial
Query!
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Query!
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Query!
Masking / blinding
Blinded (masking used)
Query!
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
Query!
Query!
Query!
Query!
Intervention assignment
Parallel
Query!
Other design features
Query!
Phase
Phase 2
Query!
Type of endpoint/s
Query!
Statistical methods / analysis
Query!
Recruitment
Recruitment status
Completed
Query!
Data analysis
Query!
Reason for early stopping/withdrawal
Query!
Other reasons
Query!
Date of first participant enrolment
Anticipated
Query!
Actual
20/09/2018
Query!
Date of last participant enrolment
Anticipated
Query!
Actual
Query!
Date of last data collection
Anticipated
Query!
Actual
25/11/2020
Query!
Sample size
Target
Query!
Accrual to date
Query!
Final
70
Query!
Recruitment in Australia
Recruitment state(s)
Query!
Recruitment hospital [1]
0
0
Site 2102 - Kogarah
Query!
Recruitment hospital [2]
0
0
Site 2105 - Nedlands
Query!
Recruitment hospital [3]
0
0
Site 2106 - Parkville
Query!
Recruitment hospital [4]
0
0
Site 2101 - Victoria Park
Query!
Recruitment hospital [5]
0
0
Site 2104 - Woodville South
Query!
Recruitment postcode(s) [1]
0
0
2217 - Kogarah
Query!
Recruitment postcode(s) [2]
0
0
6009 - Nedlands
Query!
Recruitment postcode(s) [3]
0
0
3050 - Parkville
Query!
Recruitment postcode(s) [4]
0
0
6100 - Victoria Park
Query!
Recruitment postcode(s) [5]
0
0
5011 - Woodville South
Query!
Recruitment outside Australia
Country [1]
0
0
United States of America
Query!
State/province [1]
0
0
Florida
Query!
Country [2]
0
0
United States of America
Query!
State/province [2]
0
0
Georgia
Query!
Country [3]
0
0
United States of America
Query!
State/province [3]
0
0
Massachusetts
Query!
Country [4]
0
0
United States of America
Query!
State/province [4]
0
0
Michigan
Query!
Country [5]
0
0
United States of America
Query!
State/province [5]
0
0
Minnesota
Query!
Country [6]
0
0
United States of America
Query!
State/province [6]
0
0
New York
Query!
Country [7]
0
0
Belgium
Query!
State/province [7]
0
0
Brussels
Query!
Country [8]
0
0
Belgium
Query!
State/province [8]
0
0
Leuven
Query!
Country [9]
0
0
Belgium
Query!
State/province [9]
0
0
Liège
Query!
Country [10]
0
0
Belgium
Query!
State/province [10]
0
0
Yvoir
Query!
Country [11]
0
0
Croatia
Query!
State/province [11]
0
0
Zagreb
Query!
Country [12]
0
0
Estonia
Query!
State/province [12]
0
0
Tallinn
Query!
Country [13]
0
0
Estonia
Query!
State/province [13]
0
0
Tartu
Query!
Country [14]
0
0
Germany
Query!
State/province [14]
0
0
Baden-Württemberg
Query!
Country [15]
0
0
Germany
Query!
State/province [15]
0
0
Bayern
Query!
Country [16]
0
0
Germany
Query!
State/province [16]
0
0
Freiburg im Breisgau
Query!
Country [17]
0
0
Germany
Query!
State/province [17]
0
0
Hamburg
Query!
Country [18]
0
0
Germany
Query!
State/province [18]
0
0
Hannover
Query!
Country [19]
0
0
Germany
Query!
State/province [19]
0
0
Jena
Query!
Country [20]
0
0
Germany
Query!
State/province [20]
0
0
Kirchheim Unter Teck
Query!
Country [21]
0
0
Ireland
Query!
State/province [21]
0
0
Dublin
Query!
Country [22]
0
0
Italy
Query!
State/province [22]
0
0
Milano
Query!
Country [23]
0
0
Italy
Query!
State/province [23]
0
0
Pieve Emanuele
Query!
Country [24]
0
0
Italy
Query!
State/province [24]
0
0
Reggio Emilia
Query!
Country [25]
0
0
Italy
Query!
State/province [25]
0
0
Udine
Query!
Country [26]
0
0
Netherlands
Query!
State/province [26]
0
0
Groningen
Query!
Country [27]
0
0
Netherlands
Query!
State/province [27]
0
0
Rotterdam
Query!
Country [28]
0
0
New Zealand
Query!
State/province [28]
0
0
Christchurch
Query!
Country [29]
0
0
New Zealand
Query!
State/province [29]
0
0
Wellington
Query!
Country [30]
0
0
Poland
Query!
State/province [30]
0
0
Kraków
Query!
Country [31]
0
0
Serbia
Query!
State/province [31]
0
0
Belgrade
Query!
Country [32]
0
0
Slovenia
Query!
State/province [32]
0
0
Ljubljana
Query!
Country [33]
0
0
Spain
Query!
State/province [33]
0
0
A Coruña
Query!
Country [34]
0
0
Spain
Query!
State/province [34]
0
0
Barcelona
Query!
Country [35]
0
0
Spain
Query!
State/province [35]
0
0
Bilbao
Query!
Country [36]
0
0
Spain
Query!
State/province [36]
0
0
Santa Cruz De Tenerife
Query!
Country [37]
0
0
United Kingdom
Query!
State/province [37]
0
0
Edinburgh
Query!
Country [38]
0
0
United Kingdom
Query!
State/province [38]
0
0
Essex
Query!
Country [39]
0
0
United Kingdom
Query!
State/province [39]
0
0
London
Query!
Country [40]
0
0
United Kingdom
Query!
State/province [40]
0
0
Newcastle Upon Tyne
Query!
Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Query!
Name
Kiniksa Pharmaceuticals, Ltd.
Query!
Address
Query!
Country
Query!
Ethics approval
Ethics application status
Query!
Summary
Brief summary
The primary objective of the study is to evaluate the efficacy of mavrilimumab (KPL-301)
versus placebo, co-administered with a 26-week corticosteroid taper, for maintaining
sustained remission for 26 weeks in subjects with new onset or relapsing/refractory giant
cell arteritis (GCA).
Query!
Trial website
https://clinicaltrials.gov/ct2/show/NCT03827018
Query!
Trial related presentations / publications
Query!
Public notes
Query!
Contacts
Principal investigator
Name
0
0
John Paolini, M.D.
Query!
Address
0
0
Kiniksa Pharmaceuticals, Ltd.
Query!
Country
0
0
Query!
Phone
0
0
Query!
Fax
0
0
Query!
Email
0
0
Query!
Contact person for public queries
Name
0
0
Query!
Address
0
0
Query!
Country
0
0
Query!
Phone
0
0
Query!
Fax
0
0
Query!
Email
0
0
Query!
Contact person for scientific queries
Summary Results
For IPD and results data, please see
https://clinicaltrials.gov/ct2/show/NCT03827018
Download to PDF