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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT03827018




Registration number
NCT03827018
Ethics application status
Date submitted
25/01/2019
Date registered
1/02/2019

Titles & IDs
Public title
KPL-301 for Subjects With Giant Cell Arteritis
Scientific title
A Phase 2, Randomized, Double-blind Placebo-controlled Study to Test the Efficacy and Safety of KPL-301 in Giant Cell Arteritis
Secondary ID [1] 0 0
KPL-301-C001
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Giant Cell Arteritis 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
Other interventions - mavrilimumab
Other interventions - 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.


Other interventions: mavrilimumab
1 mL of 150 mg in a pre-filled syringe

Other interventions: 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)
Intervention code [1] 0 0
Other interventions
Intervention code [2] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Time to Flare by Week 26
Timepoint [1] 0 0
Week 26
Secondary outcome [1] 0 0
Sustained Remission Rate at Week 26
Timepoint [1] 0 0
Week 26
Secondary outcome [2] 0 0
Time to Elevated Erythrocyte Sedimentation Rate (ESR) by Week 26
Timepoint [2] 0 0
Week 26
Secondary outcome [3] 0 0
Time to Elevated C-Reactive Protein (CRP) by Week 26
Timepoint [3] 0 0
Week 26
Secondary outcome [4] 0 0
Time to Signs/Symptoms of Giant Cell Arteritis (GCA) or New or Worsening Vasculitis on Imaging by Week 26
Timepoint [4] 0 0
Week 26
Secondary outcome [5] 0 0
Cumulative Corticosteroid Dose at Week 26
Timepoint [5] 0 0
Week 26
Secondary outcome [6] 0 0
Percentage of Participants Who Completed the 26-Week Corticosteroid Taper and Who Had a Normal ESR
Timepoint [6] 0 0
Week 26
Secondary outcome [7] 0 0
Percentage of Participants Who Completed the 26-Week Corticosteroid Taper and Who Had a Normal CRP Level
Timepoint [7] 0 0
Week 26
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
Timepoint [8] 0 0
Week 26
Secondary outcome [9] 0 0
Cumulative Corticosteroid Dose at the End of the Washout Safety Follow-up Period
Timepoint [9] 0 0
Final Safety Follow-up visit (Week 38)

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
Minimum age
50 Years
Maximum age
85 Years
Sex
Both males and females
Can healthy volunteers participate?
No
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.

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 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
20/09/2018
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
25/11/2020
Sample size
Target
Accrual to date
Final
70
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
Site 2102 - Kogarah
Recruitment hospital [2] 0 0
Site 2105 - Nedlands
Recruitment hospital [3] 0 0
Site 2106 - Parkville
Recruitment hospital [4] 0 0
Site 2101 - Victoria Park
Recruitment hospital [5] 0 0
Site 2104 - Woodville South
Recruitment postcode(s) [1] 0 0
2217 - Kogarah
Recruitment postcode(s) [2] 0 0
6009 - Nedlands
Recruitment postcode(s) [3] 0 0
3050 - Parkville
Recruitment postcode(s) [4] 0 0
6100 - Victoria Park
Recruitment postcode(s) [5] 0 0
5011 - Woodville South
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Florida
Country [2] 0 0
United States of America
State/province [2] 0 0
Georgia
Country [3] 0 0
United States of America
State/province [3] 0 0
Massachusetts
Country [4] 0 0
United States of America
State/province [4] 0 0
Michigan
Country [5] 0 0
United States of America
State/province [5] 0 0
Minnesota
Country [6] 0 0
United States of America
State/province [6] 0 0
New York
Country [7] 0 0
Belgium
State/province [7] 0 0
Brussels
Country [8] 0 0
Belgium
State/province [8] 0 0
Leuven
Country [9] 0 0
Belgium
State/province [9] 0 0
Liège
Country [10] 0 0
Belgium
State/province [10] 0 0
Yvoir
Country [11] 0 0
Croatia
State/province [11] 0 0
Zagreb
Country [12] 0 0
Estonia
State/province [12] 0 0
Tallinn
Country [13] 0 0
Estonia
State/province [13] 0 0
Tartu
Country [14] 0 0
Germany
State/province [14] 0 0
Baden-Württemberg
Country [15] 0 0
Germany
State/province [15] 0 0
Bayern
Country [16] 0 0
Germany
State/province [16] 0 0
Freiburg im Breisgau
Country [17] 0 0
Germany
State/province [17] 0 0
Hamburg
Country [18] 0 0
Germany
State/province [18] 0 0
Hannover
Country [19] 0 0
Germany
State/province [19] 0 0
Jena
Country [20] 0 0
Germany
State/province [20] 0 0
Kirchheim Unter Teck
Country [21] 0 0
Ireland
State/province [21] 0 0
Dublin
Country [22] 0 0
Italy
State/province [22] 0 0
Milano
Country [23] 0 0
Italy
State/province [23] 0 0
Pieve Emanuele
Country [24] 0 0
Italy
State/province [24] 0 0
Reggio Emilia
Country [25] 0 0
Italy
State/province [25] 0 0
Udine
Country [26] 0 0
Netherlands
State/province [26] 0 0
Groningen
Country [27] 0 0
Netherlands
State/province [27] 0 0
Rotterdam
Country [28] 0 0
New Zealand
State/province [28] 0 0
Christchurch
Country [29] 0 0
New Zealand
State/province [29] 0 0
Wellington
Country [30] 0 0
Poland
State/province [30] 0 0
Kraków
Country [31] 0 0
Serbia
State/province [31] 0 0
Belgrade
Country [32] 0 0
Slovenia
State/province [32] 0 0
Ljubljana
Country [33] 0 0
Spain
State/province [33] 0 0
A Coruña
Country [34] 0 0
Spain
State/province [34] 0 0
Barcelona
Country [35] 0 0
Spain
State/province [35] 0 0
Bilbao
Country [36] 0 0
Spain
State/province [36] 0 0
Santa Cruz De Tenerife
Country [37] 0 0
United Kingdom
State/province [37] 0 0
Edinburgh
Country [38] 0 0
United Kingdom
State/province [38] 0 0
Essex
Country [39] 0 0
United Kingdom
State/province [39] 0 0
London
Country [40] 0 0
United Kingdom
State/province [40] 0 0
Newcastle Upon Tyne

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Kiniksa Pharmaceuticals, Ltd.
Address
Country

Ethics approval
Ethics application status

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

Contacts
Principal investigator
Name 0 0
John Paolini, M.D.
Address 0 0
Kiniksa Pharmaceuticals, Ltd.
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)?
No
No/undecided IPD sharing reason/comment


What supporting documents are/will be available?




Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results are available at https://clinicaltrials.gov/study/NCT03827018