NCT03827018

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).

Trial Health

98
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
70

participants targeted

Target at P50-P75 for phase_2

Timeline
Completed

Started Sep 2018

Geographic Reach
14 countries

47 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

September 20, 2018

Completed
4 months until next milestone

First Submitted

Initial submission to the registry

January 25, 2019

Completed
7 days until next milestone

First Posted

Study publicly available on registry

February 1, 2019

Completed
1.5 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

August 13, 2020

Completed
3 months until next milestone

Study Completion

Last participant's last visit for all outcomes

November 25, 2020

Completed
2.9 years until next milestone

Results Posted

Study results publicly available

October 17, 2023

Completed
Last Updated

October 23, 2023

Status Verified

October 1, 2023

Enrollment Period

1.9 years

First QC Date

January 25, 2019

Results QC Date

August 2, 2023

Last Update Submit

October 17, 2023

Conditions

Keywords

GCAtemporal arteritisHorton's disease

Outcome Measures

Primary Outcomes (1)

  • Time to Flare by Week 26

    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.

    Week 26

Secondary Outcomes (9)

  • Sustained Remission Rate at Week 26

    Week 26

  • Time to Elevated Erythrocyte Sedimentation Rate (ESR) by Week 26

    Week 26

  • Time to Elevated C-Reactive Protein (CRP) by Week 26

    Week 26

  • Time to Signs/Symptoms of Giant Cell Arteritis (GCA) or New or Worsening Vasculitis on Imaging by Week 26

    Week 26

  • Cumulative Corticosteroid Dose at Week 26

    Week 26

  • +4 more secondary outcomes

Study Arms (2)

mavrilimumab

ACTIVE COMPARATOR

Subjects randomized to mavrilimumab will receive 150 mg every other week by subcutaneous injection co-administered with a 26-week corticosteroid taper.

Combination Product: mavrilimumabDrug: prednisone

placebo

PLACEBO COMPARATOR

Subjects randomized to placebo will receive placebo every other week by subcutaneous injection co-administered with a 26-week corticosteroid taper.

Combination Product: placeboDrug: prednisone

Interventions

mavrilimumabCOMBINATION_PRODUCT

1 mL of 150 mg in a pre-filled syringe

Also known as: KPL-301
mavrilimumab
placeboCOMBINATION_PRODUCT

1 mL of placebo in a pre-filled syringe

placebo

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)

mavrilimumabplacebo

Eligibility Criteria

Age50 Years - 85 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • Subjects with new-onset or relapsing/refractory GCA.
  • Westergren erythrocyte sedimentation rate \> 30 mm/hour or c-reactive protein ≥ 1 mg/ dL.
  • Remission of GCA at or before Day 0.
  • 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.
  • 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.

You may not qualify if:

  • Transplanted organs (except corneal transplant performed more than 3 months prior to randomization).
  • Concurrent enrollment in another interventional clinical study.
  • Treatment with non-biologic investigational drug therapy within 4 weeks or 5 half-lives of the study agent, whichever was longer, prior to screening.
  • 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.
  • Treatment with alkylating agents within 12 weeks prior to screening.
  • Intramuscular, Intra-articular or IV corticosteroids within 4 weeks prior to screening.
  • Receipt of live (attenuated) vaccine within the 4 weeks before Day 0.
  • Treatment with hydroxychloroquine, cyclosporine A, azathioprine, cyclophosphamide, or mycophenolate mofetil (MMF) within 4 weeks of screening.
  • Female subjects who are pregnant, intending to become pregnant, or are breastfeeding.
  • 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.
  • Positive (or 2 indeterminate) QuantiFERON test results.
  • Clinically significant active infection or infection requiring hospitalization or IV antibiotics within 12 weeks before screening or opportunistic infection within 6 months before screening.
  • Chronic active hepatitis B infection.
  • 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.
  • 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.
  • +2 more criteria

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (49)

Site 1703

Sarasota, Florida, 34239, United States

Location

Site 1708

Tampa, Florida, 33612, United States

Location

Site 1706

Atlanta, Georgia, 30342, United States

Location

Site 1701

Boston, Massachusetts, 02114, United States

Location

Site 1707

Lansing, Michigan, 48910, United States

Location

Site 1704

Saint Clair Shores, Michigan, 48081, United States

Location

Site 1702

Rochester, Minnesota, 55905-0001, United States

Location

Site 1705

New York, New York, 10021, United States

Location

Site 2102

Kogarah, 2217, Australia

Location

Site 2105

Nedlands, 6009, Australia

Location

Site 2106

Parkville, 3050, Australia

Location

Site 2101

Victoria Park, 6100, Australia

Location

Site 2104

Woodville South, 5011, Australia

Location

Site 2204

Brussels, 1070, Belgium

Location

Site 2202

Leuven, 3000, Belgium

Location

Site 2201

Liège, 4000, Belgium

Location

Site 2203

Yvoir, 5530, Belgium

Location

Site 2303

Zagreb, 10 000, Croatia

Location

Site 2401

Tallinn, 11312, Estonia

Location

Site 2402

Tartu, 50708, Estonia

Location

Site 2502

Tübingen, Baden-Wurttemberg, 72076, Germany

Location

Site 2504

Erlangen, Bavaria, 91054, Germany

Location

Site 2507

Freiburg im Breisgau, 79106, Germany

Location

Site 2506

Hamburg, 22763, Germany

Location

Site 2503

Hanover, 30625, Germany

Location

Site 2508

Jena, 07747, Germany

Location

Site 2501

Kirchheim unter Teck, 73230, Germany

Location

Site 2601

Dublin, D04 T6F4, Ireland

Location

Site 2703

Milan, 20132, Italy

Location

Site 2701

Pieve Emanuele, 20090, Italy

Location

Site 2702

Reggio Emilia, 42100, Italy

Location

Site 2704

Udine, 33100, Italy

Location

Site 2802

Groningen, 9713 GZ, Netherlands

Location

Site 2801

Rotterdam, 3015 GD, Netherlands

Location

Site 2902

Christchurch, 8083, New Zealand

Location

Site 2901

Wellington, 6021, New Zealand

Location

Site 1002

Krakow, 31-121, Poland

Location

Site 1101

Belgrade, 11000, Serbia

Location

Site 1102

Belgrade, 11000, Serbia

Location

Site 1103

Belgrade, 11000, Serbia

Location

Site 1201

Ljubljana, 1000, Slovenia

Location

Site 1303

A Coruña, 15006, Spain

Location

Site 1301

Barcelona, 08036, Spain

Location

Site 1304

Bilbao, 48013, Spain

Location

Site 1302

Santa Cruz de Tenerife, 38320, Spain

Location

Site 1604

Edinburgh, EH4 2XU, United Kingdom

Location

Site 1603

Essex, SS0 0RY, United Kingdom

Location

Site 1602

London, E11 1NR, United Kingdom

Location

Site 1601

Newcastle upon Tyne, NE7 7DN, United Kingdom

Location

Related Publications (1)

  • Cid MC, Unizony SH, Blockmans D, Brouwer E, Dagna L, Dasgupta B, Hellmich B, Molloy E, Salvarani C, Trapnell BC, Warrington KJ, Wicks I, Samant M, Zhou T, Pupim L, Paolini JF; KPL-301-C001 Investigators. Efficacy and safety of mavrilimumab in giant cell arteritis: a phase 2, randomised, double-blind, placebo-controlled trial. Ann Rheum Dis. 2022 May;81(5):653-661. doi: 10.1136/annrheumdis-2021-221865. Epub 2022 Mar 9.

MeSH Terms

Conditions

Giant Cell Arteritis

Interventions

mavrilimumabPrednisone

Condition Hierarchy (Ancestors)

Vasculitis, Central Nervous SystemAutoimmune Diseases of the Nervous SystemNervous System DiseasesCerebrovascular DisordersBrain DiseasesCentral Nervous System DiseasesVascular DiseasesCardiovascular DiseasesArteritisVasculitisSkin Diseases, VascularSkin DiseasesSkin and Connective Tissue DiseasesAutoimmune DiseasesImmune System Diseases

Intervention Hierarchy (Ancestors)

PregnadienediolsPregnadienesPregnanesSteroidsFused-Ring CompoundsPolycyclic Compounds

Results Point of Contact

Title
Clinical Operations Study Director
Organization
Kiniksa Pharmaceuticals (UK), Ltd. c/o Kiniksa Pharmaceuticals Corp.

Study Officials

  • John Paolini, M.D.

    Kiniksa Pharmaceuticals, Ltd.

    STUDY DIRECTOR

Publication Agreements

PI is Sponsor Employee
No
Restriction Type
OTHER
Restrictive Agreement
Yes

Study Design

Study Type
interventional
Phase
phase 2
Allocation
RANDOMIZED
Masking
QUADRUPLE
Who Masked
PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
Purpose
TREATMENT
Intervention Model
PARALLEL
Model Details: Upon successful completion of the screening procedures, diagnosis criteria will be entered into an interactive web response system, and eligible subjects will be stratified for randomized study treatment into two cohorts according to whether subjects have new-onset or relapsing/refractory disease.
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

January 25, 2019

First Posted

February 1, 2019

Study Start

September 20, 2018

Primary Completion

August 13, 2020

Study Completion

November 25, 2020

Last Updated

October 23, 2023

Results First Posted

October 17, 2023

Record last verified: 2023-10

Data Sharing

IPD Sharing
Will not share

Locations