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Giant Cell ArteritisPhase 2NCT03827018

KPL-301 for Subjects With Giant Cell Arteritis

A Phase 2 study testing mavrilimumab and prednisone in people with Giant Cell Arteritis, run by Kiniksa Pharmaceuticals, Ltd. at 49 sites in 15 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
Search all trials

Trial 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). Read more about this study

Who is it for?
Ages 50 to 85 years
Conditions the study is for
Giant Cell Arteritis
What is being tested?
mavrilimumab and prednisone
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
70 participants
Where
49 sitesin 15 countries
Study dates
20 Sep 2018to 25 Nov 2020
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 to 85 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Selected Inclusion Criteria:

  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 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.

Trial locations and contacts

49 sites in 15 countries.

49 sites in 15 countries

United States8 sites

  • Site 1706

    Atlanta, Georgia, 30342

  • Site 1701

    Boston, Massachusetts, 02114

  • Site 1707

    Lansing, Michigan, 48910

  • Site 1705

    New York, New York, 10021

  • Site 1702

    Rochester, Minnesota, 55905-0001

  • Site 1704

    Saint Clair Shores, Michigan, 48081

  • Site 1703

    Sarasota, Florida, 34239

  • Site 1708

    Tampa, Florida, 33612

Other trials in United States

Germany7 sites

  • Site 2504

    Erlangen, Bavaria, 91054

  • Site 2507

    Freiburg im Breisgau, 79106

  • Site 2506

    Hamburg, 22763

  • Site 2503

    Hanover, 30625

  • Site 2508

    Jena, 07747

  • Site 2501

    Kirchheim unter Teck, 73230

  • Site 2502

    Tübingen, Baden-Wurttemberg, 72076

Other trials in Germany

Australia5 sites

  • Site 2102

    Kogarah, 2217

  • Site 2105

    Nedlands, 6009

  • Site 2106

    Parkville, 3050

  • Site 2101

    Victoria Park, 6100

  • Site 2104

    Woodville South, 5011

Other trials in Australia

Belgium4 sites

  • Site 2204

    Brussels, 1070

  • Site 2202

    Leuven, 3000

  • Site 2201

    Liège, 4000

  • Site 2203

    Yvoir, 5530

Other trials in Belgium

Italy4 sites

  • Site 2703

    Milan, 20132

  • Site 2701

    Pieve Emanuele, 20090

  • Site 2702

    Reggio Emilia, 42100

  • Site 2704

    Udine, 33100

Other trials in Italy

Spain4 sites

  • Site 1303

    A Coruña, 15006

  • Site 1301

    Barcelona, 08036

  • Site 1304

    Bilbao, 48013

  • Site 1302

    Santa Cruz de Tenerife, 38320

Other trials in Spain

United Kingdom4 sites

  • Site 1604

    Edinburgh, EH4 2XU

  • Site 1603

    Essex, SS0 0RY

  • Site 1602

    London, E11 1NR

  • Site 1601

    Newcastle upon Tyne, NE7 7DN

Other trials in United Kingdom

Serbia3 sites

  • Site 1101

    Belgrade, 11000

  • Site 1102

    Belgrade, 11000

  • Site 1103

    Belgrade, 11000

Other trials in Serbia

Estonia2 sites

  • Site 2401

    Tallinn, 11312

  • Site 2402

    Tartu, 50708

Other trials in Estonia

Netherlands2 sites

  • Site 2802

    Groningen, 9713 GZ

  • Site 2801

    Rotterdam, 3015 GD

Other trials in Netherlands

New Zealand2 sites

  • Site 2902

    Christchurch, 8083

  • Site 2901

    Wellington, 6021

Other trials in New Zealand

Croatia1 site

  • Site 2303

    Zagreb, 10 000

Other trials in Croatia

Ireland1 site

  • Site 2601

    Dublin, D04 T6F4

Other trials in Ireland

Poland1 site

  • Site 1002

    Krakow, 31-121

Other trials in Poland

Slovenia1 site

  • Site 1201

    Ljubljana, 1000

Other trials in Slovenia

About this study

Official title: A Phase 2, Randomized, Double-blind Placebo-controlled Study to Test the Efficacy and Safety of KPL-301 in Giant Cell Arteritis

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).

Read the detailed description

This Phase 2 randomized, double-blind, placebo-controlled proof of concept study will evaluate the efficacy and safety of mavrilimumab co-administered with a 26-week corticosteroid taper in subjects with GCA. The study will consist of a screening period (up to 6 weeks), a 26-week double-blind placebo-controlled period during which subjects will receive blinded mavrilimumab or placebo co-administered with a 26-week corticosteroid taper, and a 12-week washout safety follow-up period during which subjects will discontinue and wash off blinded mavrilimumab or placebo.

Being tested
mavrilimumab, placebo, prednisone
Keywords
GCA, temporal arteritis, Horton's disease
Medical subject headings
Giant Cell Arteritis

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • mavrilimumabCombination product

    Also called KPL-301

    1 mL of 150 mg in a pre-filled syringe

  • placeboCombination product

    1 mL of placebo in a pre-filled syringe

  • prednisoneDrug

    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)

Study groups in NCT03827018
Study groupRoleWhat they receive
mavrilimumabActive comparatorSubjects randomized to mavrilimumab will receive 150 mg every other week by subcutaneous injection co-administered with a 26-week corticosteroid taper.
Receives: Combination Product: mavrilimumab; Drug: prednisone
placeboPlacebo comparatorSubjects randomized to placebo will receive placebo every other week by subcutaneous injection co-administered with a 26-week corticosteroid taper.
Receives: Combination Product: placebo; Drug: prednisone

What is being measured

The primary outcome is the main question the study is designed to answer.

  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.

    Measured: Week 26

Secondary outcomes (9)

  1. Sustained Remission Rate at Week 26

    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.

    Measured: Week 26

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

    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.

    Measured: Week 26

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

    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.

    Measured: Week 26

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

    Kaplan-Meier method used to estimate the survival functions for each treatment arm.

    Measured: Week 26

  5. Cumulative Corticosteroid Dose at Week 26

    Measured: Week 26

  6. Percentage of Participants Who Completed the 26-Week Corticosteroid Taper and Who Had a Normal ESR

    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.

    Measured: Week 26

  7. Percentage of Participants Who Completed the 26-Week Corticosteroid Taper and Who Had a Normal CRP Level

    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.

    Measured: Week 26

  8. 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

    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.

    Measured: Week 26

  9. Cumulative Corticosteroid Dose at the End of the Washout Safety Follow-up Period

    Measured: Final Safety Follow-up visit (Week 38)

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted1 Feb 2019
  2. Study start20 Sep 2018
  3. Primary completion13 Aug 2020
  4. Study completion25 Nov 2020

Resources

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
Randomised
Intervention model
Parallel groups
Model description
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.
Masking
Quadruple blind
Who is masked
Participant, Care provider, Investigator, Outcomes assessor
Enrolment
70 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03827018
Sponsor's protocol ID
KPL-301-C001
Status last verified
Oct 2023
First submitted
25 Jan 2019
Last updated
23 Oct 2023
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
Yes
Individual data sharing
No

Publications

  • 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.

    PubMed 35264321

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing mavrilimumab and prednisone in people with Giant Cell Arteritis, run by Kiniksa Pharmaceuticals, Ltd. at 49 sites in 15 countries.

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 23 Oct 2023. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 49 sites in United States, Germany, Australia, Belgium, Italy and 10 more countries. 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. Some participants in this study may receive a placebo. 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 Oct 2023). 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?

Time to Flare by Week 26 (time frame: Week 26). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT03827018, which also lists 1 linked publication.

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 23 Oct 2023.

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