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Study to Assess the Safety, Pharmacokinetics, and Efficacy of KPL-404 in Participants With Rheumatoid Arthritis With Inadequate Response or Intolerance to at Least One Biologic Disease-modifying Anti-rheumatic Drug or a Janus Kinase Inhibitor

Phase 2
Completed
Conditions
Arthritis, Rheumatoid
Interventions
Drug: Placebo
Registration Number
NCT05198310
Lead Sponsor
Kiniksa Pharmaceuticals, Ltd.
Brief Summary

Phase 2 study of the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD) and efficacy of KPL-404 in subjects with moderate to severe Rheumatoid Arthritis.

Detailed Description

This is a 28-week (up to 4-week screening period, 12-week treatment period, and 12-week safety follow-up period), multicenter, randomized, double-blind, placebo-controlled, multiple dose, proof-of-concept study with PK lead-in designed to assess the safety, PK, efficacy and PD of KPL-404 in subjects with moderate to severe, active Rheumatoid Arthritis (RA) who have an inadequate response to or are intolerant to a Janus kinase inhibitor (JAKi) AND/OR at least one biologic disease-modifying anti-rheumatic drug (bDMARD). The objectives of the study are to evaluate safety, efficacy, and PD of KPL-404 compared with placebo across the estimated therapeutic range and to characterize PK across varying dose levels of KPL-404.

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
145
Inclusion Criteria
  • Body weight ≥ 40 to ≤ 140 kg for all cohorts.

  • Diagnosis of RA for ≥ 3 months fulfilling the 2010 American College of Rheumatology (ACR)/European Union League Against Rheumatism (EULAR) classification criteria for RA and that is categorized as ACR RA functional Class 1-3.

  • Treated with a biological disease-modifying anti-rheumatic drug (bDMARDs) AND/OR Janus kinase inhibitor (JAKi) therapy for RA for ≥ 3 months and had inadequate response or had to discontinue bDMARD AND/OR JAKi therapy due to intolerance or toxicity, regardless of treatment duration.

  • Currently receiving conventional synthetic disease-modifying anti-rheumatic drugs (csDMARD) therapy ≥ 3 months and on a stable dose for ≥ 4 weeks before the first dose of investigational product.

    1. The following csDMARDs are allowed: oral or parenteral methotrexate ([MTX]; 7.5 to 25 mg/week), sulfasalazine (≤ 3000 mg/day), hydroxychloroquine (≤ 400 mg/day), chloroquine (≤ 250 mg/day), and leflunomide (≤ 20 mg/day).
    2. A combination of up to 2 background csDMARDs is allowed, except the combination of MTX and leflunomide.
  • Meets all of the following disease activity criteria:

    1. Six or more swollen joints (based on 66 joint counts) and ≥ 6 tender joints (based on 68 joint counts) at screening and baseline visits;
    2. Level of high-sensitivity C-reactive protein ≥ 3 mg/L (by central laboratory);
    3. Documented seropositivity for serum Rheumatoid Factor (RF) and/or Anti-citrullinated protein antibody (ACPA) (>ULN) at screening or by prior laboratory evaluation.
  • Has completed a locally approved authorized COVID-19 vaccine regimen according to local guidance at least 3 weeks before the first dose of the Investigational Product.

  • Must have discontinued all bDMARDs or JAKi prior to the first dose of investigational product. The washout period for bDMARDs or JAKi prior to the first dose of investigational product is specified below. For bDMARDs or JAKi not listed below washout should be at least 5 times the mean elimination half-life of a drug:

    1. ≥ 4 weeks for etanercept;
    2. ≥ 8 weeks for adalimumab, infliximab, certolizumab, golimumab, abatacept, tocilizumab, and sarilumab;
    3. ≥ 1 year for rituximab;
    4. ≥ 2 weeks for JAKi (either investigational or commercially available treatment).
  • Voluntarily sign and date an informed consent form approved by independent ethics committee/Institutional Review Board (IRB)

Exclusion Criteria
  • Prior exposure to any other anti-CD40/CD40L agent.

  • Inadequate response to 5 or more classes of advanced targeted therapies (bDMARD or tsDMARD; e.g., TNF inhibitors, IL-6 receptor inhibitors, T-cell costimulatory inhibitors, anti-CD-20 antibodies, JAK inhibitors). This does not include prior discontinuation due to drug intolerance.

  • Injectable corticosteroids (including intra-articular) or treatment with > 10 mg/day dose oral prednisone or equivalent within 8 weeks prior to randomization.

  • History of any arthritis with onset prior to age 16 years or current diagnosis of inflammatory joint disease other than RA (Current diagnosis of secondary Sjogren's syndrome is permitted).

  • History of thromboembolic event or a significant risk of future thromboembolic events

  • Clinically significant active infection including signs/symptoms suggestive of infection, any significant recurrent or chronic infection, or subjects at a high risk of infection

  • History of cancer within the last 5 years from screening, except for basal and squamous cell carcinoma of the skin or in situ carcinoma of the cervix treated and considered cured.

  • History of any of the following cardiovascular conditions:

    1. Moderate to severe congestive heart failure (New York Heart Association class III or IV);
    2. Recent (within past 6 months) cerebrovascular accident, myocardial infarction, coronary stenting;
    3. Uncontrolled hypertension as defined by a confirmed systolic blood pressure > 160 mmHg or diastolic blood pressure > 100 mmHg.
  • Clinically relevant or significant electrocardiogram (ECG) abnormalities, including ECG with QT interval corrected for heart rate (QTc) > 500 msec.

Study & Design

Study Type
INTERVENTIONAL
Study Design
SEQUENTIAL
Arm && Interventions
GroupInterventionDescription
Cohort 1 PlaceboPlaceboPlacebo for KPL-404 SC q2wk for 12 weeks
Cohort 2 KPL-404KPL-404KPL-404 5mg/kg SC q2wk for 12 weeks
Cohort 2 PlaceboPlaceboPlacebo for KPL-404 SC q2wk for 12 weeks
Cohort 3 KPL-404KPL-404KPL-404 5mg/kg SC qwk for 12 weeks
Cohort 3 KPL-404 and PlaceboPlaceboKPL-404 5mg/kg SC q2wk with alternating weekly administrations of KPL-404 or placebo SC for 12 weeks
Cohort 3 PlaceboPlaceboPlacebo for KPL-404 SC qwk for 12 weeks
Cohort 4 PlaceboPlaceboPlacebo for KPL-404 SC q4wk for 12 weeks
Cohort 1 KPL-404KPL-404KPL-404 2mg/kg Subcutaneous (SC) q2wk for 12 weeks
Cohort 3 KPL-404 and PlaceboKPL-404KPL-404 5mg/kg SC q2wk with alternating weekly administrations of KPL-404 or placebo SC for 12 weeks
Cohort 4 KPL-404KPL-404KPL-404 SC q4wk for 12 weeks: 600 mg loading dose at baseline followed by 400 mg at weeks 4 and 8.
Primary Outcome Measures
NameTimeMethod
Cohorts 1 and 2: Incidence of Treatment-Emergent Adverse Events (TEAEs)Up to 24 weeks
Cohorts 1 and 2: Area Under the Curve from Time 0 to the Last Measurable Concentration (AUC0-t)Predose on Days 1-169
Cohorts 1 and 2: Maximum Serum Concentration (Cmax)Predose on Days 1-169
Cohort 3 and 4: Change from Baseline in Disease Activity Score of 28 Joints Using C-reactive Protein (DAS28-CRP) at Week 12Baseline, Week 12
Secondary Outcome Measures
NameTimeMethod
Cohort 3 and 4: Incidence of TEAEsUp to 24 weeks
Cohort 3 and 4: CmaxPredose on Days 1-169
Cohort 3 and 4: AUC0-tPredose on Days 1-169
Cohorts 1 and 2: Change from Baseline in DAS28-CRP at Week 12Baseline, Week 12

Trial Locations

Locations (41)

Jacaranda Hospital

🇿🇦

Pretoria, Gauteng, South Africa

Clinresco Centres (Pty) Ltd

🇿🇦

Kempton Park, Gauteng, South Africa

Umhlanga Hospital Medical Center

🇿🇦

Umhlanga, Kwazulu-Natal, South Africa

Carewell Arthritis Center

🇺🇸

Apple Valley, California, United States

Medvin Clinical Research

🇺🇸

Whittier, California, United States

Inland Rheumatology Clinical Trials

🇺🇸

Upland, California, United States

International Medical Research

🇺🇸

Daytona Beach, Florida, United States

Millennium Research

🇺🇸

Ormond Beach, Florida, United States

Paramount Medical Research & Consulting, LLC

🇺🇸

Middleburg Heights, Ohio, United States

Altoona Center for Clinical Research

🇺🇸

Duncansville, Pennsylvania, United States

Saint Francis Hospital- Memphis

🇺🇸

Memphis, Tennessee, United States

West Broward Rheumatology Associates, Inc.

🇺🇸

Tamarac, Florida, United States

Trinity Universal Research Assoc

🇺🇸

Carrollton, Texas, United States

Arthritis and Rheumatology Research Institute

🇺🇸

Allen, Texas, United States

Southwest Rheumatology Research LLC

🇺🇸

Mesquite, Texas, United States

Rheumatology and Pulmonary Clinic

🇺🇸

Beckley, West Virginia, United States

Medical center "Artmed" LTD

🇧🇬

Plovdiv, Bulgaria

Vesalion s.r.o.

🇨🇿

Ostrava, Czechia

JSC Evex Hospitals

🇬🇪

Tbilisi, Georgia

Revmatologicky Utsav

🇨🇿

Praha 2, Czechia

Medical Plus S.R.O.

🇨🇿

Uherské Hradiště, Czechia

Aleksandre Aladashvili Clinic LLC

🇬🇪

Tbilisi, Georgia

LTD Israel-Georgian Medical Research Clinic Helsicore

🇬🇪

Tbilisi, Georgia

LTD Georgian-Dutch Hospital

🇬🇪

Tbilisi, Georgia

Szegedi Tudomanyegyetem Szent-Gyorgyi Albert Klinikai Kozpont

🇭🇺

Szeged, Csongrád, Hungary

Qualiclinic Ltd (Qualiclinic Inc)

🇭🇺

Budapest, Hungary

Magyar Honvedseg Egeszsegugyi Kozpont

🇭🇺

Budapest, Hungary

Porcika Klinika

🇭🇺

Hódmezővásárhely, Hungary

Szabolcs-Szatmar-Bereg Megyei Korhazak es Egyetemi Oktatokorhaz

🇭🇺

Nyíregyháza, Hungary

Vita Verum Medical Egeszsegugy

🇭🇺

Székesfehérvár, Hungary

Centrum Kliniczno-Badawcze

🇵🇱

Elblag, Poland

Silmedic sp. z o.o.

🇵🇱

Katowice, Poland

RCMed Oddzial Sochaczew

🇵🇱

Sochaczew, Poland

Prywatna Praktyka Lekarska Prof. UM dr hab.med. Pawel Hrycaj

🇵🇱

Poznan, Poland

Centrum Medyczne Reuma Park

🇵🇱

Warszawa, Poland

Sweet Hope Research Specialty, Inc.

🇺🇸

Hialeah, Florida, United States

San Marcus Research Clinic, Inc.

🇺🇸

Miami Lakes, Florida, United States

Arthritis & Osteoporosis Center of Coastal Bend

🇺🇸

Corpus Christi, Texas, United States

DM Clinical Research

🇺🇸

Tomball, Texas, United States

Rheumatology Clinic of Houston

🇺🇸

Tomball, Texas, United States

Panorama Medical Centre

🇿🇦

Cape Town, Western Cape, South Africa

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