Flag of the European Union EU Clinical Trials Register Help

Clinical trials

The European Union Clinical Trials Register allows you to search for protocol and results information on:
  • interventional clinical trials that are conducted in the European Union (EU) and the European Economic Area (EEA);
  • clinical trials conducted outside the EU / EEA that are linked to European paediatric-medicine development.
  • Learn   more about the EU Clinical Trials Register   including the source of the information and the legal basis.


    The EU Clinical Trials Register currently displays   42868   clinical trials with a EudraCT protocol, of which   7063   are clinical trials conducted with subjects less than 18 years old.
    The register also displays information on   18700   older paediatric trials (in scope of Article 45 of the Paediatric Regulation (EC) No 1901/2006).


    Phase 1 trials conducted solely in adults and that are not part of an agreed PIP are not public in the EU CTR (refer to European Guidance 2008/C 168/02   Art. 3 par. 2 and   Commission Guideline 2012/C 302/03,   Art. 5) .

    Clinical Trials marked as "Trial now transitioned" were transitioned to the Clinical Trial Regulation 536/2014 and can be further followed in the Clinical Trial Information System  
     
    Examples: Cancer AND drug name. Pneumonia AND sponsor name.
    How to search [pdf]
    Search Tips: Under advanced search you can use filters for Country, Age Group, Gender, Trial Phase, Trial Status, Date Range, Rare Diseases and Orphan Designation. For these items you should use the filters and not add them to your search terms in the text field.
    Advanced Search: Search tools
     

    < Back to search results

    Print Download

    Summary
    EudraCT Number:2018-001003-36
    Sponsor's Protocol Code Number:KPL-301-C001
    National Competent Authority:Croatia - MIZ
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2019-05-17
    Trial results View results
    Index
    A. PROTOCOL INFORMATION
    B. SPONSOR INFORMATION
    C. APPLICANT IDENTIFICATION
    D. IMP IDENTIFICATION
    D.8 INFORMATION ON PLACEBO
    E. GENERAL INFORMATION ON THE TRIAL
    F. POPULATION OF TRIAL SUBJECTS
    G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
    N. REVIEW BY THE COMPETENT AUTHORITY OR ETHICS COMMITTEE IN THE COUNTRY CONCERNED
    P. END OF TRIAL
    Expand All   Collapse All
    A. Protocol Information
    A.1Member State ConcernedCroatia - MIZ
    A.2EudraCT number2018-001003-36
    A.3Full title of the trial
    A Phase 2, randomized, double-blind placebo-controlled study to test the efficacy and safety of KPL-301 in giant cell arteritis
    Randomizirano, dvostruko slijepo, placebom kontrolirano ispitivanje faze 2 za procjenu učinkovitosti i
    sigurnosti lijeka KPL-301 u liječenju arteritisa divovskih stanica
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A clinical study to test treatment of KPL-301 compared to placebo in giant cell arteritis
    Kliničko ispitivanje lijeka KPL-301 u usporedbi s placebom u oboljelih od arteritisa divovskih stanica
    A.4.1Sponsor's protocol code numberKPL-301-C001
    A.7Trial is part of a Paediatric Investigation Plan No
    A.8EMA Decision number of Paediatric Investigation Plan
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorKiniksa Pharmaceuticals, Ltd. (Hamilton, Bermuda) c/o Kiniksa Pharmaceuticals Corp.
    B.1.3.4CountryUnited States
    B.3.1 and B.3.2Status of the sponsorCommercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportKiniksa Pharmaceuticals Corp.
    B.4.2CountryUnited States
    B.4.1Name of organisation providing supportKiniksa Pharmaceuticals, Ltd.
    B.4.2CountryBermuda
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationKiniksa Pharmaceuticals, Ltd. (Hamilton, Bermuda) c/o Kiniksa Pharmaceuticals Corp.
    B.5.2Functional name of contact pointKiniksa Medical Information Group
    B.5.3 Address:
    B.5.3.1Street Address100 Hayden Ave
    B.5.3.2Town/ cityLexington, Massachusetts
    B.5.3.3Post code02421
    B.5.3.4CountryUnited States
    B.5.4Telephone number17814319100
    B.5.5Fax number18885227408
    B.5.6E-mailstudyinfo@kiniksa.com
    D. IMP Identification
    D.IMP: 1
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation No
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameMavrilimumab
    D.3.2Product code KPL-301
    D.3.4Pharmaceutical form Solution for injection in pre-filled syringe
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPSubcutaneous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNMAVRILIMUMAB
    D.3.9.1CAS number 1085337-57-0
    D.3.9.2Current sponsor codeKPL-301
    D.3.9.3Other descriptive namen/a
    D.3.9.4EV Substance CodeSUB189175
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number150
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin No
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) Yes
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product Yes
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.IMP: 2
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation Yes
    D.2.1.1.1Trade name Prednisone Tablets, USP
    D.2.1.1.2Name of the Marketing Authorisation holderPar Pharmaceutical
    D.2.1.2Country which granted the Marketing AuthorisationUnited States
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product namePredniSONE Tablets, USP
    D.3.2Product code N/A
    D.3.4Pharmaceutical form Tablet
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product No
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.8 Information on Placebo
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboSolution for injection in pre-filled syringe
    D.8.4Route of administration of the placeboSubcutaneous use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    giant cell arteritis
    Arteritis divovskih stanica
    E.1.1.1Medical condition in easily understood language
    an inflammatory disease of blood vessels
    Upalna bolest krvnih žila
    E.1.1.2Therapeutic area Diseases [C] - Cardiovascular Diseases [C14]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level LLT
    E.1.2Classification code 10018250
    E.1.2Term Giant cell arteritis
    E.1.2System Organ Class 100000004866
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    The primary objective of the study is to evaluate the efficacy of KPL-301 versus placebo, coadministered with a 26-week steroid taper, for maintaining sustained remission for 26 weeks in subjects with newonset or relapsing/refractory giant cell arteritis (GCA).
    Primarni cilj ispitivanja jeste procijeniti učinkovitost lijeka KPL-301 u usporedbi s placebom, kada se primjenjuje zajedno s 26-tjednim smanjenjem terapije kortikosteroidima, za održavanje remisije tijekom 26 tjedana u ispitanika s novim ili relapsnim/refraktornim gigantocelularnim arteritisom (GCA).
    E.2.2Secondary objectives of the trial
    The secondary objectives of the study, in subjects with new-onset and relapsing/refractory GCA, are:
    a) To evaluate the effect of KPL-301 vs placebo on cumulative corticosteroid dose.
    b) To evaluate the effect of KPL-301 vs placebo on health-related quality of life (HRQoL).
    c) To evaluate the safety and tolerability of KPL-301.
    d) To evaluate the pharmacokinetics (PK) of KPL-301.
    Sekundarni ciljevi ispitivanja, u ispitanika s novim i relapsnim/refraktornim GCA-om, su:
    a) Procijeniti učinak lijeka KPL-301 u usporedbi s placebom na kumulativnu dozu kortikosteroida.
    b) Procijeniti učinak lijeka KPL-301 u usporedbi s placebom na kvalitetu života povezanu sa zdravljem (HRQoL).
    c) Procijeniti sigurnost i podnošljivost lijeka KPL-301.
    d) Procijeniti farmakokinetiku (FK) lijeka KPL-301.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Able and willing to provide written informed consent and to comply with the study protocol
    2. Age of ≥ 50 to 85 inclusive
    3. Diagnosis of new-onset or relapsing GCA classified according to the following criteria:
    New-onset: Initial diagnosis of GCA within 6 weeks of Day 0, defined as:
    a) Westergren ESR > 30 mm/hour or CRP ≥ 1 mg/dL
    b) AND at least one of the following:
    i. Unequivocal cranial symptoms of GCA (new-onset localized headache, scalp or temporal artery pain or tenderness, ischemia-related vision loss, or otherwise unexplained mouth or jaw pain upon mastication)
    ii. Unequivocal extracranial symptoms of GCA such as claudication of the extremities
    iii. Symptoms of PMR, defined as shoulder and/or hip girdle pain associated with inflammatory morning stiffness
    c) AND at least one of the following:
    i. TAB or ultrasound revealing features of GCA
    ii. Evidence of large-vessel vasculitis by angiography or cross-sectional imaging study such as MRI, CT/CTA, or PET-CT of the aorta or other great vessels
    Relapsing: Diagnosis of GCA > 6 weeks before Day 0 AND:
    Active GCA within 6 weeks of Day 0 defined as clinical sign/symptom(s) as per above and Westergren ESR> 30 mm/hour or CRP ≥ 1 mg/ dL
    Refractory nonremitting: Diagnosis of GCA > 6 weeks before Day 0 AND
    No remission since the diagnosis of disease as per clinical expectations. i.e. Presence of sign/symptoms as per above and Westergren ESR>30mm/hour or CRP ≥ 1 mg/ dL within 6 weeks of Day 0
    4. Remission of GCA at or before Day 0 (resolution of GCA symptom(s) and CRP < 1.0 mg/dL or ESR < 20 mm in the first hour), such that the subject can safely participate in the study and follow the protocol defined rocedures, including initiation of the prednisone taper at the protocol-specified starting dose (i.e., ≤60 mg/day)
    5. At Day 0, receiving or able to receive oral prednisone up to 60 mg/day for the treatment of GCA
    6. If using methotrexate (MTX), oral or parenteral up to 25mg/week is permitted in screening if started more than 6 weeks prior to Day 0 and should be tapered to zero by Day 0.
    7. Willing to receive antiplatelet therapy depending on the Investigator’s decision
    8. Willing to receive treatment for prevention of corticosteroid-induced osteopenia/osteoporosis depending on the Investigator’s decision
    9. Female subjects must be:
    a) postmenopausal, defined as at least 12 months post cessation of menses (without an alternative medical cause), or
    b) permanently sterile following documented hysterectomy, bilateral salpingectomy, bilateral ophorectomy, or tubal ligation or having a male partner with vasectomy as affirmed by the subject, or
    c) nonpregnant, nonlactating, and if sexually active having agreed to use a highly effective method of contraception (i.e., hormonal contraceptives associated with inhibition of ovulation or intrauterine device [IUD], or intrauterine hormone-releasing system [IUS], or sexual abstinence) from Screening visit until the final Washout Safety Follow-up visit 84 ± 3 days from EOT Visit.
    10. 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 (i.e., hormonal contraceptives associated with the inhibition of ovulation or intrauterine device [IUD], or intrauterine hormone-releasing system [IUS], or sexual abstinence) from Day 0 until the final Washout Safety Follow-up visit 84± 3 days from EOT Visit. Male subjects must agree to refrain from donating sperm during this time period.
    Kriteriji podobnosti:
    Kriteriji uključenja
    1. Sposobnost i spremnost davanja pisanog informiranog pristanka i pridržavanje plana ispitivanja
    2. Starost d ≥ 50 d 85 godina
    3. Dijagnoza novog ili relapsnog GCA klasificiranog prema sljedećim kriterijima:
    Novi: Početna dijagnoza GCA unutar 6 tjedana prije 0. dana definirana kao
    a) Westergrenov ESR > 30 mm/sat ili CRP ≥ 1 mg/dl
    b) I najmanje jedan od sljedećih kriterija:
    i. Nedvosmisleni kranijalni simptomi GCA (nova lokalizirana glavobolja, bol ili osjetljivost glave ili temporalne arterije, gubitak viza povezan s ishemijom ili inače neobjašnjeni bolovi u ustima ili vilici prilikom žvakanja)
    ii. Nedvosmisleni ekstrakranijalni simptomi GCA, kao što je klaudikacija ekstremiteta
    iii. Simptomi PMR-a, koja se definira kao bolovi u ramenu i/ili zdjelici povezani s jutarnjom upalnom ukočenošću
    c) I najmanje jedan od sljedećih kriterija:
    i. TAB ili ultrazvuk koji pokazuje znakove GCA
    ii. Dokazi o postojanju vaskulitisa velikih krvnih žila pomoću angiografije ili snimanja poprečnog presjeka kao što je MR, CT/CTA ili PET-CT aorte ili drugih velikih krvnih žila
    Relapsni: Dijagnoza GCA > 6 tjedana prije 0. dana:
    Aktivni GCA unutar 6 tjedana prije 0. dana što se definira kao gore navedeni klinički znak/simptom(i) i Westergrenov ESR > 30 mm/sat ili CRP ≥ 1 mg/dl

    Refraktorno nesmanjujuće: Dijagnoza GCA > 6 tjedana prije 0. dana i bez remisije od postavljanja dijagnoze bolesti prema kliničkim očekivanjima, odnosno prisutnost znakova i simptoma kako je gore navedeno i Westergrenov ESR > 30 mm/sat ili CRP ≥ 1 mg/dl unutar 6 tjedana prije 0. dana.
    4. Remisija GCA 0. dana ili prije (povlačenje GCA simptoma i CRP < 1,0 mg/dl ili ESR < 20 mm unutar prvog sata), tako da ispitanik može sigurno sudjelovati u ispitivanju i pridržavati se postupaka definiranih planom ispitivanja, uključujući početak smanjenja terapije prednizonom u početnoj dozi definiranoj planom ispitivanja (tj. ≤60 mg/dan)
    5. Primanje ili mogućnost primanja oralnog prednizona 0. dana u dozi od najviše 60 mg/dan za liječenje GCA
    6. Ako se koristi metotreksat (MTX), oralna ili parenteralna doza od najviše 25 mg/tjedan dozvoljena je prilikom probira ako se primjena započne više od 6 tjedana prije 0. dana te treba biti smanjena na 0 mg do 0. dana.
    7. Spremnost primanja antitrombocitne terapije u zavisnosti od ispitivačeve odluke
    8. Spremnost primanja liječenja za sprječavanje osteopenije/osteoporoze izazvane kortikosteroidima u zavisnosti od ispitivačeve odluke
    9. Žene ispitanice moraju:
    a) biti u postmenopauzi, koja se definira kao najmanje 12 mjeseci bez menstruacije (bez alternativnog medicinskog uzroka), ili
    b) biti trajno sterilne nakon dokumentirane histerektomije, obostrane salpingektomije, obostrane ooforektomije ili podvezivanja jajnika ili imati muškog partnera podvrgnutog vazektomiji što može potvrditi ispitanica, ili
    c) ne smiju biti trudne, ne smiju dojiti i, ako su seksualno aktivne, moraju pristati na korištenje visokoučinkovite kontracepcijske metode (tj. hormonski kontraceptivi povezani sa sprječavanjem ovulacije ili unutarmaterični umetak (IUD) ili unutarmaternični sustav otpuštanja hormona ili seksualna apstinencija) od posjeta za probir do zadnjeg posjeta iz razdoblja sigurnosnog praćenja i ispiranja lijeka 84±3 dana nakon posjeta za kraj liječenja.
    10. Muškarci ispitanici moraju biti podvrgnuti dokumentiranoj vazektomiji ili, ako su seksualno aktivni, moraju pristati na korištenje visokoučinkovite metode kontracepcije (na primjer, hormonski kontraceptivi povezani sa sprječavanjem ovulacije ili unutarmaterični umetak (IUD) ili unutarmaternični sustav otpuštanja hormona ili seksualna apstinencija) s partnericom koja može zatrudnjeti od 0. dana do zadnjeg posjeta iz razdoblja sigurnosnog praćenja i ispiranja lijeka 84±3 dana nakon posjeta za kraj liječenja. Muškarci moraju pristati na izbjegavanje doniranja sperme tijekom tog razdoblja.
    E.4Principal exclusion criteria
    General Exclusion Criteria
    1. Major surgery within 8 weeks prior to Screening or planned major surgery within 12 months after randomization
    2. Transplanted organs (except corneal transplant performed more than 3 months prior to randomization)
    3. Major ischemic event unrelated to GCA within 12 weeks of Screening
    Exclusions Related to Prior or Concomitant Therapy
    4. Concurrent enrollment in another clinical study, with the exception of observational studies
    5. Previous treatment with KPL-301
    6. Treatment with any non-biologic investigational drug therapy within 4 weeks or 5 half-lives of the study agent, whichever was longer, prior to Screening
    7. Any cell-depleting biological therapies (e.g., anti-CD20) within 12 months prior to Day 0; or previous treatment with noncell-depleting biological therapies (such as anti-tumor necrosis factor [TNF], anakinra, anti-Interleukin [IL]-6 receptor [e.g. tocilizumab], or abatacept) within 8 weeks (or 5 half-lives, whichever is longer) prior to Screening
    8. Treatment with alkylating agents within 12 weeks prior to Screening
    9. Intramuscular, IV corticosteroids, Intra-articular corticosteroids within 4 weeks prior to Screening
    10. Receipt of live (attenuated) vaccine within the 4 weeks before Day 0
    11. Treatment with hydroxychloroquine, cyclosporine A, azathioprine, cyclophosphamide, or mycophenolate mofetil (MMF) within 4 weeks of Screening Relating to Medical History
    12. Female subjects who are pregnant, intending to become pregnant, or are breastfeeding
    13. Any condition that, in the opinion of the Investigator, could interfere with evaluation of KPL-301 or interpretation of subject safety or confound the results of the study
    14. Known history of allergy or reaction to any component of the KPL-301 or placebo formulation or to any other biologic therapy or prednisone or any of its excipients
    15. Positive (or 2 indeterminate) QuantiFERON test results.
    16. Clinically significant active infection including signs/symptoms suggestive of infection, any significant recurrent or chronic infection (including positive hepatitis C virus antibody [HCVAb]), or any episode of infection requiring hospitalization or treatment with IV antibiotics within 12 weeks before Screening. Subjects with any opportunistic infection within 6 months before Screening will be excluded from the study.
    17. Subjects with chronic active hepatitis B infection as defined below will be excluded from the study:
     Hepatitis B surface antigen (HbsAg) positive
     Hepatitis B anti-core antibody positive but anti-surface antibody negative
    18. Subjects at a high risk of infection (e.g., history of hereditary or acquired immune deficiency disorder including a history of known human immunodeficiency virus [HIV] infection), a history of an infected joint prosthesis at any time with that prosthesis still in situ, leg ulcers, indwelling urinary catheter, or persistent or recurrent chest infections
    19. 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
    20. Evidence of clinically uncontrolled respiratory disease. The Investigator should review the data from subjects’ respiratory assessments including chest x-ray and pulmonary function tests (PFTs) including DLCO performed during the screening period or within 12 weeks prior to Day 0 if results of prior assessments are available. Available PFT and DLCO assessments must have had values ≥ 60% of predicted for measurements performed and no uncontrolled lung disease. A subject’s medical regimen should not have been significantly intensified to control lung disease within 12 weeks prior to Day 0.
    21. History of chronic respiratory tract infections
    22. Congestive heart failure of New York Heart Association classification III or IV
    23. At screening blood tests, any of the following:
    a) Aspartate transaminase (AST) > 2 × upper limit of normal (ULN)
    b) Alanine transaminase (ALT) > 2 × ULN
    c) Hemoglobin < 75 g/L
    d) Neutrophils < 1.5 × 109/L
    e) Creatinine clearance (CrCl) <30 mL/min
    Opći kriteriji isključenja
    1. Veći kirurški zahvat unutar 8 tjedana prije probira ili planirani veći kirurški zahvat unutar 12 mjeseci nakon randomizacije
    2. Presađeni organi (izuzev presađivanja rožnice više od 3 mjeseca prije randomizacije)
    3. Veći ishemijski događaj nepovezan s GCA unutar 12 tjedana prije probira
    Kriteriji isključenja povezani s prethodnom ili usporednom terapijom
    4. Istovremeno sudjelovanje u drugom kliničkom ispitivanju, izuzev opažajnih ispitivanja
    5. Ranije liječenje lijekom KPL-301
    6. Liječenje bilo kojim nebiološkim ispitivanim lijekom unutar 4 tjedna ili 5 poluvijekova ispitivanog sredstva, ovisno o tome što je duže, prije probira
    7. Bilo koje biološke terapije koje izazivaju depleciju stanica (npr. anti-CD20) unutar 12 mjeseci prije 0. dana; ili ranije liječenje biološkim terapijama koje ne izazivaju depleciju stanica (kao što je sredstvo protiv faktora tumorske nekroze [TNF], anakinra, sredstvo protiv interleukinskog [IL]-6 receptora [npr. tocilizumab] ili abatacept) unutar 8 tjedana (ili 5 poluvijekova, ovisno o tome što je duže) prije probira
    8. Liječenje alkilacijskim sredstvima unutar 12 tjedan prije probira
    9. Intramuskularni, intravenski ili intraartikularni kortikosteroidi unutar 4 tjedna prije probira
    10. Primanje živog (atenuiranog) cjepiva unutar 4 tjedan prije 0. dana
    11. Liječenje hidroksiklorokinom, ciklosporinom A, azatioprinom, ciklofosfamidom ili mikofenolat mofetilom (MMF) unutar 4 tjedna prije probira
    Kriteriji isključenja povezani s poviješću bolesti
    12. Žene ispitanice koje su trudne, namjeravaju zatrudnjeti ili doje
    13. Svako stanje koje bi, prema mišljenju ispitivača, utjecati na procjenu lijeka KPL-301 ili tumačenje sigurnosti ispitanika ili pomutiti rezultate ispitivanje
    14. Poznata povijest alergije ili reakcije na bilo koju komponentu pripravka lijeka KPL-301 ili placeba ili na bilo koju drugu biološku terapiju ili prednizon ili bilo koji od njegovih pomoćnih tvari
    15. Pozitivni ili 2 neodrediva rezultata na testu QuantiFERON.
    16. Klinički značajna aktivna infekcija uključujući znakove/simptome koji ukazuju na infekciju, bilo koja značajna rekurentna ili kronična infekcija (uključujući pozitivno antitijelo na virus hepatitisa C [HCVAb]) ili bilo koja epizoda infekcije koja zahtijeva hospitalizaciju ili liječenje i.v. antibioticima unutar 12 tjedana prije probira. Ispitanici s oportunističkom infekcijom unutar 6 mjeseci prije probira bit će isključeni iz ispitivanja.
    17. Ispitanici s kroničnom aktivnom infekcijom hepatitisom B koji su definirani u nastavku bit će isključeni iz ispitivanja:
    • pozitivno na površinski antigen hepatitisa B (HbsAg)
    • pozitivno na antitijelo na jezgru virusa hepatitisa B ali negativno na antitijelo na površinski antigen
    18. Ispitanici izloženi visokom riziku od infekcije (npr. povijest poremećaja nasljedne ili stečene imunodeficijencije uključujući povijest poznate infekcije virusom humane imunodeficijencije [HIV]), povijest zaražene zglobne proteze u bilo kojem trenutku dok je proteza još uvijek in situ, čirevi na nogama, trajni urinarni kateter ili perzistentne ili rekurentne infekcije prsnog koša
    19. Povijest raka u proteklih 10 godina izuzev karcinoma bazalnih stanica ili pločastih stanica kože ili in situ karcinoma cerviksa koji je tretiran i smatra se izliječenim
    20. Dokaz o postojanju klinički nekontroliranog respiratornog oboljenja. Ispitivač treba pregledati podatke iz respiratornih procjena ispitanika uključi rendgen prsnog koša i testove funkcije pluća uključujući difuzijski kapacitet za ugljični monoksid (DLCO) obavljene tijekom razdoblja probira ili unutar 12 tjedana prije 0. dana ako su dostupni rezultati prethodnih procjena. Dostupni rezultati procjena testova funkcije pluća i difuzijskog kapaciteta za ugljični monoksid moraju imati vrijednosti ≥ 60 % od predviđenih za obavljena mjerenja i ne smiju imati nekontroliranu plućnu bolest. Ispitanikov režim lijekova ne smije biti značajno pojačan radi kontroliranja plućne bolesti unutar 12 tjedana prije 0. dana.
    21. Povijest kroničnih infekcija dišnih puteva
    22. Kongestivno zatajenje srca klase III ili IV prema klasifikaciji New York Heart Association (Kardiološkog udruženja New Yorka)
    23. Bilo koje od sljedećih vrijednosti na pretragama krvi prilikom probira:
    a) aspartat transaminaza (AST) > 2 × gornja granica normale (GGN)
    b) alanih transaminaza (ALT) > 2 × GGN
    c) hemoglobin < 75 g/l
    d) neutrofili < 1,5 × 109/l
    e) klirens kreatinina (CrCl) <30 ml/min
    E.5 End points
    E.5.1Primary end point(s)
    Time to flare by week 26 defined as time from randomization to the first flare occurring within the first 26-weeks of the double-blind period
    Vrijeme do ponovne pojave bolesti do 26. tjedna definirano kao vrijeme od randomizacije prve pojave bolesti
    E.5.1.1Timepoint(s) of evaluation of this end point
    Appro. 32 weeks after the start of the study
    Približno 32 tjedna nakon početka ispitivanja
    E.5.2Secondary end point(s)
    Time to flare by week 26 from randomization to first flare occurring within the first 26 weeks of the double-blind period in the per protocol population
    Percentage of subjects who have completed the 26-week corticosteroid taper and who have a normal ESR
    Percentage of subjects who have completed the 26-week corticosteroid taper and who have a normal CRP
    Percentage of subjects who completed the 26-week corticosteroid taper and who have no signs/symptoms of GCA
    Time to corticosteroid dose of zero mg/day
    Cumulative steroid dose at Week 26 and at the end of the Washout Safety Follow-up Period
    Change in clinical GCA assessments (including NRS and FACIT) over time
    Change in quality-of-life over time
    • postotak ispitanika u 26. tjednu s normalnim ESR-om
    • postotak ispitanika na kraju randomiziranog liječenja s normalnim
    ESR-om
    • postotak ispitanika u 26. tjednu s normalnim CRP-om
    • postotak ispitanika na kraju randomiziranog liječenja s normalnim
    CRP-om
    • vrijeme do nulte doze kortikosteroida
    • kumulativna doza kortikosteroida u 26. tjednu i na kraju razdoblja
    dvostruko slijepog liječenja
    • promjena u kliničkim procjenama GCA (uključujući NRS i FACIT)
    tijekom vremena
    • promjena kvalitete života tijekom vremena
    E.5.2.1Timepoint(s) of evaluation of this end point
    Appro. 44 weeks after the start of the study
    Približno 44 tjedna nakon početka ispitivanja
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis Yes
    E.6.2Prophylaxis No
    E.6.3Therapy Yes
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic Yes
    E.6.7Pharmacodynamic Yes
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic Yes
    E.6.12Pharmacoeconomic No
    E.6.13Others No
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) No
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other No
    E.7.1.3.1Other trial type description
    E.7.2Therapeutic exploratory (Phase II) Yes
    E.7.3Therapeutic confirmatory (Phase III) No
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled Yes
    E.8.1.1Randomised Yes
    E.8.1.2Open No
    E.8.1.3Single blind No
    E.8.1.4Double blind Yes
    E.8.1.5Parallel group Yes
    E.8.1.6Cross over No
    E.8.1.7Other No
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo Yes
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial2
    E.8.3 The trial involves single site in the Member State concerned No
    E.8.4 The trial involves multiple sites in the Member State concerned Yes
    E.8.4.1Number of sites anticipated in Member State concerned1
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA36
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA Yes
    E.8.6.2Trial being conducted completely outside of the EEA No
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    Australia
    Belgium
    Croatia
    Estonia
    Germany
    Ireland
    Italy
    Netherlands
    New Zealand
    Poland
    Serbia
    Slovenia
    Spain
    United Kingdom
    United States
    E.8.7Trial has a data monitoring committee No
    E.8.8 Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial
    LVLS
    Posljednji posjet posljednjeg uključenog ispitanika
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years2
    E.8.9.1In the Member State concerned months0
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years2
    E.8.9.2In all countries concerned by the trial months0
    E.8.9.2In all countries concerned by the trial days0
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 No
    F.1.1.1In Utero No
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.3Newborns (0-27 days) No
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F.1.1.5Children (2-11years) No
    F.1.1.6Adolescents (12-17 years) No
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 20
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 40
    F.2 Gender
    F.2.1Female Yes
    F.2.2Male Yes
    F.3 Group of trial subjects
    F.3.1Healthy volunteers No
    F.3.2Patients Yes
    F.3.3Specific vulnerable populations Yes
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception Yes
    F.3.3.3Pregnant women No
    F.3.3.4Nursing women No
    F.3.3.5Emergency situation No
    F.3.3.6Subjects incapable of giving consent personally No
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state8
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 45
    F.4.2.2In the whole clinical trial 60
    F.5 Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)
    After subjects complete the 26-week double blind treatment period, they will discontinue and wash off of blinded KPL-301 or placebo during a 12-week Washout Period, which includes close safety follow-up and monitoring for potential GCA flares. After the final Washout Safety Follow-up visit (Week 38) has been completed, the subjects will exit the trial and should be transitioned to SoC per investigator judgement.
    Nakon što ispitanici završe sa sudjelovanjem u 26-tjednom dvostruko slijepom dijelu ispitivanja proći će kroz 12-tjedno razdoblje ispiranja koje uključuje siguronosno praćenje moguće pojave bolesti. Nakon zadnjeg posjeta u klopu praćenja (Tjedan 38) ispitanici će izaći iz kliničkog ispitivanja te primati standardno liječenje na temelju procjene liječnika.
    G. Investigator Networks to be involved in the Trial
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2019-04-10
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2018-10-12
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2020-11-12
    For support, visit the EMA Service Desk , log in using your EMA account and open a ticket specifying "EU CTR" in your request.
    If you do not have an account, please visit the EMA Account management page page click on "Create an EMA account" and follow the instructions.
    The status of studies in GB is no longer updated from 1.1.2021
    For the UK, as from 1.1.2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI
    EU Clinical Trials Register Service Desk: https://servicedesk.ema.europa.eu
    European Medicines Agency © 1995-2022 | Domenico Scarlattilaan 6, 1083 HS Amsterdam, The Netherlands
    Legal notice
    EMA HMA