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Een onderzoek om de werkzaamheid, veiligheid en farmacokinetiek van axatilimab-monotherapie te evalueren bij Japanse deelnemers met recidiverende of refractaire actieve chronische graft-versus-host-ziekte

1 oktober 2026 bijgewerkt door: Incyte Biosciences Japan GK

Een fase 3, open-label, multicenter onderzoek om de werkzaamheid, veiligheid en farmacokinetiek van axatilimab-monotherapie te evalueren bij Japanse deelnemers met recidiverende of refractaire actieve chronische graft-versus-host-ziekte na ten minste twee lijnen systemische therapie

This study will be conducted to determine the clinical efficacy of axatilimab in Japanese participants with chronic graft-versus-host disease (cGVHD).

Studie Overzicht

Toestand

Actief, niet wervend

Conditie

Interventie / Behandeling

Studietype

Ingrijpend

Inschrijving (Werkelijk)

21

Fase

  • Fase 3

Contacten en locaties

In dit gedeelte vindt u de contactgegevens van degenen die het onderzoek uitvoeren en informatie over waar dit onderzoek wordt uitgevoerd.

Studie Locaties

  • Japan
      • Aichi, Japan, 470-1192
        • Fujita Health University Hospital
      • Fukui, Japan, 910-1193
        • University of Fukui Hospital
      • Fukuoka, Japan
        • Kyushu University Hospital
      • Hiroshima, Japan, 734-8551
        • Hiroshima University Hospital
      • Hokkaido, Japan, 060-8648
        • Hokkaido University Hospital
      • Hyōgo, Japan, 650-0047
        • Kobe City Medical Center General Hospital
      • Kanagawa, Japan, 259-1193
        • Tokai University Hospital
      • Kumamoto, Japan, 860-0008
        • National Hospital Organization Kumamoto Medical Center
      • Miyagi, Japan, 980-8574
        • Tohoku University Hospital
      • Nagoya, Japan, 453-8511
        • Japanese Red Cross Aichi Medical Center Nagoya Daiichi Hospital
      • Okayama, Japan, 700-8558
        • Okayama University Hospital
      • Osaka, Japan, 541-8567
        • Osaka Prefectural Hospital Organization Osaka International Cancer Institute
      • Osaka, Japan, 545-8586
        • Osaka Metropolitan University Hospital
      • Saitama-shi, Saitama,, Japan, 330-8777
        • Saitama Prefectural Children'S Medical Center
      • Tokyo, Japan, 113-8677
        • Tokyo Metropolitan Cancer and Infectious Diseases Center Komagome Hospital
      • Tokyo, Japan, 157-8535
        • National Center For Child Health and Development

Deelname Criteria

Onderzoekers zoeken naar mensen die aan een bepaalde beschrijving voldoen, de zogenaamde geschiktheidscriteria. Enkele voorbeelden van deze criteria zijn iemands algemene gezondheidstoestand of eerdere behandelingen.

Geschiktheidscriteria

Leeftijden die in aanmerking komen voor studie

6 jaar en ouder (Kind, Volwassen, Oudere volwassene)

Accepteert gezonde vrijwilligers

Nee

Geslachten die in aanmerking komen voor studie

Allemaal

Beschrijving

Inclusion Criteria:

  • At least 6 years of age at the time of signing the ICF.

  • Ability to comprehend and willingness to sign a written ICF for the study.

    • For participants 6 to 17 years old, a parent/guardian must provide consent for pediatric participants; when applicable, pediatric participants should also sign an assent form.

  • Japanese participants who are allo-HSCT recipients with active, refractory, or recurrent cGVHD requiring systemic immune suppression despite at least 2 lines of prior systemic therapy.

    • Active cGVHD is defined as the presence of signs and symptoms of cGVHD per the 2014 NIH Consensus Development Project on Criteria for Clinical Trials in cGVHD.

    • Refractory disease is defined as meeting any of the following criteria:

      • The development of 1 or more new sites of disease while being treated for cGVHD.
      • Progression of existing sites of disease despite at least 1 month of standard or investigational therapy for cGVHD.
      • Participants who did not achieve a response within 3 months on prior therapy for cGVHD and for whom the treating physician believes a new systemic therapy is required.

    • Recurrent cGVHD is defined as active, symptomatic disease (after an initial response to prior therapy) based on the NIH 2014 consensus criteria by organ-specific or global assessment or for which the physician believes a new line of systemic therapy is required.

  • Participants may have persistent, active aGVHD and cGVHD manifestations (overlap syndrome), as defined by the 2014 NIH Consensus Development Project on Criteria for Clinical Trials in cGVHD.

  • Karnofsky performance score of ≥ 60 (if aged 16 years or older); Lansky performance score of ≥ 60 (if aged younger than 16 years).

  • Adequate organ and bone marrow functions evaluated during the 14 days prior to the start of study treatment.

  • Creatinine clearance ≥ 30 mL/min based on the Cockcroft-Gault formula in adult participants and Schwartz formula in pediatric participants.

  • Concomitant use of a systemic corticosteroid is allowed but not required. Topical and inhaled corticosteroid agents are allowed. If a participant is taking a corticosteroid, it must be a stable dose for at least 2 weeks prior to the start of study treatment.

  • Concomitant use of protocol-defined immunosuppressant is allowed but not required.

  • Willingness to avoid pregnancy or fathering children based on protocol-defined criteria.

Exclusion Criteria:

  • Has aGVHD without manifestations of cGVHD.
  • Any evidence (histologic, cytogenetic, molecular, hematologic, or mixed) of relapse of the underlying cancer or post-transplant lymphoproliferative disease at the time of screening.
  • History of acute or chronic pancreatitis.
  • History of myositis.
  • History or other evidence of severe illness, uncontrolled infection, allergy to excipients, or any other conditions that would make the participant, in the opinion of the investigator, unsuitable for the study.
  • Has acquired immunodeficiency syndrome.
  • History of latent or active TB based on protocol-defined criteria.
  • Active HBV or HCV infection that requires treatment, or at risk for HBV reactivation (ie, positive HBsAg).
  • Pregnant or breastfeeding.
  • Previous exposure to CSF-1R targeted therapies.
  • Use of any agent other than corticosteroids, or the immunosuppressant for the treatment of cGVHD within 2 weeks or 5 half-lives, whichever is shorter, prior to the start of study treatment.
  • Has received an investigational treatment within 28 days prior to the start of study treatment.
  • Currently participating in any other interventional study.

Studie plan

Dit gedeelte bevat details van het studieplan, inclusief hoe de studie is opgezet en wat de studie meet.

Hoe is de studie opgezet?

Ontwerpdetails

  • Primair doel: Behandeling
  • Toewijzing: NVT
  • Interventioneel model: Opdracht voor een enkele groep
  • Masker: Geen (open label)

Wapens en interventies

Deelnemersgroep / Arm
Interventie / Behandeling
Experimenteel: Axatilimab Dose

Axatilimab at the protocol-defined dose.
Geneesmiddel: INCA034176

IV infusion

Andere namen:
  • Axatilimab

Wat meet het onderzoek?

Primaire uitkomstmaten

Uitkomstmaat
Maatregel Beschrijving
Tijdsspanne
Overall Response Rate (ORR) in the First 6 Treatment Cycles, as Defined by the 2014 NIH Consensus Development Project on Criteria for Clinical Trials in cGVHD
Tijdsspanne: up to the end of Cycle 6 (up to 168 days)

ORR was defined as the percentage of participants with a physician-assessed complete response (CR) or a physician-assessed partial response (PR), evaluated as defined by the 2014 National Institutes of Health (NIH) Consensus Development Project on Criteria for Clinical Trials in chronic graft-versus-host disease (cGVHD). A participant was considered an objective responder if they had an overall response of CR or PR at any timepoint up to the beginning of Cycle 7. CR was defined as the resolution of all manifestations in each organ or site, and PR was defined as improvement in at least 1 organ or site without progression in any other organ or site.
up to the end of Cycle 6 (up to 168 days)

Secundaire uitkomstmaten

Uitkomstmaat
Maatregel Beschrijving
Tijdsspanne
Percentage of Participants With a ≥7-point Improvement in Modified Lee Symptom Scale (mLSS) Score
Tijdsspanne: before Cycle 7 Day 1 (up to 168 days); during study (up to 12.8 months)

The mLSS is a 28-item instrument comprising 7 subscales, designed to assess the impact of cGVHD on a participant's symptoms. Participants were asked to indicate the degree of "bother" during the past 7 days due to symptoms in 7 domains (skin, eyes and mouth, breathing, eating and digestion, muscles and joints, energy, emotional distress) potentially affected by cGVHD. A mLSS responder is defined as a participant who achieved at least a 7-point improvement from baseline in the normalized mLSS score. This score is on a 0-100 scale, calculated as the average of the subscale scores.
before Cycle 7 Day 1 (up to 168 days); during study (up to 12.8 months)
ORR, as Defined by the 2014 NIH Consensus Development Project on Criteria for Clinical Trials in cGVHD
Tijdsspanne: up to 12.8 months

ORR was defined as the percentage of participants with a physician-assessed CR or a physician-assessed PR, evaluated as defined by the 2014 NIH Consensus Development Project on Criteria for Clinical Trials in cGVHD. A participant was considered an objective responder if they had an overall response of CR or PR at any post-baseline visit. CR was defined as the resolution of all manifestations in each organ or site, and PR was defined as improvement in at least 1 organ or site without progression in any other organ or site.
up to 12.8 months
Duration of Response (DOR) in Participants With a Response in the First 6 Treatment Cycles
Tijdsspanne: up to 13.8 months (maximum duration of treatment [12.8 months] plus 30 days)

Duration of response was defined as the time from the first response of either CR or PR until progression of cGVHD, start of another anti-GVHD treatment, or death from any cause, whichever was earlier. CR was defined as the resolution of all manifestations in each organ or site, and PR was defined as improvement in at least 1 organ or site without progression in any other organ or site. DOR was assessed for participants whose first CR or PR occurred during the first 6 treatment cycles. A response was followed until the response ended, regardless of whether the response ended during the first 6 treatment cycles or after Cycle 6.
up to 13.8 months (maximum duration of treatment [12.8 months] plus 30 days)
Organ-specific Response Rate
Tijdsspanne: up to 12.8 months

Organ-specific response rate was defined as the percentage of participants with a CR or PR for the 9 individual organs based on the 2014 NIH Consensus Development Project on Criteria for Clinical Trials in cGVHD (skin, eyes, mouth, esophagus, upper gastrointestinal (GI) tract, lower GI, liver, lungs, and joints and fascia) in participants with each corresponding organ involvement at baseline.
up to 12.8 months
Percent Reduction in Average Daily Dose (or Equivalent) of Corticosteroids in the First 6 Treatment Cycles
Tijdsspanne: up to the end of Cycle 6 (up to 168 days)

Participants may have been taking a corticosteroid upon study entry. At the discretion of the investigator, the corticosteroid dose may have been tapered after the first 4 weeks of study drug administration for participants who achieved either a PR or CR. Percent reduction was calculated as the ([difference between the baseline value and the post-baseline value]/divided by the baseline value) x 100.
up to the end of Cycle 6 (up to 168 days)
Percentage of Participants Who Discontinued Corticosteroid Use After Study Entry in the First 6 Treatment Cycles
Tijdsspanne: up to the end of Cycle 6 (up to 168 days)

Participants may have been taking a corticosteroid upon study entry. At the discretion of the investigator, the corticosteroid dose may have been tapered after the first 4 weeks of study drug administration for participants who achieved either a PR or CR.
up to the end of Cycle 6 (up to 168 days)
Number of Participants With Any Treatment-emergent Adverse Event (TEAE)
Tijdsspanne: up to 13.8 months

An adverse event was defined as any untoward medical occurrence associated with the use of a drug in humans, whether or not it is considered drug related. An AE could therefore have been any unfavorable or unintended sign (including an abnormal laboratory finding), symptom, or disease (new or exacerbated) temporally associated with the use of study drug. For the purposes of analysis, all AEs were considered to be TEAEs unless the AE could unequivocally be defined as not treatment emergent.
up to 13.8 months
Number of Participants With Any ≥Grade 3 TEAE
Tijdsspanne: up to 13.8 months

An adverse event was defined as any untoward medical occurrence associated with the use of a drug in humans, whether or not it is considered drug related. For the purposes of analysis, all AEs were considered to be TEAEs unless the AE could unequivocally be defined as not treatment emergent. The severity of AEs was assessed using Common Terminology Criteria for Adverse Events version 5.0 (CTCAE v5.0) Grades 1 through 5. Grade 1: mild; asymptomatic or mild symptoms; clinical or diagnostic observations only; treatment not indicated. Grade 2: moderate; minimal, local, or noninvasive treatment indicated; limiting age-appropriate activities of daily living. Grade 3: severe or medically significant but not immediately life-threatening; hospitalization or prolongation of hospitalization indicated; disabling; limiting self-care activities of daily living. Grade 4: life-threatening consequences; urgent treatment indicated. Grade 5: fatal.
up to 13.8 months
Change From Baseline in Karnofsky/Lansky Performance Status
Tijdsspanne: up to 13.8 months

The Karnofsky/Lansky performance status allows participants to be classified as to their functional impairment on a scale from 0 to 100. The lower the score, the worse the survival for most serious illnesses. The score can be used to compare the effectiveness of different therapies and to assess the prognosis in individual participants. The Karnofsky scale is designed for participants aged 16 years and older, and the Lansky scale is designed for participants younger than 16 years. Change from baseline was calculated as the post-baseline value minus the baseline value.
up to 13.8 months
Percentage of Participants Positive for Anti-drug Antibodies (ADAs)
Tijdsspanne: up to 12.8 months

Antibodies to axatilimab were evaluated in plasma samples collected from all participants.
up to 12.8 months
INCA034176 Concentration
Tijdsspanne: up to 12.8 months

Plasma samples were collected for pharmacokinetic (PK) analysis.
up to 12.8 months

Medewerkers en onderzoekers

Hier vindt u mensen en organisaties die betrokken zijn bij dit onderzoek.

Sponsor

Incyte Biosciences Japan GK

Onderzoekers

  • Studie directeur: Incyte Medical Monitor, Incyte Corporation

Publicaties en nuttige links

De persoon die verantwoordelijk is voor het invoeren van informatie over het onderzoek stelt deze publicaties vrijwillig ter beschikking. Dit kan gaan over alles wat met het onderzoek te maken heeft.

Nuttige links

Studie record data

Deze datums volgen de voortgang van het onderzoeksdossier en de samenvatting van de ingediende resultaten bij ClinicalTrials.gov. Studieverslagen en gerapporteerde resultaten worden beoordeeld door de National Library of Medicine (NLM) om er zeker van te zijn dat ze voldoen aan specifieke kwaliteitscontrolenormen voordat ze op de openbare website worden geplaatst.

Bestudeer belangrijke data

Studie start (Werkelijk)

30 juli 2024

Primaire voltooiing (Werkelijk)

6 augustus 2025

Studie voltooiing (Geschat)

30 maart 2027

Studieregistratiedata

Eerst ingediend

9 februari 2024

Eerst ingediend dat voldeed aan de QC-criteria

9 februari 2024

Eerst geplaatst (Werkelijk)

16 februari 2024

Updates van studierecords

Laatste update geplaatst (Werkelijk)

5 oktober 2026

Laatste update ingediend die voldeed aan QC-criteria

1 oktober 2026

Laatst geverifieerd

1 oktober 2026

Meer informatie

Termen gerelateerd aan deze studie

Trefwoorden

Aanvullende relevante MeSH-voorwaarden

Andere studie-ID-nummers

  • INCA34176-358

Plan Individuele Deelnemersgegevens (IPD)

Bent u van plan om gegevens van individuele deelnemers (IPD) te delen?

JA

Beschrijving IPD-plan

Incyte shares data with qualified external researchers after a research proposal is submitted. These requests are reviewed and approved by a review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations. The trial data availability is according to the criteria and process described on https://www.incyte.com/our-company/compliance-and-transparency

IPD-tijdsbestek voor delen

Data will be shared after the primary publication or 2 years after the study has ended for market authorized products and indications.

IPD-toegangscriteria voor delen

Data from eligible studies will be shared with qualified researchers according to the criteria and process described in the Data Sharing section of the www.incyteclinicaltrials.com website. For approved requests, the researchers will be granted access to anonymized data under the terms of a data sharing agreement.

IPD delen Ondersteunend informatietype

  • LEERPROTOCOOL
  • SAP

Informatie over medicijnen en apparaten, studiedocumenten

Bestudeert een door de Amerikaanse FDA gereguleerd geneesmiddel

Ja

Bestudeert een door de Amerikaanse FDA gereguleerd apparaatproduct

Nee

product vervaardigd in en geëxporteerd uit de V.S.

Ja

Deze informatie is zonder wijzigingen rechtstreeks van de website clinicaltrials.gov gehaald. Als u verzoeken heeft om uw onderzoeksgegevens te wijzigen, te verwijderen of bij te werken, neem dan contact op met register@clinicaltrials.gov. Zodra er een wijziging wordt doorgevoerd op clinicaltrials.gov, wordt deze ook automatisch bijgewerkt op onze website .

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