A Study to Evaluate the Efficacy, Safety, and Pharmacokinetics of Axatilimab Monotherapy in Japanese Participants With Recurrent or Refractory Active Chronic Graft-Versus-Host Disease

NCT06263478 | Active Not Recruiting Phase 3 FDA Drug

• 1 other identifier: INCA34176-358
• Study Type: interventional
• Target: 21
• Locations: 1 country
• Sites: 16

Brief Summary

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

Conditions

Chronic Graft-versus-host-disease

Keywords

cGVHD

Outcome Measures

Primary Outcomes (1)

• Overall Response Rate in the First 6 Cycles

  The overall response rate will be assessed by the number of participants with objective response by Cycle 7 (28-day cycles), Day 1, with responses defined by the 2014 NIH consensus criteria.

  Up to Cycle 7 (Day 169)

Secondary Outcomes (10)

• Proportion of participants with a ≥ 7-point improvement in modified Lee symptom scale (mLSS) score

  Up to 2 years

• Overall Response Rate

  Up to 2 years

• Duration of Response

  Up to 2 years

• Organ-specific Response Rate

  Up to 2 years

• Percent reduction in average daily dose (or equivalent) of corticosteroids

  Up to 2 years

Study Arms (1)

Axatilimab Dose

EXPERIMENTAL

Axatilimab at the protocol-defined dose.

Drug: INCA034176

Interventions

INCA034176 DRUG

IV infusion

Also known as: Axatilimab

Axatilimab Dose

Eligibility Criteria

• Age: 6 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Incyte Biosciences Japan GK: lead

Study Sites (16)

Fujita Health University Hospital

Aichi, 470-1192, Japan

Location

University of Fukui Hospital

Fukui, 910-1193, Japan

Location

Kyushu University Hospital

Fukuoka, Japan

Location

Hiroshima University Hospital

Hiroshima, 734-8551, Japan

Location

Hokkaido University Hospital

Hokkaido, 060-8648, Japan

Location

Kobe City Medical Center General Hospital

Hyōgo, 650-0047, Japan

Location

Tokai University Hospital

Kanagawa, 259-1193, Japan

Location

National Hospital Organization Kumamoto Medical Center

Kumamoto, 860-0008, Japan

Location

Tohoku University Hospital

Miyagi, 980-8574, Japan

Location

Japanese Red Cross Aichi Medical Center Nagoya Daiichi Hospital

Nagoya, 453-8511, Japan

Location

Okayama University Hospital

Okayama, 700-8558, Japan

Location

Osaka Prefectural Hospital Organization Osaka International Cancer Institute

Osaka, 541-8567, Japan

Location

Osaka Metropolitan University Hospital

Osaka, 545-8586, Japan

Location

Saitama Prefectural Children'S Medical Center

Saitama-shi, Saitama,, 330-8777, Japan

Location

Tokyo Metropolitan Cancer and Infectious Diseases Center Komagome Hospital

Tokyo, 113-8677, Japan

Location

National Center For Child Health and Development

Tokyo, 157-8535, Japan

Location

Related Links

• A study to evaluate the Efficacy, Safety, and Pharmacokinetics of Axatilimab Monotherapy in Japanese Participants With Recurrent or Refractory Active Chronic Graft-Versus-Host Disease

MeSH Terms

Conditions

Bronchiolitis Obliterans Syndrome

Interventions

axatilimab

Condition Hierarchy (Ancestors)

Organizing Pneumonia; Bronchiolitis Obliterans; Bronchiolitis; Bronchitis; Bronchial Diseases; Respiratory Tract Diseases; Lung Diseases, Obstructive; Lung Diseases; Graft vs Host Disease; Immune System Diseases

Study Officials

• Incyte Medical Monitor

  Incyte Corporation

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 9, 2024
• First Posted: February 16, 2024
• Study Start: July 30, 2024
• Primary Completion: August 6, 2025
• Study Completion (Estimated): March 30, 2027
• Last Updated: January 7, 2026

Record last verified: 2026-01

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP
• Time Frame: Data will be shared after the primary publication or 2 years after the study has ended for market authorized products and indications.
• Access Criteria: 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.

Locations

JP (16)