A Ph1b/2 Study of Axatilimab Monotherapy in Chinese Participants With Recurrent or Refractory cGVHD

NCT06843408 | Recruiting Phase 1 FDA Drug

• 1 other identifier: INCA34176-204
• Study Type: interventional
• Target: 30
• Locations: 1 country
• Sites: 9

Brief Summary

This study will be conducted to evaluate the safety, efficacy, and pharmacokinetics of axatilimab monotherapy in Chinese participants with recurrent or refractory active chronic graft-versus-host disease after systemic therapy.

Conditions

Chronic Graft-versus-host-disease

Keywords

cGVHD

Outcome Measures

Primary Outcomes (2)

• Number of participants with Treatment-emergent Adverse Events (TEAEs)

  Defined as adverse events reported for the first time or worsening of a pre-existing event, occurring after the first dose.

  Up to 2 years and 30 days

• Objective Response 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 Development Project on Criteria for Clinical Trials in cGVHD.

  Up to Cycle 7 (Day 169)

Secondary Outcomes (12)

• 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 (2)

Part 1 Safety Evaluation

EXPERIMENTAL

Axatilimab at the protocol-defined dose.

Drug: INCA034176

Part 2 Efficacy Evaluation

EXPERIMENTAL

Axatilimab at the protocol-defined dose.

Drug: INCA034176

Interventions

INCA034176 DRUG

IV infusion

Also known as: Axatilimab

Part 1 Safety Evaluation; Part 2 Efficacy Evaluation

Eligibility Criteria

• Age: 12 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• At least 12 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 12 to 17 years old: A parent/guardian must provide consent for pediatric participants; when applicable, pediatric participants should also sign an assent form.
• Chinese participants who are allo-HSCT recipients with active, refractory, or recurrent cGVHD requiring systemic immune suppression despite prior systemic therapy, including corticosteroids and at least 1 other appropriate treatment for refractory or recurrent cGVHD.
• 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 (Jagasia et al 2015).
• 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 (Lee et al 2015) 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 as follows:
• Absolute neutrophil count ≥ 1.0 × 109/L (without growth factors within 1 week of study entry).
• Platelet count ≥ 50 × 109/L (without growth factor or transfusion within 2 weeks of study entry).

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.
• Active symptomatic myositis.
• History or other evidence of severe illness, uncontrolled infection, allergy to excipients (see formulation details in the IB), or any other conditions that would make the participant, in the opinion of the investigator, unsuitable for the study.
• Positive HIV status.
• History of latent or active TB, including either one of following:
• Signs or symptoms suggestive of active TB upon medical history and/or physical examination.
• Recent close contact with a person with active TB.
• Positive QuantiFERON and/or T-spot TB test at screening.
• Active HBV or HCV infection that requires treatment, or at risk for HBV reactivation (ie, positive HBsAg). Participants with negative HBsAg and positive total HBcAb and/or HBsAb should be excluded if quantitative HBV DNA test result is ≥ 20 IU/mL at the time of screening. Participants who are positive for HCV antibody are eligible only if PCR is negative for HCV RNA.
• Diagnosed with another malignancy (other than malignancy for which transplant was performed) within 3 years before Cycle 1 Day 1 unless previously treated with curative intent (eg, completely resected basal cell or squamous cell carcinoma of the skin, resected in situ cervical malignancy, resected breast ductal carcinoma in situ, or low-risk prostate cancer after curative resection) and approved by the sponsor's medical monitor.
• Pregnant or breastfeeding.
• Previous exposure to CSF-1R targeted therapies.
• Use of any agent other than corticosteroids, CNIs, or mTOR inhibitors for the treatment of cGVHD within 2 weeks or 5 half-lives, whichever is shorter, prior to the start of study treatment.

Sponsors & Collaborators

• Incyte Corporation: lead

Study Sites (9)

Peking University People'S Hospital

Beijing, 101149, China

RECRUITING

West China Hospital of Sichuan University

Chengdu, 610041, China

RECRUITING

The Second Affiliated Hospital, Army Medical University (Xinqiao Hospital)

Chongoing, 400037, China

RECRUITING

Zhujiang Hospital of Southern Medical University

Guangzhou, 510280, China

RECRUITING

Nanfang Hospital of Southern Medical University

Guangzhou, 510515, China

RECRUITING

The First Affiliated Hospital, Zhejiang University School of Medicine

Hangzhou, 310024, China

RECRUITING

Shanghai Children'S Medical Center

Shanghai, 200127, China

RECRUITING

Institute of Hematology, Chinese Academy of Medical Sciences

Tianjin, 301617, China

RECRUITING

Union Hospital Affiliated to Tongji Medical College of Huazhong University of Science and Technology

Wuhan, 430022, China

RECRUITING

Related Links

• A Ph1b/2 Study of Axatilimab Monotherapy in Chinese Participants With Recurrent or Refractory cGVHD

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

Central Study Contacts

Incyte Corporation Call Center (US)

CONTACT

1.855.463.3463; medinfo@incyte.com

Incyte Corporation Call Center (ex-US)

CONTACT

+800 00027423; eumedinfo@incyte.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 20, 2025
• First Posted: February 25, 2025
• Study Start: June 25, 2025
• Primary Completion (Estimated): July 3, 2026
• Study Completion (Estimated): December 31, 2027
• Last Updated: February 3, 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

CN (9)