GVHD
A study to evaluate Axatilimab and Corticosteroids as Initial Treatment for Chronic Graft-Versus-Host Disease
Incyte Study ID:
INCA34176-357
CT.gov ID:
Eudra ID:
N/A
EU CT Number:
Sponsor:
Incyte Corporation
Collaborator:
N/A
Study Contact Information:
Clinical Study Purpose
This study will be conducted to compare the efficacy of axatilimab versus
placebo in combination with corticosteroids as initial treatment for moderate or severe chronic graft-versus-host disease (cGVHD).
Clinical Study Summary
MEDICAL CONDITION(S)
PRODUCT
COLLABORATORS
N/A
DATE
Jan 2025 - Mar 2030
TYPE
Interventional
PHASE
Phase 3
SEX
Female & Male
AGE
12 Years - NA
ACCEPTS HEALTHY VOLUNTEERS
No
Clinical Study Locations
Name
PRISMA HEALTH UPSTATE
GREENVILLE, SC, US, 29615
Status
Recruiting
Name
NATIONAL HOSPITAL ORGANIZATION KUMAMOTO MEDICAL CENTER
KUMAMOTO KUMAMOTO, Japan, 860-0008
Status
Recruiting
Name
OSAKA METROPOLITAN UNIVERSITY HOSPITAL
OSAKA, Japan, 545-8586
Status
Recruiting
Name
GUNMA SAISEIKAI MAEBASHI HOSPITAL
MAEBASHI, Japan, 371-0821
Status
Recruiting
Name
KOBE CITY MEDICAL CENTER GENERAL HOSPITAL
HYOGO, Japan, 650-0047
Status
Recruiting
Name
HIROSHIMA UNIVERSITY HOSPITAL
HIROSHIMA-SHI, Japan, 734-8551
Status
Recruiting
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Key Inclusion and Exclusion Criteria
Inclusion Criteria
- ≥ 12 years of age at the time of informed consent.
- New-onset moderate or severe cGVHD, as defined by the 2014 NIH Consensus Development Project Criteria for Clinical Trials in cGVHD, requiring systemic therapy.
Exclusion Criteria
- Received more than 1 prior allo-HCT. Prior autologous HCT is allowed.
- Has overlap cGVHD, defined as simultaneous presence of features or characteristics of aGVHD in a patient with cGVHD.
Requirements information
Inclusion Criteria
- ≥ 12 years of age at the time of informed consent.
- New-onset moderate or severe cGVHD, as defined by the 2014 NIH Consensus Development Project Criteria for Clinical Trials in cGVHD, requiring systemic therapy.
- History of allo-HCT from any donor HLA type (related or unrelated donor with any degree of HLA matching) using any graft source (bone marrow, peripheral blood stem cells, or cord blood). Recipients of myeloablative, nonmyeloablative, or reduced-intensity conditioning are eligible.
- Adequate hematologic function with ANC ≥ 0.5 × 109/L independent of growth factors for at least 7 days prior to study entry.
- Willingness to avoid pregnancy or fathering children.
Exclusion Criteria
- Received more than 1 prior allo-HCT. Prior autologous HCT is allowed.
- Has overlap cGVHD, defined as simultaneous presence of features or characteristics of aGVHD in a patient with cGVHD.
- Received more than 7 days of systemic corticosteroid treatment for cGVHD or unable to begin a prednisone dose ≥ 1.0 mg/kg per day (or methylprednisolone equivalent) for cGVHD.
- Received previous systemic treatment for cGVHD, including extracorporeal photopheresis.
- Systemic treatment with CNIs or mTOR inhibitors started within 2 weeks prior to C1D1.
- Prior treatment with CSF-1R targeted therapies.
- Active, uncontrolled bacterial, fungal, parasitic, or viral infection.
- Evidence of relapse of the primary hematologic disease or treatment for relapse after the allo-HCT was performed, including DLIs for the treatment of molecular relapse.
- History of acute or chronic pancreatitis.
- Active symptomatic myositis.
- History or current diagnosis of cardiac disease indicating significant risk of safety for participation in the study, such as uncontrolled or significant cardiac disease.
- Severe renal impairment, that is, estimated CrCl < 30 mL/min measured or calculated by Cockcroft-Gault equation in adults and Schwartz formula in pediatric participants, or endstage renal disease on dialysis.
- Impaired liver function, defined as total bilirubin > 1.5 × ULN and/or ALT and AST > 3 × ULN in participants with no evidence of liver cGVHD.
- Pregnant or breastfeeding.
- Other protocol-defined Inclusion/Exclusion Criteria may apply.
Protocol Summary
Incyte Study ID:
INCA34176-357
Primary Purpose:
Treatment
Allocation:
Randomized
Study Design:
Parallel Assignment
Masking:
Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Interventions:
Enrollment:
240
Primary Outcome
Open
Event Free Survival (EFS)
Timeframe: Up to 3 years
Secondary Outcome
Open
Objective Response (OR)
Timeframe: 6 months
Event Free Survival 2
Timeframe: Up to 3 years
Proportion of participants with a ≥ 7-point improvement in mLSS total score
Timeframe: Up to 3 years
Overall Response
Timeframe: 12 Months
DOR (in responders only)
Timeframe: Up to 3 years
Best Overall Response (BOR)
Timeframe: Up to 3 years
Overall Survival (OS)
Timeframe: Up to 3 years
Nonrelapse mortality (NRM)
Timeframe: Up to 3 years
Failure-free survival (FFS)
Timeframe: Up to 3 years
Relapse of hematologic diseases
Timeframe: Up to 3 years
Time to primary hematologic disease relapse
Timeframe: Up to 3 years
Percent reduction in daily corticosteroid dose
Timeframe: 6 months
Proportion of participants who tapered off all corticosteroids
Timeframe: 6 months
Number of participants with Treatment-emergent Adverse Events (TEAEs)
Timeframe: Up to 3 years and 30 days
Change from baseline in circulating monocyte number and phenotype (CD14/16)
Timeframe: Up to 3 years and 30 days
Change from baseline in soluble markers for bone resorption and formation, including bone-specific alkaline phosphatase (BAP) and C-terminal telopeptide (CTX)
Timeframe: Up to 3 years and 30 days