Trial · NCT04710576

A Study of Axatilimab at 3 Different Doses in Participants With Chronic Graft Versus Host Disease (cGVHD)

NCT04710576, Phase 2, Recruiting, Syndax Pharmaceuticals; results first posted 2024-10-10

3 sourcestrials-2026-11-12-0.5.0

Cite this record

ConductScience Library. A Study of Axatilimab at 3 Different Doses in Participants With Chronic Graft Versus Host Disease (cGVHD). Trials, record NCT04710576. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT04710576

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Trial

As registered on ClinicalTrials.gov, in the graph's capture of the registry.

Title: A Study of Axatilimab at 3 Different Doses in Participants With Chronic Graft Versus Host Disease (cGVHD)
NCT number: NCT04710576
Status: Recruiting
Phases: Phase 2
Enrollment: 296 participants, estimated
Lead sponsor: Syndax Pharmaceuticals
Conditions: Chronic Graft-versus-host-disease
Interventions: Axatilimab
First posted: 2021-01-14
Last updated: 2026-06-16
Results first posted: 2024-10-10

Source ClinicalTrials.gov

Registered outcomes18

Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.

List	Outcome	Time frame	Description
Primary	Overall Response Rate (ORR) in the First 6 Cycles as Defined by the 2014 NIH Consensus Development Project on Criteria for Clinical Trials in Chronic Graft-Versus-Host Disease (cGVHD)	First 6 cycles (up to Cycle 7 Day 1; each cycle = 4 weeks)	The ORR was defined as the percentage of participants with objective response (complete response \[CR\] or partial response \[PR\]). CR was defined as 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.
Secondary	ORR on Study as Defined by the 2014 NIH Consensus Development Project on Criteria for Clinical Trials in cGVHD	Up to 2 years
Secondary	Number of Participants With a Clinically Significant Improvement in Normalized Score on the Modified Lee Symptom Scale	Up to 2 years
Secondary	Duration of Response	Up to 2 years	Duration of response is defined as the time from initial partial response or complete response until documented progression of cGVHD, start of new therapy, or death for any reason.
Secondary	Sustained Response Rate	Up to 2 years	Sustained response rate is defined as the number of participants with objective response lasting for at least 20 weeks (140 days) from the time of initial response. Responses will be assessed based on the 2014 NIH Consensus Development Project on Clinical Trials in cGVHD.
Secondary	Organ-specific Response Rate	Up to 2 years	Organ-specific response is defined as the number of participants with objective response for the nine individual organs based on 2014 NIH Consensus Development Project on Criteria for Clinical Trials in cGVHD (skin, eyes, mouth, esophagus, upper gastrointestinal \[GI\], lower GI, liver, lungs and joints and fascia).
Secondary	Joints and Fascia Response Rate Based on Refined NIH Response Algorithm for cGVHD	Up to 2 years
Secondary	Percent Reductions in Average Daily Doses (or Equivalent) of Corticosteroid	Up to 2 years
Secondary	Number of Participants Who Discontinue Corticosteroid Use	Up to 2 years
Secondary	Percent Reductions in Average Daily Doses (or Equivalent) of Calcineurin Inhibitors (CNI)	Up to 2 years

1 to 10 of 18Source ClinicalTrials.gov

See all 18 registered outcomes

Outcomes18

Each outcome the trial posted results for, in ClinicalTrials.gov's words.

Type	Outcome	Time frame	Population	Units	Measured as
PRIMARY	Overall Response Rate (ORR) in the First 6 Cycles as Defined by the 2014 NIH Consensus Development Project on Criteria for Clinical Trials in Chronic Graft-Versus-Host Disease (cGVHD)	First 6 cycles (up to Cycle 7 Day 1; each cycle = 4 weeks)	The ITT analysis set for each dose level included all participants randomized to the dose level.	percentage of participants	NUMBER
SECONDARY	ORR on Study as Defined by the 2014 NIH Consensus Development Project on Criteria for Clinical Trials in cGVHD	Up to 2 years
SECONDARY	Number of Participants With a Clinically Significant Improvement in Normalized Score on the Modified Lee Symptom Scale	Up to 2 years
SECONDARY	Duration of Response	Up to 2 years
SECONDARY	Sustained Response Rate	Up to 2 years
SECONDARY	Organ-specific Response Rate	Up to 2 years
SECONDARY	Joints and Fascia Response Rate Based on Refined NIH Response Algorithm for cGVHD	Up to 2 years
SECONDARY	Percent Reductions in Average Daily Doses (or Equivalent) of Corticosteroid	Up to 2 years
SECONDARY	Number of Participants Who Discontinue Corticosteroid Use	Up to 2 years
SECONDARY	Percent Reductions in Average Daily Doses (or Equivalent) of Calcineurin Inhibitors (CNI)	Up to 2 years

1 to 10 of 18Source ClinicalTrials.gov

See all 18 outcomes

Results by group3

Each measurement the trial posted, group by group, in the words and numbers it posted.

Outcome	Group	Value	Spread	Units
Overall Response Rate (ORR) in the First 6 Cycles as Defined by the 2014 NIH Consensus Development Project on Criteria for Clinical Trials in Chronic Graft-Versus-Host Disease (cGVHD)	Axatilimab 0.3 mg/kg Q2W	73.8	95% Confidence Interval 62.7 to 83.0	percentage of participants
Overall Response Rate (ORR) in the First 6 Cycles as Defined by the 2014 NIH Consensus Development Project on Criteria for Clinical Trials in Chronic Graft-Versus-Host Disease (cGVHD)	Axatilimab 1 mg/kg Q2W	66.7	95% Confidence Interval 55.3 to 76.8	percentage of participants
Overall Response Rate (ORR) in the First 6 Cycles as Defined by the 2014 NIH Consensus Development Project on Criteria for Clinical Trials in Chronic Graft-Versus-Host Disease (cGVHD)	Axatilimab 3 mg/kg Q4W	50.0	95% Confidence Interval 38.6 to 61.4	percentage of participants

Source ClinicalTrials.gov

Participant flow12

How many started, completed or left each group, period by period.

Period	Milestone	Group	Participants
Overall Study	STARTED	Axatilimab 0.3 mg/kg Q2W	80
Overall Study	STARTED	Axatilimab 1 mg/kg Q2W	81
Overall Study	STARTED	Axatilimab 3 mg/kg Q4W	80
Overall Study	Received at Least 1 Dose of Study Drug	Axatilimab 0.3 mg/kg Q2W	79
Overall Study	Received at Least 1 Dose of Study Drug	Axatilimab 1 mg/kg Q2W	81
Overall Study	Received at Least 1 Dose of Study Drug	Axatilimab 3 mg/kg Q4W	79
Overall Study	COMPLETED	Axatilimab 0.3 mg/kg Q2W	0
Overall Study	COMPLETED	Axatilimab 1 mg/kg Q2W	1
Overall Study	COMPLETED	Axatilimab 3 mg/kg Q4W	1
Overall Study	NOT COMPLETED	Axatilimab 0.3 mg/kg Q2W	80

1 to 10 of 12Source ClinicalTrials.gov

See all 12 participant flow

Adverse events630

Each adverse event the trial posted, group by group: the participants affected out of those at risk.

Type	Event	Organ system	Group	Affected	At risk
other	Anaemia	Blood and lymphatic system disorders	Axatilimab 0.3 mg/kg Q2W	10	79
other	Anaemia	Blood and lymphatic system disorders	Axatilimab 1 mg/kg Q2W	6	81
other	Anaemia	Blood and lymphatic system disorders	Axatilimab 3 mg/kg Q4W	10	79
other	Tachycardia	Cardiac disorders	Axatilimab 0.3 mg/kg Q2W	5	79
other	Tachycardia	Cardiac disorders	Axatilimab 1 mg/kg Q2W	0	81
other	Tachycardia	Cardiac disorders	Axatilimab 3 mg/kg Q4W	2	79
other	Dry eye	Eye disorders	Axatilimab 0.3 mg/kg Q2W	4	79
other	Dry eye	Eye disorders	Axatilimab 1 mg/kg Q2W	2	81
other	Dry eye	Eye disorders	Axatilimab 3 mg/kg Q4W	1	79
other	Eyelid oedema	Eye disorders	Axatilimab 0.3 mg/kg Q2W	0	79

1 to 10 of 630Source ClinicalTrials.gov

See all 630 adverse events

Adverse event totals9

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	Axatilimab 0.3 mg/kg Q2W	2	80
Total, all-cause mortality	Axatilimab 1 mg/kg Q2W	7	81
Total, all-cause mortality	Axatilimab 3 mg/kg Q4W	12	80
Total, other adverse events	Axatilimab 0.3 mg/kg Q2W	74	79
Total, other adverse events	Axatilimab 1 mg/kg Q2W	78	81
Total, other adverse events	Axatilimab 3 mg/kg Q4W	78	79
Total, serious adverse events	Axatilimab 0.3 mg/kg Q2W	30	79
Total, serious adverse events	Axatilimab 1 mg/kg Q2W	33	81
Total, serious adverse events	Axatilimab 3 mg/kg Q4W	38	79

Source ClinicalTrials.gov

Papers2

The papers the trial lists on ClinicalTrials.gov.

PMID	Listed as	Source
39292927	DERIVED	PubMed
39704205	DERIVED	PubMed

Grants0

The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.

None in this release.

Trial comparisons0

Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.

None in this release.

Tests0

The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.

None in this release.

Data dates

The day each source was copied for this release.

ClinicalTrials.gov: 2026-11-12; ClinicalTrials.gov
Results, from AACT: 2026-03-23; AACT
NIH grant links: 2026-11-13; NIH RePORTER

Sources 3

ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
ClinicalTrials.govNo license stated
NIH RePORTER's clinical studies and project filesPublic domain

Each source's terms

ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
ClinicalTrials.govnone stated
NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)

ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.

For software

Read this trial from software.

The record and each of its lists are JSON, twenty rows a page. No key.

GET https://conductscience.org/api/trials/v1/NCT04710576

GET https://conductscience.org/api/trials/v1/NCT04710576/events?page=2

API reference For AI assistants