Trial · NCT03604692
A Phase 1/2 Study to Evaluate Axatilimab in Participants With Active cGVHD
NCT03604692, Phase 1, Phase 2, Completed, Syndax Pharmaceuticals; results first posted 2024-11-27
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. A Phase 1/2 Study to Evaluate Axatilimab in Participants With Active cGVHD. Trials, record NCT03604692. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03604692
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- A Phase 1/2 Study to Evaluate Axatilimab in Participants With Active cGVHD
- NCT number
- NCT03604692
- Status
- Completed
- Phases
- Phase 1, Phase 2
- Enrollment
- 41 participants, actual
- Lead sponsor
- Syndax Pharmaceuticals
- Conditions
- Chronic Graft-versus-host-disease
- Interventions
- axatilimab
- First posted
- 2018-07-27
- Last updated
- 2025-09-15
- Results first posted
- 2024-11-27
Source ClinicalTrials.gov
Registered outcomes18
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Phase 1: Number of Participants With DLTs | Day 1 through the first 28 days from the first dose of SNDX-6352 or administration of the third dose (Cycle 2 Day 1), whichever is later (from Cycle 1 Day 1 to Cycle 2 Day 1) | A DLT was defined as the occurrence of any protocol-specified event within the first 28 days from the first dose of SNDX-6352 or administration of the third dose (Cycle 2 Day 1), whichever is later (from Cycle 1 Day 1 to Cycle 2 Day 1) and assessed by the Investigator as not being definitely attributable to underlying disease, disease progression, inter-current illness, concomitant medications or any other alternative cause. |
| Primary | Phase 1: Recommended Phase 2 Dose (RP2D) | Day 1 through the first 28 days from the first dose of SNDX-6352 or administration of the third dose (Cycle 2 Day 1), whichever is later (from C1D1 to C2D1) | The RP2D was determined in discussion with the Sponsor, Medical Monitor, and Dose Determination Phase Investigators and was based on observations from the Phase 1 of the study (clinical benefit in chronic graft versus host disease \[cGVHD\] and pharmacokinetic/pharmacodynamic effects). |
| Primary | Phase 2: Overall Response Rate (ORR) as Assessed by the Number of Participants With Complete Response (CR) or Partial Response (PR) at Cycle 7 Day 1 (Day 168) | Cycle 7 Day 1 (Day 168) | CR or PR was defined by the 2014 National Institutes of Health (NIH) Consensus Development Project on Criteria for Clinical Trials in cGVHD.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. ORR was defined as the percentage of participants achieving a best overall response of CR or PR .ORR calculated as: (number of participants with best overall response as CR or PR)/total number of participants. |
| Secondary | Phase 1: Area Under the Plasma Concentration-time Curve From Time 0 to the Last Measurable Concentration for SNDX-6352 | Cycle 1: predose, at 30 min (end of infusion), and at 1 hour and 8 hours on Day 1 and Day 15 | Blood samples were collected for determination of SNDX-6352 concentration. |
| Secondary | Phase 1: Observed Maximum Plasma Concentration for SNDX-6352 | Cycle 1: predose, at 30 min (end of infusion), and at 1 hour and 8 hours on Day 1 and Day 15 | Blood samples were collected for determination of SNDX-6352 concentration. |
| Secondary | Phase 1: Time to Observed Maximum Plasma Concentration | Cycle 1: predose, at 30 min (end of infusion), and at 1 hour and 8 hours on Day 1 and Day 15 | Blood samples were collected for determination of SNDX-6352 concentration. |
| Secondary | Phase 1: Changes From Baseline in Colony-Stimulating Factor-1 (CSF-1) and Interleukin (IL-34) Serum Concentrations | Baseline, Cycle 1 Day 8, Day 15, predose at Cycle 2 Day 1, Cycle 4 Day 1 (28-day cycles), and end of treatment (EOT) (median duration of treatment = 7 months) | Blood samples were collected for determination of serum concentrations. |
| Secondary | Phase 1: Percent Change From Baseline in Nonclassical Monocytes (CD14+CD16++) | Baseline, Day 8, Day 15, predose at Cycle 2 Day 1, Cycle 4 Day 1, and EOT (median duration of treatment = 7 months) | Blood samples were collected for determination nonclassical monocytes levels. |
| Secondary | Phase 1: Number of Participants Positive for Anti-Drug Antibodies (ADA) | Predose on Cycle 1 Day 1, Cycle 1 Day 15, Cycle 3 Day 1, Day 1 of each subsequent cycle through EOT plus 30 days after last dose (safety follow-up) (median duration of treatment = 7 months) | Blood samples were collected for ADA assessment. A participant was considered ADA positive if at least 1 postbaseline sample was ADA positive. |
| Secondary | Phase 2: Best Overall Response (BOR), as Defined by the 2014 NIH Consensus Development Project on Criteria for Clinical Trials in cGVHD | Day 1 of each 28-Day cycle up to Cycle 7 Day 1 | Physician-reported global cGVHD activity assessment and cGVHD response determination. Best overall response was calculated as percent of participants with a response of CR or PR. |
1 to 10 of 18Source ClinicalTrials.gov
See all 18 registered outcomesOutcomes18
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Phase 1: Number of Participants With DLTs | Day 1 through the first 28 days from the first dose of SNDX-6352 or administration of the third dose (Cycle 2 Day 1), whichever is later (from Cycle 1 Day 1 to Cycle 2 Day 1) | Participants who received at least 1 cycle of treatment and had at least one post-baseline response assessment. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Phase 1: Recommended Phase 2 Dose (RP2D) | Day 1 through the first 28 days from the first dose of SNDX-6352 or administration of the third dose (Cycle 2 Day 1), whichever is later (from C1D1 to C2D1) | Participants who received at least 1 cycle of treatment and had at least one post-baseline response assessment in Phase 1. | mg/kg | NUMBER |
| PRIMARY | Phase 2: Overall Response Rate (ORR) as Assessed by the Number of Participants With Complete Response (CR) or Partial Response (PR) at Cycle 7 Day 1 (Day 168) | Cycle 7 Day 1 (Day 168) | Safety Analysis Set: All participants who received at least 1 dose of study treatment. | percentage of participants | NUMBER |
| SECONDARY | Phase 1: Area Under the Plasma Concentration-time Curve From Time 0 to the Last Measurable Concentration for SNDX-6352 | Cycle 1: predose, at 30 min (end of infusion), and at 1 hour and 8 hours on Day 1 and Day 15 | Pharmacokinetic Analysis Set: All treated participants who had at least 1 plasma concentration measured. Number Analyzed = participants who were evaluable at specified timepoints | µg*h/mL | MEAN |
| SECONDARY | Phase 1: Observed Maximum Plasma Concentration for SNDX-6352 | Cycle 1: predose, at 30 min (end of infusion), and at 1 hour and 8 hours on Day 1 and Day 15 | Pharmacokinetic Analysis Set: All treated participants who had at least 1 plasma concentration measured. Number Analyzed = participants who were evaluable at specified timepoints | µg/mL | MEAN |
| SECONDARY | Phase 1: Time to Observed Maximum Plasma Concentration | Cycle 1: predose, at 30 min (end of infusion), and at 1 hour and 8 hours on Day 1 and Day 15 | Pharmacokinetic Analysis Set: All treated participants who had at least 1 plasma concentration measured. Number Analyzed = participants who were evaluable at specified timepoints | hours | MEDIAN |
| SECONDARY | Phase 1: Changes From Baseline in Colony-Stimulating Factor-1 (CSF-1) and Interleukin (IL-34) Serum Concentrations | Baseline, Cycle 1 Day 8, Day 15, predose at Cycle 2 Day 1, Cycle 4 Day 1 (28-day cycles), and end of treatment (EOT) (median duration of treatment = 7 months) | Safety Analysis Set: All participants who received at least 1 dose of study treatment. Number Analyzed = participants who were evaluable at specified timepoints | pg/mL | MEDIAN |
| SECONDARY | Phase 1: Percent Change From Baseline in Nonclassical Monocytes (CD14+CD16++) | Baseline, Day 8, Day 15, predose at Cycle 2 Day 1, Cycle 4 Day 1, and EOT (median duration of treatment = 7 months) | Safety Analysis Set: All participants who received at least 1 dose of study treatment. Number Analyzed = participants who were evaluable at specified timepoints | percent change | MEDIAN |
| SECONDARY | Phase 1: Number of Participants Positive for Anti-Drug Antibodies (ADA) | Predose on Cycle 1 Day 1, Cycle 1 Day 15, Cycle 3 Day 1, Day 1 of each subsequent cycle through EOT plus 30 days after last dose (safety follow-up) (median duration of treatment = 7 months) | Safety Analysis Set: All participants who received at least 1 dose of study treatment. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Phase 2: Best Overall Response (BOR), as Defined by the 2014 NIH Consensus Development Project on Criteria for Clinical Trials in cGVHD | Day 1 of each 28-Day cycle up to Cycle 7 Day 1 | Safety Analysis Set: All participants who received at least 1 dose of study treatment. | percentage of participants | NUMBER |
1 to 10 of 18Source ClinicalTrials.gov
See all 18 outcomesResults by group118
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Phase 1: Number of Participants With DLTs | Phase 1 Dose Escalation: SNDX-6352 0.15 mg/kg | 0 | Participants | ||
| Phase 1: Number of Participants With DLTs | Phase 1 Dose Escalation: SNDX-6352 0.5 mg/kg | 0 | Participants | ||
| Phase 1: Number of Participants With DLTs | Phase 1 Dose Escalation: SNDX-6352 1 mg/kg | 0 | Participants | ||
| Phase 1: Number of Participants With DLTs | Phase 1 Dose Escalation: SNDX-6352 3 mg/kg | 2 | Participants | ||
| Phase 1: Number of Participants With DLTs | Phase 1 Dose Escalation: SNDX-6352 3 mg/kg Q4W | 0 | Participants | ||
| Phase 1: Recommended Phase 2 Dose (RP2D) | Phase 1 Dose Determination:SNDX-6352 | 1 | mg/kg | ||
| Phase 2: Overall Response Rate (ORR) as Assessed by the Number of Participants With Complete Response (CR) or Partial Response (PR) at Cycle 7 Day 1 (Day 168) | Phase 2 Dose Expansion: SNDX-6352 1 mg/kg Q2W | 39.1 | 95% Confidence Interval 19.71 to 61.46 | percentage of participants | |
| Phase 1: Area Under the Plasma Concentration-time Curve From Time 0 to the Last Measurable Concentration for SNDX-6352 | Phase 1 Dose Escalation: SNDX-6352 0.15 mg/kg Q2W | Cycle 1 Day 1 | 30.7 | Full Range 30.7 to 30.7 | µg*h/mL |
| Phase 1: Area Under the Plasma Concentration-time Curve From Time 0 to the Last Measurable Concentration for SNDX-6352 | Phase 1 Dose Escalation: SNDX-6352 0.5 mg/kg Q2W | Cycle 1 Day 1 | 96.2 | Full Range 96.2 to 96.2 | µg*h/mL |
| Phase 1: Area Under the Plasma Concentration-time Curve From Time 0 to the Last Measurable Concentration for SNDX-6352 | Phase 1 Dose Escalation: SNDX-6352 1 mg/kg Q2W | Cycle 1 Day 1 | 1546.0 | Full Range 898.0 to 2140.0 | µg*h/mL |
1 to 10 of 118Source ClinicalTrials.gov
See all 118 results by groupParticipant flow30
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Phase 1 Dose Escalation: SNDX-6352 0.15 mg/kg Q2W | 1 | |
| Overall Study | STARTED | Phase 1 Dose Escalation: SNDX-6352 0.5 mg/kg Q2W | 1 | |
| Overall Study | STARTED | Phase 1 Dose Escalation: SNDX-6352 1 mg/kg Q2W | 3 | |
| Overall Study | STARTED | Phase 1 Dose Escalation: SNDX-6352 3 mg/kg Q2W | 6 | |
| Overall Study | STARTED | Phase 1 Dose Escalation: SNDX-6352 3 mg/kg Q4W | 7 | |
| Overall Study | STARTED | Phase 2 Dose Expansion: SNDX-6352 1 mg/kg Q2W | 23 | |
| Overall Study | Received at Least 1 Dose of Study Treatment | Phase 1 Dose Escalation: SNDX-6352 0.15 mg/kg Q2W | 1 | |
| Overall Study | Received at Least 1 Dose of Study Treatment | Phase 1 Dose Escalation: SNDX-6352 0.5 mg/kg Q2W | 1 | |
| Overall Study | Received at Least 1 Dose of Study Treatment | Phase 1 Dose Escalation: SNDX-6352 1 mg/kg Q2W | 3 | |
| Overall Study | Received at Least 1 Dose of Study Treatment | Phase 1 Dose Escalation: SNDX-6352 3 mg/kg Q2W | 6 |
1 to 10 of 30Source ClinicalTrials.gov
See all 30 participant flowAdverse events1,140
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 | Events |
|---|---|---|---|---|---|---|
| other | Fatigue | General disorders | SNDX-6352 0.15 mg/kg Q2W | 0 | 1 | |
| other | Fatigue | General disorders | SNDX-6352 0.5 mg/kg Q2W | 1 | 1 | |
| other | Fatigue | General disorders | SNDX-6352 1 mg/kg Q2W | 12 | 26 | |
| other | Fatigue | General disorders | SNDX-6352 3 mg/kg Q2W | 3 | 6 | |
| other | Fatigue | General disorders | SNDX-6352 3 mg/kg Q4W | 2 | 6 | |
| other | Oedema peripheral | General disorders | SNDX-6352 0.15 mg/kg Q2W | 1 | 1 | |
| other | Oedema peripheral | General disorders | SNDX-6352 0.5 mg/kg Q2W | 0 | 1 | |
| other | Oedema peripheral | General disorders | SNDX-6352 1 mg/kg Q2W | 4 | 26 | |
| other | Oedema peripheral | General disorders | SNDX-6352 3 mg/kg Q2W | 1 | 6 | |
| other | Oedema peripheral | General disorders | SNDX-6352 3 mg/kg Q4W | 2 | 6 |
1 to 10 of 1,140Source ClinicalTrials.gov
See all 1,140 adverse eventsAdverse event totals15
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | SNDX-6352 0.15 mg/kg Q2W | 0 | 1 |
| Total, all-cause mortality | SNDX-6352 0.5 mg/kg Q2W | 0 | 1 |
| Total, all-cause mortality | SNDX-6352 1 mg/kg Q2W | 1 | 26 |
| Total, all-cause mortality | SNDX-6352 3 mg/kg Q2W | 0 | 6 |
| Total, all-cause mortality | SNDX-6352 3 mg/kg Q4W | 0 | 6 |
| Total, other adverse events | SNDX-6352 0.15 mg/kg Q2W | 1 | 1 |
| Total, other adverse events | SNDX-6352 0.5 mg/kg Q2W | 1 | 1 |
| Total, other adverse events | SNDX-6352 1 mg/kg Q2W | 26 | 26 |
| Total, other adverse events | SNDX-6352 3 mg/kg Q2W | 6 | 6 |
| Total, other adverse events | SNDX-6352 3 mg/kg Q4W | 6 | 6 |
1 to 10 of 15Source ClinicalTrials.gov
See all 15 adverse event totalsPapers2
The papers the trial lists on ClinicalTrials.gov.
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.
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/NCT03604692
GET https://conductscience.org/api/trials/v1/NCT03604692/results?page=2