Trial results
AI generated results summary. Written by an AI model from the official source data and checked on a sample basis. It can contain mistakes, so confirm anything important against the original source. How we use AI
According to the results reported on ClinicalTrials.gov, this trial (NCT04710576) looked at a medicine called axatilimab in people with chronic graft-versus-host disease (cGVHD) — a condition that can occur after a bone marrow or stem cell transplant, where the donated cells attack the recipient's body. A total of 241 people took part, split across three groups that each received a different dose or dosing schedule of axatilimab. The trial's main goal was to measure how many participants showed a meaningful improvement in their cGVHD symptoms within the first six treatment cycles, based on a recognised set of medical criteria. The reported data shows that in the group receiving the lowest dose (0.3 mg/kg every two weeks), 73.8% of participants were recorded as having a response — meaning their condition either fully resolved or partially improved without getting worse elsewhere. In the group receiving the middle dose (1 mg/kg every two weeks), the reported response rate was 66.7%. In the group receiving the highest dose given less frequently (3 mg/kg every four weeks), the reported response rate was 50.0%. It is worth noting that very few participants formally "completed" the study as defined in the trial records (0–1 per group), and the reported data for all secondary outcome measures — including how long responses lasted, symptom relief, and organ-specific responses — was not reported in the data submitted to ClinicalTrials.gov. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.
View full results on ClinicalTrials.gov ↗ · Read the linked publication on PubMed (PMID 39292927) ↗
These are the results as reported to ClinicalTrials.gov, not medical advice. Verify independently with the trial site and discuss what they mean for you with your doctor.
Phase 2 COPD Trial, Recruiting
Who may be able to join
AI generated eligibility summary. Written by an AI model from the official source data and checked on a sample basis. It can contain mistakes, so confirm anything important against the original source. How we use AI
Who might be able to join this trial:
- People aged 2 years or older at the time of signing the consent form.
- People who have received an allogeneic stem cell transplant (a transplant using stem cells from a donor) and have active chronic graft-versus-host disease (cGVHD) — a condition where the donor cells attack the recipient's body — that requires whole-body immune-suppressing treatment.
- People whose cGVHD has not responded to, or has come back after, at least 2 different systemic (whole-body) treatments, meaning the disease kept spreading, got worse, didn't improve within 3 months, or returned after an initial response.
- People who have a combination of both acute and chronic GVHD symptoms at the same time (sometimes called "overlap syndrome").
- People with a general health/functioning score of 60 or above on standard performance scales used for their age group (confirm with trial site).
- People with kidneys, organs, and bone marrow functioning at adequate levels, as measured by blood tests done within 14 days before joining the trial.
- People with a kidney function measurement (creatinine clearance) of 30 mL/minute or above, calculated using standard medical formulas.
- People who are able to use contraception in line with local guidelines for clinical trial participants.
- People who may be taking corticosteroids (a type of immune-suppressing medication), as long as the dose has been stable for at least 2 weeks before starting the trial — topical (skin) and inhaled corticosteroids are also allowed.
- People who may be taking certain other immune-suppressing medications (CNI or mTOR inhibitors such as sirolimus or everolimus), though these are not required.
- People (or their parent/guardian, for children) who are able to give informed consent and agree to follow the trial requirements.
Who may not be able to join:
- People who have acute GVHD only, with no signs of chronic GVHD.
- People who show any signs that their original cancer has come back, or who have developed a post-transplant condition called lymphoproliferative disease.
- People with a past history of acute or chronic pancreatitis (inflammation of the pancreas).
- People with a past history of myositis (inflammation of the muscles).
- People with a serious illness, an uncontrolled infection, or any other health condition that the trial doctor considers would make participation unsafe.
- People who have AIDS (advanced HIV infection).
- People with active Hepatitis B or Hepatitis C infections, as defined by specific blood test results (confirm with trial site).
- People diagnosed with another cancer (other than the one the transplant was done for) within the 3 years before joining the trial — exceptions may apply for certain fully treated cancers, subject to approval by the trial's medical oversight team.
- People who are pregnant or breastfeeding.
- People who have previously been treated with therapies that target a protein called CSF1-R.
- People currently taking other cGVHD treatments beyond corticosteroids, CNI, or mTOR inhibitors.
- People who have taken certain other treatments recently and have not completed the required washout (clearance) period of 2 weeks or 5 half-lives of the drug, whichever is shorter.
- People who have received another experimental treatment within 28 days before joining this trial.
- People currently participating in another interventional (treatment-testing) study.
Important: Always verify eligibility with the trial site directly before applying.
Based on publicly available eligibility criteria from ClinicalTrials.gov. Verify directly with the trial site before acting. This is not medical advice.
Contact this trial
Principal Investigator: Ellen Hooper, M.D., Syndax Pharmaceuticals
Phone: 781-419-1400
Australian sites
Contact details sourced from ClinicalTrials.gov. Verify directly with the trial site before attending.
GP referral letter
Print a one-page summary to share with your doctor.
Trial details
Where this trial is recruiting
2 site(s) in Australia. Confirm current status and contact details directly with the trial site.
Primary endpoints
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)
Can't join this trial?
Data last synced from ClinicalTrials.gov: 16 July 2026. Trial status can change. Always verify current status directly with the trial site before making any decision.