Phase 2 COPD Trial, Recruiting NCT04710576 Sponsor: Syndax Pharmaceuticals Condition: COPD
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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.

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

NCT04710576
Recruiting Phase 2 🇦🇺 Australian site

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.

This is a simplified plain English summary of the eligibility criteria. Full criteria are set by the trial investigators and may include additional requirements not shown here. Never self-exclude from a trial based on this summary. Contact the trial site directly to confirm your eligibility.
Last synced 16 July 2026
Phase 2: approximately ~30% of drugs entering this phase reach regulatory approval, based on published industry-wide historical data. This is not specific to this trial.
Phase success rates shown are historical industry-wide averages based on published data. They are not a prediction for this specific trial or your individual situation.

Contact this trial

Principal Investigator: Ellen Hooper, M.D., Syndax Pharmaceuticals

Phone: 781-419-1400

Australian sites

The Royal Children's Hospital, Parkville, Victoria
Westmead Hospital, Westmead,

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

Status
Recruiting
Phase
Phase 2
Sponsor
Syndax Pharmaceuticals
Registry
ClinicalTrials.gov
Start date
4 March 2021
Est. completion
7 April 2023

Where this trial is recruiting

🇦🇺 Australia 🇧🇪 Belgium 🇨🇦 Canada 🇫🇷 France 🇩🇪 Germany 🇬🇷 Greece 🇮🇱 Israel 🇮🇹 Italy 🇵🇱 Poland 🇵🇹 Portugal 🇸🇬 Singapore 🇰🇷 South Korea 🇪🇸 Spain 🇹🇼 Taiwan 🇬🇧 United Kingdom 🇺🇸 United States

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?

Expanded access pathways

If this trial is not available to you, other access pathways may exist. In Australia, the TGA Special Access Scheme allows access to unapproved therapeutic goods for individual patients.

TGA Special Access Scheme information

Find other recruiting trials on ClinicalTrials.gov

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.

Trial recruitment status can change without notice between our nightly data updates. Always contact the trial site directly to confirm current recruitment status before making any decisions or travel arrangements.

View original record on ClinicalTrials.gov