In short
Clinical trials are investigating Axatilimab in people with chronic graft-versus-host disease and idiopathic pulmonary fibrosis. These studies are looking at safety, effectiveness, and how Axatilimab compares with placebo or best available therapy. The trials include adults and pediatric participants, and they are in Phase 2 and Phase 3.
Key points
- Clinical trials of Axatilimab are mainly focused on chronic graft-versus-host disease, including adults with later-line disease, newly diagnosed patients, and pediatric participants. One Phase 2 study is also looking at idiopathic pulmonary fibrosis. The trial designs include randomized, open-label, double-blind, and placebo-controlled studies. Some trials compare Axatilimab with best available therapy, while others compare it with placebo or study it in combination with ruxolitinib and corticosteroids. The main outcomes include overall response, event-free survival, and lung function change. Most studies are currently authorised and are testing how well Axatilimab works in these different patient groups.
Clinical trial overview
The trial data show that Axatilimab is being studied in people with chronic graft-versus-host disease and idiopathic pulmonary fibrosis. These studies are designed to look at how well the treatment works, along with safety and tolerability in some trials. The research includes both Phase 2 and Phase 3 studies.
Studies in chronic graft-versus-host disease
Most of the trials are in people with chronic graft-versus-host disease (cGVHD), a long-term condition that can happen after stem cell transplant. One Phase 3 study compares Axatilimab with best available therapy in participants who have already had at least 2 prior lines of systemic therapy, meaning at least 2 earlier body-wide treatments. Its main goal is to compare efficacy, using response at 6 months as the key measure.
Another Phase 2 study looks at Axatilimab in combination with ruxolitinib in people with newly diagnosed cGVHD. This study is randomized and multicenter, which means participants are assigned to groups by chance and the trial is run at more than one site. The study aims to see the early benefit of the combination and how much Axatilimab adds to the treatment effect.
A different Phase 3 study tests Axatilimab plus corticosteroids as the first treatment for moderate or severe cGVHD. It compares Axatilimab with placebo, so some participants receive an inactive treatment for comparison. The trial measures event-free survival, which is the time before a major study event happens, such as new treatment, death, or treatment failure.
One Phase 2 study tests 3 different Axatilimab dose schedules in people with cGVHD after failure of at least 2 prior lines of therapy. The study summary says it is looking at the overall response rate in the first 6 cycles. Another Phase 2 study is planned for pediatric participants with cGVHD after at least 2 prior lines of systemic therapy, and it compares Axatilimab with best available therapy.
Study in idiopathic pulmonary fibrosis
One Phase 2 study is in people with idiopathic pulmonary fibrosis (IPF), a lung disease that causes scarring and can make breathing harder. This study is randomized, double-blind, placebo-controlled, and multicenter. In a double-blind study, neither the participants nor the researchers know who is receiving the study treatment or the placebo during the trial.
The main outcome in this IPF study is the annualized rate of decline in forced vital capacity (FVC) over 26 weeks. FVC is a lung test that measures how much air a person can forcefully breathe out after a deep breath. The study aims to see the effect of Axatilimab compared with placebo on lung function from baseline to Week 26.
Trial designs and comparison groups
The trials use several common research designs, including randomized, open-label, double-blind, and placebo-controlled methods. Randomized means treatment groups are assigned by chance, which helps make the groups more similar at the start. Open-label means everyone knows which treatment is being given.
The comparison groups include placebo, best available therapy, corticosteroids, and ruxolitinib-based treatment. These comparisons help researchers judge whether Axatilimab improves outcomes beyond the current treatment options used in the study.
Main outcomes being measured
In cGVHD studies, the main outcome is often overall response, which includes complete response or partial response without needing new systemic therapy by 6 months. The response is assessed using the 2014 NIH criteria, which are standard rules for measuring disease improvement in cGVHD.
In the Phase 3 study of initial cGVHD treatment, the key endpoint is event-free survival. In the IPF study, the key endpoint is the yearly rate of decline in FVC over 26 weeks. These endpoints help show whether the study treatment changes the course of the disease in a meaningful way.
Who may be able to participate
The trial data show several target groups, but eligibility differs by study. Some studies are for adults with cGVHD after at least 2 prior lines of systemic therapy. One study is for newly diagnosed cGVHD, and another is for moderate or severe cGVHD as initial treatment.
One trial is specifically for pediatric participants with cGVHD after at least 2 prior lines of systemic therapy. Another study includes subjects with idiopathic pulmonary fibrosis. The trial records provided here do not give full eligibility rules, so the exact entry criteria can differ from one study to another.