Axatilimab for Graft-versus-Host Disease
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial examines the effectiveness of a new treatment, Axatilimab (a monoclonal antibody), for children and teens with chronic graft-versus-host disease (cGVHD). cGVHD can occur after a bone marrow or stem cell transplant when the donated cells attack the recipient's body. The trial compares Axatilimab to the best available therapies (BAT) commonly used by doctors. It targets children aged 2 to 17 with moderate to severe cGVHD who have already tried at least two other treatments. Children with ongoing cGVHD symptoms despite previous treatments may be suitable candidates for this trial. As a Phase 2 trial, this research measures Axatilimab's effectiveness in an initial, smaller group of patients, providing an opportunity to access a potentially effective treatment early.
Will I have to stop taking my current medications?
The trial allows the use of systemic corticosteroids if the dose is stable for at least 2 weeks before starting the trial. However, if you are on CNIs or mTOR inhibitors, you must not have started them within 2 weeks before the trial begins. Other medications may be allowed, but the protocol does not specify all details.
Is there any evidence suggesting that this trial's treatments are likely to be safe?
Research has shown that axatilimab is generally safe for patients with chronic graft-versus-host disease (cGVHD). A safety review found no new safety issues with the treatment. Over time, patients experienced fewer treatment-related side effects. Another study confirmed that axatilimab was effective and well-tolerated, with many patients experiencing positive results. These findings suggest that axatilimab is a safe option for treating cGVHD.12345
Why do researchers think this study treatment might be promising for graft-versus-host disease?
Researchers are excited about axatilimab for treating graft-versus-host disease (GVHD) because it offers a novel approach by targeting the CSF-1R pathway, which is different from traditional therapies like corticosteroids or immunosuppressants. This mechanism of action could potentially offer a more precise treatment, leading to fewer side effects. Additionally, axatilimab has shown promise in early studies for reducing inflammation and tissue damage, offering hope for improved outcomes in patients who don't respond well to current options.
What evidence suggests that Axatilimab might be an effective treatment for graft-versus-host disease?
Research has shown that Axatilimab, one of the treatments in this trial, may help treat chronic Graft-versus-Host Disease (GVHD). In one study, 58% to 65% of patients saw improvement, meaning many felt better. Additionally, about 64% of patients experienced significant relief from their symptoms. These results suggest that Axatilimab could be a good option for those who haven't had success with other treatments. Overall, early data on Axatilimab indicates strong potential for helping people with this difficult condition. Participants in this trial may receive either Axatilimab or the Best Available Treatment (BAT) as determined by the investigator.2678
Who Is on the Research Team?
Incyte Medical Monitor
Principal Investigator
Incyte Corporation
Are You a Good Fit for This Trial?
This trial is for kids aged 2-18 with chronic Graft-Versus-Host Disease who've tried at least two systemic therapies. They must be on a stable dose of corticosteroids or other approved treatments and have had an allo-HCT from any donor.Inclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive either Axatilimab or Best Available Therapy for chronic graft-versus-host disease
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- Axatilimab
Trial Overview
The study compares Axatilimab to the Best Available Therapy in children with chronic GVHD after failing previous treatments. It aims to see if Axatilimab is more effective in managing their condition.
How Is the Trial Designed?
2
Treatment groups
Experimental Treatment
Best Available Therapy (BAT) will be selected by the Investigator for each participant. BAT may not include experimental agents (i.e. those not approved for the treatment of any indication) as well as a limited number of other selected drugs in accordance with the protocol-defined requirements.
Axatilimab at the protocol-defined dose.
Find a Clinic Near You
Who Is Running the Clinical Trial?
Incyte Corporation
Lead Sponsor
Steven Stein
Incyte Corporation
Chief Medical Officer since 2015
MD from University of Witwatersrand
Hervé Hoppenot
Incyte Corporation
Chief Executive Officer since 2014
MBA from ESSEC Business School
Citations
Efficacy and Safety of Axatilimab in Chronic Graft-Versus ...
The pooled Overall Response Rate was 58% (95% CI: 45-72%). Clinically meaningful symptom improvement occurred in 64% (95% CI: 53-75%). Any-grade ...
Efficacy and safety of axatilimab in chronic graft-versus- ...
Axatilimab demonstrates robust clinical activity in refractory cGVHD. · Overall response rate reached 65 % in clinical trials. · 64 % of patients showed ...
Axatilimab in Recurrent or Refractory Chronic Graft-versus- ...
In an early-phase clinical study involving patients with recurrent or refractory chronic GVHD, axatilimab showed promising preliminary efficacy ...
4.
ashpublications.org
ashpublications.org/blood/article/142/Supplement%201/1/499054/Safety-and-Efficacy-of-Axatilimab-at-3-DifferentSafety and Efficacy of Axatilimab at 3 Different Doses in ...
Safety and efficacy of Axatilimab at 3 different doses in patients with chronic graft-versus-host disease (AGAVE-201)
5.
ashpublications.org
ashpublications.org/blood/article/146/Supplement%201/6008/549370/Safety-analysis-of-axatilimab-in-patients-withSafety analysis of axatilimab in patients with chronic graft ...
Safety analysis of axatilimab in patients with chronic graft-versus-host disease in an expanded access program Available · Ris (Zotero) ...
6.
targetedonc.com
targetedonc.com/view/axatilimab-shows-long-term-tolerability-in-pretreated-chronic-gvhdAxatilimab Shows Long-Term Tolerability in Pretreated ...
Key Takeaways · Axatilimab showed a consistent safety profile in cGVHD patients, with no new safety signals and a decrease in TEAEs over time.
Safety and Efficacy of Axatilimab in Patients with Chronic ...
Conclusion. In AGAVE-201, the primary endpoint was met. AXA 0.3 mg/kg Q2W was well tolerated and resulted in robust clinical activity and durable responses.
Safety analysis of axatilimab in patients with chronic graft- ...
Safety analysis of axatilimab in patients with chronic graft-versus-host disease in an expanded access program ... All data were summarized using ...
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