Emapalumab for Aplastic Anemia
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial aims to determine if starting with emapalumab (an immunotherapy drug) can enhance the effectiveness of standard treatments for aplastic anemia, a condition where the body stops producing enough blood cells. Participants will follow one of two approaches: taking emapalumab for 6 weeks before either continuing with standard immune-suppressing drugs or undergoing a stem cell transplant. The trial seeks individuals recently suspected of having severe aplastic anemia, characterized by low blood counts and no signs of leukemia or other specific blood disorders. As a Phase 2 trial, this research measures the treatment's effectiveness in an initial, smaller group, offering participants the opportunity to contribute to significant advancements in aplastic anemia treatment.
Will I have to stop taking my current medications?
The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the trial coordinators or your doctor.
Is there any evidence suggesting that this trial's treatments are likely to be safe?
Research has shown that emapalumab presents some safety concerns. In past studies, 53% of patients experienced serious side effects, including infections and bleeding in the stomach or intestines. However, emapalumab is already approved for other uses, indicating that its safety profile is well-studied. In this trial, participants will take emapalumab for six weeks before switching to other treatments. Prospective participants should consider these potential risks and discuss them with the medical team.12345
Why are researchers excited about this trial's treatments?
Unlike the standard treatments for aplastic anemia, which often rely on immunosuppressive therapies like equine anti-thymocyte globulin (hATG) and cyclosporin (CsA) or hematopoietic stem cell transplant (HCT), emapalumab offers a fresh approach by targeting the interferon gamma pathway. This makes it unique because it specifically inhibits a key immune signal that may contribute to the disease. Researchers are excited about emapalumab because this targeted action could potentially improve outcomes by reducing immune system overactivity more precisely and effectively than current options. Additionally, by combining emapalumab with standard treatments or HCT, there's hope for enhanced efficacy and potentially fewer side effects.
What evidence suggests that emapalumab might be an effective treatment for aplastic anemia?
Research has shown that emapalumab may help treat conditions like primary hemophagocytic lymphohistiocytosis (HLH). One study found that 63% of patients responded positively to emapalumab, meaning their condition improved. Additionally, 70% of these patients advanced to further treatment with a hematopoietic stem cell transplant (HCT). In this trial, participants will first receive emapalumab for 6 weeks. After this initial treatment, one group will receive standard immunosuppressive therapy (IST) with equine anti-thymocyte globulin (hATG) and cyclosporin (CsA) along with a lower dose of emapalumab, while another group will proceed to a standard hematopoietic stem cell transplant (HCT). Although there is limited direct information on emapalumab's effects on aplastic anemia, the drug blocks a protein called interferon gamma, which is believed to reduce harmful inflammation in the body. These early findings suggest that emapalumab might help manage severe aplastic anemia.34678
Who Is on the Research Team?
Andromachi Scaradavou, MD
Principal Investigator
Memorial Sloan Kettering Cancer Center
Are You a Good Fit for This Trial?
This trial is for children and young adults under 25 with newly diagnosed severe Aplastic Anemia (sAA), showing very low blood cell counts and bone marrow activity. They must be able to handle the treatment, have not received prior sAA treatments, and can't have leukemia or inherited bone marrow failure.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive Emapalumab for 6 weeks, followed by either standard IST or HCT
Standard IST
Participants receive standard IST with equine anti-thymocyte globulin (hATG) and cyclosporin (CsA) in addition to a lower dose of emapalumab
HCT
Participants undergo a standard hematopoietic stem cell transplant (HCT)
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- Emapalumab
Emapalumab is already approved in United States for the following indications:
- Primary hemophagocytic lymphohistiocytosis (HLH) with refractory, recurrent or progressive disease or intolerance with conventional HLH therapy
Find a Clinic Near You
Who Is Running the Clinical Trial?
Memorial Sloan Kettering Cancer Center
Lead Sponsor