Tagraxofusp + Chemotherapy for Acute Lymphoblastic Leukemia
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial explores a new treatment for certain blood cancers, specifically acute lymphoblastic leukemia and lymphoblastic lymphoma, that have returned or don't respond to treatment. It combines tagraxofusp, a targeted therapy that kills specific cancer cells, with chemotherapy to determine if this approach can better control the disease. The trial seeks participants with CD123-positive acute lymphoblastic leukemia or lymphoblastic lymphoma that has relapsed or is refractory. As a Phase 1 trial, the research focuses on understanding how the treatment works in people, offering participants the opportunity to be among the first to receive this new therapy.
Do I need to stop my current medications to join the trial?
The trial protocol does not specify if you need to stop taking your current medications. However, you cannot have taken any investigational antileukemic or chemotherapy agents in the last 7 days before joining the study, unless you have fully recovered from side effects or have life-threatening disease. Hydroxyurea and dexamethasone are exceptions and can be taken before the study.
Is there any evidence suggesting that this trial's treatments are likely to be safe?
Research has shown that tagraxofusp has a mixed safety record. Approximately 3% of patients experienced severe reactions, including capillary leak syndrome (CLS), a potentially fatal condition where fluid leaks from small blood vessels. Additionally, about 8% of patients discontinued treatment due to side effects. Common side effects included increased liver enzymes, indicating possible liver stress.
Despite these concerns, tagraxofusp is approved for treating blastic plasmacytoid dendritic cell neoplasm (BPDCN), suggesting some established safety. Participants should discuss potential risks and benefits with their doctor to make an informed decision.12345Why are researchers excited about this study treatment for acute lymphoblastic leukemia?
Tagraxofusp is unique because it targets a specific protein called CD123, which is often present on the surface of cancer cells in acute lymphoblastic leukemia (ALL). Most current treatments for ALL, like chemotherapy regimens, primarily focus on killing rapidly dividing cells non-selectively, which can affect healthy cells too. Tagraxofusp, on the other hand, is designed to more precisely target leukemia cells, potentially reducing damage to normal cells and leading to fewer side effects. Researchers are excited about this treatment because it offers a new mechanism of action that could improve outcomes for patients who may not respond well to standard therapies.
What evidence suggests that tagraxofusp and chemotherapy might be an effective treatment for acute lymphoblastic leukemia?
Research shows that tagraxofusp holds promise in treating certain cancers. It has proven effective against a rare cancer called blastic plasmacytoid dendritic-cell neoplasm (BPDCN). Tagraxofusp targets a specific marker on cancer cells, CD123, and delivers a substance that kills these cells. Early results indicate that patients treated with tagraxofusp often experience significant tumor shrinkage. In this trial, participants will receive tagraxofusp combined with chemotherapy to evaluate its potential for treating acute lymphoblastic leukemia (ALL). Although this treatment is new for ALL, its success in similar conditions suggests it could benefit patients with CD123 positive relapsed or refractory ALL.36789
Who Is on the Research Team?
Nicholas Short
Principal Investigator
M.D. Anderson Cancer Center
Are You a Good Fit for This Trial?
Adults aged 18-70 with CD123 positive relapsed or refractory acute lymphoblastic leukemia or lymphoblastic lymphoma can join. They must have certain normal blood test results, no serious heart, liver, lung diseases, HIV, hepatitis B/C and agree to use effective birth control. Pregnant women and those with other life-threatening cancers cannot participate.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Tagraxofusp and Chemotherapy Phase
Patients receive tagraxofusp and chemotherapy drugs in cycles to treat CD123 positive relapsed or refractory acute lymphoblastic leukemia or lymphoblastic lymphoma.
Tagraxofusp and Maintenance Phase
Patients receive maintenance therapy with tagraxofusp and other drugs to sustain remission.
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- Dexamethasone
- Tagraxofusp
Trial Overview
The trial is testing tagraxofusp combined with low-intensity chemotherapy in patients whose leukemia or lymphoma has returned after treatment or hasn't responded to previous treatments. Tagraxofusp targets cancer cells by linking a toxin to IL3 which is present on the cells' surface.
How Is the Trial Designed?
1
Treatment groups
Experimental Treatment
See Detailed Description
Tagraxofusp is already approved in United States, European Union for the following indications:
- Blastic plasmacytoid dendritic cell neoplasm (BPDCN)
- Blastic plasmacytoid dendritic cell neoplasm (BPDCN)
Find a Clinic Near You
Who Is Running the Clinical Trial?
M.D. Anderson Cancer Center
Lead Sponsor
Citations
Approval of tagraxofusp-erzs for blastic plasmacytoid ...
Tagraxofusp-erzs was the first agent approved for BPDCN (for patients ages 2 years and older), and importantly, established this drug as the first CD123- ...
NCT02113982 | Tagraxofusp in Patients With Blastic ...
This is a 4-stage, non-randomized, open-label, dose escalation and expansion, multicenter study. A cycle of therapy is 21 days.
Long-Term Benefits of Tagraxofusp for Patients With Blastic ...
In first-line patients with BPDCN, TAG monotherapy resulted in high and durable responses, allowing many to bridge to stem-cell transplant.
Tagraxofusp in Blastic Plasmacytoid Dendritic-Cell Neoplasm
Tagraxofusp has shown potent antitumor activity against BPDCN cells in both in vitro and in vivo models, with cytotoxicity at femtomolar concentrations.
Efficacy and manageable safety of tagraxofusp in blastic ...
These cases highlight the efficacy and safety of TAG in pediatric and AYA patients for whom there is no other approved BPDCN therapy.
Safety Profile | ELZONRIS® (tagraxofusp-erzs)
Four (3%) patients had fatal adverse reactions, all of which were related to capillary leak syndrome (CLS).1. Overall, 10 (8%) patients discontinued ...
Tagraxofusp (SL-401) therapy for blastic plasmacytoid ...
2.3.2 Safety data. The most common individual treatment emergent adverse events (TEAEs) reported to date with tagraxofusp, regardless of dose, were ALT ...
761116Orig1s000 - accessdata.fda.gov
Review of Safety. Safety Review Approach. The clinical review of safety for this BLA was based on all available safety data from studies. STML ...
9.
targetedonc.com
targetedonc.com/view/interpreting-safety-and-efficacy-data-for-tagraxofusp-in-bpdcnInterpreting Safety and Efficacy Data for Tagraxofusp in ...
BORATE: Technically you can continue [indefinitely], until you see intolerable AEs or progression. 1 Most patients end up dose reducing [or] ...
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