Asciminib for Leukemia

Age: 18+
Sex: Any
Trial Phase: Phase 1
Sponsor: City of Hope Medical Center
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)
Breakthrough TherapyThis drug has been fast-tracked for approval by the FDA given its high promise

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment called asciminib for individuals with Philadelphia chromosome positive acute lymphoblastic leukemia (ALL). The goal is to determine the safety and effectiveness of asciminib in preventing leukemia recurrence after treatments such as stem cell or CAR T cell therapies. Participants must have recently undergone cellular therapies for their leukemia and currently show no signs of the disease. 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 treatment.

Do I have to stop taking my current medications for the trial?

The trial requires that you do not take strong inducers of CYP3A, and you should switch to an alternative at least one week before starting the study treatment. Other than this, the protocol does not specify if you need to stop other medications.

Is there any evidence suggesting that asciminib is likely to be safe for humans?

Research has shown that asciminib is generally safe for use. In studies with patients who have chronic myeloid leukemia (CML), asciminib was usually well-tolerated. The most common side effect was a low platelet count, known as thrombocytopenia. However, these studies suggest that patients can use asciminib for many years without serious safety concerns.

Comparisons with other CML treatments have found asciminib to be safer and easier to tolerate, resulting in fewer or less severe side effects than some other cancer treatments. While these results are encouraging, further research is important to confirm asciminib's safety for patients with Philadelphia chromosome-positive acute lymphoblastic leukemia (ALL).12345

Why do researchers think this study treatment might be promising?

Asciminib is unique because it targets a specific protein, ABL myristoyl pocket, which is different from the way traditional leukemia treatments like tyrosine kinase inhibitors work. This novel mechanism of action may offer a more precise way to attack cancer cells with potentially fewer side effects. Researchers are excited about asciminib because it represents a new approach in the fight against leukemia, with the potential to improve patient outcomes where other treatments may have limited effectiveness.

What evidence suggests that asciminib might be an effective treatment for leukemia?

Studies have shown that asciminib, the treatment being tested in this trial, effectively treats certain types of leukemia, particularly chronic myeloid leukemia (CML). In one study, 45% of patients experienced a significant decrease in cancer indicators. Asciminib has also proven safe and effective over the long term for people with CML. It blocks specific proteins that cancer cells need to grow. While most research has focused on CML, these results suggest that asciminib might also help with other types of leukemia, such as Philadelphia chromosome-positive acute lymphoblastic leukemia (ALL).46789

Who Is on the Research Team?

IA

Ibrahim Aldoss

Principal Investigator

City of Hope Comprehensive Cancer Center

Are You a Good Fit for This Trial?

Adults with Philadelphia chromosome positive acute lymphoblastic leukemia who have had cellular therapies like stem cell transplants or CAR T cell therapy. The trial is to see if the cancer can be kept at bay after initial treatments.

Inclusion Criteria

Serum creatinine <1.5 mg/dL
Participant meets one of the following criteria:
Documented informed consent of the participant and/or legally authorized representative
See 22 more

Exclusion Criteria

Prospective participants who, in the opinion of the investigator, may not be able to comply with all study procedures (including compliance issues related to feasibility/logistics)
Fridericia's corrected QT interval (QTcF) at screening ≥ 450 msec (male patients), ≥ 470 msec (female patients)
Any other condition that would, in the Investigator's judgment, contraindicate the patient's participation in the clinical study due to safety concerns with clinical study procedures
See 13 more

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

Echocardiography and other assessments

Treatment

Participants receive asciminib orally once or twice daily on days 1-28 of each cycle. Cycles repeat every 28 days for 12 cycles in the absence of disease progression or unacceptable toxicity.

12 months
Regular visits for bone marrow biopsy, aspirate, and blood sample collection

Follow-up

Participants are monitored for safety and effectiveness after treatment

1 year
Follow-up at 30 days then every 3 months

What Are the Treatments Tested in This Trial?

Interventions

  • Asciminib
Trial Overview The trial is testing asciminib's safety, side effects, and best dose as a maintenance treatment post-cellular therapies in adults with a specific type of leukemia. It includes biospecimen collection, bone marrow procedures, heart tests, and surveys.
How Is the Trial Designed?
1Treatment groups
Experimental Treatment
Group I: Treatment (Asciminib)Experimental Treatment6 Interventions

Find a Clinic Near You

Who Is Running the Clinical Trial?

City of Hope Medical Center

Lead Sponsor

Trials
614
Recruited
1,924,000+

National Cancer Institute (NCI)

Collaborator

Trials
14,080
Recruited
41,180,000+

Citations

Real‐World Efficacy Profile of Compassionate Use ...This retrospective Italian analysis reports the efficacy and safety outcomes of asciminib in treating 77 CML patients in chronic phase (CML‐CP) ...
Treatment with Asciminib As a Second Line after One Prior ...Over a median follow-up of 50.8 weeks, MR4 or better was achieved by 45.0% of patients as best response on asciminib (resistance/suboptimal ...
Asciminib monotherapy in patients with chronic myeloid ...With up to 8.4 years of treatment, asciminib continued to demonstrate long-term safety and efficacy in this population.
Meta-Analysis Highlights Efficacy, Safety Benefits With ...Asciminib shows superior efficacy and lower adverse effects compared to other TKIs in CP-CML patients, with improved major molecular response ...
Real-world asciminib treatment outcomes in CML-CPThis study described treatment patterns and real-world clinical outcomes among patients with CML-CP treated with asciminib in US clinical practice.
6.pubmed.ncbi.nlm.nih.govpubmed.ncbi.nlm.nih.gov/40510784/
The efficacy and safety of asciminib treatment in patients ...Asciminib is effective in the treatment of patients with CML, and the most common AE during treatment is thrombocytopenia.
Asciminib (ASC) Demonstrates Favorable Safety and ...Asciminib (ASC) demonstrates favorable safety and tolerability compared with each investigator-selected tyrosine kinase inhibitor (IS TKI) in newly diagnosed ...
Updated Efficacy and Safety Data From a Phase I TrialCML-466 Asciminib Provides Durable Molecular Responses in Patients With Chronic Myeloid Leukemia ... safety data in patients with CML-CP with the T315I ...
Asciminib in Newly Diagnosed Chronic Myeloid LeukemiaAsciminib showed superior efficacy and a favorable safety profile in patients with newly diagnosed chronic-phase CML.
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