Asciminib for Chronic Myeloid Leukemia

GC
Overseen ByGhayas C. Issa
Age: 18+
Sex: Any
Trial Phase: Phase 2
Sponsor: M.D. Anderson Cancer Center
Must be taking: Tyrosine kinase inhibitors
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)
Prior Safety DataThis treatment has passed at least one previous human trial
Breakthrough TherapyThis drug has been fast-tracked for approval by the FDA given its high promise
Approved in 2 JurisdictionsThis treatment is already approved in other countries

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 chronic myeloid leukemia (CML) who are already taking a tyrosine kinase inhibitor (TKI). The researchers aim to determine if adding asciminib can more effectively stop cancer cell growth compared to using the TKI alone. Suitable participants have CML with specific gene markers, have been on nilotinib or dasatinib (types of TKI) for at least two years without recent treatment changes, and have stable but detectable cancer markers. As a Phase 2 trial, this research focuses on measuring the treatment's effectiveness in an initial, smaller group of participants.

Do I need to stop my current medications for the trial?

The trial does not specify if you need to stop your current medications, but it does require that you continue taking your current tyrosine kinase inhibitor (like nilotinib or dasatinib). If you are on medications that affect the heart's rhythm or certain liver enzymes, you may need to stop them at least one week before starting the trial.

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

Research has shown that asciminib is safe for people with chronic myeloid leukemia (CML). In earlier studies, patients using asciminib tolerated it well over a long time without serious side effects.

Another study found that asciminib is safer and easier to tolerate than traditional medications that stop cancer cell growth. This suggests asciminib might be a safer choice for some patients.

In real-life situations, even patients who didn't respond well to other treatments found asciminib effective and safe. After more than eight years of use, it has continued to show strong safety results.

Overall, evidence suggests that asciminib is generally safe and well-tolerated for treating CML.12345

Why are researchers excited about this study treatment for chronic myeloid leukemia?

Asciminib is unique because it targets a specific part of the BCR-ABL1 protein, known as the myristoyl pocket, which is different from how current treatments like dasatinib and nilotinib work. This new mechanism of action makes asciminib particularly promising, as it can potentially overcome resistance that some patients develop to existing therapies. Researchers are excited about asciminib because it might offer a more effective treatment option for chronic myeloid leukemia, especially for those who have limited responses to other treatments.

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

Research has shown that asciminib holds promise for treating chronic myeloid leukemia (CML). In this trial, participants will receive asciminib alongside standard treatments such as dasatinib or nilotinib. Studies have found that combining asciminib with these standard treatments can greatly improve outcomes. It outperforms other common therapies, leading to a greater reduction in cancer cells, which is a positive sign. Additionally, asciminib tends to cause fewer side effects, potentially making it safer for long-term use. These findings suggest that asciminib could be a strong option for effectively managing CML.13567

Who Is on the Research Team?

Ghayas C. Issa | MD Anderson Cancer Center

Ghayas Issa, MD

Principal Investigator

M.D. Anderson Cancer Center

Are You a Good Fit for This Trial?

This trial is for chronic myeloid leukemia patients who've been on tyrosine kinase inhibitor therapy for at least 2 years without needing changes in the last 6 months. They must have certain levels of disease markers and normal organ function tests. Pregnant women, those with severe heart conditions or infections like HIV, and anyone with a history of pancreatitis or other active cancers are excluded.

Inclusion Criteria

Creatinine =<1.5 x institutional upper limit of normal
I agree to use effective birth control during and after the study.
I've been on TKI therapy for 2+ years without changes in the last 6 months.
See 10 more

Exclusion Criteria

I have had a heart attack or severe heart issues in the last 6 months.
I am allergic or have had a bad reaction to ABL001 or its ingredients.
I am not taking strong medication that affects liver enzymes or cannot stop it before starting the study treatment.
See 11 more

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive asciminib orally twice daily for up to 36 months while receiving standard of care dasatinib or nilotinib

36 months

Follow-up

Participants are monitored for safety and effectiveness after treatment completion

6 months
Every 4-8 weeks

Long-term follow-up

Participants are monitored every 3-6 months for long-term outcomes

Up to 6 years

What Are the Treatments Tested in This Trial?

Interventions

  • Asciminib
Trial Overview The study is testing ABL001's effectiveness when combined with tyrosine kinase inhibitors in treating chronic myeloid leukemia. It aims to see if this combination works better than the inhibitor alone by blocking enzymes that cancer cells need to grow.
How Is the Trial Designed?
1Treatment groups
Experimental Treatment
Group I: Treatment (asciminib)Experimental Treatment1 Intervention

Find a Clinic Near You

Who Is Running the Clinical Trial?

M.D. Anderson Cancer Center

Lead Sponsor

Trials
3,107
Recruited
1,813,000+

National Cancer Institute (NCI)

Collaborator

Trials
14,080
Recruited
41,180,000+

Citations

Asciminib in Newly Diagnosed Chronic Myeloid LeukemiaHere we report the primary results of ASC4FIRST, a pivotal phase 3, randomized trial assessing the efficacy and safety of asciminib as compared ...
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) ...
Novartis Scemblix® Phase III data first to show superior ...Scemblix is the first CML treatment to show significantly better efficacy compared to investigator-selected standard-of-care TKIs.
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 ...
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.
Longer-term data for Novartis Scemblix® reinforce superior ...Asciminib Continues to Provide Superior Efficacy and Favorable Safety and Tolerability vs Tyrosine Kinase Inhibitors In Newly Diagnosed Chronic ...
A Phase I Study of Oral Asciminib (ABL001) in Patients ...The design of a phase I, open label, dose finding study was chosen in order to establish a safe and tolerated dose of single agent ABL001 in Chronic myeloid ...
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