Asciminib for Acute Lymphoblastic Leukemia

Overseen By Valerie Trujillo

Age: 18+

Sex: Any

Trial Phase: Phase 1

Sponsor: H. Lee Moffitt Cancer Center and Research Institute

Disqualifiers: Active relapsed disease, Pregnancy, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Breakthrough Therapy This drug has been fast-tracked for approval by the FDA given its high promise

Compensation: Varies

Number of Visits: Unknown

Duration: Up to 24 months

36 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new drug, asciminib, to determine its safety for individuals previously treated for Philadelphia Chromosome Positive Acute Lymphoblastic Leukemia (Ph+ B-ALL). The study focuses on two groups: those who have undergone an allogeneic stem cell transplant (a procedure replacing diseased bone marrow with healthy cells) and those who received CAR T cell therapy (a treatment using modified immune cells to fight cancer). Researchers aim to identify the safest and most effective dose of asciminib for future studies. Adults in remission with Ph+ B-ALL, having less than 5% cancer cells in their bone marrow, may be suitable for this trial. As a Phase 1 trial, participants will be among the first to receive this new treatment, aiding researchers in understanding how asciminib works in people.

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

The trial information does not specify if 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 asciminib is likely to be safe for humans?

Research has shown that asciminib is generally safe for individuals with certain blood cancers. In earlier studies, patients with Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ ALL) who took asciminib after other treatments tolerated it well. Studies have also shown that asciminib is safe for patients who haven't responded to or can't tolerate other treatments.

Another study found that combining asciminib with other drugs was safe for patients with this type of leukemia. Although this trial is in an early stage, current data suggest that asciminib is generally well-tolerated, with manageable and not too severe side effects for most people. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for leukemia?

Unlike the standard treatments for acute lymphoblastic leukemia, which often include chemotherapy and targeted therapies, asciminib offers a novel approach. It uniquely targets the ABL myristoyl pocket (STAMP), which is different from how most existing treatments work. This specific targeting has the potential to be more selective and could reduce side effects often associated with broader-acting therapies. Researchers are excited about asciminib because it represents a new mechanism of action in the fight against this type of leukemia, potentially offering new hope for patients who have undergone allogeneic stem cell transplants or CAR T cell therapy.

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

Research has shown that asciminib may effectively treat Philadelphia Chromosome Positive Acute Lymphoblastic Leukemia (Ph+ B-ALL). In earlier studies, patients unresponsive to other treatments often achieved complete remission with asciminib. Another study found that asciminib significantly improved survival in animal tests. This drug targets specific genetic changes linked to the leukemia, potentially stopping cancer growth. In this trial, researchers are studying asciminib in patients who have already tried treatments like stem cell transplants or CAR T cell therapy, suggesting it could benefit these individuals. ^{2 3 6 7 8}

Who Is on the Research Team?

Rawan Faramand, MD

Principal Investigator

Moffitt Cancer Center

Are You a Good Fit for This Trial?

This trial is for adults with a blood cancer called Philadelphia Chromosome Positive Acute Lymphoblastic Leukemia (Ph+ B-ALL) who have already undergone either an Allogeneic Stem Cell Transplant (alloHCT, cohort A) or chimeric antigen receptor T cell (CAR T) therapy (cohort B).

Inclusion Criteria

Morphologic remission (<5% marrow blasts)

I am 18 years old or older.

My leukemia is Philadelphia chromosome positive.

Exclusion Criteria

Pregnancy or breastfeeding

Prior investigational CAR T product

My cancer has returned and is active.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive asciminib maintenance therapy following alloHCT or CAR T to prevent relapse

Up to 24 months

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Asciminib

Trial Overview

The study tests the safety and appropriate dosage of asciminib as maintenance therapy to prevent relapse in patients after treatment for Ph+ B-ALL. It involves two groups based on their prior treatments: alloHCT recipients and CAR T therapy recipients.

How Is the Trial Designed?

Group I: Patients post-alloHCT Experimental Treatment 1 Intervention

Individuals who have previously undergone an allogeneic stem cell transplant (alloHCT).

Group II: Patients post-CAR T cell therapy Experimental Treatment 1 Intervention

Individuals who have received chimeric antigen receptor T cell therapy (CAR T).

Who Is Running the Clinical Trial?

H. Lee Moffitt Cancer Center and Research Institute

Lead Sponsor

Trials

576

Recruited

145,000+

Novartis

Industry Sponsor

Trials

1,646

Recruited

2,778,000+

Vasant Narasimhan

Novartis

Chief Executive Officer since 2018

MD from Harvard Medical School, Bachelor's in Biological Sciences from University of Chicago, Master's in Public Policy from John F. Kennedy School of Government

Shreeram Aradhye

Novartis

Chief Medical Officer since 2022

MD from Yale University, MSc in Clinical Epidemiology from University of Pennsylvania

Citations

1.

ashpublications.org

ashpublications.org/blood/article/142/Supplement%201/1602/503643/Asciminib-Is-Effective-Against-ABL1-Gene-Fusions

Asciminib Is Effective Against ABL1 Gene Fusions in Acute ...

Asciminib treatment significantly increased survival outcomes compared with control mice (84 vs 46d, p=0.01) by a time comparable to that of ...

2.

pmc.ncbi.nlm.nih.gov

pmc.ncbi.nlm.nih.gov/articles/PMC12482290/

Asciminib and inotuzumab ozogamicin: a new combination ...

Improved outcome for children with acute lymphoblastic leukemia: results of Dana-Farber Consortium Protocol 91-01. Blood. 2001;97(5):1211 ...

3.

sciencedirect.com

sciencedirect.com/science/article/pii/S2473952925003787

Asciminib for relapsed or refractory Philadelphia ...

In conclusion, our analysis of this first real-life cohort of patients with R/R Ph+ ALL and BCP-CML treated with ASC, showed a high CR rate and ...

4.

haematologica.org

haematologica.org/article/view/12134

Asciminib for Philadelphia chromosome-positive leukemias

Studies have demonstrated the safety and efficacy of asciminib in patients with resistance or intolerance to two or more lines of prior therapy ...

5.

clinicaltrials.gov

clinicaltrials.gov/study/NCT07040982

NCT07040982 | Asciminib as Maintenance Treatment After ...

This phase I trial tests the safety, side effects and best dose of asciminib as maintenance treatment for adults with Philadelphia chromosome ...

6.

ashpublications.org

ashpublications.org/blood/article/145/6/551/535335/Asciminib-for-Ph-ALL-a-step-forward

Asciminib for Ph + ALL: a step forward? - ASH Publications

Their dose-finding study provides firm evidence for the feasibility and safety of this drug combination for patients with newly diagnosed Ph+ ...

7.

pubmed.ncbi.nlm.nih.gov

pubmed.ncbi.nlm.nih.gov/39374521/

Asciminib plus dasatinib and prednisone for ... - PubMed

Dasatinib is an effective treatment for Philadelphia chromosome-positive (Ph+) acute leukemia, but some patients develop resistance.

8.

hematologyadvisor.com

hematologyadvisor.com/news/acute-leukemia-asciminib-dasatinib-prednisone-safe-treatment-risk/

Asciminib Plus Dasatinib and Prednisone Safe With ...

Researchers determined asciminib plus dasatinib and prednisone was safe and showed activity for patients with Ph+ acute leukemia.