Using asciminib to treat chronic myeloid leukemia patients with a specific mutation

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This Phase II, multi-center, open-label study evaluates the efficacy and safety of asciminib in patients with chronic myeloid leukemia (CML) who have the T315I mutation and are resistant, intolerant, or ineligible for ponatinib. Participants will be enrolled if they have not previously received asciminib and have documented resistance or intolerance to at least one tyrosine kinase inhibitor. The study consists of a core phase to assess the drug's effectiveness and safety, followed by an extension phase allowing continued treatment if the drug is commercialized. The goal is to provide a new therapeutic option for patients facing limited treatment choices.

Why it matters

Who can join: the full rules

Inclusion Criteria:

* Signed informed consent must be obtained prior to participation in the study.
* Male or female participants with a diagnosis of CML-CP or CML-AP ≥ 18 years of age.
* Patients with CML-CP or CML-AP with history of documented T315I mutation after at least one TKI and are resistant, intolerant, or ineligible to ponatinib (according to Investigator judgment)
* Not already treated with asciminib or another any allosteric TKI
* Failure (adapted from the 2020 \& 2013 ELN Guidelines) or intolerance to Ponatinib at the time of Screening.
* Ineligible to ponatinib according to Investigator (based on EU ponatinib SmPC)
* Evidence of typical BCR::ABL1 transcript or atypical transcripts at the time of Screening which are amenable to standardized or non-standardized RQ-PCR quantification.

Exclusion Criteria:

* Previous hematopoietic allogeneic stem-cell transplantation
* Cardiac or cardiac repolarization abnormality
* Severe and/or uncontrolled concurrent medical disease that in the opinion of the Investigator could cause unacceptable safety risks or compromise compliance with the protocol (e.g. uncontrolled diabetes, active or uncontrolled infection, pulmonary hypertension)
* History of clinical acute pancreatitis within 1 year of study entry or past medical history of chronic pancreatitis (except if ponatinib-induced and completely resolved at time of Screening)
* History of acute or chronic liver disease (i.e., cirrhosis; liver impairment)
* Known presence of significant congenital or acquired bleeding disorder unrelated to cancer
* History of other active malignancy within 3 years prior to study entry with the exception of previous or concomitant basal cell skin cancer and previous carcinoma in situ treated curatively
* Known history of Human Immunodeficiency Virus (HIV), chronic Hepatitis B Virus (HBV), or chronic Hepatitis C Virus (HCV) infection. Testing for Hepatitis B surface antigen (HBs Ag) and Hepatitis B core antibody (HBcAb / anti HBc) will be performed at Screening
* Impairment of gastrointestinal (GI) function or GI disease that may significantly alter the absorption of study drug (e.g. ulcerative disease, uncontrolled nausea, vomiting, diarrhea, malabsorption syndrome, small bowel resection, or gastric bypass surgery)
* Treatment with medications that meet one of the following criteria and that cannot be discontinued at least one week prior to the start of treatment with study treatment:

  * Moderate or strong inducers of CYP3A
  * Moderate or strong inhibitors of CYP3A
* Pregnant or nursing (lactating) women
* Women of child-bearing potential
* Compound mutant T315I resistant to asciminib monotherapy (polyclonal ABL1 mutations including T315I can be enrolled) Other protocol-defined inclusion/exclusion criteria may apply.

Where it's running

• Novartis Investigative Site
  Bordeaux, France Recruiting
• Novartis Investigative Site
  Lille, France Recruiting
• Novartis Investigative Site
  Lyon, France Recruiting
• Novartis Investigative Site
  Nantes, France Recruiting
• Novartis Investigative Site
  Paris, France Recruiting
• Novartis Investigative Site
  Vandœuvre-lès-Nancy, France Recruiting

Study contacts

• Study coordinator: Novartis Pharmaceuticals
• Email: novartis.email@novartis.com
• Phone: +41613241111

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

• Asciminib for chronic phase CML with T315I mutation

  This study tests asciminib 200 mg twice daily in adults with chronic phase CML and a T315I mutation after stem cell transplant to see if it helps control leukemia.

  Recruiting at 5 sites Phase 2 University of Jena · Aachen and 9 other locations · NCT07789574

• LP-118 in combination with standard therapy for newly diagnosed Ph+ ALL

  This study tests whether adding LP-118 to the standard therapy for adults with Philadelphia chromosome–positive ALL is safe and useful.

  Recruiting at 2 sites Phase 1 Wake Forest University Health Sciences · Charlotte, North Carolina and 1 other locations · NCT07614022

• Asciminib for Ph+ CML-CP in Germany: real-world patient experiences

  This real-world study tests how asciminib performs in adults with Philadelphia chromosome-positive chronic myeloid leukemia in chronic phase, including those newly diagnosed or after one prior TKI.

  Recruiting at 74 sites Novartis · Baden-Baden, Baden-Wurttemberg and 73 other locations · NCT07549516

• Asciminib in children and young adults with relapsed or refractory Ph+ or ABL-class Ph-like ALL

  This study tests asciminib in kids and young adults with relapsed or refractory Philadelphia-positive or ABL-class Ph-like ALL to see if it is safe and helps treat the leukemia.

  Recruiting at 4 sites Phase 1/2 Novartis · Sydney, New South Wales and 3 other locations · NCT07387926

• Scemblix (asciminib) for newly diagnosed chronic-phase CML in China

  This project will try asciminib (Scemblix) in adults newly diagnosed with chronic-phase CML to see if it controls the disease and is safe.

  Recruiting Novartis · Wuhan, Hubei · NCT07375355

• Asciminib for children and teens with Philadelphia chromosome–positive chronic myeloid leukemia in chronic phase

  This study will try asciminib as a treatment for children aged 1 to under 18 with Philadelphia chromosome–positive chronic myeloid leukemia in chronic phase, including newly diagnosed patients and those who are resistant or intolerant to prior TKIs, with or without the T315I mutation.

  Recruiting at 39 sites Phase 2 Novartis · Chicago, Illinois and 38 other locations · NCT07354074

How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.