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

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 is a multi-center, open-label, single-arm phase I/II trial evaluating asciminib at the recommended phase 2 dose in combination with low-intensity chemotherapy, followed by asciminib plus blinatumomab for consolidation in participants aged 1 to 30 years with r/r Ph+ or ABL-class Ph-like ALL. Part 1 uses a Bayesian Optimal Interval design to determine RP2D during debulking induction, while Part 2 expands at RP2D to assess safety, tolerability, and efficacy. Participants with known T315I mutation are excluded from Parts 1 and 2 and enrolled in a separate cohort. Core treatment comprises three cycles: asciminib with low-intensity chemo, then asciminib plus blinatumomab blocks, with survival follow-up ongoing.

Why it matters

Who can join: the full rules

Inclusion Criteria:

* Evidence of Ph+ ALL or ABL1 or ABL2 fusion Ph-like ALL, inclusive of participants with ABL1 T315I mutation
* Participants with CNS1, CNS2, CNS3a, or CNS3b at screening
* Active B-Cell ALL at screening defined by MFC or IG/TCR PCR of ALL blasts \>0.01% in participants with either:

  1. Primary refractory disease (\>0.01% ALL blasts present at the end of consolidation) OR
  2. Relapsed ALL with evidence of involvement of BM with ALL (MFC or IG/TCR PCR \>0.01%) after at least one line of therapy
* Documented history of CD19 expressing B-cell ALL (in peripheral blood or bone marrow by flow cytometry).

  a) For participants who received anti-CD19 targeted therapy (e.g CD19 CAR T cells or blinatumomab), CD19 expressing B-cell ALL must be documented after anti-CD19 therapy completion prior to cycle 1 day 1
* Adequate hepatic and renal function (local laboratory analysis) as defined:

  1. ALT ≤ 5x upper limit of normal (ULN) for age
  2. Total bilirubin (sum of conjugated + unconjugated) ≤ 1.5 x ULN) for age, except for participants with Gilbert's syndrome who may only be included if total bilirubin ≤ 3.0 x ULN or direct bilirubin ≤ 1.5 x ULN
  3. Estimated glomerular filtration rate (eGFR) using the Cockcroft-Gault formula in participants ≥ 18 years, OR radioisotope GFR ≥50 mL/min/1.73 m\^2, OR creatinine based on age and sex for participants \< 18 years old
* Adequate cardiac function defined as shortening fraction ≥27% by echocardiogram (ECHO) OR left ventricular ejection fraction of ≥50% by ECHO

Exclusion Criteria:

* Participants with \>3 relapses of ALL
* Extramedullary disease (non-CNS and/or isolated CNS disease)
* Participants with CNS3c (Clinical signs of CNS leukemia (such as facial nerve palsy, brain/eye involvement or hypothalamic syndrome))
* Cardiac or cardiac repolarization abnormality, including but not limited to clinically significant cardiac arrhythmias, long QT syndrome, family history of idiopathic sudden death or congenital long QT syndrome or other clinically significant heart disease (e.g., congestive heart failure, etc.)
* 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.

Other protocol defined inclusion/exclusion criteria may apply.

Where it's running

• Novartis Investigative Site
  Sydney, New South Wales, Australia Recruiting
• Novartis Investigative Site
  Ramat Gan, Israel Recruiting
• Novartis Investigative Site
  Seoul, South Korea Recruiting
• Novartis Investigative Site
  Taipei, Taiwan Recruiting

Study contacts

• Study coordinator: Novartis Pharmaceuticals
• Email: novartis.email@novartis.com
• Phone: 1-888-669-6682

Similar studies

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

• Combining blinatumomab and asciminib for treating a type of leukemia

  This study is testing if combining two drugs, blinatumomab and asciminib, can help people with a specific type of leukemia feel better and improve their chances of recovery.

  Recruiting at 2 sites Phase 1/2 M.D. Anderson Cancer Center · The Bronx, New York and 1 other locations · NCT06308588

• Asciminib maintenance after alloHCT or CAR T for adults with Ph+ ALL

  Doctors will try asciminib in adults with Ph+ ALL who are in remission after an allogeneic stem cell transplant or CAR T therapy to check safety and find the best dose to help prevent relapse.

  Recruiting Phase 1 H. Lee Moffitt Cancer Center and Research Institute · Tampa, Florida · NCT07250087

• Short-course blinatumomab treatment for B-ALL patients with measurable residual disease

  This study tests whether a short treatment with blinatumomab can help people with B-cell acute lymphoblastic leukemia who still have some cancer cells left after their initial treatment, before they get a stem cell transplant.

  Recruiting Phase 2 Hospital Universitario Dr. Jose E. Gonzalez · Monterrey, Nuevo León · NCT06886074

• Comparing treatments for Philadelphia Chromosome Positive Acute Lymphoblastic Leukemia

  This study tests whether Ponatinib or Imatinib, both with low-dose chemotherapy, can help adults with Philadelphia Chromosome Positive Acute Lymphoblastic Leukemia feel better, and looks at how Blinatumomab can be used for those who respond well or not to these treatments.

  Recruiting at 81 sites Phase 2 Goethe University · Aachen and 84 other locations · NCT06061094

• Preventing neurotoxicity in leukemia treatment with intrathecal chemotherapy

  This study is testing if giving a specific chemotherapy directly into the spine can help protect the brain from side effects while treating adults with Acute Lymphoblastic Leukemia.

  Recruiting Phase 2 Northside Hospital, Inc. · Atlanta, Georgia · NCT05519579

• Combining blinatumomab, methotrexate, cytarabine, and ponatinib for treating certain types of leukemia

  This study is testing a new combination of four medications to see if it can help people with certain types of leukemia that have come back or didn't respond to previous treatments.

  Recruiting Phase 2 M.D. Anderson Cancer Center · Houston, Texas · NCT03263572

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.