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Safety and Efficacy of Asciminib in Pediatrics and Young Adults With Relapse/Refractory (r/r) Philadelphia Positive (Ph+) or ABL-class Ph-like Acute Lymphoblastic Leukemia (ALL)

NCT07387926

RecruitingPhase 1, Phase 2

Sponsor: Novartis Pharmaceuticals

Conditions: Acute Lymphoblastic Leukemia, Leukemia, Lymphoblastic, Acute, Philadelphia-Positive, Philadelphia Chromosome Positive Acute Lymphoblastic Leukemia

Interventions: Asciminib Adult formulation, Asciminib Pediatric formulation, Dexamethasone, Vincristine, Blinatumomab

Countries: Israel

Multi-center, open-label, single arm study of asciminib in participants aged ≥1 year to ≤30 years old with r/r Ph+ or ABL-class Ph-like ALL. This study will have 2 parts: Part 1 dose escalation and Part 2 dose expansion. Part 1 dose escalation will enroll participants aged ≥1 year to ≤30 years to determine the recommended phase 2 dose (RP2D) of asciminib when administered with low intensity chemotherapy. Part 2 dose expansion will enroll participants aged ≥1 year to ≤30 years to evaluate safety, tolerability, and efficacy of asciminib at the RP2D with the treatment regimen.

Eligibility overview

Sex: ALL

Age: 1 Year to 30 Years

Healthy volunteers: No

Study type: INTERVENTIONAL

Eligibility criteria

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.

Locations (1)

Ramat Gan, Israel