A Phase 2 Study of Ruxolitinib With Chemotherapy in Children With Acute Lymphoblastic Leukemia

Status: Recruiting
DFCI Protocol ID: 17-290

This is a nonrandomized study of ruxolitinib in combination with a standard multi-agent chemotherapy regimen for the treatment of B-cell acute lymphoblastic leukemia. Part 1 of the study will optimize the dose of study drug (ruxolitinib) in combination with the chemotherapy regimen. Part 2 will evaluate the efficacy of combination chemotherapy and ruxolitinib at the recommended dose determined in Part 1.

Conducting Institutions:

Children's Hospital Boston, Dana-Farber Cancer Institute

Overall PI:

Lewis Silverman, MD, Dana-Farber Cancer Institute

Site-responsible Investigators:


Dana-Farber Cancer Institute: Childrens Hospital Pediatric Clinical Translation Investigation Program CTIP, ctip@partners.org

Eligibility Criteria

Inclusion Criteria:

  -  Eligible Ages in Australia and Canada; 2 years to 21 years

  -  De novo high-risk (HR) Ph-like B-ALL for which any of following criteria are present
     at diagnosis:

       -  Age ≥ 10 years

       -  White blood cell (WBC) ≥ 50 × 10^3/μL

       -  CNS3 leukemia

       -  Systemic steroid pretreatment without presteroid WBC documentation

  -  One of the following Ph-like ALL genetic lesions must be present in the diagnostic
     bone marrow or peripheral blood sample:

       -  CRLF2 rearrangement with JAK1 or JAK2 mutation (JAK+)

       -  CRLF2 rearrangement without JAK mutation

       -  Other JAK pathway alterations (eg, JAK2 fusions, erythropoietin receptor (EPO-R)
  fusions, SH2B3 deletions, interleukin-7 receptor-alpha (IL7RA) mutations) with or
  without CRLF2 rearrangement

  -  Completed a 4-drug Induction therapy regimen (modified aBFM regimen or equivalent) in
     Study AALL1131 or as the institutional standard of care for HR B-ALL and have had
     end-Induction minimal residual disease (MRD) assessed

  -  Male and female subjects of reproductive non childbearing potential or willing to take
     appropriate precautions to avoid pregnancy or fathering a child for the duration of
     study participation

Exclusion Criteria:

  -  Receipt of any other cytotoxic chemotherapy before Induction therapy, with exception
     of hydroxyurea or steroid pretreatment

  -  Trisomy 21 (Down syndrome)

  -  BCR-ABL1-rearranged (Ph+) ALL

  -  Calculated creatinine clearance or radioisotope glomerular filtration rate < 70
     mL/min/1.73 m^2

  -  Alanine aminotransferase ≥ 5 × upper limit of normal (ULN) for age

  -  Direct bilirubin ≥ 1.5 × ULN (may be assumed if total bilirubin is below ULN)

  -  History or evidence of cirrhosis

  -  Platelet count < 75 × 10^3/μL

  -  Absolute neutrophil count (ANC) < 750/μL

  -  Positive screen for hepatitis B or C

  -  Known human immunodeficiency virus infection

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