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Clinical Trials Search at Vanderbilt-Ingram Cancer Center

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

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.
Pediatric Leukemia
Phase II
Chemotherapy - cytotoxic, Mol. targeted/Immunotherapy/Biologics, Supportive Care
6-thioguanine (6-TG), Cyclophosphamide (CPM), Cytarabine (ARA-C), Dexamethasone, Doxorubicin, Leucovorin, Mercaptopurine, Methotrexate, Pegaspargase (PEG-ASP), Ruxolitinib, Vincristine
Friedman, Debra
Vanderbilt University


1 Years
Inclusion Criteria:

Eligible for study when participant is 1 year to 21 years at the time of diagnosis

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/L

CNS3 leukemia at diagnosis

Systemic steroid pretreatment without presteroid WBC documentation

Diagnostic bone marrow or peripheral blood sample must have gene expression profiling and downstream genetic testing performed by submitting diagnostic specimens under the COG AALL08B1 or APEC14B1 biology studies, or AALL1131 or its successor study. Specimens must demonstrate a Ph-like expression profile (ie, LDA-positive) as tested by low density microarray testing at the COG ALL reference laboratory or TriCore laboratory at the University of New Mexico AND must contain 1 of the following genetic lesions: (determined at COG ALL reference laboratories, or equivalent CAP/CLIA-certified laboratories approved by the medical monitor:

CRLF2 rearrangement* with confirmed JAK1 or JAK2 mutation (JAK+)

CRLF2 rearrangement* without JAK mutation

Other JAK pathway alterations (eg, JAK2 fusions, EPOR fusions, SH2B3 deletions, IL7RA mutations) with or without CRLF2-R, or CRLF2-R with unknown JAK status* as determined by a COG ALL Reference Laboratory

Completed a 4-drug Induction therapy regimen (modified aBFM regimen or equivalent) in Study AALL1131 or its successor study, or as per 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
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
Absolute neutrophil count (ANC)
Positive screen for hepatitis B or C

Known human immunodeficiency virus infection

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trials, call 1-800-811-8480 or complete
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