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

Registration Number
NCT02723994
Lead Sponsor
Incyte Corporation
Brief Summary

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.

Detailed Description

Not available

Recruitment & Eligibility

Status
ACTIVE_NOT_RECRUITING
Sex
All
Target Recruitment
171
Inclusion Criteria
  • Eligible for study when participant is 1 year to 21 years at the time of diagnosis

  • Eligible Ages in 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 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:

    1. CRLF2 rearrangement with confirmed JAK1 or JAK2 mutation (JAK+)
    2. CRLF2 rearrangement without JAK mutation
    3. 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 < 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

Study & Design

Study Type
INTERVENTIONAL
Study Design
SINGLE_GROUP
Arm && Interventions
GroupInterventionDescription
Ruxolitinib in combination with chemotherapyDexamethasone-
Ruxolitinib in combination with chemotherapyLeucovorin Calcium-
Ruxolitinib in combination with chemotherapyAsparaginase Erwinia Chrysanthemi-
Ruxolitinib in combination with chemotherapyDoxorubicin-
Ruxolitinib in combination with chemotherapyPegaspargase-
Ruxolitinib in combination with chemotherapyCytarabine-
Ruxolitinib in combination with chemotherapyRuxolitinib-
Ruxolitinib in combination with chemotherapyMethotrexate-
Ruxolitinib in combination with chemotherapyThioguanine-
Ruxolitinib in combination with chemotherapyVincristine Sulfate-
Ruxolitinib in combination with chemotherapyMercaptopurine-
Ruxolitinib in combination with chemotherapyPrednisone-
Ruxolitinib in combination with chemotherapyCyclophosphamide-
Primary Outcome Measures
NameTimeMethod
Part 1: Safety/tolerability of ruxolitinib in combination with chemotherapy as measured by adverse events (AEs), vital signs, clinical laboratory tests, and echocardiogramsPart 1: AEs assessed from screening through up to 30 days after the last dose of study drug, expected to be 26 months (females) or 38 months (males)
Part 2: Efficacy of ruxolitinib in combination with chemotherapy as measured by Event-free survival, defined as the percentage of patients alive without relapse, progression, or death at 3 years from study Day 1Part 2: assessed at 3 years
Secondary Outcome Measures
NameTimeMethod
Safety and tolerability of the combination treatment for subjects beginning treatment at the recommended dose for Part 2, as assessed by AEs, vital signs, clinical laboratory tests, and echocardiogramsAEs assessed from screening through up to 30 days after the last dose of study treatment, expected to be 26 months (females) or 38 months (males)

Trial Locations

Locations (108)

Sinai Hospital of Baltimore

🇺🇸

Baltimore, Maryland, United States

Childrens Hospital of Pittsburgh

🇺🇸

Pittsburgh, Pennsylvania, United States

Texas Childrens Hospital

🇺🇸

Houston, Texas, United States

Kaiser Permanente

🇺🇸

San Diego, California, United States

Rady Children'S Hospital - San Diego

🇺🇸

San Diego, California, United States

Children'S Hospitals and Clinics of Minnesota

🇺🇸

Minneapolis, Minnesota, United States

University of Texas Health Science Cente

🇺🇸

San Antonio, Texas, United States

Loma Linda University Cancer Center

🇺🇸

Loma Linda, California, United States

Miller Childrens Hospital Pharmacy

🇺🇸

Long Beach, California, United States

Kaiser Foundation Health Plan

🇺🇸

Los Angeles, California, United States

Southern California Permanente Medical Group

🇺🇸

Los Angeles, California, United States

Valley Childrens Hospital

🇺🇸

Madera, California, United States

Lucille Packard Childrens Hospital

🇺🇸

Palo Alto, California, United States

Choc Children Hospital

🇺🇸

Orange, California, United States

Kaiser Permanente Downey

🇺🇸

Santa Fe Springs, California, United States

Connecticut Children'S Medical Ctr

🇺🇸

Hartford, Connecticut, United States

Alfred I. Dupont Hospital For Children

🇺🇸

Wilmington, Delaware, United States

Nemours Childrens Clinic

🇺🇸

Pensacola, Florida, United States

Childrens Hospital of Southwest Florida

🇺🇸

Fort Myers, Florida, United States

Joe Dimaggio Children Hospital

🇺🇸

Hollywood, Florida, United States

University of Florida Health Shands Hospital

🇺🇸

Jacksonville, Florida, United States

All Childrens Hospital

🇺🇸

Saint Petersburg, Florida, United States

Egleston Children'S Health

🇺🇸

Atlanta, Georgia, United States

Saint Marys Medical Center

🇺🇸

West Palm Beach, Florida, United States

Loyola University Medical Center

🇺🇸

Maywood, Illinois, United States

Ann & Robert H. Lurie Children'S Hospital

🇺🇸

Chicago, Illinois, United States

Southern Illinois University School of Medicine

🇺🇸

Springfield, Illinois, United States

Riley Hospital For Children

🇺🇸

Indianapolis, Indiana, United States

St Vincent Hospital

🇺🇸

Green Bay, Wisconsin, United States

University of Iowa Hospital and Clinics

🇺🇸

Iowa City, Iowa, United States

Dana Farber Boston Children'S

🇺🇸

Boston, Massachusetts, United States

Johns Hopkins University

🇺🇸

Baltimore, Maryland, United States

Helen Devos Childrens Hosp

🇺🇸

Grand Rapids, Michigan, United States

Michigan State University

🇺🇸

Lansing, Michigan, United States

Washington University

🇺🇸

Saint Louis, Missouri, United States

Alliance For Childhood Diseases

🇺🇸

Las Vegas, Nevada, United States

Hackensack University Medical Center

🇺🇸

Hackensack, New Jersey, United States

Morristown Medical Center

🇺🇸

Morristown, New Jersey, United States

Rutgers Cancer Institute of Nj

🇺🇸

New Brunswick, New Jersey, United States

Roswell Park Comprehensive Cancer Center

🇺🇸

Buffalo, New York, United States

Cohen Children'S Medical Center

🇺🇸

New Hyde Park, New York, United States

Nyu Clinical Cancer Center

🇺🇸

New York, New York, United States

Suny Upstate Medical University

🇺🇸

Syracuse, New York, United States

Cincinnati Childrens Hospital Medical Center

🇺🇸

Cincinnati, Ohio, United States

Carolinas Medical Center

🇺🇸

Charlotte, North Carolina, United States

Akron Children'S Hospital

🇺🇸

Akron, Ohio, United States

Rainbow Babies & Children Hosp

🇺🇸

Cleveland, Ohio, United States

Cleveland Clinic Foundation

🇺🇸

Cleveland, Ohio, United States

Toledo Hospital

🇺🇸

Toledo, Ohio, United States

Children'S Hospital of Philadelphia

🇺🇸

Philadelphia, Pennsylvania, United States

Penn State Hershey Medical Center

🇺🇸

Hershey, Pennsylvania, United States

Hasbro Childrens Hospital

🇺🇸

Providence, Rhode Island, United States

Driscoll Children'S Hospital

🇺🇸

Corpus Christi, Texas, United States

Medical City Dallas Hospital

🇺🇸

Dallas, Texas, United States

El Paso Children'S Hospital Corporation

🇺🇸

El Paso, Texas, United States

Children'S Hospital of the

🇺🇸

Norfolk, Virginia, United States

Providence Sacred Heart Medical Center & Children'S Hospital

🇺🇸

Spokane, Washington, United States

Seattle Children'S Hospital

🇺🇸

Seattle, Washington, United States

Bc Children'S Hospital

🇨🇦

Vancouver, British Columbia, Canada

McGill University Health Centre/Glen Site/Cedars Cancer Centre

🇨🇦

Montreal, Quebec, Canada

McMaster University Medical Centre

🇨🇦

Hamilton, Canada

University Pediatric Hospital

🇵🇷

San Juan, Puerto Rico

Ucsf Pediatric Oncology

🇺🇸

San Francisco, California, United States

Vanderbilt University Medical Center

🇺🇸

Nashville, Tennessee, United States

Primary Children'S Hospital

🇺🇸

Salt Lake City, Utah, United States

Nationwide Children'S Hospital

🇺🇸

Columbus, Ohio, United States

Children'S Center For Cancer & Blood Dis

🇺🇸

Las Vegas, Nevada, United States

Children'S Hospital Los Angeles

🇺🇸

Los Angeles, California, United States

Chu Sainte-Justine

🇨🇦

Montreal, Quebec, Canada

Arkansas Childrens Hospital

🇺🇸

Little Rock, Arkansas, United States

St Josephs Regional Medical Center

🇺🇸

Paterson, New Jersey, United States

Weill Cornell Medical College

🇺🇸

New York, New York, United States

San Jorge Childrens Hospital

🇵🇷

San Juan, Puerto Rico

Alberta'S Children Hospital

🇨🇦

Calgary, Alberta, Canada

University of Mississippi Medical Center

🇺🇸

Jackson, Mississippi, United States

Albany Medical College

🇺🇸

Albany, New York, United States

Newark Beth Israel Medical Center

🇺🇸

Newark, New Jersey, United States

University of Minnesota

🇺🇸

Minneapolis, Minnesota, United States

Maine Center For Cancer Medicine

🇺🇸

Scarborough, Maine, United States

Phoenix Children'S Hospital

🇺🇸

Phoenix, Arizona, United States

University of Oklahoma Health Sciences Center

🇺🇸

Oklahoma City, Oklahoma, United States

The Childrens Hospital of Alabama

🇺🇸

Birmingham, Alabama, United States

Children'S Hospital & Medical Center

🇺🇸

Omaha, Nebraska, United States

Medical College of Wisconsin

🇺🇸

Milwaukee, Wisconsin, United States

Legacy Emanuel Hospital and Health Center

🇺🇸

Portland, Oregon, United States

Doernbecher Childrens Hospital

🇺🇸

Portland, Oregon, United States

The Hospital For Sick Children

🇨🇦

Toronto, Ontario, Canada

St Josephs Childrens Hospital Tampa

🇺🇸

Tampa, Florida, United States

Children'S Hospital Colorado

🇺🇸

Aurora, Colorado, United States

New York Medical College

🇺🇸

Valhalla, New York, United States

University of Texas Southwestern Medical Center

🇺🇸

Dallas, Texas, United States

Cs Mott Childrens Hospital

🇺🇸

Ann Arbor, Michigan, United States

Wake Forest University

🇺🇸

Winston-Salem, North Carolina, United States

Smilow Cancer Center - Yale

🇺🇸

New Haven, Connecticut, United States

Florida Hospital Orlando

🇺🇸

Orlando, Florida, United States

Arnold Palmer Hospital For Children

🇺🇸

Orlando, Florida, United States

Kosair Childrens Hospital

🇺🇸

Louisville, Kentucky, United States

The Children'S Mercy Hospital Outpatient

🇺🇸

Kansas City, Missouri, United States

Medical University of South Carolina

🇺🇸

Charleston, South Carolina, United States

University of North Carolina At Chapel Hill

🇺🇸

Chapel Hill, North Carolina, United States

Ochsner Clinic

🇺🇸

New Orleans, Louisiana, United States

Kapiolani Medical Center For Women and Children

🇺🇸

Honolulu, Hawaii, United States

Dell Childrens Medical Center of Central Texas

🇺🇸

Austin, Texas, United States

Blank Childrens Hospital

🇺🇸

Des Moines, Iowa, United States

University of Kentucky

🇺🇸

Lexington, Kentucky, United States

University of New Mexico Hospital

🇺🇸

Albuquerque, New Mexico, United States

University of Rochester Medical Center

🇺🇸

Rochester, New York, United States

Dayton Children'S Hospital

🇺🇸

Dayton, Ohio, United States

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