New combo therapy shows promise for tough childhood leukemia

NCT ID NCT02723994

Disease control Sponsor: Incyte Corporation Source: ClinicalTrials.gov ↗

First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This study tested whether adding the targeted drug ruxolitinib to standard chemotherapy helps children with a high-risk form of acute lymphoblastic leukemia (ALL). The trial involved 171 children and young adults aged 1 to 21. Researchers first found the best dose of ruxolitinib, then measured how many patients stayed cancer-free for 3 years. The goal is to improve outcomes for those with specific genetic changes that make their leukemia harder to treat.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance: Ruxolitinib (a JAK inhibitor) combined with standard chemotherapy drugs
What this could lead to: If successful, this combination could improve the chance of staying cancer-free for children with a hard-to-treat form of leukemia.
What could go wrong: This is a phase 2 trial, so results are still early. The added drug may increase side effects, and it is not yet known if it works better than chemo alone.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

171 people

The number who actually took part.

Started

Sep 2016

Finished

Mar 2026

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

1 year to 21 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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

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Conditions

The condition(s) this trial relates to.

B-cell acute lymphoblastic leukemia Burkitt lymphoma leukemia

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Leukemia

Contacts and locations

Locations

Akron Children'S Hospital

Akron, Ohio, 44308, United States
Albany Medical College

Albany, New York, 12208, United States
Alberta'S Children Hospital

Calgary, Alberta, T3B 6A8, Canada
Alfred I. Dupont Hospital For Children

Wilmington, Delaware, 19803, United States
All Childrens Hospital

St. Petersburg, Florida, 33701, United States
Alliance For Childhood Diseases

Las Vegas, Nevada, 89109, United States
Ann & Robert H. Lurie Children'S Hospital

Chicago, Illinois, 60611, United States
Arkansas Childrens Hospital

Little Rock, Arkansas, 72202, United States
Arnold Palmer Hospital For Children

Orlando, Florida, 32806, United States
Bc Children'S Hospital

Vancouver, British Columbia, V6G1R8, Canada
Blank Childrens Hospital

Des Moines, Iowa, 50309, United States
Carolinas Medical Center

Charlotte, North Carolina, 28203, United States
Children'S Center For Cancer & Blood Dis

Las Vegas, Nevada, 89109, United States
Children'S Hospital & Medical Center

Omaha, Nebraska, 68114, United States
Children'S Hospital Colorado

Aurora, Colorado, 80045, United States
Children'S Hospital Los Angeles

Los Angeles, California, 90027, United States
Children'S Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States
Children'S Hospital of the

Norfolk, Virginia, 23507, United States
Children'S Hospitals and Clinics of Minnesota

Minneapolis, Minnesota, 55404, United States
Childrens Hospital of Pittsburgh

Pittsburgh, Pennsylvania, 15224, United States
Childrens Hospital of Southwest Florida

Fort Myers, Florida, 33908, United States
Choc Children Hospital

Orange, California, 92868, United States
Chu Sainte-Justine

Montreal, Quebec, H3T 1C5, Canada
Cincinnati Childrens Hospital Medical Center

Cincinnati, Ohio, 45229, United States
Cleveland Clinic Foundation

Cleveland, Ohio, 44195, United States
Cohen Children'S Medical Center

New Hyde Park, New York, 11040, United States
Connecticut Children'S Medical Ctr

Hartford, Connecticut, 06106, United States
Cs Mott Childrens Hospital

Ann Arbor, Michigan, 48109, United States
Dana Farber Boston Children'S

Boston, Massachusetts, 02215, United States
Dayton Children'S Hospital

Dayton, Ohio, 45404, United States
Dell Childrens Medical Center of Central Texas

Austin, Texas, 78723, United States
Doernbecher Childrens Hospital

Portland, Oregon, 97239, United States
Driscoll Children'S Hospital

Corpus Christi, Texas, 78411, United States
Egleston Children'S Health

Atlanta, Georgia, 30322-1060, United States
El Paso Children'S Hospital Corporation

El Paso, Texas, 79905, United States
Florida Hospital Orlando

Orlando, Florida, 32803, United States
Hackensack University Medical Center

Hackensack, New Jersey, 07601, United States
Hasbro Childrens Hospital

Providence, Rhode Island, 02903, United States
Helen Devos Childrens Hosp

Grand Rapids, Michigan, 49503, United States
Joe Dimaggio Children Hospital

Hollywood, Florida, 33021, United States
Johns Hopkins University

Baltimore, Maryland, 21287-7049, United States
Kaiser Foundation Health Plan

Los Angeles, California, 90027, United States
Kaiser Permanente

Fontana, California, 92335, United States
Kaiser Permanente

San Diego, California, 92111, United States
Kaiser Permanente Downey

Santa Fe Springs, California, 90670, United States
Kapiolani Medical Center For Women and Children

Honolulu, Hawaii, 96826, United States
Kosair Childrens Hospital

Louisville, Kentucky, 40202, United States
Legacy Emanuel Hospital and Health Center

Portland, Oregon, 97227, United States
Loma Linda University Cancer Center

Loma Linda, California, 92350, United States
Loyola University Medical Center

Maywood, Illinois, 60153-3328, United States
Lucille Packard Childrens Hospital

Palo Alto, California, 94304, United States
Maine Center For Cancer Medicine

Scarborough, Maine, 04074, United States
McGill University Health Centre/Glen Site/Cedars Cancer Centre

Montreal, Quebec, H4A 3J1, Canada
McMaster University Medical Centre

Hamilton, L8V 5C2, Canada
Medical City Dallas Hospital

Dallas, Texas, 75230, United States
Medical College of Wisconsin

Milwaukee, Wisconsin, 53226, United States
Medical University of South Carolina

Charleston, South Carolina, 29425, United States
Michigan State University

Lansing, Michigan, 48912, United States
Miller Childrens Hospital Pharmacy

Long Beach, California, 90806, United States
Morristown Medical Center

Morristown, New Jersey, 07962, United States
Nationwide Children'S Hospital

Columbus, Ohio, 43205, United States
Nemours Childrens Clinic

Jacksonville, Florida, 32207, United States
Nemours Childrens Clinic

Pensacola, Florida, 32504, United States
New York Medical College

Valhalla, New York, 10595, United States
Newark Beth Israel Medical Center

Newark, New Jersey, 07112, United States
Nyu Clinical Cancer Center

New York, New York, 10032, United States
Ochsner Clinic

New Orleans, Louisiana, 70121, United States
Penn State Hershey Medical Center

Hershey, Pennsylvania, 17033-2360, United States
Phoenix Children'S Hospital

Phoenix, Arizona, 85016-7710, United States
Primary Children'S Hospital

Salt Lake City, Utah, 84113, United States
Providence Sacred Heart Medical Center & Children'S Hospital

Spokane, Washington, 99204, United States
Rady Children'S Hospital - San Diego

San Diego, California, 92123, United States
Rainbow Babies & Children Hosp

Cleveland, Ohio, 44106, United States
Riley Hospital For Children

Indianapolis, Indiana, 46202, United States
Roswell Park Comprehensive Cancer Center

Buffalo, New York, 14263, United States
Rutgers Cancer Institute of Nj

New Brunswick, New Jersey, 08901, United States
Saint Marys Medical Center

West Palm Beach, Florida, 33407, United States
San Jorge Childrens Hospital

San Juan, 00912, Puerto Rico
Seattle Children'S Hospital

Seattle, Washington, 98105, United States
Sinai Hospital of Baltimore

Baltimore, Maryland, 21215, United States
Smilow Cancer Center - Yale

New Haven, Connecticut, 06510, United States
Southern California Permanente Medical Group

Los Angeles, California, 90027, United States
Southern Illinois University School of Medicine

Springfield, Illinois, 62702, United States
St Josephs Childrens Hospital Tampa

Tampa, Florida, 33607, United States
St Josephs Regional Medical Center

Paterson, New Jersey, 07503, United States
St Vincent Hospital

Indianapolis, Indiana, 46260, United States
St Vincent Hospital

Green Bay, Wisconsin, 54301, United States
Suny Upstate Medical University

Syracuse, New York, 13210, United States
Texas Childrens Hospital

Houston, Texas, 77030, United States
The Children'S Mercy Hospital Outpatient

Kansas City, Missouri, 64108, United States
The Childrens Hospital of Alabama

Birmingham, Alabama, 35233, United States
The Hospital For Sick Children

Toronto, Ontario, 94611, Canada
Toledo Hospital

Toledo, Ohio, 43606, United States
Ucsf Pediatric Oncology

San Francisco, California, 94158, United States
University Pediatric Hospital

San Juan, PR, 00930, Puerto Rico
University of Florida Health Shands Hospital

Jacksonville, Florida, 32209, United States
University of Iowa Hospital and Clinics

Iowa City, Iowa, 52242, United States
University of Kentucky

Lexington, Kentucky, 40536, United States
University of Minnesota

Minneapolis, Minnesota, 55455, United States
University of Mississippi Medical Center

Jackson, Mississippi, 39216, United States
University of New Mexico Hospital

Albuquerque, New Mexico, 87131, United States
University of North Carolina At Chapel Hill

Chapel Hill, North Carolina, 27514, United States
University of Oklahoma Health Sciences Center

Oklahoma City, Oklahoma, 73104, United States
University of Rochester Medical Center

Rochester, New York, 14642, United States
University of Texas Health Science Cente

San Antonio, Texas, 78229, United States
University of Texas Southwestern Medical Center

Dallas, Texas, 75390, United States
Valley Childrens Hospital

Madera, California, 93636-8762, United States
Vanderbilt University Medical Center

Nashville, Tennessee, 37232, United States
Wake Forest University

Winston-Salem, North Carolina, 27157, United States
Washington University

St Louis, Missouri, 63130, United States
Weill Cornell Medical College

New York, New York, 10021, United States

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