Actively Recruiting
A Phase 2 Study of Blinatumomab Combined With Chemotherapy for Infants With Newly Diagnosed Acute Lymphoblastic Leukemia Randomizing KMT2A-Rearranged Patients to Venetoclax Addition
Led by National Cancer Institute (NCI) · Updated on 2026-06-03
153
Participants Needed
111
Research Sites
N/A
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating the safety and effectiveness of adding venetoclax and/or blinatumomab to standard chemotherapy for infants with newly diagnosed acute lymphoblastic leukemia (ALL), either with a KMT2A gene rearrangement (KMT2A-R) or without (KMT2A-G). Venetoclax is a medication that may stop cancer cell growth by blocking a protein needed for their survival, while blinatumomab is an antibody that may interfere with cancer cells' ability to grow and spread. This phase II trial aims to determine if combining these treatments with chemotherapy improves outcomes for this vulnerable infant population. The study includes a steroid prephase followed by multiple treatment phases. Infants with KMT2A-R ALL are randomized into two main arms: Arm A receives standard chemotherapy and blinatumomab, while Arm B receives the same plus venetoclax. Infants without the KMT2A rearrangement (KMT2A-G) are treated on Arm C with chemotherapy and blinatumomab. Treatments involve various chemotherapy drugs, intravenous and oral medications, and intrathecal therapies given on specific schedules over several months, including induction, consolidation, MARMA, blinatumomab blocks, delayed intensification, and maintenance phases lasting up to two years. Participants will undergo regular evaluations such as bone marrow aspirations, blood sample collections, heart imaging (echocardiography or MUGA scans), and may have CT, MRI, PET scans, or lumbar punctures if needed. Researchers will monitor for treatment safety, side effects, and measure outcomes like minimal residual disease (MRD) and event-free survival over up to three years following treatment. The trial includes detailed assessments of drug effects, immune function, and long-term follow-up to better understand treatment impacts in infants with this form of leukemia.
CONDITIONS
Brief Title
Testing the Addition of the Anti-cancer Drug Venetoclax and/or the Anti-cancer Immunotherapy Blinatumomab to the Usual Chemotherapy Treatment for Infants With Newly Diagnosed KMT2A-rearranged or KMT2A-non-rearranged Leukemia
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Infants aged 365 days or less at diagnosis and older than 36 weeks gestational age at enrollment
- Newly diagnosed with B-acute lymphoblastic leukemia or acute leukemia of ambiguous lineage with at least 50% B lineage
- Leukemia cells must express CD19
- Enrolled on APEC14B1 and consented to eligibility screening prior to treatment and enrollment
- Written informed consent provided by parents or legal guardians
- Meets all institutional, FDA, and NCI human study requirements
You will not qualify if you...
- Diagnosis of Down Syndrome
- Secondary B-acute lymphoblastic leukemia after prior cytotoxic chemotherapy for another cancer
- Prior cytotoxic chemotherapy for current infant ALL or any cancer before starting protocol therapy, except limited steroid or intrathecal chemotherapy pretreatment as allowed
- Pretreatment with dexamethasone within 28 days prior to protocol therapy initiation, except a single dose around sedation
- Use of steroids beyond allowed limits or inhaled/topical steroids alone
- Use of hydroxyurea for more than 72 hours in the 7 days before enrollment
- Failure to sign informed consent or meet regulatory requirements
AI-Screening
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Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - 7 days
Participants receive prednisone, prednisolone, or methylprednisolone daily for 7 days before starting induction therapy.
Daily medication administration
Duration - 28 to 35 days
Participants receive chemotherapy and other medications to induce remission, including daunorubicin, vincristine, dexamethasone, calaspargase pegol, intrathecal therapy, and for some groups venetoclax.
Multiple visits for drug administration and monitoring over induction period
Duration - 28 to 35 days
Participants receive blinatumomab infusions with dexamethasone and intrathecal therapy; those with high disease levels may discontinue protocol therapy after this stage.
Multiple visits for IV infusions and intrathecal therapy
Duration - Approximately 56 days
Participants receive further chemotherapy including cyclophosphamide, cytarabine, mercaptopurine, and intrathecal therapy; venetoclax may be added for some cohorts.
Multiple visits for drug administration and monitoring
Duration - Approximately 49 days
Participants receive mercaptopurine, high-dose methotrexate with leucovorin rescue, intrathecal therapy, high-dose cytarabine, and asparaginase; venetoclax may be included for some cohorts.
Multiple visits for infusions and medications
Duration - 28 to 35 days
Participants receive a second cycle of blinatumomab infusions with intrathecal therapy.
Multiple visits for IV infusions and intrathecal therapy
Duration - Approximately 63 days
Participants receive intensive chemotherapy including calaspargase pegol, dexamethasone, thioguanine, vincristine, daunorubicin, cytarabine, cyclophosphamide, and intrathecal therapy.
Multiple visits for drug administration and monitoring
Duration - Up to 2 years
Participants receive cycles of mercaptopurine, methotrexate, and intrathecal therapy repeated every 12 weeks for up to 2 years to maintain remission.
Regular visits every 12 weeks with ongoing medication and intrathecal therapy for first 3 cycles
Duration - Up to 3 years
After completing treatment, participants are followed for up to 3 years to monitor health and disease status.
Periodic follow-up visits
Trial Site Locations
Total: 111 locations
1
Children's Hospital of Alabama
Birmingham, Alabama, United States, 35233
Actively Recruiting
2
Providence Alaska Medical Center
Anchorage, Alaska, United States, 99508
Actively Recruiting
3
Banner Children's at Desert
Mesa, Arizona, United States, 85202
Actively Recruiting
4
Arkansas Children's Hospital
Little Rock, Arkansas, United States, 72202-3591
Actively Recruiting
5
Loma Linda University Medical Center
Loma Linda, California, United States, 92354
Actively Recruiting
6
Miller Children's and Women's Hospital Long Beach
Long Beach, California, United States, 90806
Actively Recruiting
7
UCSF Benioff Children's Hospital Oakland
Oakland, California, United States, 94609
Actively Recruiting
8
Kaiser Permanente-Oakland
Oakland, California, United States, 94611
Actively Recruiting
9
Children's Hospital of Orange County
Orange, California, United States, 92868
Actively Recruiting
10
University of California Davis Comprehensive Cancer Center
Sacramento, California, United States, 95817
Actively Recruiting
11
UCSF Medical Center-Mission Bay
San Francisco, California, United States, 94158
Actively Recruiting
12
Children's Hospital Colorado
Aurora, Colorado, United States, 80045
Actively Recruiting
13
Connecticut Children's Medical Center
Hartford, Connecticut, United States, 06106
Actively Recruiting
14
Yale University
New Haven, Connecticut, United States, 06520
Actively Recruiting
15
Alfred I duPont Hospital for Children
Wilmington, Delaware, United States, 19803
Actively Recruiting
16
Children's National Medical Center
Washington D.C., District of Columbia, United States, 20010
Actively Recruiting
17
UF Health Cancer Institute - Gainesville
Gainesville, Florida, United States, 32610
Actively Recruiting
18
Memorial Regional Hospital/Joe DiMaggio Children's Hospital
Hollywood, Florida, United States, 33021
Actively Recruiting
19
Nemours Children's Clinic-Jacksonville
Jacksonville, Florida, United States, 32207
Actively Recruiting
20
University of Miami Miller School of Medicine-Sylvester Cancer Center
Miami, Florida, United States, 33136
Actively Recruiting
21
AdventHealth Orlando
Orlando, Florida, United States, 32803
Actively Recruiting
22
Arnold Palmer Hospital for Children
Orlando, Florida, United States, 32806
Actively Recruiting
23
Nemours Children's Hospital
Orlando, Florida, United States, 32827
Actively Recruiting
24
Nemours Children's Clinic - Pensacola
Pensacola, Florida, United States, 32504
Actively Recruiting
25
Johns Hopkins All Children's Hospital
St. Petersburg, Florida, United States, 33701
Actively Recruiting
26
Saint Joseph's Hospital/Children's Hospital-Tampa
Tampa, Florida, United States, 33607
Actively Recruiting
27
Saint Mary's Medical Center
West Palm Beach, Florida, United States, 33407
Actively Recruiting
28
Children's Healthcare of Atlanta - Arthur M Blank Hospital
Atlanta, Georgia, United States, 30329
Actively Recruiting
29
Kapiolani Medical Center for Women and Children
Honolulu, Hawaii, United States, 96826
Actively Recruiting
30
Saint Luke's Cancer Institute - Boise
Boise, Idaho, United States, 83712
Actively Recruiting
31
Lurie Children's Hospital-Chicago
Chicago, Illinois, United States, 60611
Actively Recruiting
32
Advocate Children's Hospital-Oak Lawn
Oak Lawn, Illinois, United States, 60453
Actively Recruiting
33
Advocate Children's Hospital-Park Ridge
Park Ridge, Illinois, United States, 60068
Actively Recruiting
34
Southern Illinois University School of Medicine
Springfield, Illinois, United States, 62702
Actively Recruiting
35
Riley Hospital for Children
Indianapolis, Indiana, United States, 46202
Actively Recruiting
36
University of Kentucky/Markey Cancer Center
Lexington, Kentucky, United States, 40536
Actively Recruiting
37
Norton Children's Hospital
Louisville, Kentucky, United States, 40202
Actively Recruiting
38
Children's Hospital New Orleans
New Orleans, Louisiana, United States, 70118
Actively Recruiting
39
Ochsner Medical Center Jefferson
New Orleans, Louisiana, United States, 70121
Actively Recruiting
40
Maine Children's Cancer Program
Scarborough, Maine, United States, 04074
Actively Recruiting
41
Sinai Hospital of Baltimore
Baltimore, Maryland, United States, 21215
Actively Recruiting
42
Johns Hopkins University/Sidney Kimmel Cancer Center
Baltimore, Maryland, United States, 21287
Actively Recruiting
43
Dana-Farber Cancer Institute
Boston, Massachusetts, United States, 02215
Actively Recruiting
44
UMass Memorial Medical Center - University Campus
Worcester, Massachusetts, United States, 01655
Actively Recruiting
45
C S Mott Children's Hospital
Ann Arbor, Michigan, United States, 48109
Actively Recruiting
46
Corewell Health Grand Rapids Hospitals - Helen DeVos Children's Hospital
Grand Rapids, Michigan, United States, 49503
Actively Recruiting
47
Bronson Methodist Hospital
Kalamazoo, Michigan, United States, 49007
Actively Recruiting
48
University of Minnesota/Masonic Cancer Center
Minneapolis, Minnesota, United States, 55455
Actively Recruiting
49
University of Mississippi Medical Center
Jackson, Mississippi, United States, 39216
Actively Recruiting
50
Children's Mercy Hospitals and Clinics
Kansas City, Missouri, United States, 64108
Actively Recruiting
51
Washington University School of Medicine
St Louis, Missouri, United States, 63110
Actively Recruiting
52
Mercy Hospital Saint Louis
St Louis, Missouri, United States, 63141
Actively Recruiting
53
Children's Hospital and Medical Center of Omaha
Omaha, Nebraska, United States, 68114
Actively Recruiting
54
Alliance for Childhood Diseases/Cure 4 the Kids Foundation
Las Vegas, Nevada, United States, 89135
Actively Recruiting
55
Dartmouth Hitchcock Medical Center/Dartmouth Cancer Center
Lebanon, New Hampshire, United States, 03756
Actively Recruiting
56
Hackensack University Medical Center
Hackensack, New Jersey, United States, 07601
Actively Recruiting
57
Morristown Medical Center
Morristown, New Jersey, United States, 07960
Actively Recruiting
58
Rutgers Cancer Institute of New Jersey-Robert Wood Johnson University Hospital
New Brunswick, New Jersey, United States, 08903
Actively Recruiting
59
Saint Joseph's Regional Medical Center
Paterson, New Jersey, United States, 07503
Actively Recruiting
60
Albany Medical Center
Albany, New York, United States, 12208
Actively Recruiting
61
Roswell Park Cancer Institute
Buffalo, New York, United States, 14263
Actively Recruiting
62
The Steven and Alexandra Cohen Children's Medical Center of New York
New Hyde Park, New York, United States, 11040
Actively Recruiting
63
Laura and Isaac Perlmutter Cancer Center at NYU Langone
New York, New York, United States, 10016
Actively Recruiting
64
Memorial Sloan Kettering Cancer Center
New York, New York, United States, 10065
Actively Recruiting
65
NYP/Weill Cornell Medical Center
New York, New York, United States, 10065
Actively Recruiting
66
University of Rochester
Rochester, New York, United States, 14642
Actively Recruiting
67
State University of New York Upstate Medical University
Syracuse, New York, United States, 13210
Actively Recruiting
68
Montefiore Medical Center - Moses Campus
The Bronx, New York, United States, 10467
Actively Recruiting
69
New York Medical College
Valhalla, New York, United States, 10595
Actively Recruiting
70
UNC Lineberger Comprehensive Cancer Center
Chapel Hill, North Carolina, United States, 27599
Actively Recruiting
71
Carolinas Medical Center/Levine Cancer Institute
Charlotte, North Carolina, United States, 28203
Actively Recruiting
72
Duke University Medical Center
Durham, North Carolina, United States, 27710
Actively Recruiting
73
East Carolina University
Greenville, North Carolina, United States, 27834
Actively Recruiting
74
Wake Forest University Health Sciences
Winston-Salem, North Carolina, United States, 27157
Actively Recruiting
75
Sanford Broadway Medical Center
Fargo, North Dakota, United States, 58122
Actively Recruiting
76
Children's Hospital Medical Center of Akron
Akron, Ohio, United States, 44308
Actively Recruiting
77
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States, 45229
Actively Recruiting
78
Rainbow Babies and Childrens Hospital
Cleveland, Ohio, United States, 44106
Actively Recruiting
79
Dayton Children's Hospital
Dayton, Ohio, United States, 45404
Actively Recruiting
80
ProMedica Toledo Hospital/Russell J Ebeid Children's Hospital
Toledo, Ohio, United States, 43606
Actively Recruiting
81
University of Oklahoma Health Sciences Center
Oklahoma City, Oklahoma, United States, 73104
Actively Recruiting
82
Lehigh Valley Hospital-Cedar Crest
Allentown, Pennsylvania, United States, 18103
Actively Recruiting
83
Geisinger Medical Center
Danville, Pennsylvania, United States, 17822
Actively Recruiting
84
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104
Actively Recruiting
85
Children's Hospital of Pittsburgh of UPMC
Pittsburgh, Pennsylvania, United States, 15224
Actively Recruiting
86
Rhode Island Hospital
Providence, Rhode Island, United States, 02903
Actively Recruiting
87
Prisma Health Richland Hospital
Columbia, South Carolina, United States, 29203
Actively Recruiting
88
BI-LO Charities Children's Cancer Center
Greenville, South Carolina, United States, 29605
Actively Recruiting
89
Sanford USD Medical Center - Sioux Falls
Sioux Falls, South Dakota, United States, 57117-5134
Actively Recruiting
90
East Tennessee Childrens Hospital
Knoxville, Tennessee, United States, 37916
Actively Recruiting
91
Saint Jude Children's Research Hospital
Memphis, Tennessee, United States, 38105
Actively Recruiting
92
The Children's Hospital at TriStar Centennial
Nashville, Tennessee, United States, 37203
Suspended
93
Vanderbilt University/Ingram Cancer Center
Nashville, Tennessee, United States, 37232
Actively Recruiting
94
Dell Children's Medical Center of Central Texas
Austin, Texas, United States, 78723
Actively Recruiting
95
Driscoll Children's Hospital
Corpus Christi, Texas, United States, 78411
Actively Recruiting
96
Medical City Dallas Hospital
Dallas, Texas, United States, 75230
Actively Recruiting
97
UT Southwestern/Simmons Cancer Center-Dallas
Dallas, Texas, United States, 75390
Actively Recruiting
98
El Paso Children's Hospital
El Paso, Texas, United States, 79905
Actively Recruiting
99
Cook Children's Medical Center
Fort Worth, Texas, United States, 76104
Actively Recruiting
100
Baylor College of Medicine/Dan L Duncan Comprehensive Cancer Center
Houston, Texas, United States, 77030
Actively Recruiting
101
Children's Hospital of San Antonio
San Antonio, Texas, United States, 78207
Actively Recruiting
102
Methodist Children's Hospital of South Texas
San Antonio, Texas, United States, 78229
Actively Recruiting
103
University of Virginia Cancer Center
Charlottesville, Virginia, United States, 22908
Actively Recruiting
104
Children's Hospital of The King's Daughters
Norfolk, Virginia, United States, 23507
Actively Recruiting
105
VCU Massey Comprehensive Cancer Center
Richmond, Virginia, United States, 23298
Actively Recruiting
106
Carilion Children's
Roanoke, Virginia, United States, 24014
Actively Recruiting
107
Seattle Children's Hospital
Seattle, Washington, United States, 98105
Actively Recruiting
108
Providence Sacred Heart Medical Center and Children's Hospital
Spokane, Washington, United States, 99204
Actively Recruiting
109
Saint Vincent Hospital Cancer Center Green Bay
Green Bay, Wisconsin, United States, 54301
Actively Recruiting
110
Children's Hospital of Wisconsin
Milwaukee, Wisconsin, United States, 53226
Actively Recruiting
111
University Pediatric Hospital
San Juan, Puerto Rico, 00926
Actively Recruiting
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
RANDOMIZED
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
8
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