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Pediatric Hematology/Oncology, Phase I drug studies for refractory and relapsed leukemia; genomic studies, biologic risk-stratification and treatment of acute myeloid leukemia; prediction or induction response and risk of relapse using phosphoproteomics in childhood AML; novel MRD techniques in childhood ALL.
Protocol for Collecting, Banking and Distributing Human Tissue Samples: St. Jude Children's Research Hospital Tissue Resources Core Facility
The aims of this protocol are: to collect and store diseased and normal tissue and body fluid
samples from new and returning patients at St. Jude Children's Research Hospital (SJCRH),
affiliated sites and collaborating institutions; to collect and store samples from relatives
of SJCRH patients; to collect and store retrospective and prospective pertinent corresponding
clinical and laboratory data on disease characterization, treatment, and outcome; and to
serve as a source of human biological samples and corresponding laboratory and clinical data.
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Total Therapy XVII for Newly Diagnosed Patients With Acute Lymphoblastic Leukemia and Lymphoma
The overarching objective of this study is to use novel precision medicine strategies based
on inherited and acquired leukemia-specific genomic features and targeted treatment
approaches to improve the cure rate and quality of life of children with acute lymphoblastic
leukemia (ALL) and acute lymphoblastic lymphoma (LLy).
Primary Therapeutic Objectives:
- To improve the event-free survival of provisional standard- or high-risk patients with
genetically or immunologically targetable lesions or minimal residual disease (MRD) ≥ 5%
at Day 15 or Day 22 or ≥1% at the end of Remission Induction, by the addition of
molecular and immunotherapeutic approaches including tyrosine kinase inhibitors or
chimeric antigen receptor (CAR) T cell / blinatumomab for refractory B-acute
lymphoblastic leukemia (B-ALL) or B-lymphoblastic lymphoma (B-LLy), and the proteasome
inhibitor bortezomib for those lacking targetable lesions.
- To improve overall treatment outcome of T acute lymphoblastic leukemia (T-ALL) and
T-lymphoblastic lymphoma (T-LLy) by optimizing pegaspargase and cyclophosphamide
treatment and by the addition of new agents in patients with targetable genomic
abnormalities (e.g., activated tyrosine kinases or JAK/STAT mutations) or by the
addition of bortezomib for those who have a poor early response to treatment but no
targetable lesions, and by administering nelarabine to T-ALL and T-LLy patients with
leukemia/lymphoma cells in cerebrospinal fluid at diagnosis or MRD ≥0.01% at the end of
- To determine in a randomized study design whether the incidence and/or severity of acute
vincristine-induced peripheral neuropathy can be reduced by decreasing the dosage of
vincristine in patients with the high-risk CEP72 TT genotype or by shortening the
duration of vincristine therapy in standard/high-risk patients with the CEP72 CC or CT
Secondary Therapeutic Objectives:
- To estimate the event-free survival and overall survival of children with ALL and to
assess the non-inferiority of TOTXVII compared to the historical control given by
- To estimate the event-free survival and overall survival of children with LLy when ALL
diagnostic and treatment approaches are used.
- To evaluate the efficacy of blinatumomab in B-ALL patients with end of induction MRD
≥0.01% to <1% and those (regardless of MRD level or TOTXVII risk category) with the
genetic subtypes of BCR-ABL1, ABL-class fusion, JAK-STAT activating mutation,
hypodiploid, iAMP21, ETV6-RUNX1-like, MEF2D, TCF3-HLF, or BCL2/MYC or with Down
syndrome, by comparing event-free survival to historical control from TOTXVI.
- To determine the tolerability of combination therapy with ruxolitinib and Early
Intensification therapy in patients with activation of JAK-STAT signaling that can be
inhibited by ruxolitinib and Day 15 or Day 22 MRD ≥5%, Day 42 MRD ≥1%, or LLy patients
without complete response at the End of Induction and all patients with early T cell
- To use data from clinical genomic sequencing of diagnosis, germline/remission and MRD
samples to guide therapy, including incorporation of targeted agents and institution of
genetic counseling and cancer surveillance.
- To evaluate and implement deoxyribonucleic acid (DNA) and ribonucleic acid (RNA)
sequencing-based methods to monitor levels of MRD in bone marrow, blood, and
- To assess clonal diversity and evolution of pre-leukemic and leukemic populations using
DNA variant detection and single-cell genomic analyses in a non-clinical, research
- To identify germline or somatic genomic variants associated with drug resistance of ALL
cells to conventional and newer targeted anti-leukemic agents in a non-clinical,
- To compare drug sensitivity of ALL cells from diagnosis to relapse in vitro and in vivo
and determine if acquired resistance to specific agents is related to specific somatic
genome variants that are not detected or found in only a minor clone at initial
Supportive Care Objectives
- To conduct serial neurocognitive monitoring of patients to investigate the
neurocognitive trajectory, mechanisms, and risk factors.
- To evaluate the impact of low-magnitude high frequency mechanical stimulation on bone
mineral density and markers of bone turnover.
There are several Exploratory Objectives.
Safety and Dose Finding Study of Neratinib in Children and Young Adults With Cancer That Has Returned or Not Responded to Treatment
The purpose of this study is to test the safety of neratinib at different dose levels and to
find out what effects, good and bad, it has on the patients and the cancer.
Study of Venetoclax in Combination With Chemotherapy in Pediatric Patients With Refractory or Relapsed Acute Myeloid Leukemia or Acute Leukemia of Ambiguous Lineage
The purpose of this study is to test the safety and determine the best dose of venetoclax and
cytarabine when given with or without idarubicin in treating pediatric patients with acute
myeloid leukemia (AML) that did not respond to treatment (refractory) or has come back after
PRIMARY OBJECTIVE: Determine a tolerable combination of venetoclax plus chemotherapy in
pediatric patients with relapsed or refractory AML or acute leukemia of ambiguous lineage.
The primary endpoints are the recommended phase 2 doses (RP2D) of venetoclax plus cytarabine
and venetoclax plus cytarabine and idarubicin.
SECONDARY OBJECTIVE: Estimate the overall response rate to the combination of venetoclax and
chemotherapy in pediatric patients with relapsed or refractor AML or acute leukemia of
ambiguous lineage. The secondary endpoints are the rates of complete remission (CR) and
complete remission with incomplete count recovery (CRi) for patients treated at the RP2D.
A Trial of Epigenetic Priming in Patients With Newly Diagnosed Acute Myeloid Leukemia
The overall aim of this study is to determine if epigenetic priming with a DNA
methyltransferase inhibitor (DMTi) prior to chemotherapy blocks is tolerable and carries
evidence of a clinical efficacy signal as determined by minimal residual disease (MRD),
event-free survival (EFS), and overall survival (OS). Tolerability for each of the agents, as
well as total reduction in DNA methylation and outcome assessments will be done to
simultaneously obtain preliminary biological and clinical data for each DMTi in parallel.
- Evaluate the tolerability of five days of epigenetic priming with azacitidine and
decitabine as a single agent DMTi prior to standard AML chemotherapy blocks.
- Evaluate the change in genome-wide methylation burden induced by five days of epigenetic
priming and the association of post-priming genome-wide methylation burden with
event-free survival among pediatric AML patients.
- Describe minimal residual disease levels following Induction I chemotherapy in patients
that receive DMTi.
- Estimate the event-free survival and overall survival of patients receiving a DMTi prior
to chemotherapy courses.
Phase I Dose Escalation Study of CD19/CD22 Chimeric Antigen Receptor (CAR) T Cells in Children and Young Adults With Recurrent or Refractory B Cell Malignancies
This phase I trial studies the best dose and side effects of CD19/CD22 chimeric antigen
receptor (CAR) T cells when given together with chemotherapy, and to see how well they work
in treating children or young adults with CD19 positive B acute lymphoblastic leukemia that
has come back or does not respond to treatment. A CAR is a genetically-engineered receptor
made so that immune cells (T cells) can attack cancer cells by recognizing and responding to
the CD19/CD22 proteins. These proteins are commonly found on B acute lymphoblastic leukemia.
Drugs used in chemotherapy, such as fludarabine phosphate and cyclophosphamide, work in
different ways to stop the growth of cancer cells, either by killing the cells, by stopping
them from dividing, or by stopping them from spreading. Giving CD19/CD22-CAR T cells and
chemotherapy may work better in treating children or young adults with B acute lymphoblastic
A Study to Test the Safety and Efficacy of the Drug Larotrectinib for the Treatment of Tumors With NTRK-fusion in Children
The study is being done to test the safety of a cancer drug called larotrectinib in children.
The cancer must have a change in a particular gene (NTRK1, NTRK2 or NTRK3). Larotrectinib
blocks the actions of these NTRK genes in cancer cells and can therefore be used to treat
The first study part (Phase 1) is done to determine what dose level of larotrectinib is safe
for children, how the drug is absorbed and changed by their bodies and how well the cancer
responds to the drug. The main purpose of the second study part (Phase 2) is to investigate
how well and how long different cancer types respond to the treatment with larotrectininb.
Total Therapy for Infants With Acute Lymphoblastic Leukemia (ALL) I
The purpose of this study is to test the good and bad effects of the study drugs bortezomib
and vorinostat when they are given in combination with chemotherapy commonly used to treat
acute lymphoblastic leukemia (ALL) in infants. For example, adding these drugs could decrease
the number of leukemia cells, but it could also cause additional side effects. Bortezomib and
vorinostat have been approved by the US Food and Drug Administration (FDA) to treat other
cancers in adults, but they have not been approved for treating children with leukemia. With
this research, we plan to meet the following goals:
- Determine the tolerability of incorporating bortezomib and vorinostat into an ALL
chemotherapy backbone for newly diagnosed infants with ALL.
- Estimate the event-free survival and overall survival of infants with ALL who are
treated with bortezomib and vorinostat in combination with an ALL chemotherapy backbone.
- Measure minimal residual disease (MRD) positivity using both flow cytometry and PCR.
- Compare end of induction, end of consolidation, and end of reinduction MRD levels to
Interfant99 (ClinicalTrials.gov registration ID number NCT00015873) participant
Stanford is currently not accepting patients for this trial.
For more information, please contact Norman J. Lacayo, MD, 650-497-8953.
Carfilzomib in Combination With Cyclophosphamide and Etoposide for Children
This study evaluates the use of carfilzomib in combination with cyclophosphamide and
etoposide for children with relapsed/refractory solid tumors or leukemia. The medications
cyclophosphamide and etoposide are standard drugs often used together for the treatment of
cancer in children with solid tumors or leukemia.
Carfilzomib is FDA (Food and Drug Administration) approved in the United States for adults
with multiple myeloma (a type of cancer). However, this drug is not approved to treat
children with relapsed/refractory solid tumors or leukemia. With this research, we plan to
determine the DLTs and MTD of Carfilzomib given in combination with cyclophosphamide and
etoposide in pediatric patients with relapsed/refractory leukemias and solid tumors.
Efficacy and Safety of Decitabine as Epigenetic Priming With Induction Chemotherapy in Pediatric Acute Myelogenous Leukemia (AML) Subjects
The purpose of this study is to provide data on the activity of a standard daunorubicin,
cytarabine, and etoposide (ADE) induction plus epigenetic priming with decitabine as assessed
by standard measures of complete remission (CR), leukemia free survival (LFS) and overall
survival (OS), as well as, on minimal residual disease (MRD). It will also provide necessary
data on the safety and Pharmacokinetics (PK) of decitabine in pediatric patients that is
Stanford is currently not accepting patients for this trial.
For more information, please contact SPECTRUM, .
Genome, Proteome and Tissue Microarray in Childhood Acute Leukemia
We will study gene and protein expression in leukemia cells of children diagnosed with acute
leukemia. We hope to identify genes or proteins which can help us grade leukemia at diagnosis
in order to: (a) develop better means of diagnosis and (b) more accurately choose the best
therapy for each patient.
Stanford is currently not accepting patients for this trial.
For more information, please contact Norman J Lacayo, 650-723-5535.
A Study Evaluating the Safety, Tolerability, Pharmacokinetics and Preliminary Activity of Idasanutlin in Combination With Either Chemotherapy or Venetoclax in Treatment of Pediatric and Young Adult Participants With Relapsed/Refractory Acute Leukemias or Solid Tumors
This is a Phase I/II, multicenter, open-label, multi-arm study designed to evaluate the
safety, tolerability, pharmacokinetics, and preliminary efficacy of idasanutlin, administered
as a single agent or in combination with chemotherapy or venetoclax, in pediatric and young
adult participants with acute leukemias or solid tumors.
This study is divided into three parts: Part 1 will begin with dose escalation of idasanutlin
as a single agent in pediatric participants with relapsed or refractory solid tumors to
identify the maximum tolerated dose (MTD)/maximum administered dose (MAD) and to characterize
dose-limiting toxicities (DLTs). Following MTD/MAD identification, three separate safety
run-in cohorts in neuroblastoma, acute myeloid leukemia (AML), and acute lymphoblastic
leukemia (ALL) will be conducted to identify the recommended Phase 2 dose (RP2D) of
idasanutlin in each combination, with chemotherapy or venetoclax. Part 2 will evaluate the
safety and early efficacy of idasanutlin in combination with chemotherapy or venetoclax in
newly enrolled pediatric and young adult participants in neuroblastoma, AML,and ALL cohorts
at idasanutlin RP2D. Part 3 will potentially be conducted as an additional expansion phase of
the idasanutlin combination cohorts in neuroblastoma, AML, or ALL for further response and
Dose Escalation Study of CLR 131 in Children, Adolescents, and Young Adults With Relapsed or Refractory Malignant Tumors Including But Not Limited to Neuroblastoma, Rhabdomyosarcoma, Ewings Sarcoma, and Osteosarcoma
The study evaluates CLR 131 in children, adolescents, and young adults with relapsed or
refractory malignant solid tumors and lymphoma and recurrent or refractory malignant brain
tumors for which there are no standard treatment options with curative potential.