Pediatric Clinical Trials

  • Ph II of Non-myeloablative Allogeneic Transplantation Using TLI & ATG In Patients w/ Cutaneous T Cell Lymphoma

    Non-myeloablative approach for allogeneic transplant is a reasonable option, especially given that the median age at diagnosis is 55-60 years and frequently present compromised skin in these patients, which increases the risk of infection. Therefore, we propose a clinical study with allogeneic HSCT using a unique non-myeloablative preparative regimen, TLI/ATG, to treat advanced MF/SS.

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  • Feasibility Neurocognitive Outcome After Transplant

    This pilot study will primarily be evaluated by feasibility and adherence to an iPad-based neurocognitive intervention program. It will secondarily be evaluated by performance on the neurocognitive testing post-transplant and change in performance in subsequent years.

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  • Assessment of Pain in Newborns and Older Infants (Infant Pain Assessment Study =

    Pain assessments in non-verbal, critically ill infants represent an important clinical challenge. Older children or adults can easily express their pain, but infants lack that capability. They frequently experience repetitive acute pain during routine ICU care, but their analgesic management flounders on the horns of a dilemma: (a) failure to treat infant pain leads to immediate clinical instability and potentially long-term physical, behavioral, and cognitive sequelae, vs. (b) strong analgesics may increase risks for medical complications and/or impaired brain growth. Bedside nurses currently assess pain using pain scores, before taking action to ameliorate pain. Pain scores increase nursing workload and provide subjective assessments, rather than objective data for evaluating infant pain. Consequently, infants exposed to skin-breaking procedures, surgery, or other painful conditions often receive variable and inconsistent pain management in the ICU. The investigators aim to develop a multimodal pain assessment system, using sensor fusion and novel machine learning algorithms to provide an objective measure of pain that is context-dependent and rater-independent. This will enhance the quality of pain management in ICUs and allow continuous pain monitoring in real-time.

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  • Outcomes In Children With Developmental Delay And Deafness

    Children with special needs require complex, individualized therapy to maximize their long-term quality of life. One subset of children with special needs includes those with both developmental delays and deafness. Currently, there is little compelling evidence supporting the idea that cochlear implantation provides benefit to children that don't have the cognitive potential to develop normal speech and language.

    We will perform a prospective, randomized clinical trial to answer the question of which intervention provides more benefit to this population of children using validated, norm-referenced tests.

    Our long-term goal is to develop guidelines that may help when selecting a treatment for hearing loss in a child with developmental delays.

    This proposal is significant because children with special needs are deserving of evidence upon which to base treatment decision-making, but remain under-represented in the medical literature and are often not studied. This research is designed to meet the criteria for the National Institutes of Health road map because it will generate this type of objective evidence that can directly improve patient care.

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  • Bortezomib and Combination Chemotherapy in Treating Patients With Relapsed or Refractory Acute Lymphoblastic Leukemia

    This study evaluates the value of bortezomib in combination with specified chemotherapies for the treatment of patients with relapsed or refractory acute lymphoblastic leukemia. Bortezomib may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth.

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  • Risk Factors for Acquired Cardiovascular Disease in Adults With Congenital Heart Disease

    This research study is to determine the risk factors for acquired heart disease, in adults with congenital heart disease. This knowledge is important to develop and target ways to prevent or delay the onset of acquired heart disease in adults with congenital heart disease.

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  • Shotgun Sequencing in Diagnosing Febrile Neutropenia in Patients With Acute Myeloid Leukemia

    This research trial studies the shotgun sequencing of blood samples in diagnosing febrile neutropenia in patients with acute myeloid leukemia. Studying samples of blood from patients with acute myeloid leukemia in the laboratory may help identify pathogens and accurately diagnose infections such as febrile neutropenia.

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  • Cerebral Oxygenation and Autoregulation in Preterm Infants

    Premature infants are at high risk for variations in blood pressure and oxygenation during the first few days of life. The immaturity of the premature brain may further predispose these infants to death or the development of neurologic problems. The relationship between unstable blood pressure and oxygen levels and brain injury has not been well elucidated.

    This study investigates the utility of near-infrared spectroscopy (NIRS), a non-invasive oxygen-measuring device, to identify preterm infants at highest risk for brain injury or death.

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  • Pharmacology of Aminophylline for Acute Kidney Injury in Neonates

    Acute kidney injury (AKI) in critically ill neonates is common and associated with significant morbidity and mortality. No targeted therapeutic treatment strategies have been established for AKI in neonates. Within a clinical pharmacokinetic and pharmacodynamic conceptual framework, this project will examine the medication aminophylline as a potential treatment approach for AKI.

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  • Genomic Analysis of Pediatric Bone Tumors

    To determine whether gene expression analysis of primary tumor samples before and after chemotherapy are predictive of long-term survival in pediatric patients with bone sarcomas (Ewings sarcoma (ES) and Osteosarcoma(OS)).

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  • Reading in Preterm and Full-term Children: Neural Basis and Prediction

    The purpose of this study is to understand reading abilities of children born preterm: their cognitive profiles, the neural basis of good and poor reading abilities, and the behavioral and neural factors that predict persistent difficulties. The investigators hope to learn

    - what specific skills correlate reading skills

    - if preterm children have different cognitive profiles than full term children with respect to reading

    - if cognitive skills measured in kindergarten predict reading ability in the second grade

    - if parts of the brain are associated with reading skill

    - whether brain characteristics in kindergarten predict reading in second grade.

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  • 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.

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  • Study of Brexucabtagene Autoleucel (KTE-X19) for the Treatment of Individuals With Relapsed/Refractory B-Cell Malignancies

    The primary objectives of this study are:

    Cohort 1: to provide access to brexucabtagene autoleucel (KTE-X19) for individuals with relapsed or refractory (r/r) mantle cell lymphoma (MCL) until KTE-X19 is commercially available

    Cohort 2: To provide access to KTE-X19 for individuals with r/r MCL whose commercially manufactured product did not meet commercial release specification(s)

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  • Ipilimumab +Nivolumab + Cryotherapy in Metastatic or Locally Advanced Soft Tissue Sarcoma

    The purpose of this Phase II study is to

    1. find out if the study drugs (ipilimumab plus nivolumab) in combination with cryotherapy will help participants with metastatic or locally advanced soft tissue sarcoma;.

    2. find out how safe are ipilimumab plus nivolumab given in combination with cryotherapy, and what side effects may be related to treatment.

    3. find out how do the study drugs in combination with cryotherapy work in soft tissue sarcoma.

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  • A Phase 1b Master Trial to Investigate CPX-351 in Subjects With Previously Untreated Acute Myeloid Leukemia

    JZP025-101 is an open-label, multicenter, multi-arm, nonrandomized phase 1b master trial to determine the recommended phase 2 dose (RP2D) of CPX-351 when administered in combination with various targeted agents in previously untreated subjects with Acute Myeloid Leukemia (AML) who are fit to receive intensive chemotherapy (ICT). Subjects will be assigned to treatment arms based on results of AML mutation testing.

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  • Safety and Efficacy of Axicabtagene Ciloleucel in Combination With Rituximab in Participants With Refractory Large B-Cell Lymphoma

    The primary objective of this study is to estimate the efficacy of axicabtagene ciloleucel in combination with rituximab, as measured by assessment of response rates in adult participants with relapsed/refractory large B-cell lymphoma.

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  • A Dose Escalation Study Evaluating CPI-818 in Relapsed/Refractory T-Cell Lymphoma

    This is a Phase 1/1b, open-label, first in human study of CPI-818, an oral interleukin-2-inducible tyrosine kinase (ITK) inhibitor for the treatment of relapsed/refractory (R/R) T-cell lymphoma.. This trial will study the safety, tolerability, and anti-tumor activity of CPI-818 as a single drug.

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  • Safety and Efficacy of ALLO-501 Anti-CD19 Allogeneic CAR T Cells in Adults With Relapsed/Refractory Large B Cell or Follicular Lymphoma

    The purpose of the ALPHA study is to assess the safety, efficacy, cell kinetics and immunogenicity of ALLO-501 in adults with relapsed or refractory large B-cell lymphoma or follicular lymphoma after a lymphodepletion regimen comprising fludarabine, cyclophosphamide, and ALLO-647.

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  • A Study to Investigate Blinatumomab in Combination With Chemotherapy in Patients With Newly Diagnosed B-Lymphoblastic Leukemia

    This phase III trial studies how well blinatumomab works in combination with chemotherapy in treating patients with newly diagnosed, standard risk B-lymphoblastic leukemia or B-lymphoblastic lymphoma with or without Down syndrome. Monoclonal antibodies, such as blinatumomab, may induce changes in the body's immune system and may interfere with the ability of cancer cells to grow and spread. Drugs used in chemotherapy, such as vincristine, dexamethasone, prednisone, prednisolone, pegaspargase, methotrexate, cytarabine, mercaptopurine, doxorubicin, cyclophosphamide, and thioguanine, 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. Leucovorin decreases the toxic effects of methotrexate. Giving monoclonal antibody therapy with chemotherapy may kill more cancer cells. Giving blinatumomab and combination chemotherapy may work better than combination chemotherapy alone in treating patients with B-ALL. This trial also assigns patients into different chemotherapy treatment regimens based on risk (the chance of cancer returning after treatment). Treating patients with chemotherapy based on risk may help doctors decide which patients can best benefit from which chemotherapy treatment regimens.

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  • IPH4102 Alone or in Combination With Chemotherapy in Patients With Advanced T Cell Lymphoma

    This is an open label, multi-cohort, and multi-center phase II study, which evaluates the clinical activity and safety of IPH4102 in Sezary Syndrome and Mycosis fungoides as single agent.

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