Pediatric Clinical Trials

  • Trametinib in Treating Patients With Epithelioid Hemangioendothelioma That Is Metastatic, Locally Advanced, or Cannot Be Removed by Surgery

    This phase II trial studies how well trametinib works in treating patients with epithelioid hemangioendothelioma that has spread to other places in the body, nearby tissue or lymph nodes, or cannot be removed by surgery. Trametinib may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth.

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  • Combination Chemotherapy With or Without Bortezomib in Treating Younger Patients With Newly Diagnosed T-Cell Acute Lymphoblastic Leukemia or Stage II-IV T-Cell Lymphoblastic Lymphoma

    This randomized phase III trial compares how well combination chemotherapy works when given with or without bortezomib in treating patients with newly diagnosed T-cell acute lymphoblastic leukemia or stage II-IV T-cell lymphoblastic lymphoma. Bortezomib may help reduce the number of leukemia or lymphoma cells by blocking some of the enzymes needed for cell growth. It may also help chemotherapy work better by making cancer cells more sensitive to the drugs. It is not yet known if giving standard chemotherapy with or without bortezomib is more effective in treating T-cell acute lymphoblastic leukemia and T-cell lymphoblastic lymphoma.

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  • Moderately Preterm Infants With Caffeine at Home for Apnea (MoCHA) Trial

    The objective of this study is to evaluate the effect of continuing treatment with caffeine citrate in the hospital and at home in moderately preterm infants with resolved apnea of prematurity on days of hospitalization after randomization.

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  • Hydrocortisone for Term Hypotension

    This trial will evaluate the effects of a 7-day course of hydrocortisone therapy on short-term morbidity, cardiovascular function, long-term neurodevelopment, and mortality in critically ill, term and late preterm infants diagnosed with cardiovascular insufficiency as defined by a need for inotrope therapy in the first 72 hours of age.

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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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  • Sorafenib (BAY 43-9006) to Treat Children With Solid Tumors or Leukemias

    Background:

    - Sorafenib is an experimental anti-cancer drug that works by blocking proteins thought to be important for tumor growth. It has blocked the growth of tumors in test tubes and in some animal models.

    - Sorafenib is approved for the treatment of advanced kidney cancer.

    - This is the first time sorafenib is being tested in children with cancer.

    Objectives:

    - To determine the highest dose of sorafenib that can be safely given to children and young adults with cancer.

    - To determine the side effects and benefits, if any, of sorafenib in children and young adults with cancer.

    - To study how the body handles sorafenib and the drug's effects on cells and proteins in the blood.

    Eligibility:

    -Patients between 2 and 21 years of age with solid tumors or leukemias that do not respond to standard treatment.

    Patients between 2 and 21 years of age with AML and FLT3-ITD mutation for Part C of the study.

    Design:

    - Patients take sorafenib tablets every day about every 12 hours in 28-day treatment cycles for a maximum of 24 cycles.

    - The dose is increased in succeeding groups of 3 to 6 children until serious side effects occur. Subsequent patients then receive the drug at lower doses.

    - Patients have a physical exam, blood and urine tests, and CT or MRI scans periodically to monitor safety and treatment effects. Leukemia patients also have bone marrow aspirates. Patients with a solid tumor may have an MRI. Children whose bones are still growing have X-rays of the lower legs periodically to monitor bone structure.

    - Patients have additional blood studies to determine the amount of sorafenib in the blood, the effect of the drug on proteins and cells in the blood, and genetic differences that affect how individuals respond to the drug.

    Investigator

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  • A Study of Clofarabine in Combination With Etoposide and Cyclophosphamide in Children With Acute Leukemias.

    Clofarabine (injection) is approved by the Food and Drug Administration (FDA) for the treatment of pediatric patients 1 to 21 years old with relapsed or refractory acute lymphoblastic leukemia (ALL) who have had at least 2 prior treatment regimens. This use is based on the induction of complete responses. Randomized trials demonstrating increased survival or other clinical benefit have not been conducted.

    The purpose of the phase 1 portion of this study was to determine if clofarabine added to a combination of etoposide and cyclophosphamide is safe in children with relapsed or refractory acute lymphoblastic leukemia (ALL) or acute myelogenous leukemia (AML). The purpose of the phase 2 portion of the study was to measure the effectiveness of the combination therapy in children with ALL.

    Investigators

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  • Gene Replacement Therapy Clinical Trial for Patients With Spinal Muscular Atrophy Type 1

    Phase 3 pivotal US trial studying open-label intravenous administration of AVXS-101 in SMA Type 1 patients

    Investigator

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  • ID Of Prognostic Factors In Mycosis Fungoides/Sezary Syndrome

    The purpose of the study is to develop a prognostic index model for the rare disease of mycosis fungoides and sezary syndrome. This will be done by collecting standardized clinical data at various institutions. The investigators hope this will enable the identification of low- and high-risk groups for survival in order to improve patient care and outcome.

    Investigator

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  • Phase 1/2A Dose Escalation Study in CLL, SLL or NHL

    This study will identify the highest dose, and assess the safety, of cerdulatinib (PRT062070) that may be given in patients with relapsed/refractory chronic lymphocytic leukemia or non-hodgkin lymphoma

    Investigator

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  • A Safety, Tolerability and PK Study of DCC-2618 in Patients With Advanced Malignancies

    This is a Phase 1, open-label, first-in-human (FIH) dose-escalation study designed to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD) and preliminary antitumor activity of DCC-2618, administered orally (PO), in adult patients with advanced malignancies. The study consists of 2 parts, a dose-escalation phase and an expansion phase.

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  • CD19/CD22 Chimeric Antigen Receptor T Cells and Chemotherapy in Treating Patients With Recurrent or Refractory CD19 Positive Diffuse Large B-Cell Lymphoma or B Acute Lymphoblastic Leukemia

    This phase I trial studies the 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 patients with CD19 positive diffuse large B-cell lymphoma or 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 diffuse large B-cell lymphoma and B acute lymphoblastic leukemia. Drugs used in chemotherapy, such as cyclophosphamide and fludarabine phosphate, 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 patients with diffuse large B-cell lymphoma or B acute lymphoblastic leukemia.

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  • A Phase III Trial of Anlotinib in Metastatic or Advanced Alveolar Soft Part Sarcoma, Leiomyosarcoma and Synovial Sarcoma

    This study evaluates the safety and efficacy of AL3818 (anlotinib) hydrochloride in the treatment of metastatic or advanced alveolar soft part sarcoma (ASPS), leiomyosarcoma (LMS), and synovial sarcoma (SS). All participants with ASPS will receive open-label AL3818. In participants with LMS or SS, AL3818 will be compared to IV dacarbazine. Two-thirds of the participants will receive AL3818, one-third of the participants will receive IV dacarbazine.

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  • Safety Study of ABT-263 in Combination With Rituximab in Lymphoid Cancers

    This is a Phase 1 study evaluating the safety of ABT-263 administered in combination with rituximab in subjects with CD20-positive lymphoproliferative disorders. The extension portion of the study will allow active subjects to continue to receive ABT-263 for up to 7 years after the last subject transitions with quarterly study evaluations.

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  • Ibrutinib, Idarubicin and Cytarabine in Treating Patients With Relapsed or Refractory Acute Myeloid Leukemia

    This phase I trial studies the side effects and best dose of ibrutinib when given together with idarubicin and cytarabine in treating patients with acute myeloid leukemia that has returned after a period of improvement or has not responded to previous treatment. Ibrutinib may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. Drugs used in chemotherapy, such as idarubicin and cytarabine, work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Giving ibrutinib together with idarubicin and cytarabine may kill more cancer cells.

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  • S1312, Inotuzumab Ozogamicin and Combination Chemotherapy in Treating Patients With Relapsed or Refractory Acute Leukemia

    This phase I trial studies the side effects and best dose of inotuzumab ozogamicin when given together with combination chemotherapy in treating patients with relapsed or refractory acute leukemia. Immunotoxins, such as inotuzumab ozogamicin, can find cancer cells that express cluster of differentiation (CD)22 and kill them without harming normal cells. Drugs used in chemotherapy, such as cyclophosphamide, vincristine sulfate, and prednisone, work in different ways to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. Giving inotuzumab ozogamicin together with combination chemotherapy may kill more cancer cells.

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  • Lenalidomide and Ibrutinib in Treating Patients With Relapsed or Refractory Chronic Lymphocytic Leukemia or Small Lymphocytic Lymphoma

    This phase I trial studies the side effects and best dose of lenalidomide when given together with ibrutinib in treating patients with chronic lymphocytic leukemia or small lymphocytic lymphoma that has returned after a period of improvement or does not respond to treatment. Biological therapies, such as lenalidomide, may stimulate the immune system in different ways and stop cancer cells from growing. Ibrutinib may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. Giving lenalidomide together with ibrutinib may work better in treating chronic lymphocytic leukemia or small lymphocytic lymphoma.

    Investigator

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  • Elevate CLL R/R: Study of Acalabrutinib (ACP-196) Versus Ibrutinib in Previously Treated Subjects With High Risk Chronic Lymphocytic Leukemia

    This study is designed to evaluate PFS endpoint for acalabrutinib vs ibrutinib in previously treated chronic lymphocytic leukemia.

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  • Combination Chemotherapy With or Without Blinatumomab in Treating Patients With Newly Diagnosed BCR-ABL-Negative B Lineage Acute Lymphoblastic Leukemia

    This randomized phase III trial studies combination chemotherapy with blinatumomab to see how well it works compared to induction chemotherapy alone in treating patients with newly diagnosed breakpoint cluster region (BCR)-c-abl oncogene 1, non-receptor tyrosine kinase (ABL)-negative B lineage acute lymphoblastic leukemia. Drugs used in chemotherapy 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. Monoclonal antibodies, such as blinatumomab, may block cancer growth in different ways by targeting certain cells. It is not yet known whether combination chemotherapy is more effective with or without blinatumomab in treating newly diagnosed acute lymphoblastic leukemia.

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  • Elevate CLL TN: Study of Obinutuzumab + Chlorambucil, Acalabrutinib (ACP-196) + Obinutuzumab, and Acalabrutinib in Subjects With Previously Untreated CLL

    This study is evaluating the efficacy of obinutuzumab in combination with chlorambucil compared with acalabrutinib in combination with obinutuzumab (Arm B) for the treatment of previously untreated chronic lymphocytic leukemia (CLL).

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