Pediatric Clinical Trials

  • Haploid Allogeneic Transplant Using the CliniMACS System

    To assess the proportion of patients with donor neutrophil engraftment within 30 days of allogeneic transplant. To assess the incidence of acute GvHD during the first 100 days after transplantation.

    Investigators

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

    The purpose of this Phase 2 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.

    Investigator

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  • Trabectedin in Treating Young Patients With Recurrent or Refractory Soft Tissue Sarcoma or Ewing's Family of Tumors

    This phase II trial is studying how well trabectedin works in treating young patients with recurrent or refractory soft tissue sarcoma or Ewing's family of tumors. Drugs used in chemotherapy such as trabectedin use different ways to stop tumor cells from dividing so they stop growing or die.

    Investigator

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  • Inhaled Nitric Oxide (INO) for the Prevention of Bronchopulmonary Dysplasia (BPD) in Preterm Infants

    This phase 3, multi-center, double blind, placebo-controlled, randomized clinical trial will attempt to demonstrate if preterm infants who require mechanical ventilation and/or positive pressure support at any point during days 5 to 14 after birth may benefit from treatment with iNO.

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  • Phase 1/2 Study of Enasidenib (AG-221) in Adults With Advanced Hematologic Malignancies With an Isocitrate Dehydrogenase Isoform 2 (IDH2) Mutation

    The primary objectives of Phase 1 Dose Escalation/Part 1 Expansion are:

    - To assess the safety and tolerability of treatment with enasidenib administered continuously as a single agent dosed orally on Days 1 to 28 of a 28-day cycle in participants with advanced hematologic malignancies.

    - To determine the maximum tolerated dose (MTD) or maximum administered dose (MAD) and/or the recommended Phase 2 dose (RP2D) of enasidenib in participants with advanced hematologic malignancies.

    The primary objective of Phase 2 is:

    • To assess the efficacy of enasidenib as treatment for participants with relapsed or refractory (R/R) acute myelogenous leukemia (AML) with an IDH2 mutation.

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  • Epidemiology of Painful Procedures in Neonates

    Sick or premature neonates are exposed to frequent painful and stressful procedures during their stay in neonatal intensive care units. Although neonates do feel pain and may have long term effects induced by painful experiences, prevention and treatment of neonatal pain is far from optimal in many units. An epidemiological study (EPIPPAIN) conducted in neonatal and pediatric intensive care units in France in 2005 showed that painful procedures were extremely frequent and that analgesics treatments varied a lot among units. Since many guidelines have been issued by international scientific societies to manage neonatal pain, one may expect that the prevention and treatment of neonatal pain has improved over the last 6 years.

    Although awake endotracheal intubations are extremely difficult or impossible in older children or adults, such intubations are still frequently performed without sedation/analgesia in neonates. Recent studies have shown that premedications facilitate intubation conditions and greatly improve neonates tolerance of the procedure. Studies aimed at assessing the risks and benefits of different sedations/analgesia strategies are urgently needed in neonates. We also need a tool to assess at the same time the technical conditions of intubations and the tolerance of the neonate to the procedure so that data from different studies can be compared.

    The objectives of the present study are:

    1. To describe the incidence of painful and stressful procedures performed in the neonate in intensive care units as well as in neonates transported by the medical emergency system (SMUR) of the Ile-de-France region 6 years after the first EPPIPAIN study conducted in the same region and same type of population in order to assess the evolution of practices. The description of painful and stressful procedures will be completed with a real-time around-the-clock assessment of the pain induced by procedures using a validated behavioral pain scale.

    2. To link this study with The Epipage study 2 in order to look for associations between the number of painful and stressful procedures and/or analgesic treatments of the neonatal period and the neurological outcome of children that will be followed in the Epipage cohort. The Epipage study is a separate study that will follow for 13 years a cohort of premature neonates recruited in 2011.

    3. To describe the incidence of painful or stressful procedures and analgesic treatments in neonates transported by the pediatric emergency system (SMUR) of the Ile-de-France region in France.

    4. To obtain initial validity of a tool permitting to assess intubations in neonates. An observational detailed description of endotracheal intubations conditions will be conducted in neonates transported and intubated by SMUR and in neonates intubated in intensive care units

    5. To describe continuous sedation and analgesia practices in ventilated neonates in intensive care units. For these neonates, data from medical records will be recovered up to 2 months of admission in intensive care units

    6. To describe the frequency of heel sticks for glycemia measurement and blood gazes practices among centers. Relate heel stick practices to the normality or abnormality of glycemia results

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  • Study of Tisagenlecleucel in Combination With Ibrutinib in r/r Diffuse Large B-cell Lymphoma Patients

    A multi-center, open-label, phase Ib study to evaluate the safety and tolerability of the administration of tisagenlecleucel in combination with ibrutinib in patients with r/r DLBCL who have received two or more lines of systemic therapy, including an anti-CD20 and anthracycline based chemotherapy, and who have progressed after or are not candidates for ASCT.

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  • Ruxolitinib Phosphate in Treating Patients With Chronic Neutrophilic Leukemia or Atypical Chronic Myeloid Leukemia

    This phase II trial studies how well ruxolitinib phosphate works in treating patients with chronic neutrophilic leukemia (CNL) or atypical chronic myeloid leukemia (aCML). Ruxolitinib phosphate may stop the growth of cancer cells by blocking some of the enzymes needed for cells to reproduce. This trial also studies the genetic makeup of patients. Certain genes in cancer cells may determine how the cancer grows or spreads and how it may respond to different drugs. Studying how the genes associated with CNL and aCML respond to the study drug may help doctors learn more about CNL and aCML and improve the treatment for these diseases.

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  • F18PET/CT Versus TC-MDP Scanning to Detect Bone Mets

    The primary objective is to compare the diagnostic performance of 18F- Fluoride PET/CT scanning to that of conventional bone scanning for detecting cancer that has spread to the bone (bone metastasis). The intent of the study is to determine whether 18F-Fluoride PET/CT will lead to improved treatment and patient outcomes.

    Investigators

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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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  • 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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  • Extended Treatment and Follow-up of Subjects Treated With Belumosudil in Study KD025-208 or Study KD025-213

    Extended Treatment and Follow-up of Subjects Treated with Belumosudil in Study KD025-208 or Study KD025-213

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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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  • Efficacy and Safety Study of Idelalisib in Participants With Indolent B-Cell Non-Hodgkin Lymphomas

    The primary objective will be to assess the overall response rate and to evaluate the efficacy and safety of idelalisib (IDELA; GS-1101) in participants with previously treated indolent Non-Hodgkin Lymphoma (iNHL) that is refractory both to rituximab and to alkylating-agent-containing chemotherapy.

    Eligible participants will initiate oral therapy with idelalisib at a starting dose of 150 mg taken twice per day. Treatment with idelalisib can continue in compliant participants as long as the study is still ongoing and the participants appear to be benefiting from treatment with acceptable safety.

    Investigators

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  • Peripheral Blood Stem Cell Transplant vs Bone Marrow Transplant in Individuals With Hematologic Cancers (BMT CTN 0201)

    The study is designed as a Phase III, randomized, open label, multicenter, prospective, comparative trial of granulocyte colony stimulating factor (G-CSF)-mobilized peripheral blood stem cells (PBSC) versus marrow from unrelated donors for transplantation in patients with hematologic malignancies. Recipients will be stratified by transplant center and disease risk and will be randomized to either the PBSC or marrow arm in a 1:1 ratio.

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  • High Dose Chemotherapy and Allogeneic Hematopoietic Cell Transplant for Non-Hodgkin's Lymphoma

    To evaluate the role of allogeneic hematopoietic cell transplantation in the treatment of NHL.

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  • Phase 2 Total Skin Electron Beam Therapy (TSEBT 12 Gy) in Stage IB-IIIA Mycosis Fungoides

    To examine the efficacy and safety of total skin electron beam therapy to a dose of 12 Gray (TSEBT 12 Gy) in patients who have mycosis fungoides (MF) staged as IB to IIIA.

    Investigators

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  • Study of the Safety and Tolerability of PCI-32765 in Patients With Recurrent B Cell Lymphoma

    The purpose of this study is to establish the safety and optimal dose of orally administered PCI-32765 in patients with recurrent B cell lymphoma.

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  • Study of KW-0761 Versus Vorinostat in Relapsed/Refractory CTCL

    The purpose of this study is to compare the progression free survival of KW-0761 versus vorinostat for subjects with relapsed or refractory CTCL.

    Investigators

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  • Risk-Group Classification of Patients With Newly Diagnosed Acute Lymphoblastic Leukemia

    This clinical trial is studying risk-group classification of patients with newly diagnosed acute lymphoblastic leukemia. Developing a risk-group classification guide may help doctors assign patients with newly diagnosed acute lymphoblastic leukemia to treatment clinical trials.

    Investigator

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