Pediatric Clinical Trials

  • Transplantation of Ex Vivo Expanded, UCB-derived, Stem & Progenitor Cells vs. Unmanipulated UCB for HM Patients

    This study is an open-label, controlled, multicenter, international, Phase III, randomized study of transplantation of NiCord® versus transplantation of one or two unmanipulated, unrelated cord blood units in patients with acute lymphoblastic leukemia or acute myeloid leukemia, myelodysplastic syndrome, chronic myeloid leukemia or lymphoma, all with required disease features rendering them eligible for allogeneic transplantation.

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  • Radiation Therapy With or Without Combination Chemotherapy or Pazopanib Hydrochloride Before Surgery in Treating Patients With Newly Diagnosed Non-rhabdomyosarcoma Soft Tissue Sarcomas That Can Be Removed by Surgery

    This randomized phase II/III trial studies how well pazopanib hydrochloride, combination chemotherapy, and radiation therapy work and compares it to radiation therapy alone or in combination with pazopanib hydrochloride or combination chemotherapy in treating patients with newly diagnosed non-rhabdomyosarcoma soft tissue sarcomas that can be removed by surgery. Radiation therapy uses high energy x-rays to kill tumor cells. Drugs used in chemotherapy, such as ifosfamide and doxorubicin hydrochloride, 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. Pazopanib hydrochloride may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. It is not yet known whether radiation therapy works better when given with or without combination chemotherapy and/or pazopanib hydrochloride in treating patients with non-rhabdomyosarcoma soft tissue sarcomas.

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  • Ibrutinib and Obinutuzumab With or Without Venetoclax in Treating Patients With Chronic Lymphocytic Leukemia

    This phase III trial studies how well ibrutinib and obinutuzumab with or without venetoclax work in treating patients with chronic lymphocytic leukemia. Ibrutinib may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. Immunotherapy with monoclonal antibodies, such as obinutuzumab, may help the body's immune system attack the cancer, and may interfere with the ability of tumor cells to grow and spread. Drugs used in chemotherapy, such as venetoclax, 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 ibrutinib, obinutuzumab and venetoclax may work better in treating patients with chronic lymphocytic leukemia.

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  • Study to Evaluate the Safety and Efficacy of Adeno-IFN Gamma in Cutaneous B-cell Lymphoma

    The primary objective of this study is to evaluate the efficacy of a four-month dosing period of intra-lesional injection of TG1042 in patients with relapsing CBCL.

    Patients will receive intra-tumoral injections of an adenoviral vector construct containing the human interferon gamma gene (TG1042), in an attempt to enhance immune responses with anti-tumor activity. This local administration induces tumour cell killing at the injected tumour sites.

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  • FLASH [Fluorescent Light Activated Synthetic Hypericin] Clinical Study: Topical SGX301 (Synthetic Hypericin) for the Treatment of Cutaneous T-Cell Lymphoma (Mycosis Fungoides)

    To evaluate the use of SGX301, a topical photosensitizing agent, to treat patients with patch/plaque phase cutaneous T-cell lymphoma (mycosis fungoides).

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  • Pembrolizumab and Interferon Gamma-1b in Treating Patients With Stage IB-IVB Relapsed or Refractory Mycosis Fungoides and Sezary Syndrome

    This phase II trial studies how well pembrolizumab and interferon gamma-1b work in treating patients with stage IB-IVB mycosis fungoides and sezary syndrome that has come back or has not responded to previous treatment. Immunotherapy with monoclonal antibodies, such as pembrolizumab, may help the body's immune system attack the cancer, and may interfere with the ability of tumor cells to grow and spread. Interferon gamma-1b may boost the immune system activity. Giving pembrolizumab and interferon gamma-1b together may work better in treating patients with stage IB-IVB mycosis fungoides and sezary syndrome.

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  • Naloxone Hydrochloride Study for Relief of Pruritus in Patients With MF or SS Forms of CTCL

    This multi-center, double-blind, vehicle-controlled, randomized crossover design study will evaluate the safety and efficacy of topically applied naloxone lotion, 0.5%, for the treatment of pruritus in patients with the mycosis fungoides (MF) or SĂ©zary syndrome (SS) Forms of Cutaneous T-cell Lymphoma (CTCL). This study will also determine if there is systemic absorption of the drug in a subset of subjects and if so, describe the range and mean plasma levels reached after two weeks of three time daily (TID) dosing.

    Funding Source - FDA OOPD

    Investigator

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  • Dosing of Brentuximab Vedotin for Mycosis Fungoides, Sezary Syndrome Patients

    The purpose of this study is to test any good and bad effects of the study drug called brentuximab vedotin at a lower dose than is FDA-approved.

    Investigator

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  • A Trial of TTI-621 for Patients With Hematologic Malignancies and Selected Solid Tumors

    Multicenter, open-label, phase 1a/1b trial of TTI-621 in subjects with relapsed or refractory hematologic malignancies and selected solid tumors.

    Investigator

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  • A Safety, Efficacy and Pharmacokinetics Study of CD11301 for the Treatment of Cutaneous T-Cell Lymphoma (CTCL)

    To assess the efficacy, safety and pharmacokinetics in subjects treated with CD11301 gel vs. placebo for early stage CTCL (IA, IB, or IIA).

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  • Inositol to Reduce Retinopathy of Prematurity

    This is a Phase 3, randomized, double-masked, placebo-controlled study designed to determine the effectiveness of myo-Inositol 5% Injection to increase the incidence of survival without severe Retinopathy of Prematurity (ROP) through acute/final ROP determination up to 55 weeks postmenstrual age (PMA) in premature infants <28 0/7 weeks' gestation.

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  • Incubator Weaning of Moderately Preterm Infants

    The objective of this proposal is to evaluate whether weaning from an incubator to a crib at lower versus higher weight, 1600g or 1800g will result in shorter length of hospital stay among moderately preterm infants.

    The hypothesis of this study is that length of hospital stay (from birth to discharge) will be decreased among moderate preterm infants weaned from an incubator to an open crib at a lower versus higher weight, 1600g vs. 1800g.

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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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  • Pilot Lenalidomide in Adult Diamond-Blackfan Anemia Patients w/ RBC Transfusion-Dependent Anemia

    This is a single-center, single arm, open-label study of oral lenalidomide monotherapy administered to red blood cell (RBC) transfusion dependent adult subjects with Diamond-Blackfan Anemia (DBA).

    Primary Objective: To evaluate the erythroid response rate as measured by rate of red blood cell transfusion independence [MDS International Working Group (IWG) 2000 Criteria will be applied].

    Secondary Objective: 1)To evaluate the tolerability and safety profile of lenalidomide in patients with DBA and other inherited marrow failure syndromes 2) To correlate response to lenalidomide with biologic surrogates of DBA including ribosomal protein mutation status, ex vivo erythroid colony growth, and microarray gene expression

    Investigator

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  • CPG 7909 + Local Radiotherapy in Recurrent Low-Grade Lymphomas

    Brief summary TBD

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  • A Study of ACP-196 (Acalabrutinib) in Subjects With Relapsed/Refractory CLL and Intolerant of Ibrutinib Therapy

    A Phase 2 Study to evaluate the Efficacy and Safety of ACP-196 (acalabrutinib) in Subjects with Relapsed/Refractory CLL and Intolerant of Ibrutinib Therapy

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  • Study of Blood Samples From Newborns With Down Syndrome

    This research study is looking at blood samples from newborns with Down syndrome. Studying the genes expressed in samples of blood from patients with Down syndrome may help doctors identify biomarkers related to cancer.

    Investigator

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  • Genes in Predicting Outcome of Patients With DLBCL Treated With Rituximab and Combination Chemotherapy (R-CHOP)

    The investigators hypothesize that survival of newly diagnosed DLBCL (diffuse large B-cell lymphoma) patients treated with R-CHOP can be predicted by RNA or protein gene expression or by presence of biomarkers associated with the anti-tumor effects of Rituximab.

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  • Collecting and Storing Samples of Blood and Tumor Tissue From Patients With Osteosarcoma

    The purpose of this study is to collect and store samples of blood and tumor tissue from patients with osteosarcoma. Collecting and storing samples of tumor tissue and blood from patients to test in the laboratory may help the study of cancer in the future.

    Investigators

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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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