Education and Training

  • In Infants With Laryngomalacia, Does Acid-Blocking Medication Improve Respiratory Symptoms?

    All neonates, ages 0 to 4 months, presenting to LPCH pediatric ENT clinic for airway difficulties or stridor will be screened for inclusion. As is consistent with an acceptable standard of medical care, these children will undergo a flexible nasal endoscopic exam to make the diagnosis of laryngomalacia, as well as be weighed and a breastfeeding history taken. If laryngomalacia is present, the study staff with then administer the Infant Gastroesophageal Reflux Questionnaire (IGERQ) and an airway symptoms questionnaire (ASQ).

    Those babies with an IGERQ score of less than sixteen (no more than mild reflux) and an ASQ score greater than six will be eligible for randomization. The patient will then be randomly placed in the control group (placebo) or the intervention group (ranitidine 2mg/kg every 12 hours or famotidine 0.5 mg/kg daily). Patients will stay on medication for a minimum of 6 months, or until symptoms resolve. Patients will be seen in follow up at 1, 2, 3, 4, 5, 6, 8 and 10 months. At which time I-GERQ, ASQ and weights will be taken. The primary outcome measure will be the time for the ASQ score to drop to normal on ranitidine or famotidine versus placebo.

    A secondary outcome will be weight gain in percentile. If the patient's I-GERQ score goes above 16 at any time in the study, the patient will be crossed over to the treatment arm and started on medical treatment.

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  • Outcome Measures in Infant/Early Childhood Lung Disease w/ Chest CT Scanning & Lung Function Testing

    To implement a new method of performing chest CT imaging in young children at Packard Children's Hospital entitled controlled ventilation infant/young child chest CT scanning. This technique will be used to evaluate early lung disease comparing quantitative chest CT air trapping and airway measurements with lung function measurements in infants, toddlers, and young children with chronic lung disease.

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  • Oral Triple Combination Antiviral Drug Therapy for Treatment of Influenza A in Immunocompromised Subjects

    This Phase 2, open label, randomized study will investigate the virologic benefit, clinical efficacy, safety, and tolerability of amantadine and ribavirin with oseltamivir (TCAD) versus oseltamivir monotherapy for the treatment of all strains of influenza A in immunocompromised adult and pediatric subjects.

    Investigator

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  • Insulin Resistance Intervention After Stroke Trial

    The purpose of this study is to determine if pioglitazone is effective in preventing future strokes or heart attacks among non-diabetic persons who have had a recent ischemic stroke.

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  • Phase 2 Study of ISIS 681257 (AKCEA-APO(a)-LRx) in Participants With Hyperlipoproteinemia(a) and Cardiovascular Disease

    This is a multicenter, randomized, double-blind, placebo-controlled, dose-ranging study to evaluate the safety, including tolerability, of ISIS 681257 and to assess the efficacy of different doses and dosing regimens of ISIS 681257 for reduction of plasma Lipoprotein(a) [Lp(a)] levels in participants with hyperlipoproteinemia(a) and established cardiovascular disease (CVD).

    Investigator

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  • Pulmonary Interstitial Lymphography in Early Stage Lung Cancer

    The stereotactic body radiation therapy (SBRT) procedure is an emerging alternative to the standard treatment for early stage non-small cell lung cancer (NSCLC), typically lobectomy with lymphadenectomy. This procedure (lobectomy) does not fulfill the medical need as many patients are poor operative candidates or decline surgery.

    This study assesses the feasibility of stereotactic body radiation therapy (SBRT) as a tool to produce therapeutically useful computed tomography (CT) scans, using standard water-soluble iodinated compounds as the contrast agents.

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  • Observation, Radiation Therapy, Combination Chemotherapy, and/or Surgery in Treating Young Patients With Soft Tissue Sarcoma

    This phase III trial is studying observation to see how well a risk based treatment strategy works in patients with soft tissue sarcoma. In the study, patients are assigned to receive surgery +/- radiotherapy +/- chemotherapy depending on their risk of recurrence. Sometimes, after surgery, the tumor may not need additional treatment until it progresses. In this case, observation may be sufficient. Radiation therapy uses high-energy x-rays to kill tumor cells. Drugs used in chemotherapy, such as ifosfamide and doxorubicin, work in different ways to stop the growth of tumor cells, either by killing the cells or by stopping them from dividing. Giving chemotherapy and radiation therapy before surgery may make the tumor smaller and reduce the amount of normal tissue that needs to be removed. Giving these treatments after surgery may kill any tumor cells that remain after surgery.

    Investigators

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  • Internet Weight Control for Bingeing Adolescents

    The purpose of this study is to determine if an internet-based intervention designed to reduce binge eating and improve healthy weight regulation skills will result in reductions in binge eating and weight loss, or weight maintenance, in overweight adolescents who binge eat.

    Investigator

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  • Impact of Treatment Induced Premature Menopause on Quality of Life

    To understand the impact of treatment induced menopause on quality of life and sexuality following blood and marrow transplantation.

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  • Observational Study to Evaluate Allogeneic HSCT Outcomes for Cerebral Adrenoleukodystrophy (CALD)

    Study ALD-103 will be a multi-site, global, prospective and retrospective data collection study that is designed to evaluate outcomes of allo-HSCT in male subjects with CALD ≤17 years of age.

    Investigator

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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.

    Investigator

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  • Limited-English Proficiency (LEP) Virtual Reality (VR) Study

    The purpose of this study is to determine if non-invasive distracting devices (Virtual Reality headset) are more effective than the standard of care (i.e., no technology based distraction) for preventing anxiety in parental and pediatric populations with limited English proficiency (LEP).

    Investigator

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  • Ipilimumab With or Without Sargramostim in Treating Patients With Stage III or Stage IV Melanoma That Cannot Be Removed by Surgery

    This randomized phase II trial is studying how well giving ipilimumab with or without sargramostim (GM-CSF) works in treating patients with stage III or stage IV melanoma that cannot be removed by surgery (unresectable). Ipilimumab works by activating the patient's immune system to fight cancer. Colony-stimulating factors, such as sargramostim, may increase the number of immune cells found in bone marrow or peripheral blood and may help the immune system recover from the side effects of treatment. It is not yet known whether giving ipilimumab together with sargramostim is more effective than ipilimumab alone in treating melanoma.

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  • Hoarding Disorder Treatment With Virtual Reality

    This study explores whether a facilitated peer support group called Buried in Treasures (BIT) and a virtual reality decluttering practice can help individuals with clutter challenges.

    Investigator

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  • Idiopathic Focal Segmental Glomerulosclerosis (FSGS) and Treatment With ACTH

    FSGS is an immunologic disorder wherein circulating immune proteins cause damage to the kidneys and progressive injury and scarring. Corticosteroid therapy is occasionally, but not nearly universally, successful in reducing proteinuria, and when patients respond, they have a favorable prognosis. The investigators believe that ACTH therapy (H.P. Acthar Gel) can provide a more rapid, well tolerated reduction in glomerular injury.

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  • 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.

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  • Intralesional Cemiplimab for Adult Patients With Cutaneous Squamous Cell Carcinoma or Basal Cell Carcinoma

    This study is researching an experimental drug called cemiplimab. The study is focused on Cutaneous Squamous Cell Carcinoma (CSCC) and Basal Cell Carcinoma (BCC).

    The aim of the study is to evaluate the safety and tolerability (how your body reacts to the drug) of cemiplimab (also known as REGN2810).

    The first part of the study tested several different doses of cemiplimab given weekly for 12 weeks.

    The study is also looking at several other research questions, including:

    - What side effects may happen from taking the study drug

    - To see effect of cemiplimab on the tumor

    - How much study drug is in the blood at different times

    Investigator

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  • INCB7839 in Treating Children With Recurrent/Progressive High-Grade Gliomas

    This is a multicenter phase 1 trial of INCB7839 for children with recurrent or progressive high-grade gliomas, including, but not limited to, diffuse intrinsic pontine glioma (DIPG) and other diffuse midline gliomas (DMGs), after upfront therapy.

    Investigator

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  • Hepatocellular Carcinoma Study Comparing Vaccinia Virus Based Immunotherapy Plus Sorafenib vs Sorafenib Alone

    This is a randomized Phase 3 study to determine whether treatment with vaccinia virus based immunotherapy (Pexa-Vec) followed by sorafenib increases survival compared to treatment with sorafenib in patients with advanced hepatocellular carcinoma who have not received prior systemic therapy.

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  • Impact of a Ketogenic Diet on Metabolic and Psychiatric Health in Patients With Bipolar Illness

    To initiate a low-carbohydrate, high-fat (LCHF) or ketogenic dietary (KD) intervention among a cohort of outpatients with bipolar illness who also have metabolic abnormalities, overweight/obesity, and/or are currently taking psychotropic medications experiencing metabolic side effects.

    Investigator

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