Education and Training

  • Study of the Usability and Efficacy of a New Pediatric CPAP Mask

    This study will evaluate a newly developed pediatric mask (known as Pixi) on children aged 2-7 using continuous positive airway pressure (CPAP), or Non-invasive ventilation (NIV) treatment. The participants will undergo a monitored sleep study, followed by a 7 night trial of the Pixi mask in the home environment. During the study usability will be measured through questionnaires filled in by the parent and clinician.

    The study hypothesis is that the usability of the mask will be superior to the patient's usual mask.

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  • Paclitaxel + Bevacizumab (Avastin) for the Treatment of Metastatic or Unresectable Angiosarcoma

    This is an open-label, single-arm, multi-center, Phase 2 study with Paclitaxel in combination with Bevacizumab in patients with Unresectable or Metastatic Angiosarcoma. The study aims to determine the safety and effectiveness of combining two drugs Paclitaxel and Bevacizumab in the treatment of Angiosarcoma that cannot be removed by surgery, or has spread to other parts of your body. The primary objective is to evaluate 4month non progression rate. The secondary objective is to evaluate overall response rate after 3rd and 6th cycle, median duration of response, 6th and 12th month survival, toxicity of Paclitaxel and Bevacizumab combination, toxicity of maintenance Bevacizumab and to collect paraffin-embedded tumor blocks for angiogenesis markers and tissue microarray.

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  • Open Label Two-Arm Study to Evaluate Rilzabrutinib in IgG4-Related Disease Patients

    This is a Phase 2a, multi-center, open-label, two-arm study of approximately 25 patients with active IgG4-related disease (IgG4-RD). The two arms include (1) Experimental: rilzabrutinib with glucocorticoids and (2) Active Comparator: glucocorticoids only.

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  • Point-of-Care System for Determination of Bilirubin Capacity in Neonates

    The aims of this observational bench project are to validate the performance of the miniaturized and modernized hematofluorometer that measures bilirubin capacity into a product and is suitable for operation in various point of care environments w in the management of preterm neonates.

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  • Phase 1-2 of Temozolomide and Hypofractionated Radiotherapy in Tx of Supratentorial Glioblastoma Multiform

    The purpose of this study is to investigate the safety and effectiveness of a combination treatment for glioblastoma multiforme utilizing radiotherapy plus the FDA-approved chemotherapy drug temozolomide

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  • Study to Assess the Safety, Tolerability, Pharmacokinetics, and Effect on Disease Progression of BIIB078 Administered to Previously Treated Adults C9ORF72-Associated Amyotrophic Lateral Sclerosis (ALS)

    The primary objective is to evaluate the long-term safety and tolerability of BIIB078 in participants with chromosome 9 open reading frame 72-amyotrophic lateral sclerosis (C9ORF72-ALS).

    The secondary objective is to evaluate the pharmacokinectic (PK) of BIIB078 in participants with C9ORF72-ALS.

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  • Selinexor in Advanced Liposarcoma

    This is a randomized, multicenter, double-blind, placebo-controlled, Phase 2-3 study of patients diagnosed with advanced unresectable dedifferentiated liposarcoma. Approximately 342 total patients will be randomized to study treatment (selinexor or placebo).

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  • Measurements and Characterization of Doppler Signals From the Right Chest in Pediatric and Adult Patients

    Recently it has been shown that clear reproducible Doppler signals can be recorded from the lung parenchyma by means of a pulsed Doppler ultrasound system incorporating a special signal processing package parametric Doppler, TPD, EchoSense Ltd., Haifa, Israel). These lung Doppler signals (LDS) are in full synchrony with the cardiac cycle and can be obtained from the lungs, including areas remote from the heart and main pulmonary vessels. The LDS waves typically have peak velocities of up to 30 cm/s and are of relatively high power, making it possible to detect them despite the aforementioned attenuation by the air in the lungs. The LDS are thought to represent the radial wall movement of small pulmonary blood vessels, caused by pressure pulse waves of cardiac origin which propagate throughout the lung vasculature. The LDS may contain information of significant diagnostic and physiological value regarding the pulmonary parenchyma and vasculature, as well as the cardio-vascular system in general.

    Pulmonary arterial hypertension (PAH) is a condition characterized by reshaping of the small pulmonary arteries with increase in pulmonary vascular resistance, leading gradually to right-sided cardiac failure. A trans-thoracic echocardiograph (TTE) is a test classically undertaken in order to screen for pulmonary hypertension. However, the systolic pulmonary artery pressure (SPAP) values thereby obtained are often imprecise and depend upon the expertise of the individual carrying out the test. Therefore, the pulmonary arterial pressure and cardiac output values have to be ascertained with a right-sided cardiac catheterization, which is considered the gold-standard, but is invasive.

    In a pilot study of adult PAH patients (unpublished), lung Doppler signals have been shown to have the potential to diagnose pulmonary hypertension in two different ways: First, by measuring the degree of attenuation of the LDS during acute pressure rise in the chest cavity (i.e. during Valsalva maneuver). Second, by detecting differences between the LDS in patients with PAH and control subjects.

    One of the objectives of the present study is to evaluate the lung Doppler signals in pediatric patients of various age groups, with and without pulmonary vascular disease. The second objective of the study is to verify previous findings of abnormal lung Doppler signals in adult patients with pulmonary hypertension.

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  • Phase II Study of AS1411 Combined With Cytarabine to Treat Acute Myeloid Leukemia

    The overall aim of this study is to assess the efficacy and safety of AS1411, over a range of doses, when combined with cytarabine, in the treatment of patients with primary refractory or relapsed acute myeloid leukemia (AML).

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  • Patient-Centered Opioid Tapering Study

    This is an observational study of a voluntary opioid tapering protocol conducted in community outpatients taking long term prescription opioids for chronic pain. Patients who would otherwise continue with their existing opioid prescriptions were encouraged to participate in a voluntary opioid taper program. Interested patients were identified by their pain physician, Dr. Richard Stieg, and then completed an online informed consent document and baseline self-report assessments including types and doses of opioid medications as well as demographic and psychosocial measures, clinical and pain characteristics. Patients were either given or mailed a free copy of a patient book The Opioid-Free Pain Relief Kit, or Less Pain, Fewer Pills: Avoid the dangers of prescription opioids and gain control over chronic pain. Over the following weeks and up to 4 months, Dr. Stieg implemented a slow, individually tailored opioid taper in all patient participants. Follow-up online self-report surveys were completed at 4 months post enrollment. Opioid dose data were confirmed via medical chart review, and doses were converted to a standardized morphine equivalent daily dose (MEDD). Main outcome was change in opioid dose baseline to 4 months. Secondary outcome was change in pain intensity (numeric rating scale, 0-10) baseline to 4 months.

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  • Pomalidomide in Treating Younger Patients With Recurrent, Progressive, or Refractory Central Nervous System Tumors

    This phase I trial studies the side effects and best dose of pomalidomide in treating younger patients with tumors of the brain or spine (central nervous system) that have come back or are continuing to grow. Pomalidomide may interfere with the ability of tumor cells to grow and spread and may also stimulate the immune system to kill tumor cells.

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  • Safety and Efficacy of Eculizumab in Refractory Generalized Myasthenia Gravis (REGAIN Study)

    The purpose of this study is to determine if eculizumab is safe and effective for the treatment of refractory generalized Myasthenia Gravis.

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  • Sorafenib Tosylate in Treating Patients With Recurrent Aggressive Non-Hodgkin's Lymphoma

    This phase II trial is studying how well sorafenib works in treating patients with recurrent diffuse large B-cell non-Hodgkin's lymphoma. Sorafenib may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth and by blocking blood flow to the tumor.

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  • Phase 4 Study of Obeticholic Acid Evaluating Clinical Outcomes in Patients With Primary Biliary Cholangitis

    Primary Biliary Cholangitis (PBC) is a serious, life-threatening, bile acid related liver disease of unknown cause. Without treatment, it frequently progresses to liver fibrosis and eventual cirrhosis requiring liver transplantation or resulting in death. The investigational drug, Obeticholic Acid (OCA) is a modified bile acid and FXR agonist that is derived from the primary human bile acid chenodeoxycholic acid. The key mechanisms of action of OCA, including its choleretic, anti-inflammatory, and anti-fibrotic properties, underlie its hepatoprotective effects and result in attenuation of injury and improved liver function in a cholestatic liver disease such as PBC. The study will assess the effect of OCA compared to placebo, combined with stable standard care, on clinical outcomes in PBC participants.

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  • Pilot Immunotherapy Study With Letetresgene Autoleucel (Lete-cel, GSK3377794)T-cells in New York Esophageal Squamous Cell Carcinoma-1 (NY-ESO-1)/ LAGE-1a-positive Advanced Non-small Cell Lung Cancer (NSCLC) Either Alone or in Combination With Pembrolizumab

    This trial will evaluate safety and tolerability of letetresgene autoleucel (GSK3377794) with or without pembrolizumab in participants with non-small cell lung cancer.

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  • Positive Pressure Treatment of Obstructive Sleep Apnea

    Six month at home positive pressure therapy study; which mode of therapy will lead to better adherence and patient outcomes?

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  • PHIL in the Treatment of Intracranial dAVF.

    This study is a prospective, multicenter, single-arm study. Patients with Dural Arteriovenous Fistulas (dAVF) have a few choice for safe treatment. In this study, all patients with qualifying dAVFs will be treated with PHIL® Liquid Embolic material.

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  • Serial Ultrasound in Metastatic Renal Cell Carcinoma (mRCC)

    To assess whether changes in quantitative tumor perfusion parameters after 3 weeks of treatment, as measured by power Doppler ultrasound, can predict initial objective response, defined by current standard-of-care, to therapy at 12 weeks after start of treatment

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  • Study Evaluating Efficacy And Tolerability Of Veliparib in Combination With Temozolomide (TMZ) or In Combination With Carboplatin and Paclitaxel Versus Placebo in Participants With Breast Cancer Gene (BRCA)1 and BRCA2 Mutation and Metastatic Breast Cancer

    The primary objective of the study is to assess the progression-free survival (PFS) of oral veliparib in combination with TMZ or in combination with carboplatin and paclitaxel compared to placebo plus carboplatin and paclitaxel in subjects with BRCA1 or BRCA2 mutation and locally recurrent or metastatic breast cancer.

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  • Midostaurin in Treating Older Patients With Mutated Acute Myeloid Leukemia Post-Transplant

    This phase 2 trial studies the side effects and how well midostaurin works in treating older patients with acute myeloid leukemia with change in genetic material post-hematopoietic cell transplantation. Midostaruin may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. Giving midostaruin post-transplant may improve patient outcomes.

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