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The Big Picture
Active Clinical Studies at Children's Hospital & Research Center at Oakland

Children's Hospital & Research Center Oakland is committed to providing the most up-to-date information on currently active clinical studies within our institution. Tables listing all current studies, and organized by Principal Investigator, are provided and updated on a monthly basis. In addition, please feel free to review this page monthly for highlights of the most recently approved active clinical studies.

Full List of All Active CHRCO Clinical Studies
By Principal Investigator:

Clinical Studies Approved Within the Last Three Months:

A Multicenter, Open-label BMN 110 US Expanded Access Program (BMN 110 US EAP) to Provide BMN 110 to Patients Diagnosed with MPS IVA
MPS IVA is an inherited autosomal recessive disorder, which causes multiple clinical manifestations including impaired functional capacity, endurance, and quality of life. There is currently no standard accepted treatment for MPS IVA other than supportive care. Enzyme replacement therapy (ERT) with BMN 110 (rhGALNS) may be a potential new treatment option for MPS IVA patients. The objectives of this study, led by Paul Harmatz, MD, are to provide MPS IVA patients with access to BMN 110 until commercial product becomes available; and to collect additional information on BMN 110 safety and tolerability.

Full Synopsis


A Phase 1/2, Open Label Study Evaluating the Safety and Efficacy of Gene Therapy in Subjects with β-Thalassemia Major by Transplantation of Autologous CD34+ Stem Cells Transduced Ex Vivo with a Lentiviral βA-T87Q-Globin Vector (LentiGlobin® BB305 Drug Product)
Mark Walters, MD, is leading this non-randomized, open label, multi-site, single dose, phase 1/2 study to evaluate the safety and efficacy of autologous hematopoietic stem cell transplantation (HSCT) using LentiGlobin® BB305 Drug Product. Adults with β-thalassemia major who receive at least 100 mL/kg/year of packed red blood cells (pRBCs) or ≥8 transfusions of pRBCs per year in each of the 2 years preceding enrollment represent a population with severe disease at high risk of morbidity and mortality from transfusional iron-overload.

Full Synopsis


The Burden of Chikungunya and Dengue Transmission, Infection and Disease in Kenya
Outbreaks of arthropod-borne viruses, such as dengue (DENV) and chikungunya (CHIKV) viruses, demonstrate the substantial cost and health burden of these emerging/re-emerging health threats to the developing and developed world. A. Desiree LaBeaud, MD, MS, is leading this study to assess the true burden of human disease related to DENV strains 1-4 and for CHIKV across Kenya, and then to determine the drivers of viral circulation, estimate the thresholds for outbreak initiation, and provide improved outbreak risk assessment.

Full Synopsis

                 
Intracellular Magnesium and Calcium in Acute Asthma
Asthma is a major disease in pediatrics with substantial morbidity on an individual and community level, particularly in urban populations. Intravenous magnesium (Mg) has for some time been considered a safe and effective therapy for acute asthma, but the mechanisms by which Mg may be of benefit and the specific populations of patients who may particularly benefit from this therapy are incompletely studied. Sara Leibovic, MD is leading this study to investigate the levels of plasma and intracellular Mg, Ca, and the Ca:Mg ratio in patients suffering acute asthma exacerbations, patients with stable asthma between exacerbations, and in healthy controls.

Full Synopsis

              
Effects of iron overload on the mitochondrial genome in patients with thalassemia
Estaban Gomez, MD, is leading this study to investigate the relationship between iron overload and mitochondrial DNA damage in a group of well-characterized patients with beta-thalassemia major. Clinical measures of iron overload and iron toxicity will be correlated with mitochondrial DNA damage, specifically the frequency of the “common” deletion and mitochondrial genome copy number per cell. The utility of these metrics as biomarkers of iron toxicity that precedes organ damage will be studied.

Full Synopsis


Epidemiological Study of Childhood and Youth Diabetes in Developing Countries (Aim 3)
Janelle Noble, PhD, is leading this study to add to the understanding of childhood diabetes in Azerbaijan, Mali, Bangladesh, Sudan and Pakistan. The study will be done in partnership with the International Diabetes Federation Life for a Child program (based in Sydney, Australia), the Children’s Hospital Oakland Research Institute (Oakland, USA), and College of Medicine, University of Florida (Gainesville, USA).

Full Synopsis

iScreen: Evaluating Mobile Technology as a Tool to Elicit Social Determinants of Health
The iScreen study, led by Dayna Long, MD, is intented to create a simple, validated tool to help health care providers identify patient needs which extend beyond their immediate medical illness and may be contributing to their health.

Full Synopsis

A Randomized Phase II/III Study of Vorinostat (IND# 71976) and Local Irradiation OR Temozolomide and Local Irradiation OR Bevacizumab (IND# 7921) and Local Irradiation Followed by Maintenance Bevacizumab and Temozolomide in Children with Newly Diagnosed High-Grade Gliomas: A Groupwide Phase II/III Study (ACNS0822)
This study, led by Joseph Torkeldson, MD, will be done in three parts: an initial feasibility study within the phase II part to determinthe e safe and tolerable dose of vorinostatin combination with radiation therapy for patients less than 22 years of age; a phase II part that will compare 2 experimental treatment arms (bevacizumab or vorinostat during chemo-radiotherapy); and a randomized phase III part where patients will be randomized to either the chosen experimental arm or the temozolomide arm for a formal efficacy (EFS) comparison.

Full Synopsis

A Phase 3, Double-Blind, Randomized, Efficacy and Safety Comparison of Prasugrel and Placebo in Pediatric Patients with Sickle Cell Disease (Protocol H7T-MC-TADO)
Sickle cell disease (SCD) is a genetic blood disorder in which vaso-occlusive crisis (VOC) is the principle complication. A VOC can result in recurrent hospitalization and cause a variety of serious organ system complications, disabilities, and even death. The primary objective of this study, led by Lori A. Styles, MD, is to assess the efficacy of prasugrel compared to placebo in pediatric patients with SCD as measured by reduction in the rate of VOC.

Full Synopsis

Dietary Protein Effects on Trimethylamine-N-oxide (TMAO)
The aim of this protocol, led by Ronald Krauss, MD, is to test whether high red meat intake, compared to equivalent amounts of protein derived from white meat and non-meat sources, will result in higher fasting plasma levels of carnitine and TMAO, and an increase in 24-hour plasma and urinary TMAO levels following an isotope-labeled dietary carnitine and choline challenge. Findings from this study may yield a novel mechanistic basis for the relationship between high red meat intake and increased CVD risk.  

Full Synopsis

Eye-com Biosensor as a Seizure Detector
This study, led by Rachel Kuperman, MD, is to gather data to confirm that oculometric measures identified by a biosensor are as reliable as video EEG in predicting onset and real time tracking of the manifestations of seizures. Until now, EEG and clinical observations have been the only way to characterize and quantify seizures. The study will provide the foundation for the ultimate goal of developing a technology that can supplement or replace routine EEG and provide an alarm for autonomic instability in patients with epilepsy who are at risk for sudden death from epilepsy.

Full Synopsis

The Effects of Tranylcypromine on Gene and Protein Expression in Cells of Hematologic Origin
Altering gene expression may offer profound therapeutic benefits for conditions in which de-repression of gene expression permits the transcription of genes that can “rescue” a phenotype caused by a mutation in a closely related gene.  By a similar logic, genes whose transcription is repressed by pathologic mechanisms as occurs in neoplasia can be re-expressed with restoration of normal cellular functions including apoptosis. Tranylcypromine, a monoamine oxidase inhibitor, also inhibits the activity of chromatin-modifying enzymes that participate in gene expression. This study, led by Hugh Rienhoff, Jr., MD, will examine the effects TC has on hematopoietic gene expression in adult patients taking TC for the treatment of depression using a single 5 mL sample of blood.

Full Synopsis

Inpatient Outcomes and Chemotherapy Related Toxicities Among a National Cohort of Children with Acute Leukemia
National Cancer Institute (NCI) funded cooperative oncology group trials have improved overall survival for children with cancer from 10% to 85% over the past five decades, and have set standards of care for adults with malignancies. However, according to the Institute of Medicine: “the clinical trial system…is approaching a state of crisis. If [it] does not improve its efficiency and effectiveness, the introduction of new treatments for cancer will be delayed and patient lives will be lost unnecessarily.” The goal of this study, led by James Feusner, MD, is to improve toxicity monitoring, estimate treatment associated resource utilization and costs, and serve as a platform for answering important clinical epidemiology questions by merging data from the Children’s Oncology Group (COG) and the Pediatric Health Information Systems (PHIS) administrative data base for patients treated for de novo acute myeloid leukemia (AML) on recent Phase III clinical trials.

Full Synopsis

Long Term Follow Up Physical Exercise Program (LTFU-PEP)
Pamela Orren, PhD is leading this study to determine whether a 12 week long, biweekly exercise program will lead to a decrease in body mass index (BMI) and increased quality of life (QoL) in obese pediatric cancer survivors. We aim to see an increase in overall physical health, a more robust quality of life and decrease symptoms of depression and anxiety of participants who have completed the exercise program.  Results of this pilot study will inform about effective interventions to treat childhood obesity in pediatric cancer survivors.

Full Synopsis

Hemoglobin S Polymerization and Modification Screening
This study, led by Elliott Vichinsky, MD, is a collaboration between Global Blood Therapeutics and Children's aimed at idenitfying  compounds that in vitro and increase HbS oxygen affinity and decrease HbS polymerization.  These compounds will then be tested in animal models with the hope of  demonstrating a decrease in red cell sickling.

Full Synopsis

Full List of All Active CHRCO Clinical Studies
By Principal Investigator:

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