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The Big Picture
Active Clinical Studies at Children's Hospital & Research Center at Oakland
Children's Hospital & Research Center at 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. Recently vitamin D3, but not its hydroxylated metabolites, has been found to inhibit hedgehog signaling, the pathway whose abnormal activity is responsible for the development of basal cell carcinomas (BCCs), the most common human cancer. This would suggest that exposure to ultraviolet radiation, by producing high levels of vitamin D3, might restrain the growth of BCCs despite the contrary effect of increased pro-carcinogenic mutagenesis with increasing exposure to ultraviolet radiation.
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
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Clinical Studies Approved Within the Last Three Months:
A Phase II Study of IMC-A12 (Anti-IGF-I Receptor Monoclonal Antibody in Children with Relapsed/Refractory Solid Tumors (ADVL0821)
Carla Golden, MD, is the local lead investigator for this COG Phase II study of IMC-sA12 (ImClone Systems), a monoclonal antibody to Insulin-Like Growth Factor Receptor (IGF-IR), in pediatric patients with recurrent/refractory solid tumors. The investigators propose to evaluate the safety and efficacy of IMC-A12 in children and adolescents with relapsed or refractory solid tumors.
A Phase 2, 24 Week, Randomized, Open Label, Multi-Center Study to Assess the Safety, Tolerability, and Pharmaco- dynamics of FBS0701 in the Treatment of Chronic Iron Overload Requiring Chelation Therapy
This is an open-label, 24 week study led by Paul Harmatz, PD, of two daily dose levels (16 and 32 mg/kg) of FBS0701. Each treatment arm will consist of approximately 20 patients with transfusional iron overload in need of chelation therapy. Safety will be assessed in clinic, weekly for Weeks 1-4 of therapy, bi-weekly for Weeks 6-8, moving to every 4 weeks during Weeks 12-24. Safety assessments will consist of adverse event recording, vital signs measurements, blood sampling, urinalysis, physical examinations, and ECGs.
A Prospective, Randomized, Double-Blind, Multicenter Study to Establish the Safety and Tolerability of Doripenem Compared with Cefepime in Hospitalized Children with Complicated Urinary Tract Infections
The primary objective of this study, led by Julie Kulhanjiam, MD, is to establish the safety and tolerability profile of doripenem compared with that of cefepime in hospitalized children 3 months to <18 years of age with cUTI. Doripenem for injection (JNJ-38174942, S-4661) is a sterile, synthetic, parenteral antibiotic of the carbapenem class of β-lactam antibiotics with broad-spectrum, potent antibacterial activity against aerobic and anaerobic gram-positive and gram-negative bacteria that is currently approved for use in the U.S. in patients > 18 years.
A Prospective, Multicenter, Open Enrollment Study of Human Plasma-Derived Factor XIII Concentrate in Subjects with Congenital Factor XIII Deficiency
The investigators and CSL Behring LLC, the Sponsor of this study, are conducting a research study, led by Alison Matsunaga, MD, to evaluate the safety and recommended (best) amount or level of Factor XIII in patients with FXIII deficiency. Factor XIII Concentrate works by assisting the blood in the usual clotting process, thereby preventing bleeding. Factor XIII Concentrate is currently licensed in 14 countries. The purpose of this study is to evaluate the safety and recommended (best) amount or level of Factor XIII Concentrate required to prevent bleeding so that it can be licensed and sold in the United States.
An Open Label, Prospective, Pharmacokinetic/ Pharmacodynamic and Safety Evaluation of Intravenous Oseltamivir (Tamiflu®) in the Treatment of Children 1 to 12 Years of Age with Influenza Infection (NP25139B)
This study, led by Ann Petru, MD, is a prospective, open label, pharmacokinetic/ pharmacodynamic and safety evaluation of IV oseltamivir therapy to define the pharmacokinetics of oseltamivir and oseltamivir carboxylate and evaluate the safety profile following intravenous (IV) administration of oseltamivir phosphate in children between 1 and 12 years of age with influenza. The investigators will also evaluate the viral load and viral shedding and all isolates for phenotypic and, where necessary, genotypic resistance.
National Collaborative to Improve Care of Children with Complex Congenital Heart Disease: A Project of the Joint Council of Congenital Heart Disease (JCCHD)
Howard Rosenfeld, MD, is leadint the CHRCO Department of Cardiology in participating in the JCCHD National Collaboration to Improve Care of Children with Complex Congenital Heart Disease. The purpose of this initiative is to improve care and outcomes for infants with univentricular heart by developing a National Registry to gather clinical process and outcome data and to learn how to most effectively implement consensus standards into everyday practice across pediatric cardiology centers.
A Study of Intrathecal Enzyme Replacement for Cognitive Decline in Mucopolysaccharidosis I
This study, led by Paul Harmatz, MD, is a 24-month open label, prospective, randomized trial in 16 MPS I patients age six years or older who have documented evidence of cognitive decline. The study will test the safety and efficacy of intrathecal recombinant human alpha-L iduronidase (rhIDU) to reduce or stabilize cognitive decline by assessing the subjects at baseline with neuropsychological, clinical, radiological, and biochemical evaluations and then monitoring the change in these parameters during a regimen of first monthly, then quarterly, intrathecal treatments with rhIDU.
An Open-Label Treatment Protocol to Evaluate the Safety of Replagal Treatment in Patients with Fabry Disease
This is a treatment study carried out at multiple centers in the United States. Paul Harmatz, MD, is leading the local study. Patients with Fabry disease who are naïve to treatment, received Replagal under an FDA authorized emergency expanded patient access program (e-IND), have received Replagal previously with no safety concerns, or who have previously received agalsidase beta (Fabrazyme®) will be transitioned to receive Replagal treatment every other week for 12 months. The primary objective of the study is to evaluate the safety of Replagal in patients with Fabry disease.
Improving Breastmilk Feeding at Discharge: A Quality Improvement
The objective of this research project, led by Pricilla Joe, MD, is to determine the effectiveness of implementation of locally relevant literature-based best practices on the rates of breastmilk feeding in newborns with birthweight less than 1500 grams. This implementation will be supported by 10 other involved Neonatal Intensive Care Units (NICUs), as well as an Expert Panel, participating in a statewide collaborative focused on improving breastmilk feeding in very low birth-weight infants.
Universal Data and Serum Specimen (UDC) System for Hemophilia: Collecting Data on Females with Bleeding Disorders
This project, led by Alison Matsunaga, MD, will collect data on females with hemophilia and certain other blood disorders over the course of several years in order to help the Centers for Disease Cotnrol and Prevention (CDC) review and create prorams to reduce or prevent the complications of hemophilia (for example, joint disease or transmission of blood-borne viruses such as hepatitis or HIV).
A Pilot Trial of Unrelated Donor Hematopoietic Cell Transplantation for Children with Severe Thalassemia Using a Reduced Intensity Conditioning Regimen (The URTH Trial)
The primary objective of this study led by Mark Walters, MD, is to determine event-free survival (EFS) at 1 year after unrelated donor (URD) hematopoietic stem cell transplantation (HCT) using bone marrow (BM) or umbilical cord blood (UCB) in patients with transfusion dependent thalassemia.
Specimen Banking for Future Sickle Cell Disease Research
The purpose of this project, led by Carolyn Hoppe, MD, is to establish a biospecimen repository at CHRCO for samples obtained at the CHRCO Sickle Cell Disease program for use in future research on sickle cell disease.
Investigating Hypomethylation of Transposable Elements and Their Effects on Karyotype Stability in Chronic Lymphocytic Leukemia (CLL)
Drawing upon the resources of CHORI and the investigators' expertise in human genetics, cytogenetics and genomics, this project, led by Lucia Carbone, PhD, aims to study the relationship between global hypomethylation, activation of transposable elements, and chromosomal rearrangements in cancer. Transposable elements are sequences of DNA that can move and change location within the genome and cause mutations, including large-scale chromosomal rearrangements.
Correlating Immune Reconstitution of Memory CD27+ B Lymphocytes and Expression of Toll-like Receptors with Vaccine Antibody Response after Chemotherapeutic Regimens and Hematopoietic Stem Cell Transplantation in Pediatric Patients; and the Molecular Ontogeny of Human Antibody Repertoires after Hematopoietic Stem Cell Transplantation
Anu Agrawal, MD is leading this two-part study to better elucidate issues surrounding the timing of revaccination after chemotherapy and hematopoietic stem cell transplantation (HSCT). Currently practices are not guided by evidence-based guidelines and varies from Europe to the United States. This study aims to provide more evidence-based data to address the current controversy.
Hypospadias Etiology: Genetic Analysis
Hypospadias are characterized by a defect in the development of the ventral aspect of the penis and an ectopic opening on the urethral meatus. This congenital malformation, often considered as a lack of virilization of male external genitalia, is the second most frequent genital malformation in newborn males after cryptorchidism. Its incidence ranges from 1/1000 to 1/100 with significant variations according to ethnic origin. This study, led by Lawrence Baskin, MD, will analyse several candidate genes in hypospadia patient and control population to idenity genetic variations associated with risk for hypospadia.
Vapocoolant Effect in Tick Removal
This study, led by F. Ralph Berberich, MD, FAAP, will evaluate the effect of a vapocoolant analgesic spray (Pain Ease, Gebauer Company, Cleveland Ohio) on the process of tick removal. By means of a questionnaire as well as by direct observation, the investigator will determine whether the ease of removal may be enhanced and any associated discomfort is reduced by the inclusion of vapocoolant.
piRNAs and Genome Methylation in Blood Cell Lineages
This study, led by David I. Martin, MD, compares certain marks ("cytosine methylation") on the DNA of human blood cells, and the presence of small RNAs termed piRNAs in those cells. Comparison of marks and piRNAs in different cell types may reveal how the piRNAs regulate formation of blood cell types.
Defibrotide for Patients with Hepatic Veno-Occlusive Disease (VOD): A Treatment IND Study
This research study, led by Jennifer Michlitsche, MD, will evaluate an investigational drug called Defibrotide as a possible treatment for hepatic VOD, which can be a complication of bone marrow and/or stem cell transplant or high-dose chemotherapy and may be life threatening.
Northern California Network of Care Evaluation Project - Sickle Cell Disease Treatment Demonstration Program (SCDTDP)
The estimated number of individuals with sickle cell disease (SCD) in the 10 counties served by the Northern California Network of Care for Sickle Cell Disease (Network of Care) is 1,574. Access to care for adults with SCD, particularly access to a medical home, is limited. Effective strategies for tracking outcomes across health care delivery systems do not exist. The Network of Care proposes to draw on the strengths of established collaborations within the Northern California region and develop new partnerships with the goals of 1) enhancing coordination of service delivery for individuals with SCD; 2) improving access to and follow up with comprehensive and subspecialty care, with particular focus on youth and adults with SCD; and 3) increasing knowledge about and participation in the medical home model for people with SCD across the lifespan.
Modulation of Iron Deposition in Sickle Cell Disease and Other Hemoglobinopathies (Survey Study)
A consortium of expert hematology centers in the US, Canada, and UK recruited a large population of heavily iron overloaded Sickle Cell (SCD) and Thalassemia (TM) patients to participate in a natural history study to compare the frequencies of iron-related organ injury in these two disease populations. The purpose of the current study is to identify up to 100 patients from 10 centers with either SCD, TM, and DBA, 10-20 years of transfusion exposure (0.2-0.6 mg Fe/kg/day), at least 6 annual ferritin levels greater than 2500, and age of initiation of regular transfusion before age 10 who meet eligibility criteria for future studies of iron deposition.
Full List of All Active CHRCO Clinical Studies
By Principal Investigator:
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