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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 I Trial of NECTAR (Nelarabine, Etoposide and Cyclophosphamide in T-ALL Relapse): A Joint Study of TACL and POETIC
Nelarabine is a drug approved by the FDA (Food and Drug Administration) for treating children and adults with T-cell acute lymphoblastic leukemia (T-ALL) or non-hodgkins lymphoma (T-NHL). Nelarabine has been used by itself to treat children and adults with relapsed T-ALL and T-NHL. Nelarabine has not been tested in combination with the other chemotherapy drugs used in this study. In this study, researchers are doing a Phase I study. In a phase I study drugs are tested to the highest dose that can be safely given. Drugs are given at gradually increasing dosages until there are unacceptable side effects. The goal of this Phase I study, led by Jacob Garcia, MD, is to find out the doses of nelarabine, etoposide and cyclophosphamide that can be safely given for 5 days to patients with relapsed T-ALL and T-NHL.
Treatment of High Risk Renal Tumors: A Groupwide Phase II Study
Anaplastic Wilms tumor, clear cell sarcoma of the kidney (CCSK), and malignant rhabdoid tumor (MRT) comprise 7.5%, 3.5%, and 1.6% of pediatric renal tumors, respectively (data from National Wilms Tumor Studies 1-5). Although renal cell carcinoma (RCC) was not included in past National Wilms Tumor Studies, SEER data indicate that RCC comprises 5.9% of renal tumors in children and adolescents. Despite their relatively low prevalence, these tumor types account for a disproportionately high number of relapses and deaths among children with renal tumors. This study by Carla Golden, MD, is designed to improve clinical outcomes for patients with anaplastic Wilms tumor, MRT, and Stage IV CCSK, while maintaining excellent outcomes for patients with Stages I-III CCSK.
Mapping of the Genes Involved in Treatment Responses Among Rheumatoid Arthritis Patients
Recently, there have been significant improvements in treatments for rheumatoid arthritis (RA). New biologic therapies have shown a high degree of efficacy both in clinical trials and in real life settings. Nevertheless, although a considerable proportion of patients show a good response to these therapies, other patients do not respond well to the treatment. It has been hypothesized that the degree of response may be under genetic control (Lui, 2008). The goal of this study, led by Damini Jawaheer, PhD, is to map the gene loci associated with response or no-response to treatment with biologics.
Prevalence of Elevated Urine Osmolality in the Morning in Healthy U.S. Adults
The overall objective of this cross-sectional survey, led by Jodi Stookey, PhD, is to determine the prevalence of elevated urine osmolality in the morning among U.S. adults and test for associations between elevated urine osmolality and intake of beverages other than water among U.S. adults.
National Ophthalmic Genotyping and Phenotyping Network, Stage 1 -- Creation of DNA Repository for Inherited Ophthalmic Diseases
John Waterson, MD, is developing a network to collect information on eye examinations and DNA of patients with eye diseases. The network, called eyeGENE®, will be used to test samples for genes that might be involved in the eye disease of the individual subjects, as well as being placed in repository for other researchers, now and in the future, who are studying eye diseases.
A Randomized, Double-blind, Parallel-group Study to Assess the Safety and Efficacy of Asacol® (1.2 to 4.8g/day) 400 mg Delayed-release Tablets Given Twice Daily for 26 Weeks to Children and Adolescents for the Maintenance of Remission of Ulcerative Colitis
Sabina Ali, MD is leading this multi-center, multinational, 26-week study of two dose levels of Asacol consisting of a high dose and a low dose in pediatric patients in order to assess the safety and efficacy of high dose and low dose Asacol given twice daily for 26 weeks for maintaining remission in children and adolescents with ulcerative colitis (UC). The primary efficacy endpoint is the proportion of patients who have maintained complete remission through Week 26 as determined by a PUCAI score < 10 during the entire study period.
Immunologic and Genetic Factors Associated with Influenza Infection and Vaccine Response in Children with Life Threatening and Fatal Influenza (VE)
The Pediatric Acute Lung Injury and Sepsis Investigator’s (PALISI) Network, is a consortium of clinical investigators at over 75 ICUs in the US and Canada. For the current study, led by Heidi Flori, MD, 200 children will be enrolled across 36 US PALISI ICUs representing all US regions. The study will assist in understanding and defining the immunogenetic mechanisms that underlie variation in vaccine response, which may ultimately aid in interpreting patterns of vaccine effectiveness to suggest potential new vaccine designs.
Epidemiology and Immune Response to Life Threatening Influenza Infection in Children (R56)
The primary hypothesis of this study, led by Heidi Flori, MD, is that the genetic makeup of the host influences the innate immune and inflammatory response to influenza, and underlies risk for more life-threatening illness and mortality. Dr. Flori will conduct a multicenter observational cohort study across an experienced pediatric critical care research network (PALISI).
Treatment of Children with All Stages of Hepatoblastoma - A Groupwide Phase III Study (AHEP0731)
Hepatoblastoma is the most common malignant liver neoplasm in children. This study, led by Carla Golden, MD, builds on the results of the last 20 years of hepatoblastoma clinical trials and seeks to diminish toxicity in the approximately 30% of low-risk patients, increase survival in intermediate-risk patients and identify new agents(s) that may be used in high-risk and recurrent patients.
Treatment of Children with Newly-Diagnosed Low Stage Lymphocyte Predominant Hodgkin Disease (LPHD) (AHOD03P1)
Carla Golden, MD, is leading this study to see if late effects can be lowered or prevented by using less chemotherapy and radiation and to find out if using less chemotherapy and radiation to treat LPHD will still show high cure rates. Some subjects will get no chemotherapy or radiation if only one lymph node was affected by LPHD and a surgeon completely removed it.
A Phase II Pilot Study of Bortezomib (PS-341, Velcade, IND# 58,443) Combined with Reinduction Chemotherapy in Children and Young Adults with Recurrent, Refractory or Secondary Acute Myeloid Leukemia (AAML07P1)
This study, led by Carla Golden, MD, proposes to test the hypothesis that the addition of bortezomib to Acute Myeloid Leukemia (AML) relapse regimens (idarubicin/cytarabine or etoposide/cytarabine) will improve the overall response rate in children with relapsed/refractory/secondary AML without significantly increasing the toxicity.
A Phase II Pilot Trial of Bortezomib (PS-341, Velcade, IND# 58,443) in Combination with Intensive Re-Induction Therapy for Children with Relapsed Acute Lymphoblastic Leukemia (ALL) and Lymphoblastic Lymphoma (LL) (AALL07P1)
This study, led by Carla Golden, MD, phase 2 pilot study to determine the feasibility and safety of adding bortezomib to intensive induction chemotherapy for patients with relapsed B-precursor acute lymphoblastic leukemia (ALL), relapsed T-cell ALL and relapsed T-lymphoblastic lymphoma (LL). Despite significant progress in the treatment and outcome of pediatric ALL/LL, the prognosis for patients who develop recurrent disease is poor. This study aims to improve outcomes in relapsed ALL by combining bortezomib with the backbone chemotherapy regimen used for treatment of relapsed ALL in COG study AALL01P2.
Blood Collection from Patients with Hunter Syndrome Currently Participating in the Hunter Outcome Survey for use in Disease Assay Development, A Hunter Outcome Survey Sub-Study
Hunter syndrome is an X-linked recessive lysosomal storage disease caused by the deficiency of the enzyme iduronate-2-sulfatase (I2S). It is a rare disease with an estimated minimum incidence of 1 in 162,000 live births. The purpose of this study, led by Paul Harmatz, MD, is to collect a one time blood sample from Hunter syndrome patients currently participating in the Hunter Outcome Survey (HOS) and their parents to be used to develop an assay for newborn screening for Hunter syndrome.
REPLAGAL (agalsidase alfa) for Emergency Use in Fabry Disease
The enzyme therapy REPLAGAL (agalsidase alfa) is approved for treatment of Fabry disease in Europe and Canada. Both REPLAGAL and FABRAZYMW have demonstrated efficacy in human clinical trials, but different endpoints and dosing regimens used in trials prevent direct comparison of the relative efficacies of the two products. This intervention, led by Paul Harmatz, MD, for one subject who will take REPLAGAL in place of FABRAZYMW, is necessary due to the patient's declining renal function, continuing shortage of FABRAZYME, and lack of other approved therapeutic alternatives.
Gender differences in Outcomes and Genetic Associations of Rheumatoid Arthritis
The study, led by Damini Jawaheer, PhD, has two main aims: (i) to examine gender differences in disease manifestations among male and female Rheumatoid Arthritis (RA) patients in different cohorts of RA patients (analyses of coded data collected for other primary studies), and (ii) to establish a repository of DNA and serum samples from RA patients for analysis of PADI4 SNPs.
Molecular Ontogeny of Human Antibody Repertoires
Alexander Lucas, PhD, is leading this study to understand at the molecular level how protective immune responses to encapsulated bacterial pathogens are generated in infants by vaccination with polysaccharide-protein conjugate vaccines.
Orthopaedic Surgical Issues for Patients with MPS
Stephen Skinner, MD, will investigate medical and surgical interventions in relation to the orthopaedic and medical management of patients with MPS. The investigators will also conduct a retrospective review of x-rays, clinical reports, and exams from CHRCO’s MPS patients to report the natural history, medical interventions and orthopaedic surgical corrections, data and functions as they relate to orthopaedic conditions in the MPS patients.
Collection of Bone Marrow and Blood from Patients with Genetic Diseases of Blood Cells
An alternative approach to the use of allogeneic HSC transplantation for the treatment of genetic diseases of blood cells is the genetic correction of a patient's own (autologous) HSC or gene therapy. This method is being studied for the potential to have less side effects and overall risks. The purpose of this study, led by Mark Walters, MD, is to evaluate the techniques of gene transfer and expression, which is the ability to have abnormal cells take up normal genes and use the gene to produce corrected cell conditions and activity. Samples of bone marrow and blood will be used to study and improve these techniques.
Vaccine Immunity Following Splenectomy
Alexander Lucas, PhD, will lead this study involving one 3 year-old child who received conjugated polysaccharide vaccines as standard practice after vaccination. The study will evaluate the antibody response to conjugate vaccine antigens after splenectomy and vaccination using a new molecular method to clone native Fab fragments from peripheral blood.
Investigating the Influence of Pregnancy on Rheumatoid Arthritis Disease Activity
The goal of this study, led by Damini Jawaheer, PhD, is to recruit at least 100 Danish women with RA who are planning to get pregnant, and 20 age-matched Danish controls, and follow them prospectively from before conception, at intervals of 12 weeks, throughout pregnancy and until 24 months after delivery. The data collected in the study will be used to perform genetic studies that could identify the candidate genes that influence disease activity in RA.
Pharmacogenetic Prediction of Hydroxyurea Response in Sickle Cell Disease
Carolyn Hoppe, MD, is leading this effort to create a five site NYCON Pediatric Hemoglobinopathy Research Consortium to conduct four inter-related clinical/translational studies of Hydroxyurea (HU) use in sickle cell disease (SCD). Collectively, these studies focus on identifying novel indications for HU, pharmaco-genomic prediction of response, risks of and barriers to its use.
UV-Induced Vitamin D3 (D3) Production and the Risk of Basal Cell Carcinoma (BCC) Carcinogenesis
Vitamin D is an essential vitamin that has been recently hypothesized to protect against various autoimmune diseases and cancers of the prostate, colon and breast. Ervin Epstein, MD, is leading this study to determine whether changes in vitamin D levels after ultraviolet light may be able predict relative increased risk of BCC.
Trial of Late Surfactant to Prevent BPD – A Study in Ventilated Preterm Neonates Receiving Inhaled Nitric Oxide: The TOLSURF Study
Infants born prematurely are at risk for both respiratory distress syndrome (RDS) and bronchopulmonary dysplasia (BPD), defined as a continuing requirement for ventilatory support and/or supplemental oxygen at 36 weeks postmenstrual age (PMA). David Durand, MD, will lead this multi-center, randomized, controlled trial in order to determine whether late doses of surfactant, in addition to iNO, administered to Extremely Low Gestational Age Newborn (ELGAN) infants greater than 28 weeks gestation who continue to require mechanical ventilation between 7 and 14 days of age will increase survival without BPD.
A Randomized, Double-Blinded, Placebo-Controlled Study of RAD001 in the Treatment of Patients with Subependymal Giant Cell Astrocytomas (SEGA) Associated with Tuberous Sclerosis Complex (TSC)
Candida Brown, MD, will evaluate the antitumor activity of RAD001 versus placebo in patients with SEGA associated with TSC. The primary objective is to compare the SEGA response rate in patients with TSC-associated SEGA on RAD001 versus placebo.
Investigation of the Transcriptome of a Single Individual
This study, led by Dario Boffelli, PhD, will involve one male adult volunteer who will have his transcriptome analyzed.
Full List of All Active CHRCO Clinical Studies
By Principal Investigator:
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