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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 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.
Pilot Study Examining Mechanisms of Iron Trafficking and Extra-hepatic Iron Distribution in Sickle Cell Disease, Thalassemia, and other Iron Loading Anemias
Given the potential side effects of chelation therapy and limited evidence of iron-induced organ toxicity in Sickle Cell Disease (SCD) it is essential to clarify the role of organ iron toxicity in SCD patients and develop a specific therapeutic approach based on disease specific data. Elliott Vichinsky, MD, is leading this pilot study to examine the hypothesis that a chronic inflammatory state in SCD leads to hepcidin- and cytokine-mediated iron withholding within the RES, lower plasma NTBI levels, less distribution of iron to the heart in SCD, and finally lower uptake and enhanced export of NTBI by cardiomyocytes.conditioned by SCD serum. Data from these biochemical studies will enable us to complete improved estimations of sample size necessary to conduct a definitive study of the mechanisms mediating iron distribution and toxicity.
A Phase I Trial of Monoclonal Antibody HGS-ETR2 (Lexatumumab) With or Without Interferon Gamma in Patients with Refractory Pediatric Solid Tumors (P6981)
Pediatric solid tumors represent approximately one fourth of cancer diagnoses in children. Despite intensive regimens, patients with metastatic or recurrent tumors have unsatisfactory survival rates. TRAIL (TNF-related apoptosis inducing ligand) has been shown to have specific antitumor activity against a wide range of tumor cells without inducing death in normal cells. Bindu Kanathezhath, MD is leading this study to determine the MTD and tumor response of Lexatumumab in patients with refractory pediatric solid tumors as a single agent and in combination therapy with a fixed dose of interferon gamma 1b.
A Multicenter Open-Label Treatment Protocol to Observe the Safety of Gene-Activated® Human Glucocerebrosidase (GA-GCB, velaglucerase alfa) Enzyme Replacement Therapy in Newly Diagnosed
Gaucher disease is a rare lysosomal storage disorder caused by the deficiency of the enzyme glucocerebrosidase (GCB). Due to the deficiency of functional GCB, glucocerebroside accumulates within macrophages leading to cellular engorgement, organomegaly, and organ system dysfunction. The purpose of this multi-center study, led by Paul Harmatz, MD, is to observe the safety of velaglucerase alfa in patients with type 1 Gaucher disease who are either treatment naive (newly diagnosed) or who are currently being treated with the Enzyme Replacement Therapy (ERT) imiglucerase.
Sweat Testing in CF Newborns Detected by Screening
In a well defined cohort of newborns with known CFTR mutations, Karen Hardy, MD, will evaluate a uniform patient preparation protocol for sweat testing that includes salt supplementation and adequate fluid intake guidelines. The goal of the study is to determine the effect on sweat electrolytes of fluid and electrolyte status and assess for longitudinal changes in sweat electrolyte concentration and other clinical and laboratory parameters.
A Pilot Study of Low Magnitude Vibration Therapy to Increase Bone Density and Size in Patients with Thalassemia Induced Osteoporosis
Osteoporosis, or weak bones, affects patients with all types of thalassemia syndromes at all ages. It has been estimated nearly 70% of adults with thalassemia have osteopenia or osteoporosis which can lead to fracture, bone pain and decreased quality of life. Recent advances in the field have shown that low magnitude mechanical stimulation or “vibration therapy”, can also strengthen bone. This simple therapy can be conducted in the home and does not increase strain on the heart. The primary aim of this 6 month pilot and feasibility trial led by Ellen Fung, PhD, is to evaluate “vibration therapy” as an anabolic stimulus to bone in 20 patients with thalassemia.
Full List of All Active CHRCO Clinical Studies
By Principal Investigator:
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