Active Core Studies
CHRP Clinical Research Core personnel currently supports 30 active studies (9 pilot, 7 NIH, 14 industry), representing 20 investigators across 12 pediatric divisions (adolescent medicine, anesthesia, cardiology, child psychiatry, critical care, genetics, hematology/oncology, infectious disease, immunology, neonatology, pulmonary, and rheumatology).
Pilot Studies
#2008: Lysosomal Storage Disorder
Study Title: Newborn Screening for Lysosomal Storage Disorders: A Feasibility Study in the California Population
PI: Kristina Cusamo-Ozog, MD – Pediatric Genetics
The overall objective of this research is to evaluate the feasibility of newborn screening for various lysosomal storage disorders (LSDs) in the general population.
#2009: CMV Lung Tx Study Title: CMV-Specific CD4 and CD8 T cell immune responses: Implications for anti-viral prophylaxis
PI: Sharon Chen, MD – Pediatric Infectious Diseases
This pilot study evaluates CMV-specific T cell immune responses in 4 pediatric and 35 adult lung and lung/heart transplant patients. At each of 6 post-transplant visits, patients will have a brochoalveolar lavage and lung biopsy. We expect patients with detectable CMV-specific T cell immune response will demonstrate lower CMV viral loads, fewer CMV infection/rejection episodes.
#3002: Acupuncture Study Title: The effect of acupuncture on patient controlled opioid use after pediatric orthopedic surgery
PI: Jeannie Seybold, MD – Pediatric Anesthesia
The aim of this study is to determine whether acupuncture will reduce hydromorphone use via patient-controlled analgesia (PCA) by children after orthopedic surgery. Our primary endpoint is to determine if acupuncture will decrease opioid use. Secondary endpoints will be to determine if acupuncture will improve pain scores, decrease stress response as measured by salivary cortisol, and improve recovery markers, including the times to first PO intake and hospital discharge.
#3004: Insulin Delivery Study Title: Creation and application of a closed loop glucose measurement-insulin delivery system
PI: Saraswati Kache, MD – Pediatric Clinical Care
In this Pilot study, subcutaneous glucose measurements will be transmitted to a computer using a radiofrequency (RF) signal, the computer will use the PID algorithm, and then send an RF signal to control an insulin infusion pump.
#3006: ALL Study Title: Novel Genomic Approach to Risk-Stratification of Childhood Acute Lymphoblastic Leukemia (ALL)
PI: Joshua D. Schiffman, MD– Pediatric Hematology/Oncology
This pilot study will connect molecular genetics with clinical characteristics and improve risk stratification and individual treatment response for childhood leukemia.
#3007: PDA Study Title: Cerebral Monitoring and Brain Injury in Very Low Birth Weight Neonates with a Patent Ductus Arteriosus (PDA).
PI: Valerie Chock, MD – Pediatric Neonatology
This pilot study proposes to investigate brain function in preterm infants with a patent ductus arteriosus using multimodal bedside neurological monitoring (aEEG and cerebral oximetry) and correlate these innovative measures with neuroimaging outcomes.
#3008: SLIT Study Title: Sublingual Immunotherapy in Dust Mite and Timothy Grass Allergic Subjects.
PI: Kari Nadeau, MD PhD - Pediatric Allergy/Immunology
A Phase I, Single-Center, Blinded, Randomized, Placebo-Controlled study of Sublingual Immunotherapy (SLIT) in subjects with dust mites and Timothy grass allergy..
NIH Studies
#1010: APPLE Study Title: A mulitcenter, randomized, double-blind, placebo controlled study to test the safety and efficacy of Lipitor (Atorvastatin) in Reducing the progression of carotid intima media thickness (IMT) in Early childhood systemic lupus Erythematosus(SLE).
PI: Christy Sandborg, M.D. - Pediatric Rheumatology
This study evaluates the effect of Lipitor (atorvastatin) versus placebo on the rate of progression of carotid intima media thickness (IMT) in children age 10-21 years enrolled from 20 sites (220 total participants) and randomized to receive standard treatment plus Lipitor or standard treatment plus placebo.
#1011: SCCOR Study Title: Genetic Contribution to Graft and Patient Outcomes.
PI: Daniel Bernstein, M.D. - Pediatric Cardiology
This is a multi-center study of approximately 750 pediatric heart transplant patients at six large North American pediatric transplant centers. We are expecting to enroll 80 patients at LPCH. Clinical data collection will use the mechanism of the Pediatric Heart Transplant Study Group (a prospective multi-center study of outcomes and their risk factors among over 20 pediatric heart transplant centers in North America).
#1015: RIM Study Title: Rituximab for the Treatment of Refractory Adult and Juvenile Dermatomyositis (DM) and Adult Polymyositis(PM)
PI: Christy Sandborg, M.D. - Pediatric Rheumatology
The primary objective of the proposed study is to investigate whether, in patients with IIM disease; rituximab will 1) improve muscle strength and 2) improve symptoms of disease. This is a multicenter, double-blind, randomized placebo phase design (RPPD) study to compare the time to achieve the definition of improvement between two groups of rituximab treated patients.
#2003: Losartan Study Title: The Trial of Beta Blocker Therapy (Atenolol) vs. Angiotensin II Receptor Blocker Therapy (Losartan) in Individuals with Marfan
PI: David Liang, MD – Cardiology; and Danial Murphy MD – Pediatric Cardiology
#2006: CMV Study Title: A Phase III Randomized, Placebo-Controlled Blinded Investigation of Six Weeks vs. Six Months of Oral Valganciclovir Therapy in Infants with Symptomatic Congenital Cytomegalovirus Infection (CASG 112)
PI: Kathleen Gutierrez, MD – Pediatric Infectious Diseases
#2007: Viral Transplant Study Title: Posttransplant respiratory viral infections influence outcomes after pediatric lung transplant
PI: Carol Conrad, MD – Pediatric Pulmonary Medicine
#2011: PCQLI Study Title: The Pediatric Cadiac Quality of Life Inventory Testing Trial
PI: David Rosenthal MD - Pediatric Cardiology
This multi-center study aims to test the scores generated by the newly developed Pediatric Cardiac Quality of Life Inventory (PCQLI). The goal is to study the psychometric properties of the PCQLI in children (8-12 yo) and adolescents (age 13-18 years old) with acquired and congenital heart disease.
Industry-Sponsored Studies
#1009: Humira Study Title: A Multicenter, Randominzed, Double-Blind, Placebo-Controlled Study of the Safety, Efficacy, and Pharmacokinetics of the Human Anti-TNF Monoclonal Antibody Adalimumab in Children with Polyarticular Juvenile Rheumatoid Arthritis.
PI: Christy Sandborg, M.D. - Pediatric Rheumatology
In this study polyarticular juvenile rheumatoid arthritis (JRA) subjects who are either methotrexate (MTX) treated or non-MTX treated initially received Adalimumab by subcutaneous injection every ther week for a four-month open-label lead-in period. Subjects who responded to the open-label therapy were then rolled over into the double-blind portion of the study and were randomized to receive either adalimumab or placebo for an additional 32 weeks or until flare of disease, whichever is earlier.
#2001: Clarinet Study Title: International Randomized Double Blind Study Evaluating the Efficacy and Safety of Clopidogrel 0.2mg/kg Once Daily Versus Placebo in Neonates and Infants with Cyanotic Congenital Heart isease Paliated with a Systemic-to-pulmonary Artery Shunt (e.g. modified Blalock Taussig shunt)
PI: David Rosenthal MD - Pediatric Cardiology
The purpose of this study is to evaluate the safety and effectiveness of clopidogrel in neonates (newborns less than 30 days old) and infants (up to 92 days of age) after a systemic-to-pulmonary artery shunt procedure for prevention of death and illness related to blood clots.
#2014 Berlin Heart Study Title: Berlin Heart IDE Clinical Trial
PI: David Rosenthal, MD - Pediatric Cardiology
#3003 - TOPP Study Title: Tracking Outcomes and Practice in Pediatric Pulmonary Hypertension
PI: Jeffrey Feinstein, MD
International, multi-center, prospective, observational, non interventional program set up to study the natural history of pulmonary hypertension (PH) in children, focusing on diagnosis and disease management in children and adolescents suffering from PH in real world clinical settings.
Archive (Non-Active Core studies)
#1001: RV Pacing
Study Title: Resynchronization Pacing and RV Failure: A Pilot Study
PI: Anne Dubin, M.D. – Pediatric Cardiology.
#1008: Cytokine
Study Title: Diagnostic and Prognostic Biomarkers in Systemic Juvenile Arthritis
PI: Elizabeth Mellins, M.D. – Pediatric Immunology/Transplantation Biology
#1014: Glutathione
Study Title: Novel diagnostic and therapeutic monitoring techniques for mitochondrial disease
PI: Gregory Enns, M.D. - Pediatric Genetics
#1005: MEDTRONIC Study Title: Relationship of mechanical synchrony to heart rate in paced patients with congenital complete heart block.
PI: Anne Dubin, M.D. – Pediatric Cardiology
#2004: Osmolality
Study Title: Saliva Osmolality: Is it a marker of hydration status during the perioperative period in children?
PI: R J Ramamurthi, M.D. - Pediatric Anesthesia
#2013: Homozygosity
Study Title: Homozygosity mapping of a novel tumor supressor
PI: Michael Wei, MD – Pediatric Hematology/Oncology
#1002: HART Study Title: Heparin And the Risk of Thrombosis (HART) Study
PI: Stephen Roth, MD, MPH - Pediatric Cardiology
#2010: NAC Study Title: A double-blind, placebo controlled study of the effects of Glutathione and N-ACETYLCYSTEINE on Autistic children and Adolescents.
PI: Antonio Hardan, MD – Child Psychiatry
#1012: Autism 179 Study Title: A Multicenter, Double-Blind, Randomized, Placebo-Controlled, Parallel-Group Study with Three Fixed Doses of Aripiprazole in the Treatment of Children and Adolescents with Autistic Disorder
PI: Antonio Hardan, MD – Child Psychiatry
#2002: Autism 180 Study Title: A 52-Week, Open-Label, Multicenter Study of the Safety and Tolerability of Aripiprazole Flexibly Dosed in the Treatment of Children and Adolescents with Autistic Disorder
PI: Antonio Hardan, MD – Child Psychiatry
#1016: Strattera Study Title: Atomoxetine as an adjunct therapy in the treatment of comorbid ADHD in children and adolescents with bipolar I or II disorder
PI: Kiki Chang , MD – Child Psychiatry
#1004: AGA Study Title: Closure of muscular ventricular septal defects (VSD) with the AMPLATZER® Muscular VSD Occluder.
PI: Jeffrey Feinstein, M.D., M.P.H. – Pediatric Cardiology
