Pediatric Nephrology Open Protocols

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Pediatric Nephrology Open Protocols

  1. 1. Pediatric Nephrology Open Protocols (listed alphabetically by study acronym) 1. Open Label Controlled Trial of Eculizumab in Adolescent Patients With Plasma Therapy-Resistant Atypical Hemolytic Uremic Syndrome (aHUS) Purpose: Atypical Hemolytic-Uremic Syndrome (aHUS) is a serious and life-threatening rare disorder that includes anemia microangiopathic hemolytic anemia, low platelets, and ultimately kidney failure. aHUS can recur and is a chronic disease. The purpose of this study is to determine whether eculizumab is safe and effective in the treatment of adolescent patients with plasma therapy-resistant Atypical Hemolytic-Uremic Syndrome (aHUS). http://clinicaltrials.gov/ct2/show/NCT00844844?term=eculizumab&rank=3 Recruitment status: enrollment closed Sponsor: Alexion Pharmaceuticals Site PI: Dr. Larry Greenbaum, Director, Division of Pediatric Nephrology, Emory University, Atlanta, GA Site coordinator: Margo Kamel, MSPH 2. Chronic Kidney Disease in Children Prospective Cohort Study (CKiD) Purpose: This is an observational study of children with chronic kidney disease. The primary goals of this study are to determine the risk factors for decline in kidney function and to define how a progressive decline in kidney function impacts neurocognitive function and behavior; the risk factors for cardiovascular disease; and growth failure and its associated morbidity. http://clinicaltrials.gov/ct/show/NCT00327860?order=1 Recruitment status: enrollment closed at this time Sponsor: National Institute of Health (NIH)
  2. 2. Site PI: Dr. Larry Greenbaum, Director, Division of Pediatric Nephrology, Emory University, Atlanta, GA Site coordinator: Margo Kamel, MSPH 3. Pediatric Kidney Transplant Study of Sirolimus, Mycophenolate Mofetil, and Corticosteroids vs Calcineurin Inhibitor Based Immunosuppression (CNI-W) Purpose: The purpose of this experimental study is to determine whether children can safely be withdrawn from Prograf® after transplantation and changed to Rapamycin® (sirolimus). Recent research studies in adult transplantation have demonstrated that with the use of Rapamycin® (sirolimus), it is possible to discontinue the use of Prograf (tacrolimus) with no increase in rejection, with decreased scarring in the kidney, and with improvements in kidney function and survival of the kidney. http://clinicaltrials.gov/ct2/show/NCT00555373?term=cni-w&rank=1 Recruitment status: enrollment closed Participating research site with the University of Alabama Birmingham Sponsor: Wyeth, Inc. (Investigator Initiated) Site PI: Dr. Barry Warshaw, Associate Professor, Division of Pediatric Nephrology, Emory University, Atlanta, GA Site coordinator: Margo Kamel, MSPH 4. Cerebro-renal disease? A morphometric study of the brains of pediatric patients with chronic renal insufficiency (CRI MRI Study) Purpose: This study, in collaboration with the radiology department at Children’s Healthcare of Atlanta, aims to identify regions of abnormal brain development in children with chronic renal insufficiency (CRI) who would be diagnosed as normal using conventional MRI techniques. Recruitment status: actively recruiting
  3. 3. Sponsor: Children’s Healthcare of Atlanta Co-Investigator: Dr. Larry Greenbaum, Director, Division of Pediatric Nephrology, Emory University, Atlanta, GA Site coordinator: Tauri Harden, RN 5. Noninvasive Methods to Monitor Graft Survival in Kidney Transplant Patients (CTOT-01) Purpose: The purpose of this study is to test noninvasive methods to monitor the health and condition of new kidneys in people who have received kidney transplants. This observational study will investigate whether certain blood and urine tests are useful in monitoring the health of transplanted kidneys. http://www.clinicaltrials.gov/ct/show/NCT00308802?order=2 Recruitment status: enrollment closed Sponsor: National Institute of Health (NIH) Site PI: Dr. Barry Warshaw, Associate Professor, Division of Pediatric Nephrology, Emory University, Atlanta, GA Site coordinator: Margo Kamel, MSPH 6. A Randomized, Crossover, Pharmacokinetic and Pharmacodynamic Study to Determine the Safety and Efficacy of Cysteamine Bitartrate Delayed-release Capsules (RP103), Compared to Cystagon® in Patients with Nephropathic Cystinosis Purpose: Cystinosis is an inheritable disease that, if untreated, results in kidney failure as early as the first decade of life. The current marketed therapy is Cystagon® (cysteamine bitartrate), which must be taken every six hours for the rest of the patient's life to prevent complications of cystinosis. RP103 is a formulation of cysteamine bitartrate that is being studied to see if it may be able to be given less frequently, once every 12 hours, and have similar results.
  4. 4. http://clinicaltrials.gov/ct2/show/NCT01000961? Recruitment status: actively recruiting Sponsor: Raptor Pharmaceuticals Site PI: Dr. Larry Greenbaum, Director, Division of Pediatric Nephrology, Emory University, Atlanta, GA Site coordinator: Margo Kamel, MSPH 7. A Long-Term, Open-Label, Safety and Efficacy Study of Cysteamine Bitartrate Delayed-release Capsules (RP103) in Patients with Nephropathic Cystinosis Purpose: Cystinosis is an inheritable disease that, if untreated, results in kidney failure as early as the first decade of life. The current marketed therapy is Cystagon® (cysteamine bitartrate), which must be taken every six hours for the rest of the patient's life to prevent complications of cystinosis. RP103 is a formulation of cysteamine bitartrate that is being studied to see if it may be able to be given less frequently, once every 12 hours, and have similar results. http://clinicaltrials.gov/ct2/show/NCT01197378? Recruitment status: not open to enrollment yet Sponsor: Raptor Pharmaceuticals Site PI: Dr. Larry Greenbaum, Director, Division of Pediatric Nephrology, Emory University, Atlanta, GA Site coordinator: Margo Kamel, MSPH 8. Novel Therapies for Resistant FSGS: Phase II Clinical Trial (FONT II) Purpose: A significant percentage of patients with primary FSGS are resistant to corticosteroids and other immunosuppressive medications. In view of the rising incidence of this disease and the grim prognosis for patients with resistant disease, it is imperative
  5. 5. that new therapeutic approaches be evaluated in an efficient and systematic manner. This project will test whether rosiglitazone, adalimumab,and/or galactose can safely reduce proteinuria (abnormal amounts of protein in the urine) and protect kidney function better than standard treatment for patients with focal segmental glomerulosclerosis (FSGS). http://clinicaltrials.gov/ct2/show/NCT00814255? Recruitment status: open to enrollment Sponsor: National Institutes of Health (NIH)-National Institute of Diabetes and Digestive and Kidney Disease (NIDDK) Site PI: Dr. Larry Greenbaum, Director, Division of Pediatric Nephrology, Emory University, Atlanta, GA Site coordinator: Margo Kamel, MSPH 9. Antibody response to Human Papillomavirus Recombinant Vaccine (Gardasil ®) in girls and young women with chronic kidney disease Purpose: People with chronic kidney disease are known to have immune response abnormalities, including a diminished response to some vaccinations. Those with chronic kidney disease have a disproportionate burden of HPV 6-, 11-, 16- and/or 18-related genital tract disease. Due to immune response abnormalities, the CKD population may or may not respond to the recommended three- dose regimen of Gardasil®, a vaccine intended to protect against HPV 6-, 11-, 16-, and 18-related genital tract disease. The objective of this study is to measure the antibody response to Gardasil® in female patients 9-21 years of age with chronic kidney disease (CKD) (Stage 1-4), end-stage kidney disease (Stage 5 CKD), and status- post kidney transplant. Gardasil® vaccine will be administered according to the FDA-approved schedule. http://clinicaltrials.gov/ct2/show/NCT00806676? Recruitment status: open to enrollment Partner center with Johns Hopkins University
  6. 6. Sponsor: Merck & Co, Inc (Academic Grant) Site PI: Dr. Sandra Amaral, Assistant Professor of Pediatrics, Division of Pediatric Nephrology, Emory University, Atlanta, GA Site coordinator: Verena Weissenborn, RN 10. Hope as a Resource in Chronic Childhood Illness Purpose: This qualitative research is expected to provide a foundation for a critical understanding of how children conceptualize hope in the context of a chronic illness. In particular, the research will clarify what practices of care are experienced by children as nurturing hope; such clarification can provide guidance for the development of new (or more intentional) supportive practices of multidisciplinary care from a spiritual perspective that are grounded in and responsive to the lived experience of children Recruitment status: open to enrollment Sponsor: Lilly Theological Research Grants Program Site PI: Dr. Donald Batisky, Associate Professor, Division of Pediatric Nephrology, Emory University, Atlanta, GA Site coordinator: Margo Kamel, MSPH 11. Clinical Trials in Transplantation in Children (Protocol CTOTC-02) Immune Development in Pediatric Transplantation (IMPACT) Transplantation is the preferred method of treatment for end-stage renal disease (ESRD) in children. Over the past forty years, the use of newer immunosuppressive drugs has decreased the risk for organ rejection considerably, and improved short-term outcomes. However, these costly and complicated life-long treatment regimens also cause serious side effects. This has been particularly true for children, who undergo treatment with these drugs at the same time they are transitioning, physically and emotionally, from childhood to adulthood. These factors lead to significantly reduced life-spans, decreased drug regimen adherence, and an increased need for re- transplantation, as compared with adults.
  7. 7. Current immunosuppressive procedures and strategies for children mimic those for adults, despite the difference between the two populations' immune systems and needs. New strategies aimed at tailoring to an individual child's needs would both reduce the risk of complications and improve outcomes. The purpose of this study is to generate information that will help to change the current practice of pediatric transplantation into one that is more individualized and preventative. http://clinicaltrials.gov/ct2/show/NCT00951353? Recruitment status: open to enrollment Sponsor: National Institute of Allergy and Infectious Diseases (NIAID) Study PI: Dr. Allan Kirk, Professor of Surgery, Division of Transplantation, Department of Surgery, Emory University, Atlanta, GA Site PI: Dr. Barry Warshaw Associate Professor, Division of Pediatric Nephrology, Emory University, Atlanta, GA Site coordinator: Tauri Harden, RN 12. Immune Monitoring and Assay Development in Kidney Transplant Recipients Currently, a kidney biopsy is the only way to determine whether a patient with a kidney transplant has rejection of their kidney. The goals of this study are to develop and study urine and blood tests that can determine if a patient is rejecting a transplanted kidney. This will hopefully decrease the need to perform kidney biopsies and allow for earlier diagnosis of rejection. PI: Jennifer Jackson, MD, 3rd year Pediatric Nephrology Fellow, Emory University Mentor: Dr. Allan Kirk, Professor of Surgery, Division of Transplantation, Department of Surgery, Emory University, Atlanta, GA Site coordinator: Tauri Harden, RN and Verena Weissenborn, RN 13. Immunomonitoring Protocol for Adolescent Kidney Transplant Patients (IMPTK)
  8. 8. Purpose: This study is designed to investigate two new metrics for immunomonitoring, serum calcineurin enzyme activity and urinary chemokines, to assess their usefulness and validity in detecting allograft rejection and medication nonadherence in adolescents in kidney transplants. Recruitment status: actively enrolling Sponsor: NIH Site PI: Dr. Sandra Amaral, Division of Pediatric Nephrology, Emory University, Atlanta, GA Site coordinator: Verena Weissenborn, RN, BSN 14. A Molecular Pathogenesis-Driven Approach for Diagnosis and Treatment of Complement-Based Renal Diseases (KidCOM) Purpose: The purpose of this research is to build a registry of patients with aHUS and MPGN. MPGN is a rare disease that can cause problems with how the kidney filters blood and waste. Currently, little is known about how to treat MPGN. There are three different types of MPGN; MPGN I, MPGN II/Dense Deposit Disease (MPGN II/DDD), and MPGN III. Although we will look at all types of MPGN, we are focused on MPGN II/DDD because it is most closely linked to the immune system. Hemolytic Uremic Syndrome (HUS) is another rare immune disease that affects blood supply to the kidneys, and impairs their function. There are two types of HUS; typical HUS, and atypical HUS. Typical HUS, which is usually diagnosed in childhood, is due to a bacterial infection in the stomach and intestines. Atypical HUS (aHUS) is a hereditary disease that is associated with recurrent episodes. Recruitment status: open to enrollment Collaborators: Dr. Christoph Licht at The Hospital for Sick Children, Toronto, Ontario, Canada; Dr. William Smoyer at The Research Institute at Nationwide Children's Hospital, Columbus, Ohio; Dr. Patrick Brophy at University of Iowa Hospitals and Clinics, Iowa City, Iowa.
  9. 9. Sponsors: Foundation for Children with Atypical HUS, and Optherion, Inc. Site PI: Dr. Larry Greenbaum, Director, Division of Pediatric Nephrology, Emory University, Atlanta, GA Site coordinator: Margo Kamel, MSPH 15. A Phase III, Randomized, Open-Label, Parallel-Group, Dose-Ranging Clinical Trial to Study the Safety and Efficacy of MK-0954/Losartan Potassium in Pediatric Patients With Hypertension (Merck HTN study) Purpose: The purpose of this study is to test the safety and effect of the research study drug, COZAAR® (losartan potassium), on the lowering of blood pressure in children from 6 months to 6 years of age. COZAAR® is approved by the United States Food and Drug Administration (FDA) to treat high blood pressure in adults and children 6 years or older. Previous studies of COZAAR® in children with high blood pressure have provided information about the dosages that can be used to treat high blood pressure, and have shown the drug to be generally well tolerated. Recruitment status: open to enrollment http://clinicaltrials.gov/ct2/show/NCT00756938? Sponsor: Merck Pharmaceuticals Site PI: Dr. Larry Greenbaum, Director, Division of Pediatric Nephrology, Emory University, Atlanta, GA Site coordinator: Margo Kamel, MSPH 16. Genotype as a Predictor of Mycophenolate Mofetil Related Toxicity in Pediatric Transplant Recipients (MPA-GAS) Purpose: The purpose of this research study is to find out whether small differences in a person’s genes contribute to their risk of developing side effects such as low white blood cell counts while taking mycophenolate mofetil (MMF or CellCept®). Eligible
  10. 10. participants have received a kidney transplant and have been treated with mycophenolate mofetil (CellCept®) as part of their immune suppression regimen. This study looks at minor gene variations both in patients who have had a specific side effect while taking CellCept and in those who have not had that side effect while taking this medication. Recruitment status: open to enrollment Participating research site with Cincinnati Children’s Hospital. Site PI: Dr. Barry Warshaw, Associate Professor, Division of Pediatric Nephrology, Emory University, Atlanta, GA Site coordinators: Margo Kamel, MSPH, and Tauri Harden, RN 17. P-Glycoprotein and Steroid Resistance in Pediatric Nephrotic Syndrome (SRNS) Purpose: The purpose of the study is to look at the blood cells of children with nephrotic syndrome, to see if certain children have greater activity of the p-glycoprotein pump. The researchers also will look at the pump activity to see if this helps to predict whether or not the child responds to therapy with steroids. Recruitment status: open to enrollment Participating research site with the Research Institute at Nationwide Children's Hospital, Columbus, OH. Site PI: Dr. Larry Greenbaum, Director, Division of Pediatric Nephrology, Emory University, Atlanta, GA Site coordinator: Margo Kamel, MSPH 18. Helping teens with kidney transplants to prepare for adulthood Purpose: This is a longitudinal study which will be comprised of survey data collected from patients and parents every 3 months from entry into adolescent transition clinic until the patient transitions to adult care. The purpose of the study is to identify predictors for poor
  11. 11. transition and medication non-adherence. The particular battery of surveys administered aims to examine general health measures, emotional functioning, readiness for transition, adherence barriers and risk behaviors because it is believed that these measures may be the most critical in determining successful transition to adult care. Recruitment status: open to enrollment PI: Dr. Sandra Amaral, Assistant Professor of Pediatrics, Division of Pediatric Nephrology, Emory University, Atlanta, GA Site coordinator: Verena Weissenborn, RN 19. A Phase 3, Prospective, Randomized, Double-blind, Placebo controlled Multicenter Study to Evaluate the Pharmacokinetics, Safety and Efficacy of Paricalcitol Capsules in Decreasing Serum Intact Parathyroid Hormone Levels in Pediatric Subjects Ages 10 to 16 years with Moderate to Severe Chronic Kidney Disease (Zemplar study) Purpose: Single Dose Pharmacokinetic (Part I) This research study involves the use of Paricalcitol (Zemplar®) capsules, an active form of vitamin D. Zemplar® is approved by the United States Food and Drug Administration (FDA) for the prevention and treatment of hyperparathyroidism associated with chronic kidney disease. The Zemplar® capsule is currently approved for use in adult patients (18 years or older). This study is going to evaluate the use of paricalcitol capsules in a pediatric population (ages 10-16 years) to safely and effectively decrease parathyroid levels along with evaluating the pediatric subjects absorption and metabolism of the study drug. Recruitment status: open to enrollment 24-Week Safety and Efficacy (Part II) This research study involves the use of Paricalcitol (Zemplar®) capsules, an active form of vitamin D. Zemplar® is approved by the United States Food and Drug Administration (FDA) for the prevention and treatment of hyperparathyroidism associated with chronic kidney disease. The Zemplar® capsule is currently approved is for use in adult patients (18 years or older). This study is going to
  12. 12. evaluate the use of Paricalcitol capsules in a pediatric population (ages 10-16 years) to safely and effectively decrease parathyroid levels. Recruitment status: not open to enrollment yet Sponsor: Abbott Pharmaceuticals Site PI: Dr. Larry Greenbaum, Director, Division of Pediatric Nephrology, Emory University, Atlanta, GA Site coordinator: Margo Kamel, MSPH 20. Executive Function in Children with Hypertension Purpose: Studies in young adults indicate that primary hypertension is associated with decreased performance on neurocognitive testing compared with normotensive controls, particularly in the domains of attention, working memory, and executive function. These cognitive deficits can improve in adults when hypertension is subsequently well-controlled, indicating that the neurocognitive deficits seen in hypertensives may represent an early manifestation of hypertensive target organ damage of the brain. The goal of the current proposal is to investigate the relationship between primary hypertension and executive function as an emerging target of hypertensive damage in children. The overall hypothesis is that children with primary hypertension have evidence for central nervous system target organ damage, as manifested by decreased executive function. Recruitment status: not open to enrollment yet Sponsor: NIH Site PI: Dr. Donald Batisky, Director of Hypertension Program, Division of Pediatric Nephrology, Emory University, Atlanta, GA Site coordinator: Margo Kamel, MSPH
  13. 13. 21. A multicenter, randomized, double-blind, 8 week study to evaluate the dose response, efficacy and safety of aliskiren in pediatric hypertensive patients 6-17 years of age Purpose: This double-blind 8 week study will evaluate dose response, efficacy (blood pressure lowering effect) and safety of aliskiren in children 6 - 17 years old with hypertension at low, mid and high weight-based doses. The low dose ranges from 6.25 mg to 25 mg of aliskiren, the mid dose ranges from 37.5 mg to 150 mg of aliskiren and the high dose ranges from 150 mg to 600 mg of aliskiren. This study is being conducted to support monotherapy registration of aliskiren for the treatment of hypertension in children 6-17 years of age. Recruitment status: not open to enrollment yet http://clinicaltrials.gov/ct2/show/NCT01150357? Sponsor: Novartis Pharmaceuticals Site PI: Dr. Donald Batisky, Director of Hypertension Program, Division of Pediatric Nephrology, Emory University, Atlanta, GA Site coordinator: Margo Kamel, MSPH

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