Pediatric Kidney Research

Pediatric Research
Committed to excellence and innovation in pediatric kidney research

In conjunction with Emory University School of Medicine, the Children's Healthcare of Atlanta Nephrology Program is committed to excellence and innovation in pediatric kidney research.

We are devoted to developing new and better preventive, diagnostic services and treatments through clinical and lab research.

KidneyResearchOverview

Our team is active in advanced clinical research for pediatric kidney transplant, giving children better options for transplants and innovative therapies. We are involved in:

Our areas of interest include:
  • Adolescent nonadherence and transition to adult care
  • Behavioral consequences of solid organ transplant
  • Cystinosis
  • Effects of chronic kidney disease in children
  • Hypertension
  • Immunosuppressive strategies
  • Nephrotic syndrome and focal segmental glomerulosclerosis
  • New medications to prevent transplant rejection
  • Noninvasive strategies to monitor kidney transplant recipients

Children's is also at the forefront of atypical HUS research, our physicians successfully provided a kidney transplant combined with an experimental drug therapy to treat the condition, only the third patient of this kind in the U.S.

OpenProtocols

An Open-label, Multi-center Clinical Trial of Eculizumab in Pediatric Patients with Atypical Hemolytic-uremic syndrome (aHUS) (Study Number:  C10-003)
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 if eculizumab is safe and effective in the treatment of aHUS in pediatric patients who have not been adequately helped or have suffered side effects from the current available treatments. 

  • Recruitment status: on-going, not recruiting patients
  • Sponsor: Alexion Pharmaceuticals
  • Site PI: Dr. Larry Greenbaum, Director, Division of Pediatric Nephrology, Emory University, Atlanta, GA
  • Site coordinator: Gail Schwartz, RN and Margo Kamel, MSPH

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 (Aliskiren Core study)
This double-blind 8 week study will evaluate dose response, efficacy (blood pressure lowering effect) and safety of aliskiren in children 6 to 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: open to enrollment
  • Sponsor: Novartis Pharmaceuticals
  • Site PI: Dr. Donald Batisky, Director of Hypertension Program, Division of Pediatric Nephrology, Emory University, Atlanta, GA
  • Site coordinator: Irena Kizer, BS

A Multicenter, Double-blind, Randomized, 52-week, Extension Study to Evaluate the Long Term Safety, Tolerability and Efficacy of Aliskiren Compared to Enalapril in Pediatric Hypertensive Patients 6-17 Years of Age (Aliskiren Extension study)
The purpose of this study is to evaluate in a randomized, double-blind fashion, the long-term safety, tolerability and efficacy profile of aliskiren compared to the active comparator enalapril in children, 6 to17 years old with hypertension (msSBP ≥ 95th percentile for age, gender and height, at baseline in study CSPP100A2365). Patients will be randomized to receive either aliskiren or enalapril. Weight-group based doses of aliskiren or enalapril will be administered once daily and children will receive study medication in a double-blind manner. This study is being conducted to support monotherapy registration of aliskiren for the treatment of hypertension in pediatric patients 6-17 years of age (age at baseline in Study CSPP100A2365).

  • Recruitment status: open to enrollment
  • Sponsor: Novartis Pharmaceuticals
  • Site PI: Dr. Donald Batisky, Director of Hypertension Program, Division of Pediatric Nephrology, Emory University, Atlanta, GA
  • Site coordinator: Irena Kizer, BS

Chronic Kidney Disease in Children Prospective Cohort Study (CKiD)
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.

  • Recruitment status: open to enrollment
  • Sponsor: National Institute of Health (NIH)
  • Site PI: Dr. Larry Greenbaum, Director, Division of Pediatric Nephrology, Emory University, Atlanta, GA
  • Site coordinator: Irena Kizer, BS and Margo Kamel, MSPH

Cerebro-renal disease: A morphometric study of the brains of pediatric patients with chronic renal insufficiency (CRI MRI Study)
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: open to enrollment
  • Sponsor: Children’s Healthcare of Atlanta
  • Co-Investigator: Dr. Larry Greenbaum, Director, Division of Pediatric Nephrology, Emory University, Atlanta, GA
  • Site coordinator: Jessica Humani and Margo Kamel, MSPH

A Long-Term, Open-Label, Safety and Efficacy Study of Cysteamine Bitartrate Delayed-release Capsules (RP103) in Patients with Nephropathic Cystinosis
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.

  • Recruitment status: on-going, not recruiting patients
  • Sponsor: Raptor Pharmaceuticals
  • Site PI: Dr. Larry Greenbaum, Director, Division of Pediatric Nephrology, Emory University, Atlanta, GA
  • Site coordinator: Margo Kamel, MSPH

Executive Function in Children with Hypertension
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: open to enrollment
  • Sponsor: NIH
  • Site PI: Dr. Donald Batisky, Director of Hypertension Program, Division of Pediatric Nephrology, Emory University, Atlanta, GA
  • Site coordinator: Irena Kizer, BS and Margo Kamel, MSPH

Novel Therapies for Resistant FSGS: Phase II Clinical Trial (FONT II)
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 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).

  • Recruitment status: on-going, not recruiting patients
  • 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

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.

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.

  • Recruitment status: on-going, not recruiting patients 
  • 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: Monica Haughton, RN

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: Dr. Allan Kirk, Professor of Surgery, Division of Transplantation, Department of Surgery, Emory University, Atlanta, GA
  • Site coordinator: Monica Haughton, RN and Gail Schwartz, RN

International Pediatric Peritoneal Dialysis Network Study
The International Pediatric Peritoneal Dialysis Network (IPPN) was created to register and collect data regarding pediatric patients receiving peritoneal dialysis. The purpose of this study is to continuously monitor outcomes in children around the world that are on PD. This registry should provide much needed information to doctors, nurses, dieticians, psychologists, social workers, and health care administrators, and help improve the wellbeing of children currently on PD.

  • Recruitment status: open to enrollment
  • Sponsor: International Pediatric Peritoneal Dialysis Network (IPPN)
  • Site PI: Dr. Larry Greenbaum, Director, Division of Pediatric Nephrology, Emory University, Atlanta, GA
  • Site coordinator: Irena Kizer, BS                            

A Molecular Pathogenesis-Driven Approach for Diagnosis and Treatment of Complement-Based Renal Diseases (KidCOM)
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.
  • 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

Safety and Pharmocokinetics of Lisinopril in Pediatric Kidney Transplant Recipients (Lisinopril PK Study)
While limited PK data on lisinopril in children with essential hypertension are available, no data exist on lisinopril PK in pediatric renal transplant recipients. Given the kidney is the primaryroute of elimination of lisinopril, its clearance is highly influenced by GFR and this relationship may be impacted by the presence of a single transplanted kidney. For example, the impact of lisinopril on glomerular hemodynamics and GFR may be greater in pediatric transplant recipients and may be related to differences in PK. Additionally, because lisinopril may interact with other drugs, it is unclear if the available pediatric PK data applies to children with a kidney transplant who are prescribed a varying number of immunosuppressive medications whose dosage require frequent adjustment. Therefore, lisinopril PK in this population may differ from otherwise healthy children with essential hypertension and the renal transplant patient population may require specific dose adjustments according to age, weight, gender, level of GFR, or concomitant medications.

  • Recruitment status: open to enrollment
  • Sponsor: The Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
  • Site PI: Dr. Larry Greenbaum, Director, Division of Pediatric Nephrology, Emory University, Atlanta, GA
  • Site coordinators: Gail Schwartz, RN 

Genotype as a Predictor of Mycophenolate Mofetil Related Toxicity in Pediatric Transplant Recipients (MPA-GAS)
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 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 Irena Kizer, BS

Nephrotic Syndrome Study Network (NEPTUNE)
The purpose of this study is to find markers of Nephrotic Syndrome (kidney disease with too much protein in the urine). We are particularly interested in diseases called Focal and Segmental Glomerulosclerosis (FSGS), Minimal Change Disease (MCD), and Membranous Nephropathy (MN). By collecting health information and laboratory samples, our goal is to learn more about these kidney diseases and find better ways to prevent and treat people with these kidney diseases.

  • 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 coordinators: Gail Schwartz, RN  

Validation of PROMIS Pediatric Banks with Incident Nephrotic Syndrome (PROMIS NS)
Childhood onset nephrotic syndrome is a condition which affects the kidneys causing them to leak protein from the blood into the urine. Nephrotic syndrome is a disease that can improve (remission) and worsen (relapse) at different times throughout childhood. The purpose of this study is to measure how patients feel when they have active nephrotic syndrome and also when they are in remission. 

  • Recruitment status: open to enrollment
  • Sponsor: NIH
  • Site PI: Dr. Larry Greenbaum, Director, Division of Pediatric Nephrology, Emory University, Atlanta, GA
  • Site coordinator: Margo Kamel, MSPH. Irena Kizer, BS

A Comparative Single-Dose Pharmacokinetic and Safety Study of TAK-491 Between Infants, Children, and Adolescents with Hypertension and Healthy Adults (TAK-491 Study)
The purpose of this research study is to find out how safe and tolerable the study drug  Edarbi (azilsartan medoxomil) is when given to children aged 1 year to 16 years.  We want to look at how one dose of the study drug Edarbi is processed, broken down and passed out by the body over 24 hours after swallowing it. TAK-491 is an investigational drug called Edarbi which has been studied for mild to moderate high blood pressure in adults. Edarbi was approved for the treatment of hypertension in adults in February 2011 by the Food and Drug Administration (FDA). Edarbi has not been approved for children less than 18 years of age.   

  • Recruitment status: not open to enrollment yet
  • Sponsor: Takeda Global Research & Development Center, Inc
  • Site PI: Dr. Donald Batisky, Director of Hypertension Program, Division of Pediatric Nephrology, Emory University, Atlanta, GA
  • Site coordinator: Margo Kamel, MSPH

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)

  • 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 evaluate the use of Paricalcitol capsules in a pediatric population (ages 10-16 years) to safely and effectively decrease parathyroid levels.
    • Recruitment status: open to enrollment
    • Sponsor: Abbott Pharmaceuticals
    • Site PI: Dr. Larry Greenbaum, Director, Division of Pediatric Nephrology, Emory University, Atlanta, GA
    • Site coordinator: Margo Kamel, MSPH

A Phase 3, Open-Label, Multicenter Study to Evaluate the Safety of Paricalcitol Capsules in Pediatric Subjects Ages 10 to 16 with Stage 5 Chronic Kidney Disease Receiving Peritoneal Dialysis (Zemplar M11-612 study)
The objective is to evaluate the safety of paricalcitol capsules in pediatric subjects, ages 10 to 16 years old, with Stage 5 chronic kidney disease (kidney failure) receiving peritoneal dialysis and being treated for secondary hyperparathyroidism. Subjects will be in the dosing period of the study for 12 weeks in order to evaluate the incidence of hypercalcemia (high calcium levels in blood). Approximately 12 subjects will be enrolled and all 12 will receive paricalcitol capsules.

  • Recruitment status: open to enrollment
  • Sponsor: Abbott Pharmaceuticals
  • Site PI: Dr. Larry Greenbaum, Director, Division of Pediatric Nephrology, Emory University, Atlanta, GA
  • Site coordinator: Margo Kamel, MSPH