Pediatrics Health

Pediatric Clinical Trials

 Condition Description  Investigator  Contact Information 
Endocrine  TrialNet: Natural History Study of the Development of Type 1 Diabetes: Monitoring

The overall objective of this study is to perform baseline and repeat assessments over time of the metabolic and immunologic status of individuals at risk for type 1 diabetes (T1D) to: (a) characterize their risk for developing T1D and identify subjects eligible for prevention trials, (b) describe the pathogenic evolution of T1D, and (c) increase the understanding of the pathogenic factors involved in the development of T1D. 

For more information, click here.
Appiagyei-Dankah, Yaw Emily Beltz
Emily.Beltz@spectrumhealth.org
616.486.4936 

Hematology / Oncology / BMT  BMT Cord Blood Access Protocol: A Multicenter Access and Distribution Protocol for Unlicensed Cryopreserved Cord Blood Units (CBUs) in Pediatric and Adult Patients with Hematologic Malignancies and other Indications

This is an access study in which umbilical cord blood used for bone marrow transplant is only available through this access and distribution study. In October 2011, the Food and Drug Administration (FDA) began considering cord blood as a biological drug.  In the United States, drugs must meet standards set by the FDA to make sure they are safe. Cord blood units that were not collected, tested, or stored exactly according to FDA standards may be used for transplant if the transplant is done as part of a study. 

For more information, click here.
Abdel-Mageed, Aly Kathy Nystrom
Kathy.Nystrom@spectrumhealth.org
616.486.2062  
Hematology / Oncology / BMT  NYBB UCB: A multicenter safety study of unlicensed, investigational cryopreserved cord blood units (CBUs) manufactured by the National Cord Blood Program (NCBP) and provided for unrelated hematopoietic stem cell transplantation of pediatric and adult patients 

Umbilical cord blood used for bone marrow transplant which is only available through this access and distribution study. In October 2011, the Food and Drug Administration (FDA) began considering cord blood as a biological drug. In the United States, drugs must meet standards set by the FDA to make sure they are safe. Cord blood units that were not collected, tested, or stored exactly according to FDA standards may be used for transplant if the transplant is done as part of a study. 
Abdel-Mageed, Aly Kathy Nystrom
Kathy.Nystrom@spectrumhealth.org
616.486.2062  
Hematology / Oncology / BMT  Multiple Cord Blood: Allogeneic Stem Cell Transplantation Using Multiple Unrelated Donor Umbilical Cord Grafts

This research study will evaluate the ability of umbilical cord blood cells, which have been obtained from multiple unrelated donors, to serve as a source of stem cells for patients undergoing stem cell transplant. The use of a single unrelated umbilical cord blood unit is common for small pediatric patients requiring stem cell transplantation when a related or unrelated donor is not available. For larger patients, the use of more than one umbilical cord blood unit provides an adequate number of stem cells and increases the chances of successful engraftment and survival.  
Abdel-Mageed, Aly  Kathy Nystrom
Kathy.Nystrom@spectrumhealth.org
616.486.2062 
Hematology / Oncology / BMT  CliniMACS T-Cell Depletion: An Expanded Access Study of the Feasibility of Using the CliniMACS® Device for CD34+ Cell Selection and T-Cell Depletion for Graft-versus-Host Disease Prophylaxis in Alternative Donor Stem Cell Transplant Recipients

This research study will help doctors learn about how to prevent serious side effects that can happen when a patient receives a stem cell transplant that uses cells from an alternative donor. This will allow your doctor to use a potentially lifesaving investigational treatment that is not FDA-approved for use in the treatment of the recipient’s disease at this time. This protocol will also allow researchers to gather additional information about the safety and effectiveness of using the CliniMACS® device to help prevent GVHD after an alternative-donor transplant.  
Abdel-Mageed, Aly  Emily Gleason
Emily.Gleason@helendevoschildrens.org
616.486.6332 
Hematology / Oncology / BMT  Kid-DOTT: Prospective Evaluation of the Duration of Therapy

This is a research study looking at treating venous blood clots with blood thinning medication. 

For more information, click here.
Mitchell, Deanna  Emily Gleason
Emily.Gleason@helendevoschildrens.org
616.486.6332  
Hematology / Oncology / BMT  LSD-Gauchers (Registry): The Gaucher Registry, an observational registry program
 
The Gaucher Registry is one of four registries which make up the Rare Disease Registry. The Rare Disease Registry sponsored by Genzyme collects information on patients with Gaucher, Pompe, Fabry, and mucopolysaccharidosis type I (MPS I) diseases to better understand how to prevent, diagnose and treatment them.

For more information, click here.
Dickens, David  Bill Boshoven
William.Boshoven@spectrumhealth.org
616.486.6121 
Hematology / Oncology / BMT  Brincidofovir: Expanded Access Protocol to Provide Brincidofovir for the Treatment of Serious Adenovirus Infection or Disease

This study is an access protocol to provide treatment of the investigational drug called brincidofovir (BCV) to treat the virrus called adenovirus (or AdV). 

For more information, click here.
Duffner, Ulrich  Laura Paulsen
cancer.research@spectrumhealth.org
616.486.8500 
Nephrology  CKiD: Chronic Kidney Disease in Children Prospective Cohort Study 

Chronic Kidney Disease in Children Prospective Cohort Study (CKiD), two cohorts of patients (20;10) opening an amendment, committing to enrolling three cohorts of 10 more over next three years. We are one of 52 sites, E coast region led by CHOP.  Third cohort open. Children need to be under five years of age.

For more information, click here.
Quiroga Chand, Alejandro Jennifer Bartleson
Jennifer.Bartleson@spectrumhealth.org
616.391.8534
Nephrology  Genome: Genome Wide Association Study of Childhood Onset Idiopathic Nephrotic Syndrome

To investigate the genetic risk factors for idiopathic nephrotic syndrome (NS) and define common disease loci for childhood NS.  
Cai, Yi  Kathy Nystrom
Kathy.Nystrom@spectrumhealth.org
616.486.2062  
Nephrology  MWPNC Lupus Nephritis Registry: Pediatric Lupus Nephritis Registry

Systemic Lupus Erythematosis (SLE) is a chronic disease that affects more that million people in the USA. Studies show that up to 80 percent of children with SLE disease will develop lupus nephritis. The registry plans to initially study if certain laboratory results predict outcomes and if there are differences in treatment outcomes between people of different races. This registry is being done by a group of pediatric nephrologists (kidney doctors for children) and rheumatologists (doctors who specialize in autoimmune diseases) throughout the United States who have joined efforts to study this disease. 
Quiroga Chand, Alejandro  Emily Beltz
Emily.Beltz@spectrumhealth.org
616.486.4936  
Nephrology  MWNPC Neph Syndrome Registry (CNOS): Childhood Nephrotic Syndrome Observational Study

A registry of patients who have nephrotic syndrome. To gather information about how nephrotic syndrome progresses in different people and how or why that happens and to improve understanding of the causes, effects and treatment response of childhood nephrotic syndrome. 
Quiroga Chand, Alejandro  Stephanie Fagerman
Stephanie.Fagerman@spectrumhealth.org
616.486.6332 
Nephrology  Atlantis Study: ACTH for Frequently Relapsing and Steroid Dependent Nephrotic Syndrome

The purpose of this study is to determine if ACTH prevents relapses in children and young adults with nephrotic syndrome. ACTH is a hormone that is produced in the body, it has been made into a formulation called (HP Acthar Gel®) that is given by injections two times a week. The ACTH treatment will be given for 12 months. ACTH is not FDA approved for use in children. 

For more information, click here
Quiroga Chand, Alejandro  Jennifer Bartleson
Jennifer.Bartleson@spectrumhealth.org
616.391.8534
Nephrology  Cure GN: Cure Glomerulonephropathy Network

The purpose of the CureGN is to create a source of information so that researchers can easily and effectively study rare glomerular disease such as MCD, FSGS, MN and IgAN, the underlying causes, identify markers of disease, and identify and evaluate new therapies. 
Cai, Yi Bill Boshoven
William.Boshoven@spectrumhealth.org
616.486.6121  
Nephrology  MWPNC GIGA: GIGA-kids Genomics of IgA related diseases in kids

The purpose of this study will be to determine how some genes affect the development of IgAN and HSP by looking at biomarkers found in the blood. Normal, healthy children will also be enrolled in this study as matched controls and are required to perform adequate validations of biomarkers. IgA Nephropathy (IgAN) and Henoch-Schönlein Purpura (HSP) are related diseases, both thought to be due by deposits of the IgA antibody in the kidney. IgA nephropathy (IgAN) represents the leading cause of kidney failure among young adults, and the most frequent form of primary glomerulonephritis worldwide. 
Quiroga Chand, Alejandro  Bill Boshoven
William.Boshoven@spectrumhealth.org
616.486.6121
Nephrology  Gambro HF-20: Prismaflex HF20 Set and Prismaflex® System 7.10 for Acute CRRT in Children

This research study involves patients with acute kidney injury. This study collects data on the use of a CRRT device for children weighing less than 20 kg (44.09 pounds). The smaller size was designed specifically for the treatment of small children. The Gambro Prismaflex System Version 7.10 has been designed to allow for the safe treatment of children weighing down to eight kg (17.6 pounds). 

For more information, click here.
Hackbarth, Richard  Emily Gleason
Emily.Gleason@helendevoschildrens.org
616.486.6332
Nephrology  NAPRTCS Registry

The purpose of the registry is to enter scientific data on the care and treatment of pediatric kidney disease and transplantation into a national registry. Collecting this data into one nationwide registry is expected to improve medical care of children including those who have received renal transplants.
Steinke, Julia  Bill Boshoven
William.Boshoven@spectrumhealth.org
616.486.6121 
Nephrology  Tolvaptan (Hyponatremia)- 276: Study of the Safety and Effectiveness of SAMSCA® (Tolvaptan) in Children and Adolescents With Euvolemic or Hypervolemic Hyponatremia

The purpose of the study is to learn if an investigational medication called tolvaptan can safely and effectively treat hyponatremia in children and adolescents. Tolvaptan is already approved in many countries to treat adults with hyponatremia, but it is considered an investigational medication in this study because it is not known if it works the same way in children or adolescents. The investigational medication, tolvaptan, works by helping the kidneys maintain the right amount of sodium in the blood by helping the kidneys remove extra water. It is important for us to learn if tolvaptan works the same way in children and adolescents as it does in adults. 

For more information, click here
Quiroga Chand, Alejandro  Thomas Symington
Thomas.Symington@spectrumhealth.org
616.391.9366
Nephrology  Acthar Registry: Adrenocorticotropic Hormone (ACTH) for Frequently Relapsing and Steroid Dependent Nephrotic Syndrome

The purpose of the registry is to enter scientific data on the use of Acthar in pediatric patients with relapsing and steroid dependent nephrotic syndrome. 
Quiroga Chand, Alejandro  Jennifer Bartleson
Jennifer.Bartleson@spectrumhealth.org
616.391.8534 
Neurology  MP-104: Deflazacort Expanded Access Program for Children, Adolescents and Adults With Duchenne Muscular Dystrophy

This is an expanded access program for the investigational medication deflazacort, which is a glucocorticosteroid. Through the program, deflazacort is being made available for use for patients with Duchenne muscular dystrophy (DMD) while a new drug application is under preparation and review at the Food and Drug Administration. 

For more information, click here.
DeRoos, Steven  Kathy Nystrom
Kathy.Nystrom@spectrumhealth.org
616.486.2062  
Neurology  IPSS: International Paediatric Stroke Study (IPSS): Towards the Establishment of Standards of Practice and the Initiation of Multi-Center, Multi-National Clinical Trials for Neonates and Children with Stroke

The purpose of this research is to learn more about childhood stroke (sudden blockage of blood flow to a part of the brain). Since 1992, the sponsor, Hospital for Sick Children, has been running a Canadian Registry of Pediatric Stroke with children’s hospitals in Canada. Since 2002, the sponsor has also run an International Registry for childhood stroke with children’s hospitals around the world. 
Fain, Daniel  Stephanie Fagerman
Stephanie.Fagerman@spectrumhealth.org
616.486.6332  
Pulmonary: Cystic Fibrosis  CF Registry: Cystic Fibrosis Registry

This study is an ongoing data collection effort sponsored by the Cystic Fibrosis Foundation, known as the Patient Registry. The goal of the Patient Registry is to collect data to better understand the illness and ultimately improve the care and survival of those with CF. The Patient Registry was established in 1966 as a means to monitor important trends in the CF population and to improve understanding, treatment, and survival. 
Millard, Susan  Cynthia Gile
Cynthia.Gile@spectrumhealth.org
616.391.6148
Pulmonary: Cystic Fibrosis  CFFC: A Long-Term Prospective Observational Safety Study of the Incidence of and Risk Factors for Fibrosing Colonopathy in US Patients with Cystic Fibrosis Treated with Pancreatic Enzyme Replacement Therapy: A Harmonized Protocol Across Sponsors (the "Study")  

A Long-Term Prospective Observational Safety Study of the Incidence of and Risk Factors for Fibrosing Colonopathy in US Patients with Cystic Fibrosis Treated with Pancreatic Enzyme Replacement Therapy: A Harmonized Protocol Across Sponsors

For more information, click here
Millard, Susan  Cynthia Gile
Cynthia.Gile@spectrumhealth.org
616.391.6148 
Pulmonary: Cystic Fibrosis  Transition Study: Survey of Experiences with Transition to Adult Cystic Fibrosis Care

This study is a research initiative to improve the process of transitioning from pediatric to adult cystic fibrosis care. 
McCaslin, Charles  Cynthia Gile
Cynthia.Gile@spectrumhealth.org
616.391.6148 
Pulmonary: Cystic Fibrosis  Teach: Testing the Effect of Adding Chronic Oral Azithromycin to Inhaled Tobramycin in People With CF (TEACH)

People with Cystic Fibrosis (CF) commonly get chronic infections in their lungs. One bacteria or germ called Pseudomonas aeruginosa (Pa) is particularly good at living in the CF lung. Pa is often treated with the antibiotics, azithromycin and inhaled tobramycin. There is some study data that suggests that azithromycin and inhaled tobramycin do not work as well when they are used together. We are investigating if this is true.

For more information, click here
Millard, Susan  Cynthia Gile
Cynthia.Gile@spectrumhealth.org
616.391.6148 

Sleep  Sleep Apnea Microbiome study

At present, the only way to screen and diagnose obstructive sleep apnea is by performing a sleep study which is not available to all children. This study will try to evaluate if changes in the stool flora or microbiome (microorganisms normally present in your body) could be used to screen or diagnose children with obstructive sleep apnea. 
Tamae Kakazu, Maximiliano  Bill Boshoven
William.Boshoven@spectrumhealth.org
616.486.6121 

You can also visit clinicaltrials.gov for opportunities internationally.