Clinical Trials (Pediatric)

A randomized, double-blind, placebo-controlled study to evaluate the efficacy and safety of SPR001 (tildacerfont) in reducing supraphysiologic glucocorticoid use in adult subjects with classic adrenal hyperplasia

Classic adrenal hyperplasia (CAH) is a genetic disorder that results in decreased cortisol production and increased androgen (male sex hormone) production. CAH currently has no approved therapies. Tildacerfont is a new drug that may help with blocking signals from the brain to decrease hormone production from the pituitary gland. The primary objective of the study is to see if tildacerfont can reduce steroid use in patients with CAH.

Principle Investigator: Dr. Ahmed Khattab

Sponsor: Spruce

Call 732-235-7894 or visit: https://www.clinicaltrials.gov/study/NCT05714969 for more information.

Copper histidinate treatment for Menke’s disease

Menkes disease is a rare, life threatening, genetic disorder that affects copper absorption. This study investigates the clinical effects of subcutaneous copper histidinate for patients who were diagnosed with Menke’s disease. The objective is to provide additional clinical and safety data on long term treatment with copper histidinate.

Principle Investigator: Dr. Milen Velinov

Sponsor: Sentynl

Call 732-235-7894 or visit: https://www.clinicaltrials.gov/study/NCT04074512 for more information.

A phase 3, randomized, placebo-controlled, observer-blinded trial to evaluate the safety of a 6-valent OspA-based lyme disease vaccine in healthy children 5 through 17 years of age

Lyme disease is a tick born bacterial infection. If left untreated it can cause lasting effects such as arthritis and neurologic complications. The goal of this study is to help ensure that VLA15 (Lyme disease vaccine) is safe in children ages 5 through 17 years old. Patients will receive the vaccine in a 3-dose series given at months 0, 2 and 6.

Principle Investigator: Dr. Sunanda Gaur

Sponsor: Pfizer

Call 732-235-7894 or visit:  https://www.clinicaltrials.gov/study/NCT05634811 for more information.

A Multi-Center Observational Study: The RECOVER Post Acute Sequelae of SARS-CoV-2 (PASC) Pediatric Cohort Study

This study is collecting and analyzing data on children with Post Acute Sequelae of SARS-CoV-2 (PASC). PASC, also known as long covid, is not well understood at this time but includes lasting symptoms for more than 4 weeks after the initial covid-19 infection has resolved and new illness possibly due to immune dysregulation. The goal of this study is to help define and characterize PASC to help better understand it.

Principle Investigator: Lawrence Kleinman MD

Sponsor: Other Transactional Authority, National Heart, Lung, And Blood Institute (NHLBI) National Institutes Of Health (NIH)

Call 732-235-7894 or visit: https://clinicaltrials.gov/study/NCT05172024 for more information.

COVID-19 Network of Networks Expanding Clinical and Translational approaches to Predict Severe Illness in Children (CONNECT to Predict Sick Children)

This study is collecting and analyzing data on children with mild-severe covid-19 infections and post covid multi-inflammatory syndrome in children. The long-term goal of this study is to develop and deploy models to predict the severity of illness in patients.

Principle Investigator: Lawrence Kleinman MD

Sponsor: National Institutes of Health (NIH)

Call 732-235-7894 or visit: https://reporter.nih.gov/project-details/10320995 for more information.

Environmental influences on Child Health Outcomes (ECHO) Cohort Data and Biospecimen Collection Protocol

This study is collecting and analyzing data and biospecimens from children, pre-birth through 20 years old, to better understand environmental influences (physical, chemical, social, behavioral, and biological) on development. This data will go into the ECHO platform which can then be used to address research question that require population data.

Principle Investigator: Dr. Emily Barrett, PhD

Sponsor: National Institutes of Health (NIH)

Call 732-235-7894 or visit: https://www.nih.gov/echo for more information.

Observational Study for Pediatric Rheumatic Diseases: The CARRA Registry

Rheumatic diseases, including juvenile idiopathic arthritis and systemic lupus erythematosus, affect about 300,000 children in the US. This study registry prospectively collects data on patients with pediatric-onset rheumatic disease. The aim of this registry is to evaluate treatment patterns and clinical outcomes over time.

Principle Investigator: Lakshmi Nandini Moorthy, MD

Sponsor: CARRA

Call 732-235-7894 or visit: https://clinicaltrials.gov/study/NCT02418442 for more information.

Chronic childhood vasculitis: Characterizing the individual rare diseases to improve patient outcomes

Childhood chronic primary vasculitis (CPV) is a group of rare life-threatening diseases caused by inflammation of the blood vessels that lead to damage of vital organs. Due to rarity of CPV in children the individual diseases they are difficult to characterize. This study aims to collect clinical information and biological samples from children with CPV and compare them to adults with CPV, to better classify, diagnose and monitor children with CPV

Principle Investigator: Lakshmi Nandini Moorthy, MD

Sponsor: University of British Columbia

Call 732-235-7894 or visit: https://clinicaltrials.gov/study/NCT02006134 for more information.

PARADIGHM (Physicians Advancing Disease Knowledge in Hypoparathyroidism): A Registry for Patients with Chronic Hypoparathyroidism

Chronic hypoparathyroidism is a rare disease caused by decreased parathyroid hormone secretion. This results in low calcium and phosphate levels. Standard treatment is to supplement with calcium and vitamin D which can lead to fluctuating levels and potential long-term damage to the kidneys. rhPTH(1-84) is a recently developed recombinant parathyroid hormone identical to what is made in the body. It can be given as a daily injection and has been shown to reduce the amount of calcium and vitamin D needed. This registry is collecting data from patients with chronic hypoparathyroidism who have and have not received rhPTH to help understand the risks and benefits. This information can also be used to guide future treatments and research in this field.

Principle Investigator: Ian Marshall, MD

Sponsor: Takeda Development Center Americas, Inc.

Call 732-235-7894 or visit: https://clinicaltrials.gov/study/NCT01922440 for more information.

Reduction of Duration and Severity of Pediatric Gastroenteritis Through Amino Acid Fortified Oral Rehydration Therapy

Young children are at higher risk for dehydration when they have diarrhea. Children who present with dehydration are typically given Oral Rehydration Therapy (such as Pedialyte). This study is testing a fortified Oral Rehydration therapy that includes amino acids which may help decrease the diarrhea faster and speed up recovery.

Principle Investigator: Dr. Paul Breslin

Sponsor: Gerber Foundation

Call 732-235-7894 or visit: https://clinicaltrials.gov/study/NCT06137014 for more information.

The PRECIOUS Study: Predicting Crohn’s & Colitis Outcomes in the United States

Crohn’s disease and ulcerative colitis are inflammatory bowel diseases (IBD) that affect both children and adults. IBD presents as range of gastrointestinal symptoms including bloody diarrhea and can affect nutrition and growth. Treatments for IBD include steroids, and oral and IV medications that target the immune system. This study is evaluating the PredictSURE IBD blood test in a diverse US population. This test is designed to predict the severity of a patient’s disease upon diagnosis and once validated can be used to help determine a patients treatment course.

Principle Investigator: Melissa Weidner MD

Sponsor: PredictImmune Ltd. UK and the Crohn’s & Colitis Foundation, USA

Call 732-235-7894 or visit: https://clinicaltrials.gov/study/NCT03952364 for more information.

A phase 2A, 2-part, open-label, non- randomized, multicenter, single and multiple dose trial to evaluate pharmacokinetics, safety, and tolerability of aztreonam and avibactam ± metronidazole in neonates and infants from birth to less than 9 months of age with suspected or confirmed infections due to gram negative pathogens requiring intravenous antibiotics

Bacterial resistance to antibiotics is a problem for patients of all ages. This study is evaluating a combination of currently used antibiotics, aztreonam and avibactam, in patients less than 9 months old to evaluate its safety and tolerability in neonates and infants. With the data aztreonam plus avibactam may become an option for patients with resistant bacterial infections.

Principle Investigator: Dr. Surasak Puvabanditsen

Sponsor: Pfizer

Call 732-235-7894 or visit: https://www.clinicaltrials.gov/study/NCT06462235 for more information.