Clinical Trials (Pediatric)

A Phase 3, Randomized, Modified Double-blind, Active-controlled, Parallel-group, 2-arm Study to Investigate the Safety and Immunogenicity of a Single Dose of a 21-valent Pneumococcal Conjugate Vaccine in Children and Adolescents With Sickle Cell Disease

People with sickle cell disease have a higher risk of infections caused by the bacteria Streptococcus Pneumoniae. There are currently a few vaccines available to prevent this infection. This study is comparing the safety and efficacy of a new formulation of the vaccine, PCV21 with the currently recommended 20vPCV.

Principle Investigator: Dr. Archana Sharma

Sponsor: Sanofi

Call 732-235-7894 or visit: https://www.clinicaltrials.gov/study/NCT07247188 for more information.

A Phase 2/3 Study to Evaluate the Safety, Efficacy, and Pharmacokinetics of Atumelnant Treatment in Pediatric Participants With Congenital Adrenal Hyperplasia Including a Long-Term Extension

Congenital adrenal hyperplasia (CAH) is a genetic disorder resulting in low cortisol and high androgen levels. There is no cure but it is currently managed with lifelong treatments with steroids. This study is investigating the safety and efficacy of a new oral medication, Atumelnant, that works as a competitive antagonist to reduce androgen secretion.

Principle Investigator: Dr. Ahmed Khattab

Sponsor: Crinetics Pharmaceuticals Inc.

Call 732-235-7894 or visit: https://www.clinicaltrials.gov/study/NCT07159841 for more information.

A Multicenter, 12-Month, Randomized, Double Blind, Placebo-Controlled Phase 3 Efficacy and Safety Study of Daily Oral LUM-201 in Naïve-to-Treatment, Prepubertal Children with Growth Hormone Deficiency (GHD)

Growth hormone deficiency (GHD) is an uncommon disorder occurring in children that can lead to short stature along with other complications. Current treatment involves subcutaneous injections of replacement growth hormones for several years. This study is investigating a new oral medication, LUM-201, which may help certain patients secrete their own growth hormone and avoid the need for frequent injection.

Principle Investigator: Dr. Ian Marshall

Sponsor: Lumos Pharma

Call 732-235-7894 or visit: https://www.clinicaltrials.gov/study/NCT06948214 for more information.

AN INTERVENTIONAL, EFFICACY, AND SAFETY, PHASE 3 RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED STUDY WITH AN OPEN-LABEL EXTENSION TO INVESTIGATE RIMEGEPANT IN MIGRAINE PREVENTION IN ADOLESCENTS 12 TO LESS THAN 18 YEARS OF AGE WITH CHRONIC MIGRAINE

This is a global, multicenter, randomized, double-blind, placebo-controlled, parallel group study to assess the efficacy and safety of rimegepant in migraine prevention in adolescents 12 to <18 years of age with chronic migraine.
A total of approximately 200 eligible participants with chronic migraine are planned to receive rimegepant or matching placebo. Enrollment should include a similar number of participants in the 12 to 14 years of age group and in the 15 to 17 years of age group. Additionally, at least 25% of randomized participants should be male.
The total study duration is up to 76 weeks. There is an optional OLE phase following the DBT phase to evaluate the long-term safety and tolerability of rimegepant.
It is estimated that approximately 150 participants may enter the OLE phase. To ensure adequate information on safety, at least 100 participants will be treated with rimegepant for up to 1 year.

Principle Investigator: Dr. Amanda Spinner

Sponsor: Pfizer

Call 732-235-7894 or visit: https://www.clinicaltrials.gov/study/NCT06616194 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.

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.

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.