Researchers around the world celebrate Clinical Trials Day on May 20 in recognition of the incredible advances in medicine made possible by clinical trials. This observance is timed to reflect the launch of the earliest recognized clinical trial by researcher, James Lind. Published in 1753, Lind's work compared several test groups to a control group, to demonstrate the effectiveness of citrus fruit as a treatment for scurvy.

Clinical trials are research studies conducted in a healthcare setting designed to learn if a new treatment is safe, effective, or has fewer side effects than current standard of care options. As a result, physicians can confidently prescribe the most effective treatments for patients or optimize current treatment regimens.

Despite many similarities, pediatric patients can respond differently to the same treatments often used in adult medical practices. For this reason, research in a pediatric setting is vital to advancing medical care for children. Dr. David Seo, Chief Research Officer and Senior Vice President of Research at Nicklaus Children's, said “Clinical trials in pediatric medicine offer hope to children and families facing life-threatening illnesses.” For children enrolled in clinical trials at Nicklaus Children's Hospital, research represents potential relief from a chronic illness and hope for the future.

Treatments and cures that are currently available have been made possible through clinical trials. Recognizing the importance of research in advancing pediatric medicine, Dr. Seo added that “At Nicklaus Children's, we are committed to improving healthcare for our children by expanding the number of clinical trials and making them more accessible to our community.”

A growing number of clinical trials offered at Nicklaus Children's Hospital also requires dedicated staff to ensure that daily processes run smoothly and follows regulations. Nicklaus Children's Research Institute has more than 40 professionals dedicated to ensuring high quality oversight of clinical trials.

If you are at Nicklaus Children's Hospital the week of May 18, please watch for the dedicated members of the research team and take a moment to thank them for the tireless work they do each day to improve children's lives.

Interventional Studies Open for Recruitment in 2025

Randomized Study in Children and Adolescents With Migraine: Acute Treatment
NCRI ID: NEU23013
Condition: Migraine
Age for Inclusion: 6-17 years old

Phase 3 Efficacy and Safety Study of GTX-102 in Pediatric Subjects With AS (Aspire)
NCRI ID: NEU24001
Condition: Angelman Syndrome
Age for Inclusion: 4-17 years old

A Study to Test the Long-term Safety and Tolerability of Brivaracetam in Study Participants With Childhood Absence Epilepsy or Juvenile Absence Epilepsy
NCRI ID: NEU24007
Condition: Absence Epilepsy
Age for Inclusion: 2+ years old

A Study With Tovorafenib (DAY101) as a Treatment Option for Progressive, Relapsed, or Refractory Langerhans Cell Histiocytosis
NCRI ID: ONC23006
Condition: Recurrent or Refractory Langerhans Cell Histiocytosis
Age for Inclusion: 180 days and above

Studying the Effect of Levocarnitine in Protecting the Liver From Chemotherapy for Leukemia or Lymphoma
NCRI ID: ONC23150
Condition: Leukemia or Lymphoma
Age for inclusion: 15+ years old

A Study Comparing Abemaciclib Plus Temozolomide to Temozolomide Monotherapy in Children and Young Adults With High-grade Glioma Following Radiotherapy
NCRI ID: ONC23162
Condition: Glioma
Age for Inclusion: 0+ years old

PEP-CMV Vaccine Targeting CMV Antigen to Treat Newly Diagnosed Pediatric HGG and DIPG and Recurrent Medulloblastoma
NCRI ID: ONC23174
Condition: Glioma and Medulloblastoma
Age for Inclusion: 3+ years old

A Study Using Nivolumab, in Combination With Chemotherapy Drugs to Treat Nasopharyngeal Carcinoma (NPC)
NCRI ID: ONC24032
Condition: Stage II, III, or IV Nasopharyngeal Carcinoma
Age for Inclusion: ≤ 21 years old.

A Study of Lower Radiotherapy Dose to Treat Children With CNS Germinoma
NCRI ID: ONC25006
Condition: Germinoma
Age for Inclusion: 3+ years old

Developmental Dysplasia of the Hip: Observation Vs. Bracing
NCRI ID:  ORT23125
Condition: Hip Dysplasia
Age for Inclusion: 1-12 weeks old