In recognition of World Kidney Month in March and Irritable Bowel Syndrome (IBS) Awareness Month in April, this month's Research Roundup is dedicated to the incredible research being conducted in Nephrology and Gastroenterology. Research in these departments is advancing pediatric medicine affecting thousands of children in the U.S. diagnosed with rare disorders like Focal Segmental Glomerulosclerosis (FSGS)1 and school-aged children reporting IBS-like symptoms2.
Dr. Alisa Muniz Crim, Nicklaus Children's Pediatric Specialists Chief of Gastroenterology, emphasized the importance of research saying “Research allows us to identify and create novel ways to diagnose and treat conditions. Increasing our understanding of a condition improves health outcomes for children with conditions like Inflammatory Bowel Disease.”
Combined, these teams currently have five studies in development and six interventional studies in operation. Studies like these make innovative treatments available to patients of Nicklaus Children's Health System that may have previously had limited options.
Dr. Ana Paredes, a Nephrologist at Nicklaus Children's, said that “Access to cutting-edge treatments, sometimes only available through research, enables us to intervene earlier in the progression of conditions like FSGS. This significantly improves odds of successfully treating these diseases.”
Dr. Lina Felipez Marrero, the Director of Research for the Inflammatory Bowel Disease (IBD) Program, is most excited about research currently in development. Her team will use a patient's genetic profile to individualize treatment for IBD. Innovative research like this “enhances our understanding of conditions like IBD and ultimately leads to better care.”
Research at a pediatric hospital in a city like Miami contributes to research in other ways. Dr. Paredes stressed the importance of pediatric research saying “Most research results are based on an adult population. Pediatric clinical trials are critically important to ensuring optimal dosage, pharmacokinetics, and safety.” Adding to this, Dr. Muniz Crim recognized that “Inclusion of the Hispanic population unique to South Florida, ensures that research results for conditions like IBD can be applied with confidence to the patients here at Nicklaus Children's and families around the world.”
The Research Institute anticipates great success in advancing pediatric medicine because of research conducted by leaders like these. As Dr. Muniz Crim said “By contributing to research, you really are changing the future!”
Currently Recruiting Studies
Nephrology Studies
Study of Efficacy and Safety of Iptacopan in Patients With C3 Glomerulopathy. (APPEAR-C3G)
NCRI ID: NEP23121
Condition: Complement 3 Glomerulopathy (C3G)
Age for Inclusion: 12+ years old
Study of Sparsentan Treatment in Pediatrics With Proteinuric Glomerular Diseases (EPPIK)
NCRI ID: NEP99184
Condition: Focal Segmental Glomerulosclerosis (FSGS), Minimal Change Disease, Immunoglobulin A Nephropathy, IgA Vasculitis, Alport Syndrome.
Age for Inclusion: 1-17 years old
Gastroenterology Studies
DIET- Treatment and Acceptability of Specific Carbohydrate Diet for Children with Active Crohn's Disease or Ulcerative Colitis in the South Florida Population
NCRI ID: GAS99043
Condition: Crohn's Disease, Ulcerative Colitis
Age for Inclusion: 8-21 years old
Evaluation of Oral Tofacitinib in Children Aged 2 to 17 Years Old Suffering From Moderate to Severe Ulcerative Colitis
NCRI ID: GAS99042
Condition: Ulcerative Colitis
Age for Inclusion: 2-17 years old
Environmental Risk Factors Associated with Pediatric Eosinophilic Esophagitis (EoE) in a Tertiary Care Center in South Florida
NCRI ID: GAS23157
Condition: Eosinophilic Esophagitis (EoE),
Age for Inclusion: <21 years old
ABTECT-2 - ABX464 Treatment Evaluation for Ulcerative Colitis Therapy -2
NCRI ID: GAS23135
Condition: Ulcerative Colitis
Age for Inclusion: 16+ years old
Recently Activated Studies
Randomized Study in Children and Adolescents With Migraine: Acute Treatment
NCRI ID: NEU23013
Condition: Migraine
Age for Inclusion: 6-17 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 of Lower Radiotherapy Dose to Treat Children With CNS Germinoma
NCRI ID: ONC25006
Condition: Germinoma
Age for Inclusion: 3+ 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
Developmental Dysplasia of the Hip: Observation Vs. Bracing
NCRI ID: ORT23125
Condition: Hip Dysplasia
Age for Inclusion: 1-12 weeks old