Clinical Trials

At the Children’s Foundation Research Institute, we investigate the latest medications and devices by participating in clinical trials exploring the efficacy of these new treatments. Below is a list of our trials that are currently enrolling patients.

If you have questions regarding one of the clinical trials below, contact us.

Le Bonheur patient Charlie Byrd being cared for in clinical trials by Dr. Tracee Ridley Pryor

Ligelizumab drug trial

  • Principal Investigator: Jay A. Lieberman, MD
  • A 52 week, multi-center, randomized double-blind placebo-controlled study to assess the clinical efficacy and safety of ligelizumab (QGE031) in decreasing the sensitivity to peanuts in patients with peanut allergy
  • Ages 6 to 17 years
  • ClinicalTrials.gov identifier: NCT04984876
  • Study Coordinator: Laura Boywid 901.287.5230 or laura.boywid@lebonheur.org

Aminophylline drug trial

  • Principal Investigator: Umar Boston, MD
  • Effect of aminophylline on preventing acute kidney injury in neonates and infants with congenital heart disease undergoing open heart surgery
  • Neonates less than 1 year needing cardiac surgery for congenital heart defect OR less than 18 years needing heart transplant
  • ClinicalTrials.gov identifier: NCT04984876

      Be Empowered Grow Intentionally Now

      • Principal Investigator: Kathryn Sumpter, MD
      • Device to track continuous glucose monitoring
      • Ages 8 to 17, patient of Le Bonheur’s Endocrine Clinic, poor management of diabetes, enrolled in TennCare health insurance

      TrialNET: Natural History Study of the Development of Type 1 Diabetes

      • Principal Investigator: Alicia Diaz-Thomas, MD
      • 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:
        • Characterize their risk for developing T1D and identify subjects eligible for prevention trials
        • Describe the pathogenic evolution of T1D
        • Increase the understanding of the pathogenic factors involved in the development of T1D

            Regeneron COVID-19 study (not currently enrolling)

            • Principal Investigator: Sandra Arnold, MD
            • Determine the efficacy of REGEN-COV antibody cocktail treatment 
            • Ages 0 to 17 years with at least one risk factor, who have tested positive within the last three days for COVID-19 and are not currently or previously hospitalized due to COVID-19
            • Study coordinator: Lauren Davis (901) 287-4594 or lauren.davis2@lebonheur.org

              ContraFect staph study

              • Principal Investigator: Sandra Arnold, MD
              • Determine the efficacy and safety of a single dose of exebacase in patients receiving standard of care antibiotics for the treatment of staphylococcus aureus bloodstream infections (bacteremia), including right-sided infective endocarditis
              • ClinicalTrials.gov identifier: NCT04160468f

              Nirmatrelvir/Ritonavir COVID-19 study

              • Principal Investigator: Sandra Arnold, MD
              • A phase 2/3 interventional safety, pharmacokinetics and efficacy, open-label multi-center, single-arm study to investigate orally administered PF-07321332 (Nirmatrelvir)/Ritonavir in nonhospitalized symptomatic pediatric participants with COVID-19 who are at risk of progression to severe disease
              • Study coordinator: Katie Ryan (901) 287-5425 or kathryn.ryan@lebonheur.org
              • ClinicalTrials.gov identifier: NCT05261139

                RSV Enanta study

                • Principal Investigator: Sandra Arnold, MD
                • Evaluate EDP-938 regiments 
                • Ages 28 days to 24 months infected with respiratory syncytial virus (RSV)
                • ClinicalTrials.gov identifier: NCT04816721

                  PIVOTAL Pilot Study

                  • Principal Investigator: Mark Weems, MD
                  • Device trial for ages 14-28 days with significant patent ductus arteriosus (hsPDA)

                  Amplitude integrate electroencephalogram (aEEG) in newborns exposed to prenatal illicit substances

                  • Principal Investigator: Divya Rana, MD
                  • All infants and their mothers, admitted to The Newborn Center’s well baby nursery (WBN) or neonatal intensive care unit (NICU) with history of maternal illicit or prescription substance use during pregnancy

                  Brivaracetam drug trial

                  • Principal Investigator: James Wheless, MD
                  • Long-term study to evaluate safety and efficacy of brivaracetam used as adjunctive treatment in pediatric patients with epilepsy
                  • Must have completed prior participation in other related study

                  RESPONSE study

                  • Principal Investigator: James Wheless, MD
                  • RNS System Responsive Stimulation for Adolescents with Epilepsy (RESPONSE), sponsored by NeuroPace
                  • Demonstrate that the RNS system is safe and effective as an adjunctive therapy in adolescents 12 to 17 years with medically refractory partial onset epilepsy
                  • ClinicalTrials.gov identifier: NCT04839601

                  Aucta TSC face cream drug trial

                  • Principal Investigator: James Wheless, MD
                  • Ages 2 to 18 years with diagnosis of tuberous sclerosis complex (TSC) and visible facial angiofibromas

                  Human Tissue Study

                  • Principal Investigator: James Wheless, MD
                  • Use of extra tissue samples obtained during brain surgery to look for any changes in proteins and genes in the tissue
                  • Ages 4 to 13 years with epilepsy and receiving epilepsy surgery

                  Raise

                  • Principal Investigator: James Wheless, MD
                  • Evaluate the efficacy and safety of intravenous ganaxolone in status epilepticus
                  • 12 years of age with status epilepticus (SE)
                  • ClinicalTrials.gov identifier: NCT04391569

                  MONARCH (Stoke Therapeutics)

                  • Principal Investigator: James Wheless, MD
                  • Actively recruiting via a prescreen waitlist
                  • Open-label study investigating the safety of single and multiple ascending doses of antisense oligonucleotide STK-001 in children and adolescents with Dravet syndrome
                  • Ages 2 to 18 years with Dravet syndrome
                  • ClinicalTrials.gov identifier: NCT04442295

                  Swallowtail (Stoke Therapeutics)

                  • Principal Investigator: James Wheless, MD
                  • Extension study - enrolling by invitation
                  • An open-label extension study for patients with Dravet syndrome who previously participated in studies of STK-001 investigating the safety of single and multiple ascending doses
                  • Ages 2.5 years and older with Dravet syndrome
                  • ClinicalTrials.gov identifier: NCT04740476

                  SKIP 039

                  • Principal Investigator: James Wheless, MD
                  • Safety and efficacy extension study of cenobamate in pediatric subjects with partial-onset (focal) seizures
                  • Ages 2 to 18 years with partial-onset seizures
                  • ClinicalTrials.gov identifier: NCT05067634

                  SKIP 025

                  • Principal Investigator: James Wheless, MD
                  • Randomized, double-blind study to evaluate efficacy and safety of cenobamate adjunctive therapy in primary generalized tonic-clonic seizures
                  • Older than 18 years with primary generalized tonic-clonic seizures
                  • ClinicalTrials.gov identifier: NCT 03678753

                  Novel side positioner vest device trial

                  • Principal Investigator: Stephanie Einhaus, MD
                  • Efficacy and safety of a novel side vest positioner in treating and preventing human infant occipital plagiocephaly
                  • Healthy infants ages 1 to 10 months

                  Low-frequency TMS treatment for refractory complex partial epilepsy

                  • Principal Investigator: Shalini Narayana, PhD
                  • 5 years or older and diagnosed with refractory epilepsy with partial-onset seizures

                  Functional brain mapping using transcranial magnetic stimulation

                  • Principal Investigator: Shalini Narayana, PhD
                  • Healthy volunteers and patients with neurological and psychiatric disorders ages 3 to 17 years

                  Setmelanotide (RM-493) drug trial

                  • Principal Investigator: Ahlee Kim, MD
                  • Treatment of obesity and hyperphagia in patients diagnosed with Bardet Biedl Syndrome (BBS) or Alstrom Syndrome (AS) in ages 6 years or older

                  Setmelanotide (RM-493) extension drug trial

                  • Principal Investigator: Ahlee Kim, MD
                  • Long term extension trial of setmelanotide for patients who have completed a trial of setmelanotide for the treatment of obesity associated with genetic defects upstream of the MC4 receptor in the leptin-melanocortin pathway
                  • Must have completed participation in related setmelanotide study

                  Setmelanotide (RM-493) drug trial for rare genetic disorders of obesity

                  • Principal Investigator: Ahlee Kim, MD
                  • Ages 6 years and older with specific genotypes for rare genetic disorders of obesity

                  Post-operative pain management drug trial

                  • Principal Investigator: Lindsey Locke, MSN, CPNP-PC
                  • NSAIDS vs. opioids in post-operative pain management
                  • Ages 4 to 13 years with diagnosis of supracondylar humerus fracture needing percutaneous pinning
                  • ClinicalTrials.gov identifier: NCT04905563

                  Braces in adolescent idiopathic scoliosis (AIS)

                  • Principal Investigator: Denis Diangelo, PhD
                  • Evaluation of conventional strap tension, constant-tension units and a modified brace for AID
                  • Ages 5 to 17 years with a diagnosis of idiopathic scoliosis and a new or current brace patient

                  Complementary alternative medicine with idiopathic scoliosis

                  • Principal Investigator: Lindsey Locke, MSN, CPNP-PC
                  • Studying if the use of complementary alternative medicine improves patient outcomes in adolescents with idiopathic scoliosis undergoing posterior spinal fusion
                  • Ages 12 to 21 years with a diagnosis of adolescent idiopathic scoliosis and undergoing posterior spinal fusion

                  Scoliosis analog model for evaluation of bracing technology

                  • Principal Investigator: Denis Diangelo, PhD
                  • Ages 5 to 17 years diagnosed with idiopathic scoliosis

                  Intra-operative ketorolac drug trial

                  • Principal Investigator: Anthony Sheyn, MD
                  • Intra-operative ketorolac to decrease pain and narcotic use in immediate post-operative period following tonsillectomy or adeno-tonsillectomy
                  • Ages 2 to 18 years

                  Fibrin Sealant Grifols drug trial

                  • Principal Investigator: Max Langham, MD
                  • Evaluate the safety and efficacy of Fibrin Sealant Grifols as an adjunct to hemostasis during pediatric surgeries
                  • Elective open (non-laparoscopic) surgery for pelvic, abdominal or thoracic where a hemostatic agent is needed, under 18 years

                  Minimally invasive pilonidal excision trial

                  • Principal Investigator: Tim Jancelewicz, MD, MA, MS
                  • Multi-center, non-randomized controlled trial
                  • Patients with acute pilonidal abcess may also be included provided they undergo a procedure more extensive than simple incision and drainage.
                  • Ages 0 to 24 years
                  • ClinicalTrials.gov identifier: NCT03772873

                  Improving the clinical diagnosis of Hirschsprung Disease

                  • Principal Investigator: Ankush Gosain, MD, PhD

                  The Hirschsprung Disease Research Collaboration

                  • Principal Investigator: Ankush Gosain, MD, PhD
                  • ClinicalTrials.gov identifier: NCT004788712

                  Perioperative Recovery Protocol for Inflammatory Bowel Disease ENRICH Study

                  • Principal Investigator: Ankush Gosain, MD, PhD
                  • Assess the effectiveness and implementation of a perioperative enhanced recovery protocol for inflammatory bowel disease
                  • Aim to measure clinical outcomes of length of hospital stay, opioid use, surgical complications and post-discharge health care utilization
                  • Ages 10 to 17 years
                  • ClinicaTrials.gov identifier: NCT04060303

                  Contrast enhance voiding urosonograpy (ceVUS) trial

                  • ceVUS for diagnosis of vesicoureteral reflux in children
                  • Ages 0 to 17 years


                  Updated April 11, 2022