Investigator-Initiated Trials

Some clinical studies are investigator-initiated trials (IITs). This type of clinical research is designed and led by a medical researcher or clinician. IITs often explore new uses for existing treatments, compare different treatments or investigate rare diseases. Nationwide Children’s is passionate about making a difference in the lives of children not only here in Columbus, Ohio, but across the country and around the world.

Child with cancer who has lost his hair but is still smiling.

Investigator-Initiated Cancer Clinical Studies

Nationwide Children’s acknowledges the significant role research has in improving child health, with a focus on translating discoveries into best outcomes for all kids. That’s why Nationwide Children’s is a national leader in cancer research. In addition to participating in national and international cancer studies, Nationwide Children’s supports investigator-initiated cancer clinical trials. IITs provide physician researchers new opportunities to study and identify new ways to use existing treatments. And for the rare diseases and cancers that affect children, this cutting-edge research often provides the next option for families who need it most.

Open Investigator-Initiated Trials

Central Nervous System (CNS) Tumors

Phase I/II Study of Lutathera in Pediatric and Young Adult Patients with Recurrent and/or Progressive CNS Tumors which Express Somatostatin Type 2A Receptors and Demonstrate Uptake on DOTATATE PET

In this research study, we want to find out if the study drug Lutathera, an IV infusion drug, is effective in treating different kinds of brain cancer.

Gliomas

Researchers Studying Oral Medicine to Treat Newly Diagnosed High-Grade Glioma With NTRK Fusion

Help researchers learn more about the effects of a study drug on children with brain tumors.

Researchers Studying Two Oral Drugs Given After Radiotherapy to Patients Just Diagnosed With High-Grade Glioma (HGG), including DIPG

The goal of this study is to determine the efficacy of the study drugs ribociclib and everolimus to treat pediatric and young adult patients newly diagnosed with a high-grade glioma (HGG), including DIPG, that have genetic changes in pathways (cell cycle, PI3K/mTOR) that these drugs target.

Targeted Pediatric High-Grade Glioma Therapy

The study aims to analyze the genes in brain tumors from kids, teens, and young adults who have just been diagnosed with a severe type of brain cancer.

Hematology

Registry and DNA Biorepository for Patients With Hereditary Antithrombin Deficiency

The goal of this study is to learn more about the natural history of hereditary antithrombin deficiency and to analyze genetic defects that can cause antithrombin deficiency and correlate with the natural history of the disease.

Young Women’s Health Study

We are testing the ability of quick tools (bleeding symptom survey and/or ferritin levels) to predict an underlying diagnosis of von Willebrand Disease in teens with HMB.

Hematopoietic Stem Cell Transplantation

How to Use a Medical Device That Helps Prepare Stem Cells for Transplant to Treat Patients and Prevent Graft vs Host Disease (GvHD) During the Transplant Process

The purpose of the study is to study the CliniMACS® CD34 Reagent System.  It's designed to prevent a serious complication called Graft vs Host Disease (GvHD), which can occur after a type of stem cell transplant known as allogeneic hematopoietic stem cell transplantation (HSCT).

 

TB19DHCT: Depletion of αβT Cells and CD19+ B Cells in Alternative Donor Stem Cell Transplant for Children and Young Adults with Blood Cancers

Help researchers study the one-year overall survival of patients undergoing allogeneic HSCT using the T-Cell Receptor (TCR) αβ/CD19+ depleted platform and grafts from alternative donors (MUD, MMUD and haploidentical).

How the Recovery of Certain Immune Cells Affects Virus Infections in Kids After a Stem Cell Transplant

This study will use immune profiling to identify allo-HSCT patients at highest risk for developing HAdV end organ disease.

 

Infectious Diseases

An Initial Study Where Doctors Use Specific Immune Cells From Partially Matched Donors to Treat Severe Adenovirus Infections in Hospitalized Patients

This study will test the safety and effectiveness of using specific immune cells from related donors to treat adenovirus infections.

Leukemia/Lymphoma

Collecting Bone Marrow and Blood for Possible Enrollment in Leukemia and Lymphoma Research Studies

The purpose of the study is to collect additional bone marrow and peripheral blood prior to diagnosis of new or recurrent leukemia/lymphoma.

Neuroblastoma

Researchers Studying Universal Donor NK Cells in Combination with IV Drug Therapy in Patients With Relapsed or Refractory Neuroblastoma

Help researchers study the safety, tolerability, and efficacy of a fixed dose of UD TGFβi NK cells in combination with standard irinotecan, temozolomide, and dinutuximab IV therapy.

Sarcoma

Researchers Studying Donor NK Cell Infusions Combined With IV Medications in Patients With Relapsed or Refractory Pediatric Sarcomas

Help researchers determine if adding infusions of a type of immune cell called a “natural killer,” or NK cell to the sarcoma chemotherapy regimen GEM/DOX (gemcitabine and docetaxel) is safe and can improve outcomes in people with childhood sarcomas that have relapsed or not responded to prior therapies.

Solid Tumors

A Phase 2 Study of Oral Medication Cabozantinib to Help Maintain Treatment in Children With High-Risk Solid Tumors

This study will look at how cabozantinib works on more types of pediatric cancers, including neuroblastomas, which are very serious and have few treatment options.

von Willebrand Disease

Young Women’s Health Study

We are testing the ability of quick tools (bleeding symptom survey and/or ferritin levels) to predict an underlying diagnosis of von Willebrand Disease in teens with HMB.