Center for Pediatric Research
Clinical Trials
Natural History of Wilson Disease: Registry for Patients with Wilson Disease
This study is currently enrolling.
You are invited to donate your blood, urine, research data, and related information to be stored (banked) in a research specimen/data database or bank where it can be used for future research projects. The specimens/data collec ...
STARZL NETWORK FOR EXCELLENCE IN PEDIATRIC TRANSPLANTATION (snept) to improve the health of pediatric LIVER TRANSPLANT PATIENTS
This study is currently enrolling.
Pediatric liver transplantation has become the standard of care for many children with liver diseases. However, different institutions often treat and manage children with liver transplant in different ways. Currently, there ar ...
A Feasibility and Randomized Phase 2/3 Study of the VEFGR2/MET Inhibitor Cabozantinib in Combination with Cytotoxic Chemotherapy for Newly Diagnosed Osteosarcoma
This study looks at how well cabozantinib in combination with MAP work when given to children and young adults with newly diagnosed OST disease. Cabozantinib is experimental because it has not been proven to work in a situation ...
A multicenter access and distribution protocol for unlicensed cryopreserved cord blood units (CBUs) for transplantation in pediatric and adult patients with hematologic malignancies and other indications
The primary purposes of this study are to: (a) Provide access to cord blood units for recipients whose best choice for a cord blood unit(s) do not meet all FDA standards, but do meet standards set by the NMDP on this study; and ...
A Phase 1, open-label, non-comparative, multicenter clinical study to evaluate the safety, tolerability, and pharmacokinetics of ceftolozane/tazobactam (MK-7625A) in pediatric participants with nosocomial pneumonia
Ceftolozane/tazobactam, also known as Zerbaxa, is a combination product, and is available by prescription for use in adults for the treatment of complicated urinary tract infections, including pyelonephritis (serious infection ...
A Phase 2 Study of Reduced Therapy for Newly Diagnosed Average-Risk WNT-Driven Medulloblastoma Patients
The overall goal of this study is to find out if a reduced dose of radiation to the whole brain and spine and less chemotherapy can be given to children and young adults with WNT subtype medulloblastoma without increasing the r ...
A Phase 2 Study of the JAK1/JAK2 Inhibitor Ruxolitinib with Chemotherapy in Children with De Novo High-Risk CRLF2-Rearranged and/or JAK Pathway-Mutant Acute Lymphoblastic Leukemia
The overall goal of this study are to: Part 1 - Find the best dose of ruxolitinib to use with standard chemotherapy in children, adolescents, and young adults with HR B-ALL; Part 2 - Find out if the investigational drug ruxolit ...
A Phase 2/3, Multicenter, Randomized, Double-blind Study to Evaluate the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of Oral Ozanimod (RPC1063) in Pediatric Participants with Moderately to Severely Active Crohn’s Disease with an Inadequate Response to Conventional Therapy
The purpose of this study is to see whether ozanimod (RPC1063, also referred to as the ‘study drug’ or ‘study treatment’) is safe and effective for the possible treatment of Crohn’s Disease (CD) in a pediatric population. To do ...
A Phase 3 Randomized Non-Inferiority Study of Carboplatin and Vincristine versus Selumetinib (NSC# 748727, IND# 77782) in Newly Diagnosed or Previously Untreated Low-Grade Glioma (LGG) not associated with BRAFV600E Mutations or Systemic Neurofibromatosis Type 1 (NF1)
This study looks at how well a drug called selumetinib works when given to children and young adults with LGG. The study doctors want to see if selumetinib works as well as CV for getting rid of or shrinking LGGs and stopping t ...