Active Clinical Trials at Norton Children’s

Learn more about participating in a clinical trial.

Active clinical trials at Norton Children’s and the UofL School of Medicine include:

A Phase 1, Open-Label, Dose-Finding Study to Evaluate Safety, Tolerability, and Immunogenicity and Phase 2/3 Placebo-Controlled, Observer-Blinded Safety, Tolerability, and Immunogenicity Study of A SARS-COV-2 RNA Vaccine Candidate Against Covid-19 in Heal

Acute Lymphoblastic Leukemia (ALL)/Lymphoblastic L

A Randomized Web-based Physical Activity Intervention among Children and Adolescents with Acute Lymphoblastic Leukemia

Acute Lymphoblastic Leukemia (ALL)/Lymphoblastic L

An Open-Label, Multicenter Study of RC-P in Patients with Acute Lymphoblastic Leukemia (ALL)/Lymphoblastic Lymphoma (LBL) Following Hypersensitivity to E. coli-derived Asparaginases

Acute Myeloid Leukemia (AML)

A Phase 1/2 Study of CPX-351 (NSC# 775341; IND #129443) Alone Followed by Fludarabine, Cytarabine, and G-CSF (FLAG) for Children with Relapsed Acute Myeloid Leukemia (AML)

Asthma

IAA – Vitamin D Supplementation in Children with Obesity Related Asthma (VDORA1)

Autism

Treatment of children with autism spectrum disorders and epileptiform EEG with divalproex sodium. (VPA Autism Barnes 2013)

B-Acute Lymphoblastic Leukemia (B-ALL)

A Phase 2 Study of Inotuzumab Ozogamicin (NSC# 772518, IND#133494) in Children and Young Adults with Relapsed or Refractory CD22+ B-Acute Lymphoblastic Leukemia (B-ALL)

B-Acute Lymphoblastic Leukemia (B-ALL)

A Phase 3 Trial Investigating Blinatumomab (IND# 117467, NSC# 765986) in Combination with Chemotherapy in Patients with Newly Diagnosed Standard Risk or Down syndrome B-Lymphoblastic Leukemia (B-ALL) and the Treatment of Patients with Localized B-Lymphobl

B-Acute Lymphoblastic Leukemia (B-ALL)

AALL1732: A Phase 3 Randomized Trial of Inotuzumab Ozogamicin (IND#:133494, NSC#: 772518) for Newly Diagnosed High-Risk B-ALL; Risk-Adapted Post-Induction Therapy for High-Risk B-ALL, Mixed Phenotype Acute Leukemia, and Disseminated B-LLy

Bacterial Infection

An Open label, Dose-finding, Pharmacokinetics, Safety, and Tolerability Study of Oritavancin Single Dose Infusion in Pediatric Subjects Less Than 18 Years of Age with Suspected or Confirmed Bacterial Infections

Bacterial Infection

A Phase 1, Open-Label, Two-Part Study to Evaluate the Pharmacokinetics, Safety, and Tolerability of a Single Dose of Intravenous Lefamulin in Hospitalized Pediatric Subjects Receiving Standard of Care Antibiotic Therapy for Confirmed or Suspected Infectio

Bronchiolitis

EFFICACY OF MAGNESIUM SULFATE BOLUS IN PEDIATRIC PATIENTS WITH BRONCHIOLITIS

Catecholaminergic Polymorphic Ventricular Tachycar

Improving Diagnosis and Treatment in CPVT

Catheter Ablation

Catheter Ablation with Reduction or Elimination of Fluoroscopy (CAREFL)—A Multi-center Registry

Central Nervous System Germ Cell Tumors (CNS GCT)

Phase 2 Trial of Response-Based Radiation Therapy for Patients with Localized Central Nervous System Germ Cell Tumors (CNS GCT)

Classical Hodgkin Lymphoma (cHL)

An Open-label, Uncontrolled, Multicenter Phase II Trial of MK-3475 (Pembrolizumab) in Children and Young Adults with Newly Diagnosed Classical Hodgkin Lymphoma with Inadequate (Slow Early) Response to Frontline Chemotherapy (KEYNOTE 667).

Congenital Heart Disease

Advanced Cardiac Therapies Improving Outcomes Network to Improve the Health of Pediatric and Congenital Heart Disease Patients with Heart Failure

Cystic Fibrosis

A Master Protocol to Test the Impact of Discontinuing Chronic Therapies in People With Cystic Fibrosis on Highly Effective CFTR Modulator Therapy (SIMPLIFY)

Data Collection

University of Louisville Kosair Charities Pediatric Clinical Research Unit Research Information for Subjects

Epilepsy

A double-blind, randomized, placebo-controlled study to evaluate the efficacy and safety of intravenous ganaxolone in status epilepticus

Epilepsy and Autism

Safety and Tolerability of GWP42006 in Subjects with Drug Resistant Epilepsy and Autism—Extension Safety Study

Genomics

NCI-COG PEDIATRIC MATCH (MOLECULAR ANALYSIS FOR THERAPY CHOICE) MASTER VERSION CONTROL PROTOCOL

Germ Cell Tumors

A randomised phase 3 trial of accelerated versus standard BEP chemotherapy for patients with intermediate and poor-risk metastatic germ cell tumours

Heart Failure

Pharmacologic Reversal of Ventricular Remodeling in Childhood Cancer Survivors at Risk for Heart Failure (PREVENT-HF): A Phase 2b Randomized Placebo-Controlled (Carvedilol) Trial

Hepatic Malignancy

Pediatric Hepatic Malignancy International Therapeutic Trial (PHITT)

Hepatitis C Virus (HCV)

A Long Term Follow-up Registry for Adolescent and Pediatric Subjects Who Received a Gilead Hepatitis C Virus Direct Acting Antiviral (DAA) in Gilead-Sponsored Chronic Hepatitis C Infection Trials

High-Grade Glioma

A Phase 2 Study of Dabrafenib (NSC# 763760) with Trametinib (NSC# 763093) after Local Irradiation in Newly Diagnosed BRAFV600-Mutant High-Grade Glioma (HGG) (IND #145355)

Intermediate Risk (IR) Rhabdomyosarcoma (RMS)

COG ARST1431 A Randomized Phase 3 Study of Vincristine, Dactinomycin, Cyclophosphamide (VAC) Alternating with Vincristine and Irinotecan (VI) Versus VAC/VI Plus Temsirolimus (TORI, Torisel, NSC# 683864, IND# 122782) in Patients with Intermediate Risk (IR)

Intravascular

Bedside Ultrasound for Assessment of Intravascular Volume Status in Critically Ill Pediatric Patients

Invasive Aspergillosis or Invasive Mucormycosis

A Phase 2, Open-Label, Non-Comparative, Multicenter Study to Evaluate the Safety and Tolerability, Efficacy and Pharmacokinetics of Isavuconazonium Sulfate for the Treatment of Invasive Aspergillosis (IA) or Invasive Mucormycosis (IM) in Pediatric Subject

Jaundice

A phase 2B, mulit-center, single dose, blinded, randomized, placebo-controlled, dose escalation, safety and efficacy trial of Stannsoporfin in neonates with Hyperbilirubinemia.

Juvenile Arthritis

OBSERVATIONAL STUDY OF PEDIATRIC RHEUMATIC DISEASES:THE CARRA REGISTRY

Juvenile Idiopathic Arthritis (JIA)

A Randomized, Open Label, Multi-Center, Phase 3 Efficacy Study of Sub-Q Abatacept (Orencia) in Preventing Extension of Oligoarticular Juvenile Idiopathic Arthritis JIA (LIMIT-JIA)

Langerhans Cell Histiocytosis

International Collaborative Treatment Protocol For Children And Adolescents With Langerhans Cell Histiocytosis

Leukemia

Risk-stratified Therapy for Acute Myeloid Leukemia in Down Syndrome

Leukemia

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

Low-Grade Glioma (LGG)

A Phase 3 Randomized Study of Selumetinib (IND # 77782) versus Carboplatin/Vincristine in Newly Diagnosed or Previously Untreated Neurofibromatosis Type 1 (NF1) Associated Low-Grade Glioma (LGG)

Low-Grade Glioma (LGG)

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

Medulloblastoma

NEWLY DIAGNOSED CHILDREN (LESS THAN 10 YEARS OLD) WITH MEDULLOBLASTOMA AND OTHER CENTRAL NERVOUS SYSTEM EMBRYONAL TUMORS: CLINICAL AND MOLECULAR RISK-TAILORED INTENSIVE AND COMPRESSED INDUCTION CHEMOTHERAPY FOLLOWED BY CONSOLIDATION WITH EITHER SINGLE CYC

Medulloblastoma

Phase II Trial of Eflornithine/DFMO as Maintenance Therapy for Molecular High Risk/Very High Risk and Relapsed/Refractory Medulloblastoma

Multiple Sclerosis (MS)

An Open-Label, Parallel-Group Study To Evaluate Safety, Tolerability, Pharmacokinetics, And Pharmacodynamic Effects Of Ocrelizumab In Children And Adolescents With Relapsing-Remitting Multiple Sclerosis

Neuroblastoma

Utilizing Response and Biology Based Risk Factors to Guide Therapy in Patients with Non-High Risk Neuroblastoma

Neuroblastoma

PEDS-PLAN – Pediatric Precision Laboratory Advanced Neuroblastoma Therapy (A Study Using Molecular Guided Therapy with Induction Chemotherapy followed by a Randomized Controlled Trial of standard immunotherapy with or without DFMO followed by DFMO mainten

Neuroblastoma

A Phase II Preventative Trial of DFMO as a Single Agent in Patients with High Risk Neuroblastoma in Remission (NMTT- Neuroblastoma Maintenance Therapy Trial Using Difluoromethylornithine (DFMO))

Neuroblastoma

A Phase 2 Randomized Study of Irinotecan/Temozolomide/Dinutuximab with or without Eflornithine (DFMO) (IND# 141913) in Children with Relapsed, Refractory or Progressive Neuroblastoma

Neurofibromatosis Type 1 (NF1)

A Phase 2b Trial of the MEK 1/2 Inhibitor (MEKi) PD-0325901 in Adult and Pediatric Patients with Neurofibromatosis Type 1 (NF1)-Associated Inoperable Plexiform Neurofibromas (PNs) that are Causing Significant Morbidity

Neuromuscular Disorders

Muscular Dystrophy Association Neuromuscular Observational Research (MOVR) Data Hub Protocol

Pediatric Peritoneal Dialysis

North American Pediatric Renal Transplant Cooperative Study (NAPTRCS)

Philadelphia Chromosome Ph+ALL

International Phase 3 trial in Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ALL) testing imatinib in combination with two different cytotoxic chemotherapy backbones.

Respiratory Distress Syndrome (RDS)

A Partially-Blind, Randomized, Controlled, Parallel-Group Dose Ranging Study to Determine the Efficacy, Safety and Tolerability of AeroFactTM (SF-RI 1 surfactant for inhalation combined with a dedicated drug delivery system) in Preterm Infants at Risk of

Respiratory Syncytial Virus (RSV)

A Phase 2/3 Randomized, Double-blind, Palivizumab-controlled Study to Evaluate the Safety of MEDI8897, a Monoclonal Antibody With an Extended Half-life Against Respiratory Syncytial Virus, in High-risk Children (MEDLEY)

Sepsis

The NICU Antibiotics and Outcomes Trial (NANO)

Septic Shock

Stress Hydrocortisone in Pediatric Septic Shock (SHIPSS)

Solid Tumors

A5481092 PHASE 1/2 STUDY TO EVALUATE PALBOCICLIB(IBRANCE®) IN COMBINATION WITH IRINOTECAN AND TEMOZOLOMIDE AND/OR IN COMBINATION WITH TOPOTECAN AND CYCLOPHOSPHAMIDE IN PEDIATRIC PATIENTS WITH RECURRENT OR REFRACTORY SOLID TUMORS

Solid Tumors

A Phase 1/2 Study of Lenvatinib in Combination With Everolimus in Recurrent and Refractory Pediatric Solid Tumors, Including CNS Tumors

Spinal Muscular Atrophy (SMA)

A Prospective Long-Term Registry of Patients with a Diagnosis of Spinal Muscular Atrophy (SMA)

Supraventricular Tachycardia (SVT)

CARDIAC SOLAR Infant SVT Registry

Type 2 Diabetes (T2D)

Efficacy and safety of oral semaglutide versus placebo both in combination with metformin and/or basal insulin in children and adolescents with type 2 diabetes

Type 2 Diabetes Mellitus (T2DM)

A double-blind, randomized, placebo-controlled, parallel group trial to evaluate the efficacy and safety of empagliflozin and linagliptin over 26 weeks, with a double-blind active treatment safety extension period up to 52 weeks, in children and […]

Research

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