A Phase I/II Study Using Eflornithine (DFMO) and AMXT 1501 for Relapsed and Refractory Neuroblastoma, CNS Tumors, and Sarcomas
Milton S. Hershey Medical Center
Summary
The purpose of this study is to evaluate the investigational oral drug AMXT 1501 in combination with oral eflornithine (DFMO). An investigational drug is one that has not been approved by the U.S. Food \& Drug Administration (FDA), or any other regulatory authorities around the world for use alone or in combination with any drug, for the condition or illness it is being used to treat. The goals of this part of the study are: * Establish a recommended dose of AMXT 1501 in combination with DFMO * Test the safety and tolerability of AMXT 1501 in combination with DFMO * To determine the activity of study treatments chosen based on: * How each subject responds to the study treatment * How long a subject lives without their disease returning/progressing
Eligibility
- Age range
- Up to 26 years
- Sex
- All
- Healthy volunteers
- No
Inclusion Criteria: 1. Age: All participants : Must be a maximum of 26 years of age at diagnosis Age at enrollment by Phase: 1. Safety Run-in (Dose level 1)-The first three (3) participants enrolled will be ≥ 12 years of age at enrollment. Once evaluated for safety by DSMB, we will move on to the next three (3) participants enrolled who will be ≥6 years of age at enrollment. Once evaluated for safety by DSMB, we will move on to the Phase I. 2. Phase I and II: ≤ 26 years of age at diagnosis. 2. Pathology All participants must have a confirmed pathologic diagnosis of tumor typ…
Interventions
- DrugEflornithine (DFMO)
Oral DFMO capsules
- DrugAMXT 1501 Dicaprate
Capsule
Locations (12)
- University of Alabama/Children's of AlabamaBirmingham, Alabama
- Arkansas Children's HospitalLittle Rock, Arkansas
- Connecticut Children's HospitalHartford, Connecticut
- Nicklaus Children's HospitalMiami, Florida
- Arnold Palmer Hospital for ChildrenOrlando, Florida
- St. Joseph's Children's HospitalTampa, Florida