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
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.
Phase I and II: ≤ 26 years of age at diagnosis.
Pathology
Relapsed/refractory Neuroblastoma (NB)
BSA of <0.25 m2
Investigational Drugs: Participants who are currently receiving another investigational drug are excluded from participation.
Anti-cancer Agents: Participants who are currently receiving other anticancer agents are not eligible. Participants must have fully recovered from the hematological and bone marrow suppression effects of prior chemotherapy.
Infection: Participants who have an uncontrolled infection are not eligible until the infection is judged to be well controlled in the opinion of the investigator.
Crossover
Eflornithine (DFMO)
DrugOral DFMO capsules
AMXT 1501 Dicaprate
DrugCapsule
Phase I- Number of Participants with Adverse Events as a Measure of Safety and Tolerability
To evaluate the safety, tolerability and recommended phase 2 dose (RP2D) of AMXT 1501 in combination with oral DFMO in pediatric and young adult participants.
Phase II- Number of Cohort 1 participants with progression free survival (PFS) during study
To evaluate, in a prospective randomized clinical trial, the efficacy of eflornithine (DFMO) in combination with AMXT 1501 compared to DFMO alone in neuroblastoma (Cohort 1) based upon Progression Free Survival (PFS)
Phase II- Number of Cohort 2-4 participants with progression free survival (PFS) during study
To evaluate the efficacy of eflornithine (DFMO) in combination with AMXT 1501 in non-randomized (Cohorts 2-4) based upon Progression Free Survival (PFS): 2\. Cohort 2-Relapsed/refractory Embryonal Tumor with Multilayered Rosettes (ETMR) Atypical Teratoid Rhabdoid Tumor (ATRT) 3. Cohort 3-Diffuse Intrinsic Pontine Glioma (DIPG) at diagnosis after standard of care radiation therapy 4. Cohort 4- Relapsed/refractory Ewing Sarcoma (EWS) and Osteosarcoma (OST)
Phase I- Number of participants with progression free survival (PFS) during study
To evaluate the activity of AMXT 1501 in combination with DFMO based on Progression free survival (PFS)
Phase I- Determine the Overall Response Rate (ORR) of Participants using INSS Response
To evaluate the activity of AMXT 1501 in combination with DFMO based on Overall response rate (ORR)
Phase II- Determine the Overall Response Rate (ORR) of Participants using INSS Response
To evaluate the efficacy of eflornithine (DFMO) in combination with AMXT 1501 (Cohort 2-4) and in a randomized arm compared to DFMO alone in neuroblastoma (Cohort 1), based upon overall response rate (ORR).
Phase II- Length of time that participants experience Overall Survival (OS)
To evaluate the efficacy of eflornithine (DFMO) in combination with AMXT 1501 (Cohort 2-4) and in a randomized arm compared to DFMO alone in neuroblastoma (Cohort 1), based upon overall Survival (OS).
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Milton S. Hershey Medical Center
Lead sponsor
Aminex Therapeutics, Inc.
Collaborator
Penn State University
Collaborator
