Actively Recruiting
A Dose Escalation Study Using Eflornithine (DFMO) and AMXT 1501 Followed by a Randomized Controlled Trial of DFMO With or Without AMXT 1501 for Neuroblastoma, CNS Tumors, and Sarcomas
Led by Milton S. Hershey Medical Center · Updated on 2026-06-03
289
Participants Needed
10
Research Sites
104 weeks
Total Duration
On this page
Sponsors
M
Milton S. Hershey Medical Center
Lead Sponsor
A
Aminex Therapeutics, Inc.
Collaborating Sponsor
AI-Summary
What this Trial Is About
Researchers are evaluating the investigational oral drug AMXT 1501 combined with oral eflornithine (DFMO) in children and young adults with certain cancers including neuroblastoma, central nervous system tumors, and sarcomas. This study aims to establish the recommended dose of AMXT 1501 with DFMO, assess their safety and tolerability, and determine how well these treatments work based on patient response and progression-free survival. The study includes both phase 1 dose escalation and phase 2 randomized components to better understand these treatments' effects. Participants are divided into groups based on age and tumor type. Initial phase 1 includes adolescents and young adults aged 12 or older, and a pediatric cohort under 12, both receiving up to twenty-four 28-day cycles of AMXT 1501 combined with DFMO starting at a dose of 350 mg/m2 twice daily. In phase 2, one group receives the combination at the recommended phase 2 dose, while another group receives DFMO alone. Certain tumor cohorts are assigned to the combination treatment. Patients in the DFMO alone group who experience disease progression may switch to the combination therapy. Throughout the study, participants undergo tumor assessments, safety monitoring, and evaluations of treatment response and survival. Researchers will track adverse events during treatment cycles and assess progression-free survival over two years with an additional five years of follow-up. Participants' ability to swallow capsules and stable steroid use if applicable are required. The total study duration includes up to twenty-four treatment cycles, safety monitoring, and long-term follow-up to gather comprehensive data on treatment effects and tolerability.
CONDITIONS
Brief Title
Eflornithine (DFMO) and AMXT 1501 for Neuroblastoma, CNS Tumors, and Sarcomas
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Must be 21 years old or younger at diagnosis
- Phase I AYA cohort: 12 years or older at enrollment
- Phase I pediatric cohort: younger than 12 years at enrollment
- Confirmed diagnosis of relapsed/refractory neuroblastoma, ETMR, ATRT, newly diagnosed DIPG (radiologic diagnosis accepted), relapsed/refractory Ewing sarcoma, or osteosarcoma
- Disease staging completed during the 28-day screening period before first dose
- Relapsed or refractory disease as defined for each tumor type, with stable or better disease status if active
- Ability to swallow capsules
- Stable steroid dose for CNS disease patients for at least one week
- Fully recovered from prior chemotherapy and other treatments within specified timeframes
- Lansky or Karnofsky Performance Scale score of 60 or higher
- Adequate organ function including hematological, liver, cardiac, and renal criteria
- Negative pregnancy test and agreement to use birth control for subjects of childbearing potential
- Written informed consent obtained
You will not qualify if you...
- Body surface area less than 0.25 m2
- Currently receiving another investigational drug
- Currently receiving other anticancer agents
- Uncontrolled infection
- Inability or unlikely compliance with safety monitoring requirements
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Up to twenty-four 28-day cycles
Participants receive oral doses of AMXT 1501 combined with DFMO or DFMO alone in up to twenty-four 28-day cycles depending on their cohort and randomization. Subjects may cross over to combination treatment if disease progresses on DFMO alone and criteria are met.
Repeated visits each cycle for up to 24 cycles
Duration - Up to 5 years after treatment
Participants are monitored for progression-free survival, overall survival, and safety after treatment ends, with follow-up lasting up to 5 years.
Periodic visits during follow-up
Trial Site Locations
Total: 10 locations
1
University of Alabama/Children's of Alabama
Birmingham, Alabama, United States, 35233
Actively Recruiting
2
Arkansas Children's Hospital
Little Rock, Arkansas, United States, 72202
Actively Recruiting
3
Connecticut Children's Hospital
Hartford, Connecticut, United States, 06106
Actively Recruiting
4
Nicklaus Children's Hospital
Miami, Florida, United States, 33155
Actively Recruiting
5
Arnold Palmer Hospital for Children
Orlando, Florida, United States, 32806
Actively Recruiting
6
St. Joseph's Children's Hospital
Tampa, Florida, United States, 33614
Actively Recruiting
7
Kapiolani Medical Center for Women and Children
Honolulu, Hawaii, United States, 96813
Actively Recruiting
8
Penn State Milton S. Hershey Medical Center and Children's Hospital
Hershey, Pennsylvania, United States, 17033
Actively Recruiting
9
Monroe Carrell Jr. Children's Hospital at Vanderbilt
Nashville, Tennessee, United States, 37232
Actively Recruiting
10
Children's Medical Center
Dallas, Texas, United States, 75235
Actively Recruiting
Research Team
B
BCC Enroll
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
RANDOMIZED
Model
CROSSOVER
Primary Purpose
TREATMENT
Number of Arms
4
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