Study of Oral AMXT 1501 Combined with Eflornithine DFMO for Treating Neuroblastoma, CNS Tumors, and Sarcomas in Patients Up to 26 Years Old

What this Trial Is About

Researchers are evaluating the investigational oral drug AMXT 1501 combined with oral eflornithine DFMO to treat relapsed and refractory neuroblastoma, central nervous system tumors, and sarcomas in participants up to 26 years old. The study aims to find a safe and tolerable dose of AMXT 1501 with DFMO, assess the activity of these treatments based on participant responses, and measure how long participants live without disease progression. The study begins with a safety run-in phase involving six participants divided by age groups, followed by a Phase I dose-escalation stage using a 33 design where participants receive up to twenty-four 28-day cycles of AMXT 1501 and DFMO. In Phase II, cohort 1 participants are randomized to receive either both drugs at the recommended Phase II dose or DFMO alone, while other cohorts automatically receive the combination. Participants may cross over to combination therapy if disease progresses on DFMO alone. Participants will undergo disease staging during a 28-day screening before treatment. Study involvement includes oral treatment cycles, safety and tolerability assessments, and monitoring progression-free survival and overall response rates over up to 2 years with an additional 5 years follow-up. Safety monitoring covers adverse events and organ function tests. The total study duration may extend until 2035, with participants monitored closely throughout treatment and follow-up periods.

Eflornithine (DFMO) and AMXT 1501 for Neuroblastoma, CNS Tumors, and Sarcomas