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

Conditions

• Atypical Teratoid/Rhabdoid Tumor
• DIPG Brain Tumor
• Diffuse Intrinsic Pontine Glioma
• Embryonal Tumor With Multilayered Rosettes
• Ewing Sarcoma
• Neuroblastoma
• Osteosarcoma

Eligibility Criteria

Sex

ALL

Age

N/A - 26 Years

Healthy Volunteers

Not accepted

Interventions

• Eflornithine (DFMO) — DRUG
  Oral DFMO capsules
• AMXT 1501 Dicaprate — DRUG
  Capsule

Study Details

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

Key Dates

Start date

May 13, 2026

Status verified

Jun 2026

Primary completion

May 31, 2033

Completion

May 31, 2035

Study Design

Enrollment

289 participants (estimated)

Allocation

RANDOMIZED

Intervention model

CROSSOVER

Primary purpose

TREATMENT

Arms

• Experimental: Safety Run-in
  This study will include a safety run-in of 6 participants. The first 3 participants will be ≥ 12 years of age. The next 3 participants will be ≥ 6 years of age. The study will then move on to the Phase I.
• Experimental: Phase I
  Phase I will use a standard 3+3 design in which groups of 3 participants per dose level will be treated and assessed. Participants will receive up to twenty-four (24), 28-day cycles of AMXT 1501 combined with DFMO. Participants will receive oral AMXT 1501 at a starting dose of 350 mg/m2 BID each day. The dose escalation scheme for subsequent groups and modifications for dose limiting toxicities (DLT) are detailed in the protocol.
• Experimental: Phase II- Arm A: AMXT 1501 + DFMO
  In this portion of the study, cohort 1 will be randomized to either receive Arm A: oral AMXT 1501 at the recommended phase 2 dose (RP2D) found in the Phase I along with oral DFMO at the RP2D found in the Phase I on each day of study or Arm B: oral DFMO alone at the recommended phase 2 dose (RP2D) found in the Phase I. Participants will receive up to twenty-four (24), 28-day cycles of their assigned treatment. Cohorts 2 (ETMR/ATRT), 3 (DIPG), and 4 (Sarcomas) will automatically be assigned to Arm A with AMXT 1501 in combination with DFMO. Participants in cohort 1 who progress on DFMO alone (and have met the primary PFS endpoint) may cross over to AMXT 1501+DFMO.
• Active Comparator: Phase II- Arm B: DFMO Alone
  In this portion of the study, cohort 1 will be randomized to either receive Arm A: oral AMXT 1501 at the recommended phase 2 dose (RP2D) found in the Phase I along with oral DFMO at the RP2D found in the Phase I on each day of study or Arm B: oral DFMO alone at the recommended phase 2 dose (RP2D) found in the Phase I. Participants will receive up to twenty-four (24), 28-day cycles of their assigned treatment. Cohorts 2 (ETMR/ATRT), 3 (DIPG), and 4 (Sarcomas) will automatically be assigned to Arm A with AMXT 1501 in combination with DFMO. Participants in cohort 1 who progress on DFMO alone (and have met the primary PFS endpoint) may cross over to AMXT 1501+DFMO.

Primary Outcome Measure

Phase I- Number of Participants with Adverse Events as a Measure of Safety and Tolerability [ Time Frame: 28 days ]

Central Contacts

• BCC Enroll
  7175310003
  [email protected]

Locations (11)

Find similar trials in Birmingham, AL

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