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New treatment option for Atypical Teratoid/Rhabdoid Tumor
Official title Eflornithine (DFMO) and AMXT 1501 for Neuroblastoma, CNS Tumors, and Sarcomas
ClinicalTrials.gov ID: NCT06465199
What this study is testing
What is Eflornithine (DFMO)?
Eflornithine (DFMO) is an investigational medicine, given as a pill taken by mouth, being studied as a potential treatment for atypical teratoid/rhabdoid tumor.
Also referred to as Difluoromethylornithine.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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.
- Phase 2: a mid-size study of how well it works
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages up to 26
You may be able to join if
- Age: All participants : Must be a maximum of 26 years of age at diagnosis Age at enrollment by Phase:
- 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...
- Phase I and II: ≤ 26 years of age at diagnosis.
- Pathology All participants must have a confirmed pathologic diagnosis of tumor type (except for DIPG):
- Relapsed/refractory Neuroblastoma (NB)
You likely can't join if
- 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...
- Infection: Participants who have an uncontrolled infection are not eligible until the infection is judged to be well controlled in the opinion of the...
- Participants who, in the opinion of the investigator, may not be able to comply with the safety monitoring requirements of the study, or in whom...
See the full eligibility criteria
Who can join
- Age: All participants : Must be a maximum of 26 years of age at diagnosis Age at enrollment by Phase:
- 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...
- Phase I and II: ≤ 26 years of age at diagnosis.
- Pathology All participants must have a confirmed pathologic diagnosis of tumor type (except for DIPG):
- Relapsed/refractory Neuroblastoma (NB)
- Relapsed/refractory Embryonal tumor with multilayer rosettes (ETMR)
- Relapsed/refractory Atypical teratoid rhabdoid tumor (ATRT)
- Newly diagnosed Diffuse Intrinsic Pontine Glioma (DIPG)- radiologic diagnosis acceptable
- Relapsed/refractory Ewing Sarcoma (EWS)
- Relapsed/refractory Osteosarcoma (OST)
- Tumor assessment: Disease staging must be performed at baseline during the 28 day screening period prior to first dose of study drug.
- Disease Status: Relapsed or Refractory Neuroblastoma Relapsed disease defined as: High-risk neuroblastoma that was previously in remission after standard therapy (at least 4 cycles of aggressive multi-drug induction...
- Received at least one recent treatment for their relapse/refractory disease and is stable (SD) or better on this treatment.
- Participants must not have disease in any organs (including lungs, liver, or brain). Relapsed or refractory ETMR/ATRT Participants that have relapsed following standard of care therapy or having progressed during...
- Received at least one recent treatment for their relapse/refractory disease and is stable (SD) or better on this treatment. Newly Diagnosed Diffuse Intrinsic Pontine Glioma (DIPG) Participants with DIPG to start greater...
- Received at least one recent treatment for their relapse/refractory disease and is stable (SD) or better on this treatment.
- Participants must be able to swallow capsules.
- Participants with CNS disease currently taking steroids must have been on a stable dose of steroids for at least one week and must not have progressive hydrocephalus at enrollment.
- Participants must have fully recovered from the acute toxic effects of all prior anti- cancer chemotherapy and be within the following timelines:
- Myelosuppressive chemotherapy: Must not have received within 2 weeks of enrollment onto this study (6 weeks if prior nitrosourea).
- Small Molecule Inhibitor (anti-neoplastic agent): At least 7 days since the completion of therapy with a small molecule inhibitor. For agents that have known adverse events occurring beyond 7 days after administration...
- Immunotherapy: At least 4 weeks since the completion of any type of immunotherapy, e.g. tumor vaccines, CAR-T cells except for anti-GD2 Monoclonal antibodies (ex. naxitamab, dinutuximab, etc.) which should be at least 2...
- XRT: At least 14 days since the last treatment except for radiation delivered with palliative intent to a non-target site. Note: Participants with DIPG will be required to have had up front standard of care radiation...
- Stem Cell Transplant:
- Allogeneic: No evidence of active graft vs. host disease
- Allo/Auto: ≥ 45 days must have elapsed since transplant.
- MIBG Therapy: At least 6 weeks since treatment with MIBG therapy.
- Participants must have a Lansky or Karnofsky Performance Scale score of \>/= 60
- Participants must have adequate organ function at the time of enrollment:
- Hematological: Hematological recovery as defined by ANC ≥750/μL (unsupported- \>24 hrs off G-CSF and 7 days off neulasta)
- Liver: Adequate liver function as defined by AST and ALT \<10x upper limit of normal
- Cardiac: all participants must have:
- Normal serum Cardiac Troponin Concentration
- Normal BNP (B-type natriuretic peptide) Level
- A QTcF ≤ 470 msec (or EKG with no significant findings)
- Normal ECHO defined as: i. Shortening fraction of ≥ 27% by echocardiogram, or ii. Ejection fraction of ≥ 50% by echocardiogram or radionuclide angiogram
- Renal: Participants must have adequate renal function defined as:
- For participants \< 17 years old: estimated Glomerular Filtration rate (eGFR) as calculated from the Bedside Schwartz equation (in units of mL/min/1.73 m2) or via radioisotope GFR of ≥ 70 mL/min/1.73 m2. The Bedside...
- For participants ≥17 years old: estimated Glomerular Filtration rate (eGFR) as calculated from the Cockcroft and Gault formula (in units of mL/min/1.73 m2) or via radioisotope GFR of ≥ 70 mL/min/1.73 m2. The Cockcroft...
- Participants of childbearing potential must have a negative pregnancy test. Participants of childbearing potential must agree to use an effective birth control method. Participants who are lactating must agree to stop...
- Written informed consent in accordance with institutional and FDA guidelines must be obtained from all participants (or participants' legal representative).
What rules you out
- 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...
- 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.
- Participants who, in the opinion of the investigator, may not be able to comply with the safety monitoring requirements of the study, or in whom compliance is likely to be suboptimal, should be excluded.
The study team makes the final eligibility decision.
Where it's taking place
- Birmingham, Alabama, United States
- Little Rock, Arkansas, United States
- Hartford, Connecticut, United States
- Miami, Florida, United States
- Orlando, Florida, United States
- Tampa, Florida, United States
- Honolulu, Hawaii, United States
- Kansas City, Kansas, United States
- St Louis, Missouri, United States
- Hershey, Pennsylvania, United States
- Nashville, Tennessee, United States
- Dallas, Texas, United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, up to 26 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Birmingham, Alabama, United States; Little Rock, Arkansas, United States; Hartford, Connecticut, United States; Miami, Florida, United States; Orlando, Florida, United States; Tampa, Florida, United States and 6 more location(s). Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.