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New treatment option for Neuroblastoma
Official title Silmitasertib (CX-4945) in Combination With Chemotherapy for Relapsed Refractory Solid Tumors
ClinicalTrials.gov ID: NCT06541262
What this study is testing
What is Silmitasertib?
Silmitasertib is an investigational medicine, given as a pill taken by mouth, being studied as a potential treatment for neuroblastoma.
Also referred to as CX-4945.
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 drug, silmitasertib (a pill taken by mouth), in combination with FDA approved drugs for solid tumors. 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 30
You may be able to join if
- Age: Less than 30 years old at initial diagnosis
- Pathology All people must have a confirmed diagnosis of tumor type. Phase I: Relapsed/refractory solid tumors: Neuroblastoma, Ewing Sarcoma...
- Relapsed/refractory Neuroblastoma
- Relapsed/refractory Ewing sarcoma
- Tumor assessment: Disease assessment is required for eligibility and must be done after last dose of previous therapy and prior to first dose of...
You likely can't join if
- Investigational Drugs: people who are currently receiving another investigational drug are excluded from participation.
- Anti-cancer Agents: people who are currently receiving other anticancer agents are not eligible. people must have fully recovered from the...
- people who are currently receiving Vitamin K antagonists (warfarin).
- people who are currently receiving the class of lipid-lowering medications HMG-CoA reductase inhibitors (statins).
- Infection: people who have an uncontrolled infection are not eligible until the infection is judged to be well controlled in the opinion of the...
- people 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...
See the full eligibility criteria
Who can join
- Age: Less than 30 years old at initial diagnosis
- Pathology All people must have a confirmed diagnosis of tumor type. Phase I: Relapsed/refractory solid tumors: Neuroblastoma, Ewing Sarcoma, Osteosarcoma, Rhabdomyosarcoma, Liposarcoma Phase II:
- Relapsed/refractory Neuroblastoma
- Relapsed/refractory Ewing sarcoma
- Tumor assessment: Disease assessment is required for eligibility and must be done after last dose of previous therapy and prior to first dose of study drug.
- Disease Status: Relapsed/Refractory Neuroblastoma Relapsed disease defined as neuroblastoma that was previously in remission after standard therapy (at least 4 cycles of aggressive multi-drug induction chemotherapy...
- Any age with International Neuroblastoma Risk Group (INRG) Stage L2, MS, or M with MYCN amplification
- Age ≥ 547 days and INRG Stage M regardless of biologic features
- Any age initially diagnosed with INRG Stage L1 MYCN amplified NBL who have progressed to Stage M without systemic chemotherapy
- Age ≥ 547 days of age initially diagnosed with INRG Stage L1, L2, or MS who have progressed to Stage M without systemic chemotherapy Relapsed/refractory Sarcoma people that have relapsed following standard of care...
- Measurable or evaluable disease, including at least one of the following:
- Measurable tumor by CT or MRI
- MIBG or PET that is positive for disease
- Bone Marrow biopsy/aspirate that is positive for disease
- Timing from prior therapy: people must have fully recovered from the acute toxic effects of all prior anti- cancer therapy and be within the following timelines:
- Myelosuppressive chemotherapy: Must not have received within 2 weeks of enrollment onto this study.
- Small Molecule Inhibitors (anti-neoplastic agent): At least 2 weeks from the completion of therapy with a small molecule inhibitor.
- Immunotherapy: At least 4 weeks since the completion of any type of immunotherapy, e.g. tumor vaccines, CAR-T cells, anti-GD2 Monoclonal antibodies (ex. naxitamab, dinutuximab, etc.).
- Radiotherapy: At least 30 days since the last treatment except for radiation delivered with palliative intent to a non-target site.
- Stem Cell Transplant:
- Allogeneic: No evidence of active graft vs. host disease
- Allogeneic/Autologous: ≥ 2 months must have elapsed since transplant.
- MIBG Therapy: At least 6 weeks since treatment with MIBG therapy.
- people must have a Lansky or Karnofsky Performance Scale score of \>/= 50.
- people must have adequate organ function at the time of enrollment:
- Cardiac: people must have a QTcF ≤ 480 msc.
- Hematological: Hematological recovery as defined by ANC ≥750/μL
- Liver: Adequate liver function as defined by AST and ALT \<5x upper limit of normal
- Renal: people must have adequate renal function defined as:
- estimated Glomerular Filtration rate (eGFR) as calculated from the Bedside Schwartz equation (for people \< 17 years old) (in units of mL/min/1.73 m2) or via radioisotope GFR of ≥ 70 mL/min/1.73 m2. The Bedside Schwartz...
- estimated Glomerular Filtration rate (eGFR) as calculated from the Cockcroft and Gault formula (for people ≥17 years old (in units of mL/min/1.73 m2) or via radioisotope GFR of ≥ 70 mL/min/1.73 m2. The Cockcroft and...
- OR a 24 hour urine Creatinine clearance ≥ 70 mL/min/1.73 m2
- people of childbearing potential must have a negative serum pregnancy test. people of childbearing potential must agree to use effective measures to avoid pregnancy.
- Written informed consent in accordance with institutional and FDA guidelines must be obtained from all people (or people' legal representative).
What rules you out
- Investigational Drugs: people who are currently receiving another investigational drug are excluded from participation.
- Anti-cancer Agents: people who are currently receiving other anticancer agents are not eligible. people must have fully recovered from the hematological and bone marrow suppression effects of prior therapy.
- people who are currently receiving Vitamin K antagonists (warfarin).
- people who are currently receiving the class of lipid-lowering medications HMG-CoA reductase inhibitors (statins).
- Infection: people who have an uncontrolled infection are not eligible until the infection is judged to be well controlled in the opinion of the investigator.
- people 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.
- people with any clinically significant unrelated systemic illness (serious infections or significant cardiac, pulmonary, hepatic or other organ dysfunction), that in the opinion of the investigator would compromise the...
- people with any of the following gastrointestinal disorders:
- Active malabsorption (e.g. short gut) syndrome.
- Uncontrolled diarrhea (excess of 4 stools/day)
- Gastritis, ulcerative colitis, Chron's disease or hemorrhagic coloproctitis
- History of gastric or small bowel surgery involving any extent of gastric or small bowel resection
- Lactating people are not eligible unless they have agreed to not breastfeed their infants. There is an unknown but potential risk for adverse events in nursing infants secondary to treatment of the nursing subject with...
- people with a history of any other malignancy.
The study team makes the final eligibility decision.
Where it's taking place
- Birmingham, Alabama, United States
- Phoenix, Arizona, United States
- Oakland, California, United States
- San Diego, California, United States
- Hartford, Connecticut, United States
- Gainesville, Florida, United States
- Miami, Florida, United States
- Orlando, Florida, United States
- St. Petersburg, Florida, United States
- Tampa, Florida, United States
- Honolulu, Hawaii, United States
- Louisville, Kentucky, United States
- Kansas City, Missouri, United States
- St Louis, Missouri, United States
- Hackensack, New Jersey, United States
- Hershey, Pennsylvania, United States
- Providence, Rhode Island, United States
- Nashville, Tennessee, United States
- Dallas, Texas, United States
- Salt Lake City, Utah, United States
+ 2 more site(s).
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 30 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Birmingham, Alabama, United States; Phoenix, Arizona, United States; Oakland, California, United States; San Diego, California, United States; Hartford, Connecticut, United States; Gainesville, Florida, United States and 16 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.