BridgeMD
New treatment option for Neuroblastoma
Official title Tipifarnib and Naxitamab for Relapsed/Refractory Neuroblastoma
ClinicalTrials.gov ID: NCT06540963
What this study is testing
What is Tipifarnib?
Tipifarnib is an investigational medicine, given as an infusion into a vein, being studied as a potential treatment for neuroblastoma.
Also referred to as R115777.
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, tipifarnib (a pill taken by mouth), in combination with the Food and Drug Administration (FDA) approved drug, naxitimab, administered intravenously (IV; a liquid that continuously goes into your body through a tube that has been placed during a surgery into one of your veins). Naxitamab is FDA approved for pediatric patients 1 year of age and older and adult patients with relapsed or refractory high-risk neuroblastoma in the bone or bone marrow who have demonstrated a partial response, minor response, or stable disease to prior therapy, it may not be approved in the type of disease used in this study.
- 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 1 to 21
You may be able to join if
- Age: people must be age ≤ 21 years at initial diagnosis. people must be \>12 months of age at enrollment. Safety Run-In (first 6 people) must be age...
- Pathology: All people must have a pathologically confirmed diagnosis of neuroblastoma at any point in their treatment.
- Tumor assessment: Disease staging must be performed. This disease assessment is required for eligibility and must be done within a maximum of 4 weeks...
- Disease Status: Relapsed/Refractory Neuroblastoma Relapsed disease defined as neuroblastoma that was previously in remission after standard therapy...
- Any age with International Neuroblastoma Risk Group (INRG) Stage L2, MS, or M with MYCN amplification
You likely can't join if
- people who are less than 1 year of age
- BSA of \<0.25 m2
- 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...
- 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: people must be age ≤ 21 years at initial diagnosis. people must be \>12 months of age at enrollment. Safety Run-In (first 6 people) must be age 6 years or older.
- Pathology: All people must have a pathologically confirmed diagnosis of neuroblastoma at any point in their treatment.
- Tumor assessment: Disease staging must be performed. This disease assessment is required for eligibility and must be done within a maximum of 4 weeks before 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 neuroblastoma (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
- Measurable Disease: people must be relapsed or refractory with active disease. people must have measurable or evaluable disease, including at least one of the following: Measurable tumor \>10mm by computed tomography...
- people with central nervous system (CNS) disease currently taking steroids must have been on a stable dose of steroids for at least one week prior to their biopsy and must not have progressive hydrocephalus at...
- Timing from prior therapy: people 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).
- Hematopoietic growth factors: At least 5 days since the completion of therapy with a growth factor.
- Small Molecule Inhibitors (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, anti-GD2 Monoclonal antibodies (ex. naxitamab, dinutuximab, etc.).
- XRT (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
- Allo/Auto: ≥ 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:
- Hematological: Hematological recovery as defined by absolute neutrophil count (ANC) ≥750/μL, platelets ≥30/μL (may be transfused).
- Liver: Normal liver function as defined by Aspartate transferase (AST), Alanine transaminase (ALT), and total bilirubin (TBL) all within upper limit of normal
- Renal: people must have adequate renal function defined as Creatinine clearance (in units ml/min) or radioisotope GFR ≥ 70. The formula to be used: Adjusted Glomerular Filtration Rate (GFR)=(Estimated GFR×BSA/1.73)...
- Cardiac: people must have a QTcF ≤ 470 msc.
- people of childbearing potential must have a negative pregnancy test. people of childbearing potential must agree to use an effective birth control method.
- people who are lactating must agree to stop breast-feeding. (NOTE: breast milk cannot be stored for future use while the mother is being treated on study.)
- Written informed consent in accordance with institutional and FDA guidelines must be obtained from all people (or people' legal representative).
What rules you out
- people who are less than 1 year of age
- BSA of \<0.25 m2
- 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 chemotherapy.
- 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.
- Previous Gr.4 allergic or anaphylactic reaction to naxitamab, leading to the discontinuation of naxitamab during prior therapy.
The study team makes the final eligibility decision.
Where it's taking place
- Little Rock, Arkansas, United States
- Hartford, Connecticut, United States
- Miami, Florida, United States
- Orlando, Florida, United States
- Honolulu, Hawaii, United States
- St Louis, Missouri, United States
- Durham, North Carolina, United States
- Portland, Oregon, United States
- Hershey, Pennsylvania, United States
- Nashville, Tennessee, United States
- Austin, Texas, 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, 1 year to 21 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Little Rock, Arkansas, United States; Hartford, Connecticut, United States; Miami, Florida, United States; Orlando, Florida, United States; Honolulu, Hawaii, United States; St Louis, Missouri, 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.