NCT06540963 · Giselle Sholler
Tipifarnib and Naxitamab for Relapsed/Refractory Neuroblastoma
What this study is about
The purpose of this study is to evaluate the experimental drug, tipifarnib (a pill taken by mouth), in combination with the Food and Drug Administration (FDA) approved drug, naxitimab, administered given through a vein (IV) (IV; a liquid that continuously goes into your body through a tube that has been placed during a surgery into one of your veins).
View original scientific description
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).
Interventions
DRUG
Tipifarnib
Tablet
DRUG
Naxitamab
IV
Primary outcome measures
Determine the Overall Response Rate (ORR) of Participants using INSS Response
Time frame: 6 months
To evaluate the activity of Tipifarnib in combination with Naxitamab based on Overall response rate (ORR)
Who can participate
This study lists these criteria on ClinicalTrials.gov. A study coordinator reviews eligibility during screening — this page does not determine whether you qualify.
Inclusion criteria
- Age: Subjects must be age ≤ 21 years at initial diagnosis. Subjects must be \>12 months of age at enrollment. Safety Run-In (first 6 subjects) must be age 6 years or older. 2. Pathology: All subjects must have a pathologically confirmed diagnosis of neuroblastoma at any point in their treatment. 3. 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. 4. 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, with or without radiation and surgery, followed by immunotherapy, or according to a standard high-risk treatment/neuroblastoma protocol) and has now relapsed and is in any number of relapses. Refractory disease defined as High-risk neuroblastoma as defined by the In
Where
- Little Rock, Arkansas
- Hartford, Connecticut
- Miami, Florida
- Orlando, Florida
- Honolulu, Hawaii
- St Louis, Missouri
- Durham, North Carolina
- Portland, Oregon
- Hershey, Pennsylvania
- Nashville, Tennessee
- Austin, Texas
- Dallas, Texas
Related conditions & keywords
Frequently asked questions
What is a clinical trial?
A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.
Is it safe to participate?
Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.
Will I be compensated?
Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.
Will I receive a placebo instead of treatment?
When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.
Can I leave a trial if I change my mind?
Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.
How long does a clinical trial last?
Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.
Data: ClinicalTrials.gov · synced Apr 28, 2026 · Source of record for eligibility and locations