Actively Recruiting
Tipifarnib and Naxitamab for Relapsed/Refractory Neuroblastoma
Led by Giselle Sholler · Updated on 2026-04-28
98
Participants Needed
12
Research Sites
573 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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. The goals of this part of the study are: * Test the safety and tolerability of tipifarnib in combination with naxitimab in patients with cancer * 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
CONDITIONS
Official Title
Tipifarnib and Naxitamab for Relapsed/Refractory Neuroblastoma
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Patients must be older than 12 months and 21 years or younger at initial diagnosis, with the first 6 patients in the safety run-in phase being at least 6 years old
- Pathologically confirmed diagnosis of neuroblastoma
- Disease staging completed within 4 weeks before first dose
- Relapsed neuroblastoma after standard therapy or refractory high-risk neuroblastoma as defined by INRG criteria
- Measurable or evaluable active disease with tumor >10mm by CT or MRI, positive MIBG or PET scan, or positive bone marrow biopsy/aspirate
- Stable dose of steroids for at least one week if CNS disease present, without progressive hydrocephalus
- Recovery from prior therapies within specified timeframes, including chemotherapy, immunotherapy, radiotherapy, stem cell transplant, and MIBG therapy
- Lansky or Karnofsky Performance Scale score of 50 or higher
- Adequate organ function including blood counts, liver, kidney, and cardiac function
- Negative pregnancy test for those of childbearing potential and agreement to use effective birth control
- Agreement to stop breastfeeding during study treatment
- Written informed consent obtained
You will not qualify if you...
- Patients younger than 1 year old
- Body surface area less than 0.25 m2
- Currently receiving other investigational drugs or anticancer agents
- Uncontrolled infections
- Previous severe allergic or anaphylactic reaction to naxitamab leading to discontinuation
- Inability or likely inability to comply with safety monitoring requirements
AI-Screening
AI-Powered Screening
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Trial Site Locations
Total: 12 locations
1
Arkansas Children's Hospital
Little Rock, Arkansas, United States, 72202
Actively Recruiting
2
Connecticut Children's Hospital
Hartford, Connecticut, United States, 06106
Actively Recruiting
3
Nicklaus Children's Hospital
Miami, Florida, United States, 33155
Actively Recruiting
4
Arnold Palmer Hospital for Children
Orlando, Florida, United States, 32806
Actively Recruiting
5
Kapiolani Medical Center for Women and Children
Honolulu, Hawaii, United States, 96813
Actively Recruiting
6
Cardinal Glennon Children's Medical Center
St Louis, Missouri, United States, 63104
Actively Recruiting
7
Duke University
Durham, North Carolina, United States, 27708
Actively Recruiting
8
Randall Children's Hospital
Portland, Oregon, United States, 97227
Actively Recruiting
9
Penn State Milton S. Hershey Medical Center and Children's Hospital
Hershey, Pennsylvania, United States, 17033
Actively Recruiting
10
Monroe Carrell Jr. Children's Hospital at Vanderbilt
Nashville, Tennessee, United States, 37232
Actively Recruiting
11
Dell Children's Blood and Cancer Center
Austin, Texas, United States, 78723
Actively Recruiting
12
Children's Medical Center
Dallas, Texas, United States, 75235
Actively Recruiting
Research Team
B
BCC Enroll
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
2
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