Pediatric Precision Therapy for Neuroblastoma
This study is for children and young adults up to 22 years old with newly diagnosed high-risk neuroblastoma (a type of cancer that forms in nerve tissue). It aims to see if using specific drugs based on a patient's tumor characteristics, combined with standard treatments, is safe and effective. You would receive one of four drugs – Ceritinib, dasatinib, sorafenib, or vorinostat – chosen based on molecular tests. This is followed by other treatments, including immunotherapy and DFMO, with DFMO continuing for two years as maintenance. Researchers will track how long it takes for the cancer to return, how many patients receive a targeted drug, and how well patients stick to the medication schedule.
- Study design
- This is an open-label, multi-center study with a planned enrollment of 500 participants. It combines a feasibility study with a randomized controlled trial.
- What's involved
- Not specified in the trial record.
- Compensation
- Not stated in the trial record.
- Follow-up
- Participants will be followed for up to 8 years to track the time to first relapse.
AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.
Pediatric Precision Laboratory Advanced Neuroblastoma Therapy
At a glance
Conditions
Where it's being run
28 sites across 21 statesStudy leadership
- Giselle Sholler, MD · STUDY_CHAIR · Beat Childhood Cancer
Who to contact
Opens a ready-to-send draft in your own email app — review before sending.
What this trial measures
- Number of days from start of therapy to date of first relapseUp to 8 years
To measure the response of treatments chosen based on: • Event free survival (EFS)
- Number of subjects that have a targeted agent chosen for treatment.2 years
At completion of the induction therapy, the investigators will determine feasibility of adding molecularly guided targeted therapy to standard of care chemotherapy. Feasibility will be defined as: 1. Subject has a targeted agent identified 2. Receives 75% of dosing of medications while on study protocol during cycles 3-6 3. Subject is not removed from study due to targeted agent drug related toxicity.
- Number of subjects that receive 75% of dosing of medications while on study protocol during cycles 3-6.2 years
At completion of the induction therapy, the investigators will determine feasibility of adding molecularly guided targeted therapy to standard of care chemotherapy. Feasibility will be defined as: 1. Subject has a targeted agent identified 2. Receives 75% of dosing of medications while on study protocol during cycles 3-6 3. Subject is not removed from study due to targeted agent drug related toxicity.