NCT02780128

Next Generation Personalized Neuroblastoma Therapy

Next Generation Personalized Neuroblastoma Therapy (The NEPENTHE Trial)

• Status: Terminated
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 66 (actual)
• Sponsor: Yael P Mosse · Academic / Other
• Sex: All
• Age: 1 Year – 21 Years
• Healthy volunteers: Not accepted

Summary

The purpose of this research study is to match genomic aberrations in tumor cells at time of relapse to rationally designed combinations of molecularly targeted agents. This study will be done in two parts: Part I: Tumor will be accessed at study entry via a biopsy and subjected to deep sequencing to identify protocol-specified biomarkers for therapy assignment. Part II: If the tumor contains a genetic change defined by the study as being actionable, and other criteria are met, participants will be assigned to therapy based upon the genetic changes identified in the tumor biopsy.

Conditions

• Neuroblastoma
• Cancer

Interventions

Type	Name	Description
PROCEDURE	Biopsy	Needle or incisional tumor biopsy
GENETIC	Next Generation Sequencing	Tumor tissue will be sent to Foundation Medicine laboratory for molecular profiling.
PROCEDURE	Tumor Scans	Participants will undergo different types of scans to look at your tumor. These scans include CT (computerized tomography), MIBG (meta-iodobenzylguanidine) PET (positron emission tomography), and MRI (magnetic resonance imaging). Participants may have more than one type of scan.
PROCEDURE	Bone marrow Tests	Participants will have needles inserted through their hip bone to remove fluid from inside the bone marrow. This test determines if participants have tumor in the bone marrow.
OTHER	Physical Exam	The exam includes taking participant weight, height, blood pressure, heart rate and respiratory rate and performing a examination of the participants body. The investigators may also check the participants vision with an eye chart.
OTHER	Eye Exam	Participants will have their eyes will be evaluated using different instruments. Participants will also be asked to read an eye chart. The exams will take about 15 minutes.
OTHER	Labs	Standard blood tests will be done to measure different types of blood cells, to measure the amount of certain substances, and tests to check how well liver and kidneys are working. When possible, the investigators will take blood from the participants central line. If this is not possible, the investigators will take blood from a vein in the participants arm. First, the investigators will put some cream on the skin so that drawing blood will not be painful. Then the investigators will put a thin needle into the vein to draw the blood.
OTHER	Pregnancy Test	If the participant is 11 years old or older or has already started having periods, the participant will be asked to take a pregnancy test before starting this study. The results will be shared with the participant but not with the participants' parent(s). We strongly encourage the participant to share the results with the parents. If the participant is found to be pregnant, the participant will not be able to continue participation in the study. About 1 teaspoon of blood (or urine if a urine test) will be needed.
BEHAVIORAL	Interviews	A team member will take the participant's medical history, along with a listing of any medications that are being taken. Throughout the study, participants will be asked to report if they think that anything bad has happened as a result of the study.
OTHER	ECG	This is a test of electrical activity of the heart. The investigators will put electrodes (sticky pads attached to wires) on the participant's chest, arms and legs. The electrocardiogram (ECG) will not be uncomfortable, but the participant will have to lie still. It does not hurt and takes about 15 minutes.
OTHER	Echocardiogram	The participant will have an Echocardiogram (ECHO), an ultrasound of the heart, taken to assess heart function. The investigators will put some gel on the skin and use a machine to take pictures of the heart.
DRUG	Ribociclib	Participants will take ribociclib once per day orally for Days 1-21 of a 28-day cycle.
DRUG	Ceritinib	Participants will take ceritinib once per day orally for 28 days of a 28-day cycle.

Timeline

• Start date: 2016-07-01
• Primary completion: 2022-08-01
• Completion: 2022-08-01
• First posted: 2016-05-23
• Last updated: 2026-03-10

Locations

1 site across 1 country: United States