Next Generation Personalized Neuroblastoma Therapy

Part of paid clinical trials in Philadelphia, Pennsylvania.

Sponsor
Yael P Mosse
Study ID
NCT02780128
Phase
PHASE1
Status
Terminated

Conditions

Eligibility Criteria

Sex
ALL
Age
1 Year - 21 Years
Healthy Volunteers
Not accepted

Interventions

  • Biopsy — PROCEDURE
    Needle or incisional tumor biopsy
  • Next Generation Sequencing — GENETIC
    Tumor tissue will be sent to Foundation Medicine laboratory for molecular profiling.
  • Tumor Scans — PROCEDURE
    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.
  • Bone marrow Tests — PROCEDURE
    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.
  • Physical Exam — OTHER
    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.
  • Eye Exam — OTHER
    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.
  • Labs — OTHER
    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.
  • Pregnancy Test — OTHER
    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.
  • Interviews — BEHAVIORAL
    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.
  • ECG — OTHER
    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.
  • Echocardiogram — OTHER
    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.
  • Ribociclib — DRUG
    Participants will take ribociclib once per day orally for Days 1-21 of a 28-day cycle.
  • Ceritinib — DRUG
    Participants will take ceritinib once per day orally for 28 days of a 28-day cycle.

Study Details

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.

Key Dates

Start date
Jul 31, 2016
Status verified
Mar 2026
Primary completion
Aug 31, 2022
Completion
Aug 31, 2022

Study Design

Enrollment
66 participants (actual)
Allocation
NON_RANDOMIZED
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Other: Molecular Analysis
    All participants with relapsed or refractory neuroblastoma will have a tumor biopsy to identify genetic mutations. There is no drug given in this arm of the trial.
  • Experimental: Group 1: ALK
    Qualified participants whose tumors show certain mutations in the anaplastic lymphoma kinase (ALK) pathway (based on genetic sequencing results) will receive a combination therapy of ceritinib and ribociclib, to be administered orally in 28-day cycles. Two different doses of ceritinib and three different doses of ribociclib will be evaluated. Once the investigators have identified the highest safe dose of both drugs that can be given at the same time, additional participants will be enrolled in the study at this dose level. It is possible that if starting at a lower dose, participants may take a higher dose once that dose has been deemed safe.

Primary Outcome Measure

Incidence of dose limiting toxicities when combining ceritinib with ribociclib [ Time Frame: At the end of Cycle 1 (each cycle is 28 days) ]

Locations (1)

FacilityCityStateZIPSite coordinators
The Children's Hospital of PhiladelphiaPhiladelphiaPennsylvania19104-

Find similar trials in Philadelphia, PA

By condition
Neuroblastoma
By specialty
OncologyPediatric oncology
By research site
The Children's Hospital of Philadelphia· Philadelphia, PA

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