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Next Generation Personalized Neuroblastoma Therapy (The NEPENTHE Trial) (NCT02780128)

Novartis Pharmaceuticals
Foundation Medicine
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.
  • Procedure: Biopsy
    Needle or incisional tumor biopsy
    • Genetic Sequencing
  • Drug: Ceritinib
    Participants will take ceritinib once per day orally for 28 days.
    • LDK378
  • Drug: Trametinib
    Participants will take trametinib once per day orally for 28 days.
  • Drug: HDM201
    Participants will take HDM201 orally on Day 1 and Day 8 of a 28-day cycle.
    • Genetic: Next Generation Sequencing
      Tumor tissue will be sent to Foundation Medicine laboratory for molecular profiling.
      • Molecular Profiling
      • Genetic Sequencing
    • 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.
      • Radiation: X-ray
        Participants will have x-rays of the wrist and leg.
        • 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.
          • Bone marrow aspiration
        • 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.
            • Ophthalmic Exam
          • 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.
            • Blood tests
          • 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.
                • EKG
                • electrocardiogram
              • Other: Echocardiogram
                The participant will have 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.
                • echo
              • Drug: Ribociclib
                Participants will take ribociclib once per day orally for 21 days, followed by 7 days rest.
                • LEE011
              Ages eligible for Study
              1 Years to 21 Years
              Genders eligible for Study
              Accepts Healthy Volunteers
              Inclusion Criteria:
              • Aged ≥1 years to ≤ 21 years
              • Relapsed or refractory neuroblastoma
              • A sufficient interval between the last dose of prior anti-cancer therapy (including cytotoxic and biological therapies) and enrollment in this study, to allow recovery from the acute toxic effects of all prior anti-cancer therapy. Please contact site for specific details
              • Adequate bone marrow function (bone marrow may be involved with tumor. Contact site for specific details)
              • Adequate renal function, defined as Creatinine clearance or radioisotope glomerular filtration rate (GFR) ≥ 70 mL/min/1.73 m2 OR serum creatinine based on age/gender normal (contact site for details)
              • Adequate liver function, defined as total serum bilirubin ≤ 1.5 times the upper limit of normal AND alanine transaminase (ALT) ≤ 110 U/L.
              • Adequate cardiac function, defined as corrected QT interval (QTc) ≤ 480 msec AND shortening fraction > 27%
              • Males and females who are sexually active must agree to use effective contraception during and for 3 months after treatment
              Exclusion Criteria:
              • Subjects taking certain drugs or herbal medications that impact drug metabolism and/or cardiac function that cannot be discontinued (contact site for details).
              • Subjects with concurrent severe and/or uncontrolled concurrent medical conditions that could compromise participation in the study (contact site for details)
              • Other concomitant therapies:
              • Corticosteroids initiated for tumor therapy within 7 days prior to study enrollment
              • Other anti-cancer agents
              • Other investigational drugs
              • Hematological growth factors
              • Radiation therapy
              • Subjects < 0.5m2
              • Pregnant or lactating females
              • Sexually active males unless they use a condom during intercourse while taking study drug/s and for 3 months after study drug discontinuation and thus do not attempt to father a child in this period.

              1 locations

              United States (1)
              • The Children's Hospital of Philadelphia
                Philadelphia, Pennsylvania, United States, 19144
              30 June, 2016
              29 January, 2018
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