This site became the new ClinicalTrials.gov on June 19th. Learn more.
Show more
ClinicalTrials.gov Menu IMPORTANT: Listing of a study on this site does not reflect endorsement by the National Institutes of Health. Talk with a trusted healthcare professional before volunteering for a study. Read more...
ClinicalTrials.gov Menu IMPORTANT: Talk with a trusted healthcare professional before volunteering for a study. Read more...
ClinicalTrials.gov Menu
Give us feedback
Trial record 7 of 7 for:    DFMO and neuroblastoma

Pediatric Precision Laboratory Advanced Neuroblastoma Therapy (PEDS-PLAN)

This study is currently recruiting participants.
See Contacts and Locations
Verified April 2017 by Giselle Sholler, Spectrum Health Hospitals
Sponsor:
Collaborators:
Dell, Inc.
Beat NB Cancer Foundation
Because of Ezra
Information provided by (Responsible Party):
Giselle Sholler, Spectrum Health Hospitals
ClinicalTrials.gov Identifier:
NCT02559778
First received: September 22, 2015
Last updated: April 25, 2017
Last verified: April 2017
  Purpose
A prospective open label, multicenter study to evaluate the feasibility and acute toxicity of using molecularly guided therapy in combination with standard therapy followed by maintenance therapy with DFMO in subjects with newly diagnosed high risk neuroblastoma.

Condition Intervention Phase
Neuroblastoma Drug: bortezomib Drug: crizotinib Drug: dasatinib Drug: lapatinib Drug: sorafenib Drug: vorinostat Drug: DFMO Phase 1

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: No masking
Primary Purpose: Treatment
Official Title: PEDS-PLAN - Pediatric Precision Laboratory Advanced Neuroblastoma Therapy- A Pilot Study Using Molecular Guided Therapy With Induction Chemotherapy Followed by Maintenance With DFMO for Subjects With Newly Diagnosed High-Risk Neuroblastoma

Resource links provided by NLM:


Further study details as provided by Giselle Sholler, Spectrum Health Hospitals:

Primary Outcome Measures:
  • Number of subjects that have a targeted agent chosen for treatment. [ Time Frame: 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. [ Time Frame: 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 required to go off therapy due to treatment-related adverse events as assessed by CTCAE v4.0. [ Time Frame: 1 year ]

    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 are able to complete 2 years of DFMO therapy. [ Time Frame: 3 years ]

Secondary Outcome Measures:
  • Number of participants with treatment-related adverse events as assessed by CTCAE v4.0 [ Time Frame: 3 years ]
  • Overall Response Rate (ORR) of Participants by the presence of radiologically assessable disease by cross-sectional CT or MRI imaging and/or by MIBG or PET scans. [ Time Frame: 3 years ]
    • To measure the response of treatments chosen based on:
    • Overall response rate (ORR) after induction therapy
    • Progression free survival (PFS)

  • Number of days that subjects remain disease free [ Time Frame: 3 years ]
    • To measure the response of treatments chosen based on:
    • Overall response rate (ORR) after induction therapy
    • Progression free survival (PFS)


Estimated Enrollment: 24
Study Start Date: September 2015
Estimated Study Completion Date: September 2025
Estimated Primary Completion Date: September 2020 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Addition of Targeted Agent
The addition of one targeted therapy (bortezomib, crizotinib, dasatinib, lapatinib, sorafenib or vorinostat), determined using genomic profiling of tumor will be combined with standard upfront treatment during cycles 3-6 of induction chemotherapy. This will be followed by consolidation, immunotherapy and then 2 years of DFMO as maintenance.
Drug: bortezomib
One of the following drugs will be chosen for each subject based on molecular guided results: bortezomib, crizotinib, dasatinib, lapatinib, sorafenib or vorinostat. This will be followed by consolidation, immunotherapy, and then DFMO for 2 years as maintenance.
Other Name: Velcade
Drug: crizotinib
One of the following drugs will be chosen for each subject based on molecular guided results: bortezomib, crizotinib, dasatinib, lapatinib, sorafenib or vorinostat. This will be followed by consolidation, immunotherapy, and then DFMO for 2 years as maintenance.
Other Name: XALKORI
Drug: dasatinib
One of the following drugs will be chosen for each subject based on molecular guided results: bortezomib, crizotinib, dasatinib, lapatinib, sorafenib or vorinostat. This will be followed by consolidation, immunotherapy, and then DFMO for 2 years as maintenance.
Other Name: Sprycel
Drug: lapatinib
One of the following drugs will be chosen for each subject based on molecular guided results: bortezomib, crizotinib, dasatinib, lapatinib, sorafenib or vorinostat. This will be followed by consolidation, immunotherapy, and then DFMO for 2 years as maintenance.
Other Name: Tykerb
Drug: sorafenib
One of the following drugs will be chosen for each subject based on molecular guided results: bortezomib, crizotinib, dasatinib, lapatinib, sorafenib or vorinostat. This will be followed by consolidation, immunotherapy, and then DFMO for 2 years as maintenance.
Other Name: Nexavar
Drug: vorinostat
One of the following drugs will be chosen for each subject based on molecular guided results: bortezomib, crizotinib, dasatinib, lapatinib, sorafenib or vorinostat. This will be followed by consolidation, immunotherapy, and then DFMO for 2 years as maintenance.
Other Name: ZOLINZA
Drug: DFMO
DFMO will be given for 2 years as maintenance to all subjects completing immunotherapy.
Other Name: Eflornithine, α-difluoromethylornithine

  Eligibility

Ages Eligible for Study:   12 Months to 21 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Diagnosis: Subjects must have a diagnosis of high-risk (defined in protocol) neuroblastoma or ganglioneuroblastoma (nodular or intermixed) verified by histology or demonstration of clumps of tumor cells in bone marrow with elevated urinary catecholamine metabolites.
  • Subjects must be age ≤ 21 years at initial diagnosis
  • Subjects must not have had prior systemic therapy except for localized emergency radiation to sites of life-threatening or function-threatening disease and/or no more than 1 cycle of chemotherapy per a low or intermediate risk neuroblastoma regimen (as per P9641, A3961, ANBL0531, or similar) prior to determination of MYCN amplification status and histology.
  • Specimens will be obtained only in a non-significant risk manner and not solely for the purpose of investigational testing.
  • Adequate liver function must be demonstrated (defined in protocol)
  • Subjects must have adequate renal function defined as a Serum creatinine based on age/gender (defined in protocol)
  • Adequate Cardiac Function (defined in protocol)
  • Ability to tolerate PBSC collection: No known contraindication to PBSC collection.
  • A negative serum pregnancy test is required for female participants of child bearing potential (≥13 years of age or after onset of menses)
  • Both male and female post-pubertal study subjects need to agree to use one of the more effective birth control methods during treatment and for six months after treatment is stopped. These methods include total abstinence (no sex), oral contraceptives ("the pill"), an intrauterine device (IUD), levonorgestrol implants (Norplant), or medroxyprogesterone acetate injections (Depo-provera shots). If one of these cannot be used, contraceptive foam with a condom is recommended.
  • Informed Consent: All subjects and/or legal guardians must sign informed written consent. Assent, when appropriate, will be obtained according to institutional guidelines

Exclusion Criteria:

  • Subjects who are 12-18 months of age with INSS Stage 4 and all stage 3 subjects with favorable biologic features (ie, nonamplified MYCN, favorable pathology, and DNA index > 1) are not eligible.
  • Lactating females are not eligible unless they have agreed not to breastfeed their infants.
  • Subjects receiving any investigational drug concurrently.
  • Subjects with any other medical condition, including but not limited to malabsorption syndromes, mental illness or substance abuse, deemed by the Investigator to be likely to interfere with the interpretation of the results or which would interfere with a subject's ability to sign or the legal guardian's ability to sign the informed consent, and subject's ability to cooperate and participate in the study
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT02559778

Contacts
Contact: Genevieve Bergendahl, MSN 616-267-0334 genevieve.bergendahl@helendevoschildrens.org
Contact: Alyssa VanderWerff, MS 616-267-0334 alyssa.vanderwerff@helendevoschildrens.org

Locations
United States, Arkansas
Arkansas Children's Hospital Recruiting
Little Rock, Arkansas, United States, 72202
Contact: Janet Storment, RN    501-364-2760    StormentJanetS@uams.edu   
Principal Investigator: Kathleen Neville, MD         
United States, California
Rady Children's Hospital Recruiting
San Diego, California, United States, 92123
Contact: Mehrzad Milburn    858-966-8155      
Principal Investigator: William Roberts, MD         
United States, Connecticut
Connecticut Children's Hospital Recruiting
Hartford, Connecticut, United States, 06106
Contact: Sharon Huie-White    860-545-9337    shuie@connecticutchildrens.org   
Principal Investigator: Michael Isakoff, MD         
United States, Hawaii
Kapiolani Medical Center for Women and Children Recruiting
Honolulu, Hawaii, United States, 96813
Contact: Andrea Siu, MPH    808-535-7169    andrea.siu@kapiolani.org   
Principal Investigator: Randal Wada, MD         
United States, Michigan
Helen DeVos Children's Hospital Recruiting
Grand Rapids, Michigan, United States, 49503
Contact: Shannon MacKeigan    616-267-1162    shannon.mackeigan@helendevoschildrens.org   
Principal Investigator: Deanna Mitchell, MD         
Principal Investigator: Giselle Sholler, MD         
United States, Minnesota
Children's Hospital and Clinics on Minnesota Recruiting
Minneapolis, Minnesota, United States, 55404
Contact: Courtney Haller    612-813-5913    courtney.haller@childrensmn.org   
Principal Investigator: Jawhar Rawwas, MD         
United States, Missouri
Cardinal Glennon Children's Medical Center Recruiting
Saint Louis, Missouri, United States, 63104
Contact: Katherine Maxwell, RN    314-268-4000    maxwellk@slu.edu   
Principal Investigator: William Ferguson, MD         
United States, North Carolina
Levine Children's Hospital Recruiting
Charlotte, North Carolina, United States, 28204
Contact: Elizabeth Arroyo-Green    980-442-2312    Elizabeth.ArroyoGreen@carolinashealthcare.org   
Principal Investigator: Javier Oesterheld, MD         
United States, Pennsylvania
Penn State Milton S. Hershey Medical Center and Children's Hospital Recruiting
Hershey, Pennsylvania, United States, 17033
Contact: Suzanne Treadway       streadway@hmc.psu.edu   
Principal Investigator: Valerie Brown, MD         
United States, South Carolina
Medical University of South Carolina Recruiting
Charleston, South Carolina, United States, 29425
Contact: Liz Shewfelt    843-792-2957    shewfelt@musc.edu   
Principal Investigator: Jaqueline Kraveka, MD         
United States, Texas
Dell Children's Blood and Cancer Center Recruiting
Austin, Texas, United States, 78723
Contact: Letitia Holden, RN    512-628-1902    lholden@sfcaustin.com   
Principal Investigator: Virginia Harrod, MD         
Children's Medical Center Recruiting
Dallas, Texas, United States, 75235
Contact: Michelle Rivas    214-456-0004    michelle.rivas@childrens.com   
Principal Investigator: Ted Laetsch, MD         
Sponsors and Collaborators
Giselle Sholler
Dell, Inc.
Beat NB Cancer Foundation
Because of Ezra
Investigators
Study Chair: Giselle Sholler, MD NMTRC
  More Information

Additional Information:
Responsible Party: Giselle Sholler, NMTRC Chair, Spectrum Health Hospitals
ClinicalTrials.gov Identifier: NCT02559778     History of Changes
Other Study ID Numbers: NMTRC012
Study First Received: September 22, 2015
Last Updated: April 25, 2017

Additional relevant MeSH terms:
Neuroblastoma
Eflornithine
Neuroectodermal Tumors, Primitive, Peripheral
Neuroectodermal Tumors, Primitive
Neoplasms, Neuroepithelial
Neuroectodermal Tumors
Neoplasms, Germ Cell and Embryonal
Neoplasms by Histologic Type
Neoplasms
Neoplasms, Glandular and Epithelial
Neoplasms, Nerve Tissue
Sorafenib
Lapatinib
Vorinostat
Bortezomib
Dasatinib
Crizotinib
Antineoplastic Agents
Protein Kinase Inhibitors
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action
Trypanocidal Agents
Antiprotozoal Agents
Antiparasitic Agents
Anti-Infective Agents
Ornithine Decarboxylase Inhibitors
Histone Deacetylase Inhibitors

ClinicalTrials.gov processed this record on June 28, 2017