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July 10, 2018; 91 (2 Supplement 1) ArticleOpen Access

Clinical trial design for cutaneous neurofibromas

Ashley Cannon, Kurt Jarnagin, Bruce Korf, Brigitte C. Widemann, Denise Casey, Hon-Sum Ko, Jaishri O. Blakeley, Sharad K. Verma, Dominique C. Pichard
First published July 9, 2018, DOI: https://doi.org/10.1212/WNL.0000000000005790
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Article Information

vol. 91 no. 2 Supplement 1 S31-S37
DOI: 
https://doi.org/10.1212/WNL.0000000000005790
PubMed: 
29987133
Published By: 
Wolters Kluwer Health, Inc. on behalf of the American Academy of Neurology
Print ISSN: 
0028-3878
Online ISSN: 
1526-632X
History: 
  • Received November 1, 2017
  • Accepted in final form April 13, 2018
  • First Published July 9, 2018.
Copyright & Usage: 
© 2018 The Author(s). Published by Wolters Kluwer Health, Inc. on behalf of the American Academy of Neurology. This is an open access article distributed under the terms of the Creative Commons Attribution License 4.0 (CC BY), which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited.

Author Disclosures

    1. Ashley Cannon, PhD, MS, CGC,
    2. Kurt Jarnagin, PhD,
    3. Bruce Korf, MD, PhD,
    4. Brigitte C. Widemann, MD,
    5. Denise Casey, MD,
    6. Hon-Sum Ko, MD,
    7. Jaishri O. Blakeley, MD,
    8. Sharad K. Verma, PhD and
    9. Dominique C. Pichard, MD
  1. Ashley Cannon, PhD, MS, CGC,
    2. Scientific Advisory Boards:

    1. NONE

    Gifts:

    1. NONE

    Funding for Travel or Speaker Honoraria:

    1. NONE

    Editorial Boards:

    1. NONE

    Patents:

    1. NONE

    Publishing Royalties:

    1. NONE

    Employment, Commercial Entity:

    1. NONE

    Consultancies:

    1. NONE

    Speakers' Bureaus:

    1. NONE

    Other Activities:

    1. NONE

    Clinical Procedures or Imaging Studies:

    1. NONE

    Research Support, Commercial Entities:

    1. NONE

    Research Support, Government Entities:

    1. NONE

    Research Support, Academic Entities:

    1. NONE

    Research Support, Foundations and Societies:

    1. NONE

    Stock/Stock Options/Board of Directors Compensation:

    1. NONE

    License Fee Payments, Technology or Inventions:

    1. NONE

    Royalty Payments, Technology or Inventions:

    1. NONE

    Stock/Stock Options, Research Sponsor:

    1. NONE

    Stock/Stock Options, Medical Equipment & Materials:

    1. NONE

    Legal Proceedings:

    1. NONE

  2. Kurt Jarnagin, PhD,
    3. Scientific Advisory Boards:

    1. NONE

    Gifts:

    1. NONE

    Funding for Travel or Speaker Honoraria:

    1. NONE

    Editorial Boards:

    1. NONE

    Patents:

    1. NONE

    Publishing Royalties:

    1. NONE

    Employment, Commercial Entity:

    1. BioPharm Tech a consulting organization 2017-present -- no relationship to this work Pfizer -- 2016-2017 -- no relationship to this work Anacor Pharmaceuticals -- 2008-2016 -- no relationship to this work

    Consultancies:

    1. NONE

    Speakers' Bureaus:

    1. NONE

    Other Activities:

    1. NONE

    Clinical Procedures or Imaging Studies:

    1. NONE

    Research Support, Commercial Entities:

    1. Jarnagin servers as a general consultant to BioPharma industry. Those activities cover many areas, but none within the topic areas of the papers at issue here.

    Research Support, Government Entities:

    1. NONE

    Research Support, Academic Entities:

    1. NONE

    Research Support, Foundations and Societies:

    1. NONE

    Stock/Stock Options/Board of Directors Compensation:

    1. Anacor was purchased by Pfizer in 2016. As such Jarnagin received stock option-based compensation in 2016. No relationship to this work.

    License Fee Payments, Technology or Inventions:

    1. NONE

    Royalty Payments, Technology or Inventions:

    1. NONE

    Stock/Stock Options, Research Sponsor:

    1. NONE

    Stock/Stock Options, Medical Equipment & Materials:

    1. NONE

    Legal Proceedings:

    1. NONE

  3. Bruce Korf, MD, PhD,
    4. Scientific Advisory Boards:

    1. Accolade Genome Medical Envision Genomics

    Gifts:

    1. NONE

    Funding for Travel or Speaker Honoraria:

    1. NONE

    Editorial Boards:

    1. editorial boards: Current Protocols in Human Genetics, American Journal of Human Genetics (editor)

    Patents:

    1. NONE

    Publishing Royalties:

    1. Wiley: Current Protocols in Human Genetics Human Genetics and Genomics Medical Genetics at a Glance Elsevier: Emery and Rimoin's Principles and Practice of Medical Genetics

    Employment, Commercial Entity:

    1. NONE

    Consultancies:

    1. Novartis

    Speakers' Bureaus:

    1. NONE

    Other activities:

    1. President, American College of Medical Genetics and Genomics Foundation Board of Directors: Children's Tumor Foundation Senior Advisor: Neurofibromatosis Therapeutic Acceleration Project

    Clinical Procedures or Imaging Studies:

    1. NONE

    Research Support, Commercial Entities:

    1. Novartis - Tuberous Sclerosis Database

    Research Support, Government Entities:

    1. W81XWH-12-1-0155, Department of Defense Korf, Bruce (PI) 08/15/17-08/14/21 The NF Clinical Trials Consortium This is a multicenter clinical trials award intended to perform clinical trial studies in patients with NF. UAB is a patient recruitment site, but also serves as the coordinating center for the consortium. Note: This award has been renewed for another five years beginning May 2017. Role: PI 1T32HG008961, NHGRI Korf, Bruce (PI) 06/01/16-05/30/21 UAB-HudsonAlpha Genomic Medicine Training Program The UAB-HudsonAlpha Genomic Medicine Research Training Program trains postdoctoral fellows from MD, PhD, and MDPhD backgrounds, focusing on genomic medicine in clinical research. Role: PI OT2OD025284, NIH Korf, Bruce (PI) 08/26/17-05/31/18 Southern All of Us Network I am PI of the Southern All of Us Network, which consists of a set of health provider organizations in Alabama, Mississippi, and Louisiana that will be recruiting participants for the national All of Us program. Role: PI U01HG007301, NIH Cooper, Gregory (PI) 08/01/17-07/31/21 Clinical sequencing across communities in the Deep South This project is part of the NHGRI Clinical Sequencing Exploratory Research Program. We are using whole genome sequencing to establish diagnoses of newborns in the special care nursery and testing the ability to support nursery staff in return of genomic results to parents. Role: CPI

    Research Support, Academic Entities:

    1. NONE

    Research Support, Foundations and Societies:

    1. Children's Tumor Foundation Funding to support testing related to a clinical trial

    Stock/Stock Options/Board of Directors Compensation:

    1. Accolade Envision Genomics Genome Medical

    License Fee Payments, Technology or Inventions:

    1. NONE

    Royalty Payments, Technology or Inventions:

    1. NONE

    Stock/Stock Options, Research Sponsor:

    1. NONE

    Stock/Stock Options, Medical Equipment & Materials:

    1. NONE

    Legal Proceedings:

    1. NONE

  4. Brigitte C. Widemann, MD,
    5. Scientific Advisory Boards:

    1. Member scientific advisory board member of the Neurofibromatosis Therapeutic Acceleration Program (NTAP). Non for profit organization I receive no honorarium. Member external Advisory Board for Gilbert Family Neurofibromatosis Institute. Non for profit organization I receive no honorarium. Member external Advisory Board for Pacific Pediatric Neuro-Oncology Consortium (PNOC). Non for profit organization I receive no honorarium. Non for profitorganization I receive no honorarium.

    Gifts:

    1. NONE

    Funding for Travel or Speaker Honoraria:

    1. NONE

    Editorial Boards:

    1. NONE

    Patents:

    1. NONE

    Publishing Royalties:

    1. NONE

    Employment, Commercial Entity:

    1. NONE

    Consultancies:

    1. NONE

    Speakers' Bureaus:

    1. NONE

    Other Activities:

    1. NONE

    Clinical Procedures or Imaging Studies:

    1. NONE

    Research Support, Commercial Entities:

    1. NONE

    Research Support, Government Entities:

    1. NONE

    Research Support, Academic Entities:

    1. NONE

    Research Support, Foundations and Societies:

    1. NONE

    Stock/Stock Options/Board of Directors Compensation:

    1. NONE

    License Fee Payments, Technology or Inventions:

    1. NONE

    Royalty Payments, Technology or Inventions:

    1. NONE

    Stock/Stock Options, Research Sponsor:

    1. NONE

    Stock/Stock Options, Medical Equipment & Materials:

    1. NONE

    Legal Proceedings:

    1. NONE

  5. Denise Casey, MD,
    6. Scientific Advisory Boards:

    1. NONE

    Gifts:

    1. NONE

    Funding for Travel or Speaker Honoraria:

    1. NONE

    Editorial Boards:

    1. NONE

    Patents:

    1. NONE

    Publishing Royalties:

    1. NONE

    Employment, Commercial Entity:

    1. NONE

    Consultancies:

    1. NONE

    Speakers' Bureaus:

    1. NONE

    Other Activities:

    1. NONE

    Clinical Procedures or Imaging Studies:

    1. NONE

    Research Support, Commercial Entities:

    1. NONE

    Research Support, Government Entities:

    1. NONE

    Research Support, Academic Entities:

    1. NONE

    Research Support, Foundations and Societies:

    1. NONE

    Stock/Stock Options/Board of Directors Compensation:

    1. NONE

    License Fee Payments, Technology or Inventions:

    1. NONE

    Royalty Payments, Technology or Inventions:

    1. NONE

    Stock/Stock Options, Research Sponsor:

    1. NONE

    Stock/Stock Options, Medical Equipment & Materials:

    1. NONE

    Legal Proceedings:

    1. NONE

  6. Hon-Sum Ko, MD,
    7. Scientific Advisory Boards:

    1. NONE

    Gifts:

    1. NONE

    Funding for Travel or Speaker Honoraria:

    1. NONE

    Editorial Boards:

    1. NONE

    Patents:

    1. NONE

    Publishing Royalties:

    1. NONE

    Employment, Commercial Entity:

    1. NONE

    Consultancies:

    1. NONE

    Speakers' Bureaus:

    1. NONE

    Other Activities:

    1. NONE

    Clinical Procedures or Imaging Studies:

    1. NONE

    Research Support, Commercial Entities:

    1. NONE

    Research Support, Government Entities:

    1. NONE

    Research Support, Academic Entities:

    1. NONE

    Research Support, Foundations and Societies:

    1. NONE

    Stock/Stock Options/Board of Directors Compensation:

    1. NONE

    License Fee Payments, Technology or Inventions:

    1. NONE

    Royalty Payments, Technology or Inventions:

    1. NONE

    Stock/Stock Options, Research Sponsor:

    1. NONE

    Stock/Stock Options, Medical Equipment & Materials:

    1. NONE

    Legal Proceedings:

    1. NONE

  7. Jaishri O. Blakeley, MD,
    8. Scientific Advisory Boards:

    1. I served in an uncompensated role on a Scientific Advisory Board for Novartis in 2010. I served as a paid consultant for a medical advisory board for Abbvie June 2015 and as a paid consultant for Abbvie for clinical trial design for a glioblastoma quality of life study in 2016. I currently sit on the Medical Advisory Council for the Children's Tumor Foundation

    Gifts:

    1. NONE

    Funding for travel or speaker honoraria:

    1. Travel to a scientific conference in Novemebr 2017 during which I participated in a panel discussion about the drug selumetinib for brain tumors and plexiform neurofibromas. Travel expenses were paid for by AstraZenica.

    Editorial Boards:

    1. Guest editor, Experimental Neurology, January 2018

    Patents:

    1. NONE

    Publishing Royalties:

    1. NONE

    Employment, Commercial Entity:

    1. NONE

    Consultancies:

    1. I served as a compensated consultant to Abbvie in June 2015 for glioblastoma study design and as a paid consultant for Abbvie for clinical trial design for glioblastoma quality of life in 2016.

    Speakers' Bureaus:

    1. NONE

    Other Activities:

    1. NONE

    Clinical Procedures or Imaging Studies:

    1. NONE

    Research Support, Commercial Entities:

    1. Glaxo Smith Kline non-salary research support for vestibular schwannoma clinical trial with lapatinib; Sanofi non-salary research support for glioma clinical trial; Lily non-salary research support for glioma clinical trial

    Research Support, Government Entities:

    1. NIH/NIBIB, R01EB009731; Co-investogator, 07/01/09- 04/30/13; Department of Defense Neurofibromatosis Clinical Trial Consortium site PI

    Research Support, Academic Entities:

    1. NONE

    Research Support, Foundations and Societies:

    1. Children's Tumor Fundation; Brain Tumor Funders Collaborative; American Society of Clinical Oncology

    Stock/Stock Options/Board of Directors Compensation:

    1. NONE

    License Fee Payments, Technology or Inventions:

    1. NONE

    Royalty Payments, Technology or Inventions:

    1. NONE

    Stock/Stock Options, Research Sponsor:

    1. NONE

    Stock/Stock Options, Medical Equipment & Materials:

    1. NONE

    Legal Proceedings:

    1. NONE

  8. Sharad K. Verma, PhD and
    9. Scientific Advisory Boards:

    1. NONE

    Gifts:

    1. NONE

    Funding for Travel or Speaker Honoraria:

    1. NONE

    Editorial Boards:

    1. (1) Translational Genetics and Genomics, Editorial Advisory board member (non-compensated), 2016 - present.

    Patents:

    1. NONE

    Publishing Royalties:

    1. NONE

    Employment, Commercial Entity:

    1. NONE

    Consultancies:

    1. (1) Genentech (2) Bristol-Myers-Squibb

    Speakers' Bureaus:

    1. NONE

    Other Activities:

    1. NONE

    Clinical Procedures or Imaging Studies:

    1. NONE

    Research Support, Commercial Entities:

    1. NONE

    Research Support, Government Entities:

    1. NONE

    Research Support, Academic Entities:

    1. NONE

    Research Support, Foundations and Societies:

    1. (1) National Comprehensive Cancer Network Stock/Stock Options, Medical Equipment & Materials: (1) GlaxoSmithKline (2008 - present) (2) Epizyme (2016 - present)

    Stock/Stock Options/Board of Directors Compensation:

    1. NONE

    License Fee Payments, Technology or Inventions:

    1. NONE

    Royalty Payments, Technology or Inventions:

    1. NONE

    Stock/Stock Options, Research Sponsor:

    1. NONE

    Stock/Stock Options, Medical Equipment & Materials:

    1. NONE

    Legal Proceedings:

    1. NONE

  9. Dominique C. Pichard, MD
    10. Scientific Advisory Boards:

    1. NONE

    Gifts:

    1. NONE

    Funding for Travel or Speaker Honoraria:

    1. NONE

    Editorial Boards:

    1. NONE

    Patents:

    1. NONE

    Publishing Royalties:

    1. NONE

    Employment, Commercial Entity:

    1. NONE

    Consultancies:

    1. NONE

    Speakers' Bureaus:

    1. NONE

    Other Activities:

    1. NONE

    Clinical Procedures or Imaging Studies:

    1. NONE

    Research Support, Commercial Entities:

    1. CRADA with Incyte to provide study drug for a clinical trial unrelated to the topic of this manuscript.

    Research Support, Government Entities:

    1. Intramural NIH employee

    Research Support, Academic Entities:

    1. NONE

    Research Support, Foundations and Societies:

    1. NONE

    Stock/Stock Options/Board of Directors Compensation:

    1. NONE

    License Fee Payments, Technology or Inventions:

    1. NONE

    Royalty Payments, Technology or Inventions:

    1. NONE

    Stock/Stock Options, Research Sponsor:

    1. NONE

    Stock/Stock Options, Medical Equipment & Materials:

    1. NONE

    Legal Proceedings:

    1. NONE

  1. From the Department of Genetics (A.C., B.K.), University of Alabama at Birmingham; BioPharm Tech (K.J.), San Mateo, CA; Pediatric Oncology Branch (B.C.W.) and Dermatology Branch, Center for Cancer Research (D.C.P.), National Cancer Institute, NIH, Bethesda; Division of Oncology Products (D.C.) and Division of Dermatology and Dental Products (H.-S.K.), Food and Drug Administration, Silver Spring; and Department of Neurology (J.O.B., S.K.V.), The Neurofibromatosis Therapeutic Acceleration Program, The Johns Hopkins University School of Medicine, Baltimore, MD.
  1. Correspondence
    Dr. Pichard picharddc{at}nih.gov
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