Systematically Assessing Changes in Plexiform Neurofibroma Related Disfigurement From Photographs of Subjects With Neurofibromatosis Type 1 on a Phase 2 Clinical Trial

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Brief Title

Systematically Assessing Changes in Plexiform Neurofibroma Related Disfigurement From Photographs of Subjects With Neurofibromatosis Type 1 on a Phase 2 Clinical Trial

Official Title

Systematically Assessing Changes in Plexiform Neurofibroma Related Disfigurement From Photographs of Subjects With Neurofibromatosis Type 1 on a Phase 2 Clinical Trial

Brief Summary

      Background:

      People with Neurofibromatosis type 1 (NF1) have an increased risk of developing plexiform
      neurofibromas (PNs). PNs are tumors that form in the tissue. They can form anywhere in the
      body. They can become visible and cause deformations. Researchers want to see if selumetinib
      changes how PNs look in people with NF1. They also want to test a rating system for the
      visibility of these tumors.

      Objective:

      To see if treatment with selumetinib can improve the appearance of visible PNs in people with
      NF1, as determined by people who are/are not familiar with NF1.

      Eligibility:

      People with NF1 who have one or more visible PNs and have been enrolled in study 11C0161 or
      08C0079. Clinicians and non-clinicians with and without experience in NF1 are also needed to
      serve as raters.

      Design:

      Participants are people with NF1 who had photos taken on study 11C0161 or 08C0079. Raters are
      people who will evaluate the PNs in the photos. They will rate the tumors on a scale from 1
      to 10, from less to most visible.

      Participants medical records will be reviewed. Their photos will be shown to 28 raters.

      Raters will fill out a survey about their demographics, place of work, and if they are
      familiar with NF1. They will view sample photos to learn how PNs look and how to rate PNs.

      Raters will view photos of PNs taken before and after selumetinib treatment. They will also
      view photos of PNs that were not treated. They will rate PNs for up to 40 participants. They
      will have 1-2 sessions. Each session will last 1 hour....
    

Detailed Description

      Background:

      Up to 50% of subjects with Neurofibromatosis Type 1 (NF1) will develop histologically benign
      plexiform neurofibromas (PN).

      One of the most common morbidities associated with PN is a disfigurement that has the
      potential to pose formidable obstacles to the medical and psychosocial well-being of subjects
      with NF1.

      The phase 2 trial of the MEK inhibitor selumetinib for inoperable PN (NCT01362803) have shown
      volumetric shrinkage of PN in approximately 74% of subjects with at least one PN morbidity,
      with most of the responsive tumors decreasing in size by 20-50% volume from baseline. Both
      trials have completed enrollment and primary study objectives.

      In this selumetinib trial, standardized photography of visible PN was performed over time to
      assess for changes in disfigurement over time. This trial did not include criteria to measure
      changes in disfigurement and no validated criteria for measurement of PN-related
      disfigurement exist.

      We developed a scale to rate the extent of a disfigurement from NF1 PN and will apply this
      scale to photographs obtained on the phase II selumetinib trial.

      Objective:

      To determine if selumetinib treatment is associated with improvement in PN-related
      disfigurement, as determined by raters reviewing photographs before treatment and just prior
      to cycle 13 of treatment.

      Eligibility:

      Four groups of adult raters to rate disfigurement who are not directly involved in the
      conduct of the selumetinib treatment study (NCT01362803) and can read and write in English.

      Design:

      Methods

      Photographs of consenting individuals with PN, who may or may not have received selumetinib
      will be reviewed.

      Raters will be presented pre and post treatment photographs of persons who received
      selumetinib as well as control photographs of persons not receiving selumetinib.

      Each rater is presented with the same set of photographs though placement and order may
      differ.

      Each rater will look at photographs of each individual with PN for at least 10 seconds, then
      with photos still visible, raters will verbally answer a questionnaire.

      The rater will be asked questions at the end of the study to assess the feasibility of the
      questionnaire.

      Statistical Analysis

      In each cohort of raters, for each subject s slide set evaluated at a time point, the mean of
      the raters scores for each subject will be formed.

      For each person with PN that received selumetinib, the paired difference between baseline and
      pre-cycle 13 of these mean scores will be determined.

      For the full set of approximately 35 paired scores, the difference will be assessed for
      statistical importance by means of a paired t-test, or a Wilcoxon signed rank test.
    


Study Type

Observational


Primary Outcome

Evaluation of the degree of changes in disfigurement measures

Secondary Outcome

 Agreement among raters within a cohort with respect to the evaluation of disfigurement to assess the reliability of a novel disfigurement scale

Condition

Neurofibromatosis 1


Study Arms / Comparison Groups

 1/ Cohort 1A
Description:  Clinician raters familiar with NF1

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information



Estimated Enrollment

68

Start Date

May 12, 2021

Completion Date

June 30, 2022

Primary Completion Date

June 30, 2022

Eligibility Criteria

        -  INCLUSION CRITERIA:

          -  Clinician Raters - those who are professionally involved with direct patient care and
             have a career in the medical field and familiar with NF1 (Cohort 1A), OR

        Clinician Raters - those who are professionally involved with direct patient care and have
        a career in the medical field but are not familiar with NF1 (Cohort 1B), OR

        Non-clinician Raters - those who are not professionally involved in direct patient care or
        do not have a career in the medical field, but who are a first or second degree relative of
        a subject with NF1 or are otherwise closely associated with a subject with NF1 and familiar
        with NF1 (Cohort 2A), OR

        Non-clinician Raters - those who are not professionally involved in direct patient care or
        do not have a career in the medical field, and who are not a first or second degree
        relative of a subject with NF1 and are otherwise not closely associated with a subject with
        NF1 (Cohort 2B)

          -  Age greater than or equal to18 years.

          -  The ability of a subject to understand and the willingness to sign a written informed
             consent document.

          -  Ability to read and write in English.

        EXCLUSION CRITERIA:

        -Raters should not be directly involved in the NCI Phase 1/2 study of selumetinib
        (11-C-0161), either as an investigator or as a subject or first-degree family member of a
        subject enrolled on the trial.
      

Gender

All

Ages

7 Years - N/A

Accepts Healthy Volunteers

Accepts Healthy Volunteers

Contacts

Andrea M Gross, M.D., (240) 858-7013, [email protected]

Location Countries

United States

Location Countries

United States

Administrative Informations


NCT ID

NCT04879160

Organization ID

10000173

Secondary IDs

000173-C

Responsible Party

Sponsor

Study Sponsor

National Cancer Institute (NCI)


Study Sponsor

Andrea M Gross, M.D., Principal Investigator, National Cancer Institute (NCI)


Verification Date

August 31, 2021