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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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ClinicalTrials.gov Identifier: NCT04879160
Recruitment Status : Recruiting
First Posted : May 10, 2021
Last Update Posted : July 13, 2022
Sponsor:
Information provided by (Responsible Party):
National Institutes of Health Clinical Center (CC) ( National Cancer Institute (NCI) )

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....


Condition or disease
Neurofibromatosis 1

Show Show detailed description

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Study Type : Observational
Estimated Enrollment : 68 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: Systematically Assessing Changes in Plexiform Neurofibroma Related Disfigurement From Photographs of Subjects With Neurofibromatosis Type 1 on a Phase 2 Clinical Trial
Actual Study Start Date : May 12, 2021
Estimated Primary Completion Date : June 30, 2023
Estimated Study Completion Date : June 30, 2023


Group/Cohort
1/ Cohort 1A
Clinician raters familiar with NF1
2/ Cohort 1B
Clinician raters without specific NF1 familiarity
3/ Cohort 2A
Non-clinician raters familiar with NF1
4/ Cohort 2B
Non-clinician raters without specific NF1 familiarity
5/ Cohort 3
Subjects with NF1



Primary Outcome Measures :
  1. Evaluation of the degree of changes in disfigurement measures [ Time Frame: ongoing ]
    Mean scores for disfigurement as determined by raters reviewing photographs before treatment and just prior to cycle 13 of treatment.


Secondary Outcome Measures :
  1. Differences in the disfigurement scores assigned by raters between cohorts of clinical raters who differ by NF1 familiarity and non-clinical raters who differ by NF1 familiarity [ Time Frame: ongoing ]
    mean scores for disfigurement will be compared using a paired t-test or Wilcoxon signed rank test

  2. Correlation between achieving a partial response on selumetinib and having an improvement in PN-related disfigurement [ Time Frame: ongoing ]
    changes in score will be compared using a Wilcoxon rank sum test

  3. Intra-reliability of a novel disfigurement scale among raters [ Time Frame: ongoing ]
    scores for disfigurement at initial evaluation and a subsequent evaluation to assess reliability will be obtained and subtracted to form the difference in each score at the two time points

  4. Correlation between the degree of change in disfigurement and percentage of PN volume shrinkage [ Time Frame: ongoing ]
    Spearman correlation analysis to assess the degree of change in disfigurement and percentage of PN volume shrinkage from baseline

  5. Factors most important in causing change in disfigurement [ Time Frame: ongoing ]
    descriptive statistical analysis of factors

  6. Agreement among raters within a cohort with respect to the evaluation of disfigurement to assess the reliability of a novel disfigurement scale [ Time Frame: ongoing ]
    Full set of 15 pairwise Kappa statistics, taken two at a time will be constructed. For the full set of 15 Kappa statistics, the mean value along with a two-sided 95% confidence interval, will be reported separately for each of the 4 cohorts.

  7. Feasibility of understanding and completing a novel disfigurement scale [ Time Frame: ongoing ]
    descriptive findings of questionnaire responses

  8. Overall difference among the 4 cohorts of raters with respect to scores assigned for disfigurement [ Time Frame: ongoing ]
    mean scores for disfigurement will be compared using ANOVA or Kruskal-Wallis test as appropriate

  9. PN-related disfigurement rating consistency with subject/parent responses to Global Impression of Change (GIC) [ Time Frame: ongoing ]
    descriptive findings of ratings compared to Global Impression of Change (GIC) responses



Information from the National Library of Medicine

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Ages Eligible for Study:   7 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   Yes
Sampling Method:   Non-Probability Sample
Study Population
Clinicians and non-clinician raters familiar with NF1; Participants enrolled on NIH protocols 11-C-0161 and 08-C-0079 with NF1 and visible plexiform neurofibromas
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 employees of the NIH.
  • 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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04879160


Contacts
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Contact: Andrea (Andy) F Gillespie, R.N. (240) 760-6185 gillesan@mail.nih.gov
Contact: Andrea M Gross, M.D. (240) 858-3853 andrea.gross@nih.gov

Locations
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United States, Maryland
National Institutes of Health Clinical Center Recruiting
Bethesda, Maryland, United States, 20892
Contact: For more information at the NIH Clinical Center contact National Cancer Institute Referral Office    888-624-1937      
Sponsors and Collaborators
National Cancer Institute (NCI)
Investigators
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Principal Investigator: Andrea M Gross, M.D. National Cancer Institute (NCI)
Additional Information:
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Responsible Party: National Cancer Institute (NCI)
ClinicalTrials.gov Identifier: NCT04879160    
Other Study ID Numbers: 10000173
000173-C
First Posted: May 10, 2021    Key Record Dates
Last Update Posted: July 13, 2022
Last Verified: April 25, 2022
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
Plan Description: .All IPD recorded in the medical record will be shared with intramural investigators upon request.
Supporting Materials: Study Protocol
Statistical Analysis Plan (SAP)
Informed Consent Form (ICF)
Time Frame: Clinical data available during the study and indefinitely.
Access Criteria: Clinical data will be made available via subscription to BTRIS and with the permission of the study PI.

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by National Institutes of Health Clinical Center (CC) ( National Cancer Institute (NCI) ):
Selumetinib
novel disfigurement scale
Natural History
Additional relevant MeSH terms:
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Neurofibromatoses
Neurofibromatosis 1
Neurofibroma
Neurofibroma, Plexiform
Nerve Sheath Neoplasms
Neoplasms, Nerve Tissue
Neoplasms by Histologic Type
Neoplasms
Neoplastic Syndromes, Hereditary
Neurocutaneous Syndromes
Nervous System Diseases
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Genetic Diseases, Inborn
Peripheral Nervous System Diseases
Neuromuscular Diseases
Peripheral Nervous System Neoplasms
Nervous System Neoplasms