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
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.
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.
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.
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|
|Study Type :||Observational|
|Estimated Enrollment :||68 participants|
|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|
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
- 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.
- 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
- 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
- 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
- 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
- Factors most important in causing change in disfigurement [ Time Frame: ongoing ]descriptive statistical analysis of factors
- 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.
- Feasibility of understanding and completing a novel disfigurement scale [ Time Frame: ongoing ]descriptive findings of questionnaire responses
- 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
- 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
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
|Contact: Andrea (Andy) F Gillespie, R.N.||(240) email@example.com|
|Contact: Andrea M Gross, M.D.||(240) firstname.lastname@example.org|
|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|
|Principal Investigator:||Andrea M Gross, M.D.||National Cancer Institute (NCI)|