NCT04879160

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

Completed Last updated 22 March 2024

What this trial tests

trial in Neurofibromatosis 1 in 52 participants. Completed in 15 August 2022.

Timeline

12 May 2021

Primary endpoint
15 August 2022

15 August 2022

Quick facts

Lead sponsor	National Cancer Institute (NCI)
Status	Completed
Study type	OBSERVATIONAL
Enrollment	52
Start date	12 May 2021
Primary completion	15 August 2022
Estimated completion	15 August 2022
Sites	1 location across United States

Conditions studied

• Neurofibromatosis 1 — all drugs for Neurofibromatosis 1 →

Sponsor

National Cancer Institute (NCI)

Who can join

7 and older, any sex, with Neurofibromatosis 1. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

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....

Publications & conference data

1 peer-reviewed publication reference this trial (live from Europe PMC):

1. Development and pilot validation of a novel disfigurement severity scale for plexiform neurofibromas in children with neurofibromatosis type 1.
   John L, Singh G, Dombi E, Wolters PL, et al · · 2024 · PMID 37877369 · DOI 10.1177/17407745231206402