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Protocol Details

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

This study is NOT currently recruiting participants.

Summary | Eligibility | Citations | Contacts

Summary

Number

000173-C

Sponsoring Institute

National Cancer Institute (NCI)

Recruitment Detail

Type: Completed Study; data analyses ongoing
Gender: Male & Female
Min Age: 7 Years
Max Age: N/A

Referral Letter Required

No

Population Exclusion(s)

None

Keywords

Selumetinib;
novel disfigurement scale;
Natural History

Recruitment Keyword(s)

None

Condition(s)

Neurofibromatosis 1

Investigational Drug(s)

None

Investigational Device(s)

None

Intervention(s)

None

Supporting Site

National Cancer Institute

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.

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Eligibility

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.


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Citations:

Not Provided

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Contacts:

Principal Investigator

Referral Contact

For more information:

Andrea M. Gross, M.D.
National Cancer Institute (NCI)
NIHBC 10 - CRC BG RM 1-5742
10 CENTER DR
BETHESDA MD 20892
(240) 858-3853
andrea.gross@nih.gov

Jo H. Hurtt, R.N.
National Cancer Institute (NCI)
National Institutes of Health
Building 10
Room 3-2341
10 Center Drive
Bethesda, Maryland 20892
(240) 858-7012
jo.hurtt@nih.gov

NCI Referral Office
National Institute of Health Clinical Center (CC), 9000 Rockville Pike, Bethesda, Maryland 20892, United States: NCI Clinical Trials Referral Office
1-888-NCI-1937

Clinical Trials Number:

NCT04879160

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